Category Archives: Cell Medicine

Passage Of California Stem Cell Proposition Boosts Research

For the last decade and more, Stem Cell research and regenerative medicine have been the rave of the healthcare industry, a delicate area that has seen steady advancements over the last few years.

The promise of regenerative medicine is simple but profound that one day medical experts will be able to diagnose a problem, remove some of our body cells called stem cells and use them to grow a cure for our ailment. Using our body cells will create a highly personalized therapy attuned to our genes and systems.

The terminologies often used in this field of medicine can get a bit fuzzy for the uninitiated, so in this article, I have relied heavily on the insights of Christian Drapeau, a neurophysiologist and stem cell expert.

Drapeau was one of the first voices who discovered and began to speak about stem cells being the bodys repair system in the early 2000s. Since then, he has gone on to discover the first stem cell mobilizer, and his studies and research delivered the proof of concept that the AFA (Aphanizomenon flos-aquae) extract was capable of enhancing repair from muscle injury.

Christian Drapeau is also the founder of Kalyagen, astem cell research-based company, and the manufacturers of Stemregen. This stem cell mobilizer combines some of the most effective stem cell mobilizers Drapeau has discovered to create an effective treatment for varying diseases.

How exactly do stem cell-based treatments work? And how is it delivering on its promise of boosting our abilities to regenerate or self-heal?

Drapeau explains the concept for us;

Stem cells are mother cells or blank cells produced by the bone marrow. As they are released from the bone marrow stem cells can travel to any organ and tissue of the body, where they can transform into cells of that tissue.Stem cells constitute the repair system of the body.

The discovery of this function has led scientists on a long journey to discover how to use stem cells to cure diseases, which are essentially caused by cellular loss. Diseases like Diabetes and age-related degenerative diseases are all associated with the loss of a type of cell or cellular function.

However, what Drapeaus research has unearthed over the last few decades is that there are naturally occurring substances that show a demonstrated ability to induce the release of stem cells from the bone marrow. These stem cells then enter the bloodstream, from where they can travel to sites of cell deficiency or injury in the body to aid healing and regeneration. This process is referred to as Endogenous Stem Cell Mobilization (ESCM).

Stemregen is our most potent creation so far, explains Drapeau, and it has shown excellent results with the treatment of problems in the endocrine system, muscles, kidneys, respiratory systems, and even with issues of erectile dysfunction.

Despite the stunning advancements that have been made so far, a concern that both Drapeau and I share is how this innovation can be merged with another exciting innovation; AI.

Is it even a possibility? Drapeau, an AI enthusiast, explains that AI has already been a life-saver in stem cell research and has even more potential.

On closer observation, there are a few areas in which AI has greatly benefited stem cell research and regenerative medicine.

One obstacle that scientists have consistently faced with delivering the full promise of regenerative medicine is the complexity of the available data.Cells are so different from each other that scientists can struggle with predicting what the cells will do in any given therapeutic scenario. Scientists are faced with millions of ways that medical therapy could go wrong.

Most AI experts believe that in almost any field, AI can provide a solution whenever there is a problem with data analysis and predictive analysis.

Carl Simon, a biologist at the National Institute of Standards and Technology (NIST) and Nicholas Schaub recentlytested this hypothesiswhen they applied Deep Neural Networks (DNN), an AI program to the data they had collected in their experiments on eye cells. Their research revolved around causes and solutions for age-related eye degeneration. The results were stunning; the AI made only one incorrect prediction about cell changes out of 36 predictions it was asked to make.

Their program learned how to predict cell function in different scenarios and settings from annotated images of cells. It soon could rapidly analyze images of the lab-grown eye tissues to classify the tissues as good or bad. This discovery has raised optimism in the stem cell research space.

Drapeau explains why this is so exciting;

When we talk about stem cells in general, we say stem cells as if they were all one thing, but there are many different types of stem cells.For example, hair follicle and dental pulp stem cells contain neuronal markers and can easily transform into neurons to repair the brain. Furthermore, the tissue undergoing repair must signal to attract stem cells and must secrete compounds to stimulate stem cell function. A complex analysis of the tissue that needs repair and the conditions of that tissue using AI, in any specific individual, will help select the right type of stem cells and the best cells in that stem cell population, along with the accompanying treatment to optimize stem cell-based tissue repair.

Christian Drapeau

Ina study published in Februaryof this year inStem Cells, researchers from Tokyo Medical and Dental University (TMDU) reported that their AI system, called DeepACT, had successfully identified healthy, productive skin stem cells with the same accuracy that a human could. This discovery further strengthens Drapeaus argument on the potentials of AI in this field.

This experiment owes its success to AIs machine learning capabilities, but it is expected that Deep Learning can be beneficially introduced into regenerative medicine.There are many futuristic projections for these possibilities, but many of them are not as far-fetched as they may first seem.

Researchers believe that AI can help fast-track the translation of regenerative medicine into clinical practice; the technology can be used to predict cell behavior in different environments. Therefore, hypothetically, it can be used to simulate the human environment. This means that researchers can gain in-depth information more rapidly.

Perhaps the most daring expectation is the possibility of using AI to pioneer the 3D printing of organs. In a world where organ shortage is a harsh reality, this would certainly come in handy. AI algorithms can be utilized to identify the best materials for artificial organs, understand the anatomic challenges during treatment, and design the organ.

Can stem cells actually be used along with other biological materials to grow functional 3D-printed organs? If this is possible, then pacemakers will soon give way to 3D-printed hearts. A 3D-printedheart valvehas already become a reality in India, making this even more of an imminent possibility.

While all of these possibilities excite Drapeau, he is confident that AIs capabilities with data analysis and prediction, which is already largely in use, would go down as its most beneficial contribution to stem cell research;

It was already shown that stem cells laid on the connective tissue of the heart, the soft skeleton of the heart, can lead the entire formation of a new heart. Stem cells have this enormous regenerative potential. AI can take this to another level by helping establish the conditions in which this type of regeneration can be orchestrated inside the body.But we have to be grateful for what we already have, over the last 20 years, I have studied endogenous stem cell mobilization and today the fact that we have such amazing results with Stemregen is testament that regenerative medicine is already a success.

As AI continues to scale over industry boundaries, we can only sit back and hope it delivers on its full potential promise. Who knows? Perhaps AI really can change the world.

Read more:
How The Overlap Between Artificial Intelligence And Stem Cell Research Is Producing Exciting Results - Forbes

-Initiation of patient enrollment expected by year-end-

ZUG, Switzerland and CAMBRIDGE, Mass. and SAN DIEGO, Nov. 16, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and ViaCyte, Inc., a clinical-stage regenerative medicine company developing novel cell replacement therapies to address diseases with significant unmet needs, today announced that Health Canada has approved the companies Clinical Trial Application (CTA) for VCTX210, an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). Initiation of patient enrollment is expected by year-end.

With the approval of our CTA, we are excited to bring a first-in-class CRISPR-edited cell therapy for the treatment of type 1 diabetes to the clinic, an important milestone in enabling a whole new class of gene-edited stem cell-derived medicines, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. The combination of ViaCytes leading stem cell capabilities and CRISPR Therapeutics pre-eminent gene-editing platform has the potential to meaningfully impact the lives of patients living with type 1 diabetes.

Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies, said Michael Yang, President and Chief Executive Officer of ViaCyte. This approach builds on previous accomplishments by both companies and represents a major step forward for the field as we strive to provide a functional cure for this devastating disease.

The Phase 1 clinical trial of VCTX210 is designed to assess its safety, tolerability, and immune evasion in patients with T1D. This program is being advanced by CRISPR Therapeutics and ViaCyte as part of a strategic collaboration for the discovery, development, and commercialization of gene-edited stem cell therapies for the treatment of diabetes. VCTX210 is an allogeneic, gene-edited, stem cell-derived product developed by applying CRISPR Therapeutics gene-editing technology to ViaCytes proprietary stem cell capabilities and has the potential to enable a beta-cell replacement product that may deliver durable benefit to patients without requiring concurrent immune suppression.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

About ViaCyteViaCyte is a privately held clinical-stage regenerative medicine company developing novel cell replacement therapies based on two major technological advances: cell replacement therapies derived from pluripotent stem cells and medical device systems for cell encapsulation and implantation. ViaCyte has the opportunity to use these technologies to address critical human diseases and disorders that can potentially be treated by replacing lost or malfunctioning cells or proteins. ViaCytes first product candidates are being developed as potential long-term treatments for patients with type 1 diabetes to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. To accelerate and expand ViaCytes efforts, it has established collaborative partnerships with leading companies, including CRISPR Therapeutics and W.L. Gore & Associates. ViaCyte is headquartered in San Diego, California. For more information, please visitwww.viacyte.comand connect with ViaCyte onTwitter,Facebook, andLinkedIn.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni and Mr. Yang in this press release, as well as regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of our various clinical programs including our VCTX210 program; (ii) the status of clinical trials (including, without limitation, activities at clinical trial sites) and expectations regarding data from clinical trials; (iii) the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies, including as compared to other therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Therapeutics Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

ViaCyte Investor Contact: David Carey, Lazar-FINN Partners+1-212-867-1768david.carey@finnpartners.com

ViaCyte Media Contact: Glenn Silver, Lazar-FINN Partners+1-973-818-8198glenn.silver@finnpartners.com

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CRISPR Therapeutics and ViaCyte, Inc. to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes -...

Dr Pengyi Yang uses computational expertise to build virtual cells.

DrPengyiYanghasreceived one of two annual $55,000 Metcalf Prizes from the National Stem Cell Foundation of Australia inrecognition of his leadership in the field.

DrYangholds a joint position with the University of SydneySchool of Mathematics & Statistics, theCharles Perkins Centreand theChildren's MedicalResearch Institute. His work aims toremove much of the guesswork from stemcell science and eventually stemcell medicine.

Todays stem cell treatmentshave beenthe product of trial anderror, DrYang said.

My virtual stem cell will allow us to understand whats happening inside a single stem cell that makes it decide what type of cell it will becomesuch as, but not limited to,hair, skin, muscle, nerveorbloodcells.

He is mapping the many, complex influencescontrollingstem cells andthe waythey specialise into different cell types.

Stem cells are amazing because they can produce any kind of cell in the body. Theyre fundamental toregenerative medicine,DrYang said.

But, when theircontrols fail,rogue stem cells can lead to cancer.

Allhumanlifestartsas a single stem cell. It goes on to produce cells that eventually become every type of tissue and organ of the human body. Even in adulthood, stem cellsrepairandreplacetissue all the time.

People are excited about the potential of stem cell medicine, but thereality is extremely complicated. Thousands of genes, complex gene networks, environmental factors, and an individuals own health are all involved in pushing stem cells to become specific cell types,DrYang said.

DrYang, a computerscientist turned stem cell researcher, uses computational science and statistics to understand how stem cells function at a fundamental level work that will be useful forthe entire stem cell field ofresearch.

We need a computermodel to bring all of these influences togetherso we can identify the specific gene networks that drive the stem cells towards each cell type,he said.

Read the rest here:
Dr Pengyi Yang wins National Stem Cell Foundation Metcalf Prize - News - The University of Sydney

Gavreto is the first and only precision medicine approved in the EU for first-line treatment of people with RET fusion-positive advanced NSCLC

Conditional approval is based on results from the phase I/II ARROW study, in which Gavreto led to durable responses in people with RET fusion-positive advanced NSCLC

Basel, 19 November 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has granted conditional marketing authorisation for Gavreto (pralsetinib) as a monotherapy for the treatment of adults with rearranged during transfection (RET) fusion-positive advanced non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor. Gavreto is the first and only precision medicine approved in the European Union (EU) for the first-line treatment of people with RET fusion-positive advanced NSCLC.1

Todays approval represents an important step forward in delivering precision medicine to people with RET fusion-positive advanced non-small cell lung cancer, for whom treatment options have historically been limited," said Levi Garraway, M.D., Ph.D., Roches Chief Medical Officer and Head of Global Product Development. By using cancer genomic profiling upfront, healthcare professionals may identify specific genetic alterations that predict clinical benefit of targeted treatment options like Gavreto in the first-line setting.

The approval is based on results of the ongoing phase I/II ARROW study, in which Gavreto led to durable responses in people with advanced RET fusion-positive NSCLC.2 In 75 treatment-nave patients, Gavreto demonstrated an overall response rate (ORR) of 72.0% (95% CI: 60.4%, 81.8%), and median duration of response (DOR) was not reached (NR) (95% CI: 9.0 months, NR).2 In 136 patients who had previously received platinum-based chemotherapy, Gavreto demonstrated an ORR of 58.8% (95% CI: 50.1%, 67.2%), and median DOR was 22.3 months (95% CI: 15.1 months, NR).2 Gavreto was also generally well-tolerated, with a low rate of treatment discontinuation; common grade 3-4 adverse reactions were neutropenia (reported in 20.1% of patients), anaemia (17.6%) and hypertension (16.1%).2

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Approximately 37,500 people are diagnosed with RET fusion-positive NSCLC worldwide each year; the disease often affects people with minimal to no history of smoking, and who are typically younger than the average person diagnosed with lung cancer.3,4,5 Roche is committed to providing a tailored treatment option for every person with lung cancer, no matter how rare or difficult-to-treat their type of disease. Gavreto in RET fusion-positive advanced NSCLC, along with Alecensa (alectinib) in ALK-positive advanced NSCLC and Rozlytrek (entrectinib) in ROS1-positive advanced NSCLC, is part of Roches growing portfolio of precision medicines. Together, they offer personalised treatment options for almost one in ten people with advanced NSCLC, and biomarker testing is the most effective way to identify those people who may benefit.6

Beyond NSCLC, RET alterations are also key disease drivers in other cancer types, such as thyroid cancers. Gavreto has shown activity across multiple solid tumour types, reflecting tumour-agnostic potential.7 It is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with metastatic RET fusion-positive NSCLC, and for the treatment of adult and paediatric patients 12 years of age and older with advanced RET-altered thyroid cancers. Gavreto is also approved in Canada, mainland China and Switzerland. In the EU, a submission for RET-altered thyroid cancers is planned. Regulatory submissions for advanced RET fusion-positive NSCLC and RET-altered thyroid cancers are also underway in multiple countries worldwide.

Blueprint Medicines and Roche are co-developing Gavreto globally, with the exception of certain territories in Asia, including China.* Blueprint Medicines and Genentech, a wholly owned member of the Roche Group, are commercialising Gavreto in the US and Roche has exclusive commercialisation rights for Gavreto outside of the US, with the exception of certain territories in Asia, including China.*

About the ARROW study8ARROW is an ongoing phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer, RET fusion-positive thyroid cancer and other RET-altered solid tumours. ARROW is being conducted at multiple sites across the United States, Europe and Asia.

About rearranged during transfection (RET)-altered cancersRET gene alterations, such as fusions and mutations, are key disease drivers in many types of cancer, including non-small cell lung cancer (NSCLC) and several types of thyroid cancer. There are approximately 2.21 million cases of lung cancer diagnosed each year worldwide,3 of which approximately 1.8 million are NSCLC and RET fusions are present in approximately 1-2% of these patients,4,5 meaning RET fusion-positive NSCLC affects up to 37,500 people each year. Additionally, approximately 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer) have RET fusion-positive tumours,9 and roughly 90% of people with advanced medullary thyroid cancer (a less prevalent form of thyroid cancer) carry RET mutations.10 Oncogenic RET fusions also are observed at low frequencies in other cancers, including cholangiocarcinoma, colorectal, neuroendocrine, ovarian, pancreatic and thymus cancers.

About Gavreto (pralsetinib)Gavreto is a once-daily, oral precision medicine designed to selectively target rearranged during transfection (RET) alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment. Blueprint Medicines and Roche are co-developing Gavreto for the treatment of people with various types of RET-altered cancers.

About Roche in lung cancerLung cancer is a major area of focus and investment for Roche, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have six approved medicines to treat certain kinds of lung cancer, and a pipeline of investigational medicines to target the most common genetic drivers of lung cancer, or to boost the immune system to combat the disease.

About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve peoples lives. The combined strengths of pharmaceuticals and diagnostics, as well as growing capabilities in the area of data-driven medical insights help Roche deliver truly personalised healthcare. Roche is working with partners across the healthcare sector to provide the best care for each person.

Roche is the worlds largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. In recent years, the company has invested in genomic profiling and real-world data partnerships and has become an industry-leading partner for medical insights.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the thirteenth consecutive year, Roche has been recognised as one of the most sustainable companies in the pharmaceutical industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

*CStone Pharmaceuticals retains all rights to the development and commercialisation of Gavreto in these territories (mainland China, Taiwan, Hong Kong and Macau) under its existing collaboration with Blueprint Medicines.

All trademarks used or mentioned in this release are protected by law.

References[1] Gavreto, Summary of Product Characteristics. 2021.[2] Roche data on file.[3] World Health Organization. Cancer [Internet; cited 2021 Nov]. Available from: https://www.who.int/news-room/fact-sheets/detail/cancer%5B4%5D American Cancer Society. Key Statistics for Lung Cancer [Internet; cited 2021 Nov]. Available from: https://www.cancer.org/cancer/lung-cancer/about/key-statistics.html%5B5%5D Drilon A, et al. Brief Report: Frequency of Brain Metastases and Multikinase Inhibitor Outcomes in Patients With RET-Rearranged Lung Cancers. J Thorac Oncol. 2018;13:1595-601. [6] Pakkala S, Ramalingam SS. Personalized therapy for lung cancer: striking a moving target. JCI Insight. 2018;3(15):e120858.[7] Subbiah V, et al. Clinical activity and safety of the RET inhibitor pralsetinib in patients with RET fusion-positive solid tumors: Update from the ARROW trial. Presented at the American Society of Clinical Oncology (ASCO) Annual Meeting 2021; 04-08 Jun, 2021. Abstract #3079.[8] ClinicalTrials.gov. Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors (ARROW) [Internet; cited 2021 Nov]. Available from: https://clinicaltrials.gov/ct2/show/NCT03037385%5B9%5D Santoro M, et al. RET Gene Fusions in Malignancies of the Thyroid and Other Tissues. Genes. 2020;11(4):424.[10] Romei C, et al. RET mutation heterogeneity in primary advanced medullary thyroid cancers and their metastases. Oncotarget. 2018;9(11):9875-84.

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European Commission approves Roches Gavreto (pralsetinib) for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer -...

COVID-19 has been all-consuming. For nearly two years, the world has been focused on the race for vaccines, the pressures on providers, the best testing protocols, and simply staying safe.

COVID-19 also slowed some research efforts, but scientists still managed to seek solutions for many other pressing concerns Alzheimers disease, maternal mortality, and prostate cancer among them that have bedeviled patients for decades.

Below are eight medical advances that may not have grabbed your attention but could ultimately improve the lives of millions.

Assessing a stroke demands a rapid, life-or-death assessment: Is the culprit a clot, which requires a blood thinner, or bleeding in the brain, which requires surgery? Now, a portable MRI device can help make that assessment right at a patients bedside and in much less time than required by a trip to a standard machine.

The Swoop MRI which was created with input from Yale Medicine in New Haven, Connecticut received Food and Drug Administration (FDA) approval in August 2020 and is already at work in several U.S. hospitals.

The new portable machine offers many advantages over its massive cousin, says Yale neurologist Kevin Sheth, MD.

The very strong magnets in regular MRIs bring a lot of challenges, he explains. You need intensive power and cooling, precautions like a shielded room, and a lot of training. If you use a weaker magnet, all those problems go away.

The weaker magnet is effective, according to an August 2021 study, which asked clinicians to identify various cerebral pathologies using Swoop images. The goal is not to be as good as a high-magnet MRI, but to be good enough for clinical decisions, says Sheth, who co-authored the study but has no financial interest in Hyperfine, the Connecticut-based company that produces the machine.

Swoops size its smaller than some refrigerators eliminates the need to move frail patients down hospital hallways. Whats more, its cost around $100,000 compared to $1 million for the bigger machine puts it within reach of hospitals and regions with fewer resources. This could essentially democratize brain imaging, argues Sheth.

Prostate cancer strikes 1 out of 8 U.S. men, and it is expected to take more than 34,000 lives this year alone. When it metastasizes, the disease is almost always incurable, leaving physicians focused only on postponing death and improving patients lives.

A promising new approach has succeeded at both goals and did so among men with an advanced form of the disease whose condition had deteriorated despite receiving standard treatments.

In fact, it more than doubled how long patients lived without their cancer worsening, according to a paper published in September. The study, which followed 831 men in 10 countries for a median of 20 months, compared patients who continued to receive standard care with ones who got the new treatment.

The treatments name is complex: lutetium-177-PSMA-617. But its approach is straightforward: Drive radiation directly into a cancer cell while sparing healthy tissue around it.

The method uses a compound called PSMA-617 to hone in on a protein found almost exclusively in prostate cancer cells, explains Oliver Sartor, MD, study co-lead investigator and medical director of Tulane Cancer Center in New Orleans. Then, a radioactive particle carried by the compound blasts the cancer cells, wherever they are.

Its like a little smart bomb, says Sartor.

In September, the FDA granted the treatment priority review status, according to drug manufacturer Novartis, which funded the study. An answer is expected in the first half of 2022.

Sartor feels hopeful. Ive been working in prostate cancer for more than 30 years, and this is the largest advance Ive ever been associated with.

For more than 5,000 years, sickle cell disease (SCD) has caused untold suffering in people of African descent. In patients with the genetic illness, red blood cells are not round but crescent-shaped like a sickle and can clog blood vessels, depriving the body of oxygen and causing tremendous pain. For a long time, the only cure has been a bone marrow transplant, but new gene-editing techniques now may offer a safe and effective alternative.

In research conducted at Boston Childrens Hospital, scientists used a virus to switch off the gene that triggers cells sickling, according to a January 2021 study. The patients subsequently produced healthy red blood cells and nearly all were able to discontinue the blood transfusions SCD often requires.

One participant used to have transfusions every month but has not needed any in three years, says David Williams, MD, chief of the Division of Hematology/Oncology at Boston Childrens and head of the research team. This has completely changed his life.

The study followed six patients for a median of 18 months and found that the treatment completely halted the diseases more severe symptoms.

Im so happy for my sickle-cell patients. This is a terrible disease, notes Williams.

Next up for Williams is a trial with 25 patients. Meanwhile, SCD researchers elsewhere are studying other gene-editing techniques. All these approaches look promising, and we need a lot more research to determine if one or another is better, Williams says.

This is a very exciting time. In the past, we havent had any particularly good treatments, and now we have several possibilities," he adds.

When a womans uterus fails to contract after childbirth, tremendous blood loss can ensue, possibly leading to an emergency hysterectomy or even death. In fact, postpartum hemorrhage affects 3% to 10% of all childbirths in the United States and causes more than one-third of childbirth-related maternal deaths worldwide.

Treatment options include medications that dont always work and inserting a balloon to put pressure on the uterus much like exerting pressure on a cut that comes with risks and must remain in place for a day.

But providers now have another option.

A new vacuum device aids natural post-birth contractions, putting pressure on leaking blood vessels. The FDA approved the device the Jada vacuum uterine tamponade in September 2020 following a 12-site research study.

The vacuum approach is very logical since its like what the body is supposed to do, says Dena Goffman, MD, the primary investigator at Columbia University Irving Medical Center in Manhattan. Also, the vacuum is used for less time than the balloon roughly two or three hours. For moms, thats a big deal because it makes it easier to breastfeed, get out of bed, and bond with their child, she adds.

The vacuum controlled bleeding in a median of three minutes and successfully treated 94% of participants, according to the study, which was funded by the devices manufacturer, Alydia Health. In comparison, other research puts the balloons effectiveness at 87%.

When a patient has a postpartum hemorrhage and youre the doctor at the bedside, its scary because you know how quickly things can deteriorate, says Goffman. Using this device, when you see the bleeding slowing quickly and you can feel the uterus contracting, its just incredible.

Tearing an anterior cruciate ligament (ACL) the flexible band inside the knee that helps stabilize it can upend a sports career and sideline weekend athletes. Between 100,000 and 200,000 ACL tears occur each year in the United States.

The most effective repair option has been removing the ruptured ACL, harvesting a graft from the shin or elsewhere, sewing that tissue into the knee, and hoping both surgical sites heal well.

In December 2020, the FDA approved a simpler, more natural method: the Bridge-Enhanced ACL Restoration (BEAR).

We basically stimulate the ACL to heal itself, says Martha Murray, MD, orthopedic surgeon-in-chief at Boston Childrens Hospital and BEARs creator.

The approach involves placing a protein-based sponge, prepared with some of the patients own blood, between the torn ACL ends. Murray explains that the blood promotes the connection of the two ACL pieces to the sponge and, ultimately, to each other.

So far, the approach has been tested on more than 100 patients. In a May 2020 study, patients and physicians reported that BEAR performed as well as the standard repair and without the graft surgery that can cause ongoing pain or weakness at the donor site. Miach Orthopaedics, which has the worldwide exclusive license for the BEAR implant, has already begun making it available through orthopedic surgeons in the United States.

For Murray, the experience has highlighted the value of serving as a physician-researcher. When youre faced with a patient with a problem and the current solution is imperfect, its great to be able to say, Were working on a better solution. Its incredibly gratifying.

For the first time since 2014, a new obesity medication has hit the market, offering hope to the 78 million Americans who face the many risks of excess weight: cancer, heart disease, diabetes, and complications from COVID-19, among others.

And the new medication semaglutide, also known as Wegovy is significantly more powerful than its predecessors, according to research that helped it garner approval from the FDA in June.

Weve seen 1 to 2 times the amount of weight loss compared to other medications, says Robert Kushner, MD, a researcher at Northwestern University Feinberg School of Medicine who has led semaglutide studies. That's a leapfrog advance.

In fact, semaglutide recipients lost nearly 15% of their body weight on average compared with 2.4% among controls, according to one study of nearly 2,000 patients.

Semaglutide an injectable medication is not entirely new. A synthetic version of a natural hormone that quells appetite, its already used to treat Type 2 diabetes. But the obesity trials, paid for by pharmaceutical company Novo Nordisk, used a much higher dose.

High doses havent been studied long enough to identify long-term side effects, notes Kushner, a paid consultant to Novo Nordisk. But the recent research reported mild-to-moderate gastrointestinal issues that lessened over time.

Now Kushner hopes semaglutide will help spark interest in obesity medications.

Over 40% of U.S. adults have obesity, and the number who are getting a pharmacologic treatment is under 3%, he says. Part of the challenge is educating primary care providers that providing evidence-based obesity care includes consideration of medication."

Randall Bateman, MD, a Washington University School of Medicine in St. Louis (WUSTL) neurologist, is thrilled to have contributed to the first blood test for Alzheimer's disease a devastating condition that affects as many as 5.8 million Americans.

Back in 2017, though, as Bateman geared up to share the discovery that would enable the test, he worried about his peers reaction. After all, scientists were convinced that the blood marker he studied couldnt predict the disease.

But the WUSTL method was much more sensitive and direct than prior approaches. The resultant test called PrecivityAD effectively detects the amyloid plaques that are a hallmark of Alzheimers disease and has proven as accurate as the previously used tools of a spinal tap or positron emission tomography (PET) scan, which are far more costly and complex.

The test, developed by a company called C2N Diagnostics that Bateman co-founded, has been available to physicians since October 2020, when it received approval through a federal lab certification program. It now awaits additional approval from the FDA.

Weve been hoping for a test to diagnose Alzheimers for more than 20 years, says Bateman, WUSTLs Charles F. and Joanne Knight distinguished professor of neurology. Currently, up to half of people with Alzheimers are misdiagnosed.

The road to success in science is paved with hard work and great uncertainty, he adds. Its a real gamble. Youre investing your life in this work, and you hope it will have a positive impact. And then its like, Wow, it worked!

Anger, fear, recurring nightmares, and intense flashbacks are among the many symptoms that can batter patients with post-traumatic stress disorder (PTSD). The condition, which affects about 15 million U.S. adults in a given year, can be extremely difficult to treat.

A potentially groundbreaking PTSD treatment now lies in a seemingly unlikely source: MDMA, better known as the illegal drugs ecstasy and molly that fueled all-night dance raves and caused potentially fatal side effects.

In June, a study in Nature Medicine reported that patients with severe PTSD combat veterans, first responders, and victims of sexual assault and mass shootings, among others experienced significant relief from MDMA.

In fact, two months after treatment, 67% of subjects who received MDMA together with talk therapy no longer qualified for a diagnosis of PTSD. I saw this amazing transformation in patients, says Jennifer Mitchell, PhD, the studys lead author and a University of California, San Francisco, School of Medicine neurology professor.

The treatment involved three eight-hour sessions a month apart during which patients ingested MDMA and processed painful memories and emotions in talk therapy.

MDMA releases a powerful supply of serotonin and stimulates hormones associated with emotional bonding, Mitchell explains. The idea is that it helps patients be open in a way that enables them to connect well with therapists and work through their problems more quickly.

Before the drug can receive FDA approval for PTSD, researchers need to complete one more clinical trial. Even if it succeeds, Mitchell is aware that MDMA still bears stigma from its party drug image.

I hope people are going to be open-minded and look at the data, which included no abuse potential or other serious side effects from MDMA as used in the study. We are talking about use in a controlled, therapeutic situation, she says. Using drugs recreationally is entirely different. Otherwise, people would come back from [the art and community event] Burning Man cured of their psychological issues.

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8 medical advances you may have missed during COVID-19 - AAMC

Sponsored Article

Although stem cells are known to work wonders, there is still a lot of misunderstanding about what they are, what they do, and how they work.

The good news is that StemCells21 can clear everything up for you. SC21 produces all of its cellular medications in-house, and all of its treatments are performed at its cutting-edge medical centre in Bangkok. Its a one-stop shop that adheres to high-quality standards.

This company will be on display at the Thailand International Boat Show, which will be hosted at Royal Phuket Marina from January 6 to 9 next year. Staff from StemCells21 will be on hand to walk you through the producers, pricing, and techniques.

StemCells21s laboratory is a full-scale culture & analysis laboratory specialising in the production & treatment of Mesenchymal Stem Cells (StemCells21), and Natural Killer Cells (ImmuneCells21). It has also launched a new generation of regenerative medicine called Pluripotent Stem Cells (iPSC21), which hold great potential for impacting chronic diseases in the quest for anti-ageing.

The lab has seven scientists & stem cell researchers, a couple of who have worked with Professor Shinya Yamanaka, who was awarded the Nobel Prize in Physiology or Medicine in 2012 for the discovery that mature cells can be reprogrammed to become pluripotent (iPS cells).

Photo Via: Stemcells 21

Before StemCells21 was created, Managing Director Paul Collier and co-founder Sergei Dmitrievs experienced the power of stem cells either first hand or through the treatment of someone close to them. They knew that stem cells could deliver positive health results, and also knew stem cell treatmentsand the clinics that administered themhad room for improvement.

After deep laboratory investigation, they came to see that most clinics utilised relatively low-quality stem cells and incomplete treatments. While these clinics could deliver a certain level of positive results, they were only scratching the surface of the promise that stem cell treatments could deliver.

Furthermore, the clinics themselves frequently provided a less-than-ideal patient experience. Clinics were generally hectic, unprofessional, and unwelcoming. Patients were often administered a single treatment and sent on their way, unsure if they had experienced an efficacious treatment or if they had travelled and paid for nothing.

StemCells21 was created to offer superior results and give you a welcoming experience. It was set up to provide the global community with access to treatments that few people are aware of, and to offer health benefits that are superior to what most people ever imagined were possible.

The SC21 complex in Bangkok houses the StemCells21, ImmuneCells21, and IPS21 laboratories, as well as the premium 5* IntelliHealth+ (IH+) Clinic.

IntelliHealth+ is a state-of-the-art medical centre licensed by the Thai medical authorities. The luxurious design, efficient workflow layouts, and modern treatments make it the ideal choice for customers seeking a premium level of healthcare in 5* settings.

The centre treats patients from all over the world and has staff who speak fluent English, Arabic, Chinese, Russian, Thai and Spanish.

Furthermore, SC21s come from all corners of the globe for these cutting edge treatments. Many VIPs travel to the clinic including presidents, prime ministers, sports stars, football managers, bank owners and heads of major corporations, many of whom return every six to twelve months and have been doing so for years.

Recently, SC21 treated a ten-year-old British boy who had Ewing sarcoma develop in his arm, which then spread to other areas. He had tried every treatment option in the UK. His trip and treatment were sponsored by UK football teams and the public. Since he started treatment hes put on weight, hes vibrant, and his demeanour has totally changed. Various tests and scans have shown he is responding very well to the immunotherapy course and will perform another round in a few months time.

SC21 focuses on three main areas: anti-ageing and longevity; orthopaedic and muscular-skeletal issues (knee, hip, back & shoulder); and chronic diseases (diabetes, liver cirrhosis, lung, respiratory, hearing & vision disorders). Aside from that, the clinic can also help with chronic fatigue and burn-out syndrome.

Outpatient services for anti-ageing, immunotherapy and regenerative medicine are available at the centre. The anti-ageing clinic has a cutting-edge approach to skin rejuvenation, dermatology, detoxification, and wellbeing. A youthful appearance, more energy, improved mental capacity and mobility, reduced aches and pains, and a stronger immune system are among the benefits.

Photo Via: Stemcells 21

The high level of traditional medicine and the unique protocols designed by the IH+ teams give patients real therapeutic benefits and longevity.

According to Paul Collier, a client typically receives two sessions of stem cell injections during a treatment intravenous for systemic and local to the target and is required to stay in Bangkok for two days following their procedure to monitor any complications that may arise. Then theyre given a two-month take-home kit that comprises self-administered injections (similar to insulin) that target specific growth factors in organs or tissues that need to be repaired. These can also be taken orally, but they are less effective.

He goes on to say that stem cells are the foundation of the human body. They split over and over to produce humans from an embryo at the start of our lives. They restore cells in your blood, bone, skin, and organs throughout your life to keep you alive and functioning. Stem cells have two distinct properties that distinguish them from other types of cells in our bodies.

First, they can self-renew (mitosis), which is a stage of the cell cycle in which replicated chromosomes are divided into two new nuclei. As a result, identical duplicated cells are produced.

Secondly, they have the ability to differentiate into specialized cells such as cartilage, heart cells, liver cells, and neurons. No other cell in the body has the natural ability to generate new cell types.

Mesenchymal Stem Cells (MSCs) are at the core of StemCells21s regenerative programs. They are multipotent stem cells derived from various adult and fetal tissues. A large number of studies have shown the beneficial effects of MSC-based therapies to treat different pathologies, including neurological disorders, cardiac ischemia, diabetes, and bone and cartilage diseases.

StemCells21 also has arthritis treatment, which reduces inflammation & joint pain, increases cartilage growth, improves mobility & joint stability and lessens dependence on medication. The clinics degenerative spine treatments help discs regenerate and stabilize the spine.

On top of that, it provides lung & liver disease treatment as well as treatments for autism, cerebral palsy, diabetes, motor neuron disease, multiple sclerosis and immune disorders.

Theres even eye treatment, which reduces blurred vision & field of vision defects, improves night vision & enhances colour texture.

Photo Via: Stemcells 21

SC21 can even help with certain types of cancer by taking a clients blood and growing their natural killer cells (immunotherapy) over a 21-day period. Through various stimuli, their cytotoxicity is increased which kills cancer and virally-affected cells.

Paul says stem cell therapy should be looked at before undergoing any kind of invasive surgery. The type of medicine should certainly be an intervention before surgery. If you are looking at knee replacement, why not consider an injection of a biologic that would only take a couple of days and has the potential to remodel the cartilage, because once you perform surgery there is no going back.

SC21 also produces a wide range of stem-cell extract-based cosmetics and nutritional supplements, which are available at their medical centres and online under the brand SC21 Biotech.

The Thailand International Boat Show will feature Paul Collier and his team. Theyll be able to answer any of your questions about the cost, procedure, and treatment. On top of that, they will also assist you in educating yourself and managing your expectations so that you do not expect more than stem cell therapy can provide. If you want to get treatment, they will also provide you with a complete report on all treatments. SC21 is fully compliant with international regulations and guidelines.

http://www.stemcells21.com http://www.intellihealthplus.com

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SC21- 21st century cellular medicines specialists - The Thaiger

UNC-Chapel Hill and UC San Francisco scientists have published work in Nature, laying the groundwork for better anti-itching medications with fewer side effects. The work was led by UNC School of Medicine scientists Bryan L. Roth, MD, PhD, Jonathan Fay, PhD, and Can Cao, PhD.

CHAPEL HILL, NC Ever wonder whats going on when you get itchy skin, whether from a rash or medication or some other bodily reaction? And why do some strong anti-itching medications make us nauseous, dry-mouthed zombies? Scientists at the UNC School of Medicine and the University of California at San Francisco conducted research showing in precise detail how chemicals bind to mast cells to cause itch, and the scientists figured out the detailed structure of receptor proteins on the surface of these cells when a compound is bound to those proteins.

This work, published in Nature, was led by the labs of Bryan L. Roth, MD, PhD and Jonathan Fay, PhD at UNC-Chapel Hill, and Brian Shoichet, PhD, at UC San Francisco, co-senior authors who have collaborated on previous studies of important cell receptors protein complexes that chemicals (including drugs) bind to cause or stop a reaction inside cells.

Our work provides a template for the design of new anti-itch medications, said Roth, the Michael Hooker Distinguished Professor of Pharmacology. Also, our research team did a truly remarkable job showing precisely how chemically distinct compounds induce itching through one of two distinct receptors known to be involved in itching.

First author Can Cao, PhD, a postdoctoral research in the Roth lab, and co-senior author Jonathan Fry, PhD, now an assistant professor in the UNC Department of Biochemistry and Biophysics, led the experiments during the COVID pandemic.

On the surface of cells sit receptor proteins you can think of as complex locks. When a chemical key enters the lock, not only does the cell open, but the chemical causes a chain reaction of signals inside cells. Many chemicals do this, from naturally occurring dopamine in the brain to caffeine and cocaine.

When it comes to itch, Roths lab identified two receptors called MRGPRX2 on the surface of mast cells and MRGPRX4 on itch-sensing neurons that live in connective tissue and play roles in allergies, immune tolerance, wound healing and other factors in health and disease.

Several drugs unintentionally flood these receptors to trigger the release of histamines, causing the side effect of itching. Drugs such as

nateglinide for diabetes, as well as morphine, codeine, and the cough suppressant dextromethorphan are known to cause this reaction. Antihistamines are designed to tamp down the itch response, but they and other anti-itching medications do so clumsily, tripping other cell signaling pathways to cause side effects such as drowsiness, blurred vision, dry mouth, nausea, etc.

The researchers used the experimental technique electron microscopy to create high-resolution maps of these complex receptor proteins when bound to a compound that causes the release of histamines to cause itchiness. They also clarified how drugs bind to MRGPRX4 to cause itch related to various drugs and liver diseases. The researchers used the CryoEM Core Facility at UNC-Chapel Hill to determine the receptor structures.

Knowing precisely how all this plays out at the molecular level will help us and others create better ways to control the role of these two receptors in itchiness and other conditions, Roth said.

MRGPRX2 and MRGPRX4 have also been implicated in inflammation arising from the nervous system, eczema, ulcerative colitis, and pain.

The relatively potent agonists and antagonists described in our Nature paper provide chemical probes we can use to explore the biology of these receptors, Roth said, And the structures we revealed so far should accelerate the search for specific medications targeting MRGPRs.

The National Institutes of Health funded this research.

Media contact: Mark Derewicz, 919-923-0959

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Researchers Reveal Structure of Itch Receptors on Cells | Newsroom - UNC Health and UNC School of Medicine

KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Mercks anti-PD-1 therapy, for the adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions. The approval is based on data from the pivotal Phase 3 KEYNOTE-564 trial, in which KEYTRUDA demonstrated a statistically significant improvement in disease-free survival (DFS), reducing the risk of disease recurrence or death by 32% (HR=0.68 [95% CI, 0.53-0.87]; p=0.0010) compared to placebo. Median DFS has not been reached for either group.

Despite decades of research, limited adjuvant treatment options have been available for earlier-stage renal cell carcinoma patients who are often at risk for recurrence. In KEYNOTE-564, pembrolizumab reduced the risk of disease recurrence or death by 32%, providing a promising new treatment option for certain patients at intermediate-high or high risk of recurrence, said Dr. Toni K. Choueiri, director, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute, and professor of medicine, Harvard Medical School. With this FDA approval, pembrolizumab may address a critical unmet treatment need and has the potential to become a new standard of care in the adjuvant setting for appropriately selected patients.

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue and can affect more than one body system simultaneously. Immune-mediated adverse reactions can occur at any time during or after treatment with KEYTRUDA, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions. Early identification and management of immune-mediated adverse reactions are essential to ensure safe use of KEYTRUDA. Based on the severity of the adverse reaction, KEYTRUDA should be withheld or permanently discontinued and corticosteroids administered if appropriate. KEYTRUDA can also cause severe or life-threatening infusion-related reactions. Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. For more information, see Selected Important Safety Information below.

KEYTRUDA is foundational for the treatment of patients with certain advanced cancers, and this approval marks the fourth indication for KEYTRUDA in earlier stages of cancer, said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. KEYTRUDA is now the first immunotherapy approved for the adjuvant treatment of certain patients with renal cell carcinoma. This milestone is a testament to our commitment to help more people living with cancer.

In RCC, Merck has a broad clinical development program exploring KEYTRUDA, as monotherapy or in combination, as well as other investigational products across multiple settings and stages of RCC, including adjuvant and advanced or metastatic disease.

Data Supporting the Approval

KEYTRUDA demonstrated a statistically significant improvement in DFS in patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions compared with placebo (HR=0.68 [95% CI, 0.53-0.87]; p=0.0010). The trial will continue to assess overall survival (OS) as a secondary outcome measure.

In KEYNOTE-564, the median duration of exposure to KEYTRUDA was 11.1 months (range, 1 day to 14.3 months). Serious adverse reactions occurred in 20% of these patients receiving KEYTRUDA. Serious adverse reactions (1%) were acute kidney injury, adrenal insufficiency, pneumonia, colitis and diabetic ketoacidosis (1% each). Fatal adverse reactions occurred in 0.2% of those treated with KEYTRUDA, including one case of pneumonia. Adverse reactions leading to discontinuation occurred in 21% of patients receiving KEYTRUDA; the most common (1%) were increased alanine aminotransferase (1.6%), colitis and adrenal insufficiency (1% each). The most common adverse reactions (all grades 20%) in the KEYTRUDA arm were musculoskeletal pain (41%), fatigue (40%), rash (30%), diarrhea (27%), pruritus (23%) and hypothyroidism (21%).

About KEYNOTE-564

KEYNOTE-564 (ClinicalTrials.gov, NCT03142334) is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial evaluating KEYTRUDA as adjuvant therapy for RCC in 994 patients with intermediate-high or high risk of recurrence of RCC or M1 no evidence of disease (NED). Patients must have undergone a partial nephroprotective or radical complete nephrectomy (and complete resection of solid, isolated, soft tissue metastatic lesion[s] in M1 NED participants) with negative surgical margins for at least four weeks prior to the time of screening. Patients were excluded from the trial if they had received prior systemic therapy for advanced RCC. Patients with active autoimmune disease or a medical condition that required immunosuppression were also ineligible. The major efficacy outcome measure was investigator-assessed DFS, defined as time to recurrence, metastasis or death. An additional outcome measure was OS. Patients were randomized (1:1) to receive KEYTRUDA 200 mg administered intravenously every three weeks or placebo for up to one year until disease recurrence or unacceptable toxicity.

About Renal Cell Carcinoma (RCC)

Renal cell carcinoma is by far the most common type of kidney cancer; about nine out of 10 kidney cancer diagnoses are RCCs. Renal cell carcinoma is about twice as common in men than in women. Most cases of RCC are discovered incidentally during imaging tests for other abdominal diseases. Worldwide, it is estimated there were more than 431,000 new cases of kidney cancer diagnosed and more than 179,000 deaths from the disease in 2020. In the U.S., it is estimated there will be more than 76,000 new cases of kidney cancer diagnosed and almost 14,000 deaths from the disease in 2021.

About Mercks Early-Stage Cancer Clinical Program

Finding cancer at an earlier stage may give patients a greater chance of long-term survival. Many cancers are considered most treatable and potentially curable in their earliest stage of disease. Building on the strong understanding of the role of KEYTRUDA in later-stage cancers, Merck is studying KEYTRUDA in earlier disease states, with approximately 20 ongoing registrational studies across multiple types of cancer.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,600 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications in the U.S.

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS 1)] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):

Non-muscle Invasive Bladder Cancer

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:

Cervical Cancer

KEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).

KEYTRUDA is indicated for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.

Tumor Mutational Burden-High Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.

Triple-Negative Breast Cancer

KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.

KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the PD-1 or the PD-L1, blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of antiPD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. For patients with TNBC treated with KEYTRUDA in the neoadjuvant setting, monitor blood cortisol at baseline, prior to surgery, and as clinically indicated. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA as a Single Agent

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA with Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT 3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT 3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT 3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism.

Type 1 Diabetes Mellitus (DM), Which Can Present With Diabetic Ketoacidosis

Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold KEYTRUDA depending on severity. Type 1 DM occurred in 0.2% (6/2799) of patients receiving KEYTRUDA. It led to permanent discontinuation in <0.1% (1) and withholding of KEYTRUDA in <0.1% (1) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Immune-Mediated Nephritis With Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.1%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 89% (8/9) of patients. Nephritis led to permanent discontinuation of KEYTRUDA in 0.1% (3) and withholding in 0.1% (3) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Nephritis resolved in 56% of the 9 patients.

Immune-Mediated Dermatologic Adverse Reactions

KEYTRUDA can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome, drug rash with eosinophilia and systemic symptoms, and toxic epidermal necrolysis, has occurred with antiPD-1/PD-L1 treatments. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes. Withhold or permanently discontinue KEYTRUDA depending on severity. Immune-mediated dermatologic adverse reactions occurred in 1.4% (38/2799) of patients receiving KEYTRUDA, including Grade 3 (1%) and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 40% (15/38) of patients. These reactions led to permanent discontinuation in 0.1% (2) and withholding of KEYTRUDA in 0.6% (16) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 6% had recurrence. The reactions resolved in 79% of the 38 patients.

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received KEYTRUDA or were reported with the use of other antiPD-1/PD-L1 treatments. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis; Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barr syndrome, nerve paresis, autoimmune neuropathy; Ocular: Uveitis, iritis and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: Pancreatitis, to include increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: Myositis/polymyositis, rhabdomyolysis (and associated sequelae, including renal failure), arthritis (1.5%), polymyalgia rheumatica; Endocrine: Hypoparathyroidism; Hematologic/Immune: Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% of 2799 patients receiving KEYTRUDA. Monitor for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion for Grade 1 or Grade 2 reactions. For Grade 3 or Grade 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

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FDA Approves Merck's KEYTRUDA (pembrolizumab) as Adjuvant Therapy for Certain Patients With Renal Cell Carcinoma (RCC) Following Surgery - Business...

The COVID-19 pandemic is what historians refer to as an "inflection point," ora singleevent that has a dramatic and sweeping effect on the human story. In the case of this particular event, itchanged the way we work, raised awareness about public health policy, contributed to the toppling of a president and, in the field of medicine, resulted in a leap forward for vaccine technology. Indeed,one of the great unsung achievements amid the pandemicwas how scientists from around the world worked together to create multipleeffective vaccines in less than a year.

Yet what may provemost historicisthe biotechnologythat emerged from the pandemic. Specifically,the vision of an mRNA vaccine went from dream to reality. And the successful creation of a viable mRNA vaccine couldhave repercussions for the way diseases are treated for centuries.

That technology, whose development was quickened by the pandemic, is already being studied to treatother diseases.Earlier this month, scientists at Yale Universitycreated a prototype mRNA vaccinethat protected guinea pigs from tick-borne diseases by training their immune systems to recognize and fight proteins found in tick saliva. They hope that, with some further development, this could be used to help humans avoid developing Lyme disease if a tick bites them.

Yet this is merely one example of mRNA vaccines'potential, revealing howthey have far more utility than merely fighting COVID-19. Indeed, mRNA vaccines are something of a holy grail of medical innovation and researchers believe thatmRNA vaccines and their underlying biotechnology could be used tofight diseases like HIV, cancer, and influenza.

The promise of mRNA vaccines

As their name suggests, mRNA vaccines depend on the nucleic acid known asRNA.RNA isa molecule similar to DNA, but it is single-stranded (DNA is double-stranded)and plays a large number of roles in keeping your cells alive and healthy. But don't think they are unique to humans: They are found in all living things. There are alsocertain types ofviruses like SARS-CoV-2, which causes COVID-19 that could be characterized as little more than RNA strands surrounded by protein shells. Like all viruses, theytake over cells and force them to churn out other copies of themselves, the worst kind of mooch you can imagine.

Yet RNA and mRNA are not precisely the same thing.mRNA refers to "messenger RNA," a specific type of RNA that (as indicated by its name) transmits information from genetic codes in the nucleus to the cytoplasm where proteins are manufactured.

This hints at how mRNA vaccines work, which is essentially by giving your cells a blueprint of a part of a virus, and then having them manufacture what they need on their own. Previously,vaccines contained either a dead or weakened version of a pathogen, which the immune system would then learn to recognize. But mRNA vaccines don't actually contain any of a live or dead virus; instead, they contain a set of instructions (in mRNA) that infects some of the host's cells and makes them spit out a piece of protein associated with a pathogen. One's cells never manufacture the actual virus; only a piece of its "shell," say. Those pieces are then detected by the immune system and identified and destroyed. It would be a bit like learning the presence of a criminal by identifying the look of their clothing, rather than the criminal themselves.

In the case of the mRNA vaccines manufactured by Pfizer and Moderna, the mRNA contains instructions for one's cells on how to create the spike protein. The spike proteins are the little points that emerge out of the coronavirus, like spines jutting from a sea urchin,and they are what the SARS-CoV-2 virus uses to enter your cells and get you sick with COVID-19.The mRNA vaccineshave been extraordinarily successful in protecting the vaccinated; even though they have not entirely thwartedbreakthrough cases, they significantly reduce the likelihood of getting sick, and the people who do develop infectionswith evasivemutant variants rarely become seriously ill. Most notably, mRNA vaccines were the first ones to be released on the market, with Pfizer/BioNTech and Moderna winning the vaccine race exactly one year ago this month.

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The next mRNA vaccines

In terms of their world-changing potential,mRNA vaccines have two key characteristics: They are quick to make, as demonstrated by the speed with which Pfizer/BioNTech and Moderna came out with their products, and by their very nature they are versatile.

As the Association of American Medical Colleges (AAMC) wrote in March, mRNA vaccine technology has the potential to treat diseases like malaria and cystic fibrosis, tuberculosis and hepatitis B. All scientists will have to do is modify RNA strands as needed to account for the different antigens (foreign substancesrecognized by the immune system as threats) produced by each pathogen.Instead of making do with the materials immediately available to them, mRNA vaccines make it possible for scientists to create more specialized weapons based on detailed knowledge of their enemies'specific characteristics.

Take the influenza epidemic. Oneso-called "holy grail"of immunology is a universal influenza vaccine. Right now there are four influenza viruses in circulation, all of which evolve so quickly that vaccines which were effective in one year may be obsolete by the next. This puts manufacturers in a crunch, as it takes at least six months to create the conventional vaccinationswith attenuated viruses grown inside chicken eggs. The final product, though almost always safe, has a very hit-and-miss rate ofeffectiveness. An mRNA vaccine, by contrast, could in theory be designed to effectively fight all four strains and be quickly modified as necessary when they evolve. In addition, while conventional vaccine platforms have to hit a precise target in order to destroy a givenintruder,an mRNA vaccine couldtargetmultiple parts of an influenza virus at once, overwhelming it with a full-body assault that can't be easily shaken off.

In fact, we already know that the early stages of mRNA flu vaccines were effective because scientists used that research to help develop their COVID-19 vaccines. This speaks to how malleable the platform is: While conventional vaccine platforms require patients to hope that the pathogen injected into their body is similar enough to a possible flu infection to be effective, mRNA vaccines could be precisely designed to meet the specific characteristics of each new strain as it emerges.

There will be challenges to pulling this off, of course. Anna Blakney, an RNA bioengineer at the University of British Columbia, told the journal Nature that there is no guarantee mRNA will be an effective vehicle for transportinghaemagglutinin glycoproteins, the protein that flu vaccines use to fight the different bugs. As Blakney put it,"Did we just get really incredibly lucky with COVID vaccines because of the antigen design and the immunodominancy of that protein? Or have we stumbled on something that's functional for other viral glycoproteins as well?"

In addition to aiding in the war against influenza, mRNA vaccines could also be a game-changer in the fight against cancer. In the pre-mRNA vaccine world, the mere notion of a "cancer vaccine" would have seemed ludicrous; vaccines work by protecting your body against a foreign invader, and cancers (as far as we know) are caused by your own body producing mutated cells. Yet just as an mRNA vaccine can help your immune system recognize and destroy proteins associated with dangerous pathogens, they could in theory be developed to identify and eliminate proteins associated with cancer cells and, of course, the cancer cells themselves.

"A successful therapeutic cancer vaccine should induce strong T cell responses, particularly with CD8+ T cells, which have a known capacity to kill malignant cells," Dr. Norbert Pardi, whose research led to the develop of the Pfizer and Moderna vaccines, explained to the University of Pennsylvania. "Therapeutic cancer vaccines would be given to cancer patients with the hope that those vaccine-induced cytotoxic T cells would clear tumor cells."

HIV mRNA vaccines are theorized to be possible, though there are massive hurdles to be overcome. The challenge so far has been that none of the vaccine candidates developed up to this point have produced broadly neutralizing antibodies, which are vital to blocking HIV in target cells. Scientists hope that an mRNA vaccine would create an immunogen (an antigen that induces an immune response) that resembles the HIV virus and can help the body develop those broadly neutralizing antibodies against it. Unfortunately, researchers are still very early in working through this, and it seems like a HIV vaccine using this technology is not in the near future.

"We certainly think that an HIV vaccine will be far and away the most complicated vaccine that we've ever had to put into the population," Derek Cain of Duke University's Human Vaccine Institute told The Guardian. "We don't expect it to work 100% or 90% like the Covid vaccines, but even if we can get to 50-60% that would be a success; 70% would be amazing."

What comes next

The future for mRNA technology is not one of unbounded promise. As the AAMC noted, each virus poses its own individual puzzle, which makes it unlikely that other ailments can be treated with the rapid success that occurred when fighting COVID-19. Similarly, although the COVID-19 vaccines have so far not caused widespread serious side effects, this may not be true for other mRNA vaccines; more research will definitely be needed. In addition, the COVID-19 pandemic was such an overwhelmingand serious crisis that the international community collaborated in fruitful ways that may not repeat themselves if a future outbreakseemsless urgent.

There are also logistical factors to take into consideration. The supply chain breakdowns prompted by the pandemic are poised to get worse due to climate change, and experts are already concerned that mRNA vaccines will get destroyed as they are transported because they must be kept in very clean and ultra-cold conditions. It is hard to imagine that the impendingsupply chain deteriorations won't exacerbate that problem, as will the ongoing disease of misinformation. Since anti-vaccine advocates can alter their baseless beliefs as easily as viruses change their genetic composition, some of this misinformation specifically targets mRNA platforms. One particularly prevalent myth right now is that mRNA vaccines change your DNA, even though (as the above explanation makes clear) this betrays a deep ignorance about how vaccines, viruses, cellular biology and the immune system actually work.

Finally, as with all biotechnology, governments and businesses will have to adequately invest.

"Despite the promise of mRNA vaccines, we caution that they are far from a silver bullet for future pandemics," Michael J. Hoganand Norbert Pardiwrite in anAnnual Review of Medicine article."Comprehensive pandemic preparedness requires significant new investments in viral surveillance, proactive clinical testing of vaccines for pandemic-potential viruses, new diagnostic technologies, broad-spectrum antiviral treatments, and stockpiling of materials."

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mRNA vaccines changed the course of the pandemic. Now, they could cure all kinds of other diseases - Salon

The new technique, which was developed at the Washington University School of Medicine in St Louis, was shown to convert human stem cells into cells producing insulin. The natural hormone is produced in the pancreas and allows the body to use glucose (sugar) from food for energy. People who suffer from diabetes struggle to produce enough insulin, which leads to a build-up of sugar in the bloodstream.

The St Louis researchers, however, believe their new technique can be used to effectively control blood sugar levels using converted stem cells.

The technique has so far been successfully tested on mice injected with the converted cells.

According to a report that is due to be published on February 24 in the online edition of the journal Nature Biotechnology, the mice were "functionally cured" for nine months.

Dr Jeffrey R. Millman, the principal investigator and assistant professor of medicine and of biomedical engineering, said: "These mice had very severe diabetes with blood sugar readings of more than 500 milligrams per deciliter of blood levels that could be fatal for a person and when we gave the mice the insulin-secreting cells, within two weeks their blood glucose levels had returned to normal and stayed that way for many months."

The same team of researchers has previously discovered how to convert human stem cells into so-called pancreatic beta cells to make insulin.

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When these cells are injected into the bloodstream, they secret the much-needed hormone.

However, the technique was found to have its limitations and was not proven to effectively control the disease in mice.

Their new research has now proven to be much more efficient and effective.

Embryonic stem cells are a type of cell that can be instructed to develop into all sorts of specialised cells.

These can range from simple tissue and muscle cells, to even brain cells.

Scientists worldwide believe stem cell research could unlock many new therapies for ailments such as Alzheimer's disease and HIV.

Dr Millman said: "A common problem when youre trying to transform a human stem cell into an insulin-producing beta cell or a neuron or a heart cell is that you also produce other cells that you dont want."

"In the case of beta cells, we might get other types of pancreas cells or liver cells."

Pancreas and liver cells do not cause any harm when injected into mice but they do not fight the disease either.

Dr Millman added: "The more off-target cells you get, the less therapeutically relevant cells you have.

"You need about a billion beta cells to cure a person of diabetes.

"But if a quarter of the cells you make are actually liver cells or other pancreas cells, instead of needing a billion cells, youll need 1.25 billion cells.

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"It makes curing the disease 25 percent more difficult."

With their new technique, the researchers found fewer off-target cells were produced and the beta cells that were created had improved.

The technique specifically targets the cell's so-called internal scaffolding or cytoskeleton.

The cytoskeleton is what gives cells their shape and allows them to interact with their environment.

Dr Millman said: "Its a completely different approach, fundamentally different in the way we go about it.

"Previously, we would identify various proteins and factors and sprinkle them on the cells to see what would happen.

"As we have better understood the signals, weve been able to make that process less random."

Although the study's results are promising, the expert added there is a long way to go before the technique can be developed into a treatment for humans.

The converted cells will need to be tested over longer periods of time and in bigger animals.

According to Diabetes UK, some 5.5 million people are estimated to have diabetes in the UK by 2030.

Right now, more than 4.9 million people are affected by the disease and 13.6 million people are at increased risk of type 2 diabetes.

About 90 percent of people with the disease have type 2 diabetes, and only about eight percent have type 1 diabetes.

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Diabetes breakthrough: Revolutionary stem cell technique treated 'severe' disease in study - Daily Express