Category Archives: Cell Medicine

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted Global Blood Therapeutics (GBT) a British marketing authorisation for Oxbryta also known as voxelotor for both adult and paediatric patients 12 years of age and older requiring treatment of haemolytic anaemia due to sickle cell disease (SCD).

The authorisation supports the use of Oxbryta as either a monotherapy or for use in combination with hydroxycarbamide, also known as hydroxyurea.

Oxbryta is an oral treatment taken once daily and is the first medicine authorised in Britain that directly blocks sickle haemoglobin (HbS) polymerisation, which is the molecular foundation of sickling and the destruction of red blood cells in SCD.

In the UK, approximately 15,000 people are affected by SCD, a progressive and complex condition which can cause serious complications, including organ damage. For those living with the condition, it is common to experience economic disadvantages and health inequalities as SCD can inflict negative societal impacts in areas like healthcare, education and employment.

Beginning in early childhood, SCD complications can include neurocognitive impairment, acute chest syndrome and silent and overt stroke. It has been shown that early intervention and treatment of SCD can potentially change the course of this disease and, in turn, reduce symptoms and events while preventing long-term organ damage and extending life expectancy.

The marketing authorisation from the MHRA follows the European Commission (EC) authorisation which was made earlier this year and is based on data taken from the phase 3 HOPE trial. The results demonstrated clinically meaningful and statistically significant improvements in haemoglobin (Hb) levels, accompanied by a reduction of haemolysis markers, for patients treated with Oxbryta.

In 2021, Oxbryta was the first SCD treatment to be granted a Promising Innovative Medicine (PIM) designation from the MHRA, which then allowed the medicine to receive a positive scientific opinion under the Early Access to Medicines Scheme (EAMS). Healthcare professionals were then able to treat selected patients with Oxbryta ahead of market authorisation, based on clinical factors to address a clear unmet medical need.

Dr Beatriz Pujol, vice president, head of medical affairs EU & GCC at GBT, said: Following this marketing authorisation by the MHRA, we look forward to working with the National Institute of Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) with the goal of helping to facilitate rapid access to voxelotor for people living with sickle cell disease who may benefit from this important treatment.

Link:
MHRA grants marketing authorisation to Global Blood Therapeutics for sickle cell disease drug - PMLiVE

Ten years ago, Stephan Grupp, MD, PhD, plunged into an unexplored area of pediatric cancer treatment with a 6-year-old patient for whom every treatment available for her acute lymphoblastic leukemia (ALL) had been exhausted.

Dr Stephan Grupp

Grupp, a pioneer in cellular immunotherapy at Children's Hospital of Philadelphia (CHOP), had just got the green light to launch the first phase 1 trial of chimeric antigen receptor (CAR) T-cell therapy for children.

"The trial opened at the absolute last possible moment that it could have been helpful to her," he told Medscape Medical News. "There was nothing else to do to temporize her further.... It had to open then or never."

The patient was Emily Whitehead, who has since become a poster girl for the dramatic results that can be achieved with these novel therapies. After that one CAR T-cell treatment back in 2012, she has been free of her leukemia and has remained in remission for more than 10 years.

Grupp says that he is, at last, starting to use the "cure" word.

"I'm not just a doctor, I'm a scientist and one case isn't enough to have confidence about anything," he said. "We wanted more patients to be out longer to be able to say that thing which we have for a long time called the 'c word.'

"CAR T-cell therapy has now been given to hundreds of patients at CHOP, and we are unique in this we have a couple dozen patients who are 5, 6, 7, 9 years out or more without further therapy. That feels like a cure to me," he commented.

Emily was the first patient with ALL to receive the novel treatment, and also the first child.

There wasaprecedent, however. After having been "stuck" for decades, the CAR T-cell field had recently made a breakthrough, thanks to research by Grupp's colleague Carl June and his team at the University of Pennsylvania. By tweaking two key steps in the genetic modification of T cells, June's team had successfully treated three adults with chronic lymphocytic leukemia (CLL), two of whom were in complete remission.

But using the treatment for a child and for a different type of leukemia was a daunting prospect. Grupp recalls that he was candidwith Emily's parents, Tom and Kari Whitehead, emphasizing that there are no guarantees in cancer treatment, particularly in a phase 1 trial.

But the Whiteheads had no time to waste and nowhere else to turn. Her father, Tom, recalls saying: "This is something outside the box, this is going to give her a chance."

Grupp, who describes himself as being "on the cowboy end" of oncology care, was ready to take the plunge.

Little did any of them know that the treatment would make Emily even sicker than she already was, putting her in intensive care. But thanks to a combination of several lucky breaks and a lot of brain power, she would make a breathtakingly rapid recovery.

CAR T-cell therapy involves harvesting a patient's T cells and modifying them in the lab with a chimeric antigen receptor to target CD19, a protein found on the surface of ALL cancer cells.

Before the University of Pennsylvania team tweaked the process, clinical trials of the therapy yielded only modest results because the modified T cells "were very powerful in the short term but had almost no proliferative capacity" once they were infused back into the patient, Grupp explained.

"It does not matter how many cells you give to a patient, what matters is that the cells grow in the patient to the level needed to control the leukemia," he said.

June's team came up with what Grupp calls "the magic formula": a bead-based manufacturing process that produced younger T-cell phenotypes with "enormous" proliferative capacity, and a lentiviral approach to the genetic modification, enabling prolonged expression of the CAR-T molecule.

"Was it rogue? Absolutely, positively not," said Grupp, thinking back to the day he enrolled Emily in the trial. "Was it risky? Obviously...we all dived into this pool without knowing what was under the water, so I would say, rogue, no, risky, yes. And I would say we didn't know nearly enough about the risks."

The gravest risk that Grupp and his team encountered was something they had not anticipated. At the time, they had no name for it.

The three adults with CLL who had received CAR T-cell therapy had experienced a mild version that the researchers referred to as "tumor lysis syndrome" (N Engl J Med. 2011;365:725-33).

But for Emily, on day 3 of her CAR T-cell infusion, there was a ferocious reaction storm that later came to be called cytokine release syndrome (CRS).

"The wheels just came off then," said Tom. "I remember her blood pressure was 53 over 29. They took her to the ICU, induced a coma, and put her on a ventilator. It was brutal to watch. The oscillatory ventilator just pounds on you, and there was blood bubbling out around the hose in her mouth.

"I remember the third or fourth night, a doctor took me in the hallway and said, 'There's a one-in-a-thousand chance your daughter is alive when the sun comes up,' " Tom told Medscape Medical News. "And I said, 'Alright, I'll see you at rounds tomorrow, because she'll still be here.' "

"We had some vague notion of toxicity...but it turned out not nearly enough," said Grupp. The ICU "worked flat out" to save her life, he recalls. "They had deployed everything they had to keep a human being alive and they had nothing more to add. At some point, you run out of things that you can do, and we had run out."

It was then that the team ran into some good luck. The first break was when they decided to look at her cytokines. "Our whole knowledge base came together in the moment, on the fly, at the exact moment when Emily was so very sick," he recalls. "Could we get the result fast enough? The lab dropped everything to run the test."

They ordered a broad cytokine panel that included 30 analytes. The results showed that a number of cytokines "were just unbelievably elevated," he said. Among them was interleukin-6 (IL-6).

"IL-6 isn't even made by T cells, so nobody in the world would have guessed that this would have mattered. If we'd ordered a smaller panel, it might not even have been on it. Yet this was the one cytokine we had a drug for tocilizumab so that was chance. And then, another chance was that the drug was at the hospital, because there are rheumatology patients who get it.

"So, we went from making the determination that IL-6 was high and figuring out there was a drug for it at 3:00 o'clock to giving the drug to her at 8:00 o'clock, and then her clinical situation turned around so quickly I mean hours later."

Emily woke up from a 14-day medically induced coma on her seventh birthday.

Eight days later, her bone marrow showed complete remission. "The doctors said, 'We've never seen anyone this sick get better any faster,' " said Tom.

She had already been through a battery of treatments for her leukemia. "It was 22 months of failed, standard treatment, and then just 23 days after they gave her the first dose of CAR T-cells that she was cancer free," he added.

Now that Emily, 17, has remained in remission for 10 years, Grupp is finally willing to use the word "cure" but it has taken him a long time.

Now, he says, the challenge from the bedside is to keep parents' and patients' expectations realistic about what they see as a miracle cure.

"It's not a miracle. We can get patients into remission 90-plus percent of the time but some patients do relapse and then there are the risks [of the cytokine storm, which can be life-threatening].

"Right now, our experience is that about 12% of patients end up in the ICU, but they hardly ever end up as sick as Emily...because now we're giving the tocilizumab much earlier," Grupp said.

Since their daughter's recovery, Tom and Kari Whitehead have dedicated much of their time to spreading the word about the treatment that saved Emily's life. Tom testified at the US Food and Drug Administration's advisory committee meeting in 2017 when approval was being considered for the CAR T-cell product that Emily received. The product was tisagenlecleucel-T (Novartis); at that meeting, there was a unanimous vote to recommend approval. This was the first CAR T cell to reach the market.

As co-founders of the Emily Whitehead Foundation, Tom and Karis have helped raise more than $2 million to support research in the field, and they travel around the world telling their story to "move this revolution forward."

Despite their fierce belief in the science that saved Emily, they also acknowledge there was luck and faith. Early in their journey, when Emily experienced relapse after her initial treatments, Tom drew comfort from two visions, which he calls "whispers," that guided them through several forks in the road and through tough decisions about Emily's treatment.

Several times he and Kari refused treatment that was offered to Emily, and once they had her discharged against medical advice. "I told Kari she's definitely going to beat her cancer I saw it. I don't know how it's going to happen, but we're going to be in the bone marrow transplant hallway [at CHOP] teaching her to walk again. I know a lot of doctors don't want to hear anything about 'a sign,' or what guided us, but I don't think you have to separate faith and science, I think it takes everything to make something like this to happen."

The key to the CAR T-cell breakthrough that gave rise to Emily's therapy was cell proliferation, and the effect is enduring, beyond all expectations, said Grupp. The modified T cells are still detectable in Emily and other patients in long-term remission.

"The fundamental question is, are the cells still working, or are the patients cured and they don't need them?" said Grupp. "I think it's the latter. The data that we have from several large datasets that we developed with Novartis are that if you get to a year and your minimal residual disease testing both by flow and by next-generation sequencing is negative and you still have B-cell aplasia, the relapse risk is close to zero at that point."

While it's still not clear if and when that risk will ever get to zero, Emily and Grupp have successfully closed the chapter.

"Oncologists have different notions of what the word 'cure' means. If your attitude is you're not cured until you've basically reached the end of your life and you haven't relapsed, well, that's an impossible bar to hit. My attitude is, if your likelihood of having a disease recurrence is lower than the other risks in your life, like getting into your car and driving to your appointment, then that's what a functional cure looks like," he said.

"I'm probably the doctor that still sees her the most, but honestly, the whole conversation is not about leukemia at all. She has B-cell aplasia, so we have to treat that, and then it's about making sure there's no long-term side effects from the totality of her treatment. Generally, for a patient who's gotten a moderate amount of chemotherapy and CAR T, that should not interfere with fertility. Has any patient in the history of the world ever relapsed more than 5 years out from their therapy? Of course. Is that incredibly rare? Yes, it is. You can be paralyzed by that, or you can compartmentalize it."

Tom, Emily, and Kate Whitehead

As for the Whiteheads, they are focused on Emily's college applications, her new driver's license, and her project to co-write a film about her story with a Hollywood filmmaker.

Tom says the one thing he hopes clinicians take away from their story is that sometimes a parent's instinct transcends science.

Kate Johnson is a Montreal-based freelance medical journalist who has been writing for more than 30 years about all areas of medicine.

For more news, follow Medscape on Facebook, Twitter, Instagram, and YouTube.

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CAR T-Cell Therapy Turns 10 and Finally Earns the Word 'Cure' - Medscape

Kira MacDougall, MD, and Muhammad Rafay Khan Niazi, MD, spoke with CancerNetwork about their research into the significance of peripheral blood biomarkers of response to immunotherapy in nonsmall cell lung cancer published in the journal ONCOLOGY.

Kira MacDougall, MD, a first year fellow at the University of Oklahoma, and Muhammad Rafay Khan Niazi, MD, a third year resident of Internal Medicine at Staten Island University Hospital, spoke with CancerNetwork about research published in the journal ONCOLOGY titled, The Prognostic Significance of Peripheral Blood Biomarkers in Patients With Advanced NonSmall Cell Lung Cancer Treated With Pembrolizumab: A Clinical Study.

MacDougall and Niazi discuss the clinical utility of absolute lymphocyte count (ALC) and the ratio of absolute neutrophil count to ALC for predicting outcomes with pembrolizumab (Keytruda) in advanced nonsmall cell lung cancer. They also talked about future research in the space and what unanswered questions remain in this treatment setting.

Dont forget to subscribe to the Oncology Peer Review On-The-Go podcast on Apple Podcasts, Spotify, or anywhere podcasts are available.

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Oncology Peer Review On-The-Go: The Prognostic Significance of Peripheral Blood Biomarkers in Patients With Advanced NonSmall Cell Lung Cancer Treated...

image:Chromosome segregation In dividing cells. Cell cytoskeleton is depicted in red, DNA is depicted in blue and a protein that marks dividing cells is depicted in green. view more

Credit: Tom Winkler, Ben David lab.

The researchers caution: "The CRISPR genome editing method is very effective, but not always safe. Sometimes cleaved chromosomes do not recover and genomic stability is compromised which in the long run might promote cancer."

A new study from TAU identifies risks in the use of CRISPR therapeutics an innovative, Nobel-prize-winning method that involves cleaving and editing DNA, already employed for the treatment of conditions like cancer, liver and intestinal diseases, and genetic syndromes. Investigating the impact of this technology on T-cells white blood cells of the immune system, the researchers detected a loss of genetic material in a significant percentage up to 10% of the treated cells. They explain that such loss can lead to destabilization of the genome, which might cause cancer.

The study was led by Dr. Adi Barzel from the School of Neurobiology, Biochemistry and Biophysics at TAU's Wise Faculty of Life Sciences and Dotan Center for Advanced Therapies, a collaboration between the Tel Aviv Sourasky Medical Center (Ichilov) and Tel Aviv University, and by Dr. Asaf Madi and Dr. Uri Ben-David from TAU's Faculty of Medicine and Edmond J. Safra Center for Bioinformatics. The findings were published in the leading scientific journal Nature Biotechnology.

The researchers explain that CRISPR is a groundbreaking technology for editing DNA cleaving DNA sequences at certain locations in order to delete unwanted segments, or alternately repair or insert beneficial segments. Developed about a decade ago, the technology has already proved impressively effective in treating a range of diseases cancer, liver diseases, genetic syndromes, and more. The first approved clinical trial ever to use CRISPR, was conducted in 2020 at the University of Pennsylvania, when researchers applied the method to T-cells white blood cells of the immune system. Taking T-cells from a donor, they expressed an engineered receptor targeting cancer cells, while using CRISPR to destroy genes coding for the original receptor which otherwise might have caused the T-cells to attack cells in the recipient's body.

In the present study, the researchers sought to examine whether the potential benefits of CRISPR therapeutics might be offset by risks resulting from the cleavage itself, assuming that broken DNA is not always able to recover.

Dr. Ben-David and his research associate Eli Reuveni explain: "The genome in our cells often breaks due to natural causes, but usually it is able to repair itself, with no harm done. Still, sometimes a certain chromosome is unable to bounce back, and large sections, or even the entire chromosome, are lost. Such chromosomal disruptions can destabilize the genome, and we often see this in cancer cells. Thus, CRISPR therapeutics, in which DNA is cleaved intentionally as a means for treating cancer, might, in extreme scenarios, actually promote malignancies."

To examine the extent of potential damage, the researchers repeated the 2020 Pennsylvania experiment, cleaving the T-cells' genome in exactly the same locations chromosomes 2, 7, and 14 (of the human genome's 23 pairs of chromosomes). Using a state-of-the-art technology called single-cell RNA sequencing they analyzed each cell separately and measured the expression levels of each chromosome in every cell.

In this way, a significant loss of genetic material was detected in some of the cells. For example, when Chromosome 14 had been cleaved, about 5% of the cells showed little or no expression of this chromosome. When all chromosomes were cleaved simultaneously, the damage increased, with 9%, 10%, and 3% of the cells unable to repair the break in chromosomes 14, 7, and 2 respectively. The three chromosomes did differ, however, in the extent of the damage they sustained.

Dr. Madi and his student Ella Goldschmidt explain: "Single-cell RNA sequencing and computational analyses enabled us to obtain very precise results. We found that the cause for the difference in damage was the exact place of the cleaving on each of the three chromosomes. Altogether, our findings indicate that over 9% of the T-cells genetically edited with the CRISPR technique had lost a significant amount of genetic material. Such loss can lead to destabilization of the genome, which might promote cancer."

Based on their findings, the researchers caution that extra care should be taken when using CRISPR therapeutics. They also propose alternative, less risky, methods, for specific medical procedures, and recommend further research into two kinds of potential solutions: reducing the production of damaged cells or identifying damaged cells and removing them before the material is administered to the patient.

Dr. Barzel and his PhD student Alessio Nahmad conclude: "Our intention in this study was to shed light on potential risks in the use of CRISPR therapeutics. We did this even though we are aware of the technology's substantial advantages. In fact, in other studies we have developed CRISPR-based treatments, including a promising therapy for AIDS. We have even established two companies one using CRISPR and the other deliberately avoiding this technology. In other words, we advance this highly effective technology, while at the same time cautioning against its potential dangers. This may seem like a contradiction, but as scientists we are quite proud of our approach, because we believe that this is the very essence of science: we don't 'choose sides.' We examine all aspects of an issue, both positive and negative, and look for answers."

Link to the article:

https://www.nature.com/articles/s41587-022-01377-0

Nature Biotechnology

Frequent aneuploidy in primary human T cells after CRISPRCas9 cleavage

30-Jun-2022

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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CRISPR therapeutics can damage the genome - EurekAlert

image:Drug B binds to the surface of the cell and is transported to the Golgi apparatus. The drug contains an endoplasmic reticulum (ER)-specific sequence to further guide its transport to the ER. Transport to the ER is stopped if the drug bears N-glycosylation Y. view more

Credit: Ayano Satoh from Okayama University

Proteins usually undergo modifications during or after their synthesis in the endoplasmic reticulum (ER) and Golgi apparatus network inside eukaryotic cells. One such modification is glycosylation, whereby sugars, such as glycans, are added to newly synthesized proteins. Glycans allow proteins to fold properly, in turn making them stable and biologically active for various cell processes. However, the exact mechanism of glycosylation in the ER and Golgi are still not known. One way to study the process of glycosylation during protein synthesis is to deliver synthetic proteins to specific cell organelles and observe their subcellular dynamics. But this is often hindered by the lack of specific delivery methods to organelles like the ER and Golgi.

To this end, Dr. Ayano Satoh from Okayama University and Dr. Yuta Maki, Kazuki Kawata, Dr. Yanbo Liu, Kang-Ying Goo, Dr. Ryo Okamoto, and Prof.Dr. Yasuhiro Kajihara from Osaka University, Japan investigated the feasibility of modifying cholera toxin (CT) for targeted delivery to the ER and Golgi. CT is a protein produced by the bacterium Vibrio cholerae and is responsible for the hallmark symptoms of diarrhearepeated loose, watery stools. The toxin is made up of two subunits: CTA, which causes diarrhea, and CTB, which helps the toxin enter cells. CT enters the cell through the membrane into small cellular vehicles called endosomes that deliver it to the Golgi bodies. From there, an ER-specific amino acid sequence of CTA takes CT into the ER, where the toxin springs into action to cause diarrhea. CT is a protein that naturally gets delivered specifically to the Golgi and ER. This made it an attractive candidate for our investigation, says Dr. Satoh, explaining the reason behind selecting this protein for their study, which was first published on May 23, 2022, in Chemistry A European Journal.

The team synthesized an artificial, glycosylated form of the non-toxic CTB and tracked its intracellular journey using the HiBiT bioluminescence system engineered from the luciferase enzyme. In the system that the team used, the larger fragment of luciferase was added to particular receptors on the ER and Golgi. CTB was tagged with the smaller fragment of luciferase. The system works by emitting light when the two fragments bind to each other. Thus, the team tracked the artificial CTBs movement through the organelles in real time by checking for the emittance of light. Talking about the highlights of their study, Dr. Satoh says, We designed and chemically synthesized the glycosyl-CTB and demonstrated its trafficking into the ER and Golgi of living cells. We also established a method to quantitatively monitor the trafficking of CTB to these organelles.

The successful monitoring and delivery of the artificial CTB may pave the way for a new phase of research in understanding protein modification in compartments of living cells. The team emphasizes that their method of preparing CTB allows for developing various mutant forms of the protein as well as CTB bearing different glycans on its surface to help investigate the functions of N-glycan in cells.

Not only the study of glycans but CTB-mediated delivery can also be a promising tool for target-specific drug delivery in cells and organelles. Dr. Satoh observes, Our system for targeting specific organelles may help treat diseases caused by the absence of enzymes localized in specific organelles.

What is her vision for the future? Current drug delivery techniques are limited because they only target the cell surface. Our system may extend the limits of current technology and enable the delivery of drug wherever it is needed, says a hopeful Dr. Satoh.

We have our fingers crossed for her vision to come true and revolutionize the field of medicine!

About Okayama University, Japan

As one of the leading universities in Japan, Okayama University aims to create and establish a new paradigm for the sustainable development of the world. Okayama University offers a wide range of academic fields, which become the basis of the integrated graduate schools. This not only allows us to conduct the most advanced and up-to-date research, but also provides an enriching educational experience.

Website: https://www.okayama-u.ac.jp/index_e.html

About Professor Ayano Satoh from Okayama University, Japan

Dr. Ayano Satoh is an Associate Professor of the Graduate School of Interdisciplinary Science and Engineering in Health Systems and the Faculty of Engineering at the Okayama University, Okayama, Japan. She obtained her Bachelors and Masters degrees in Chemistry from Ochanomizu University, Tokyo, Japan. She then received her PhD at the Graduate School of Humanities and Sciences, Ochanomizu University, where she worked on affinity interactions among lipids, glycans, and proteins with Dr. Isamu Matsumoto. Before joining Okayama University, she worked with Dr. Graham Warren at Yale University, first as a Postdoctoral Researcher, and then as a Research Scientist. At Yale University, she worked on transport within the Golgi apparatus.

Chemistry - A European Journal

Experimental study

Cells

Design and Synthesis of Glycosylated Cholera Toxin B Subunit as a Tracer of Glycoprotein Trafficking in Organelles of Living Cells

23-May-2022

The authors declare no conflict of interest.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

The rest is here:
To cell surface and beyond: Tracing subcellular glycoprotein transport using modified cholera toxin - EurekAlert

Event Date:July 27, 2022Time:11:00 am 3:00 pm ET

Advanced cell therapies continue to be a fantastic success story for both the biotech and life sciences industries, but most importantly for patients who have witnessed first-hand the lifesaving potential of these biotherapeutics. In addition, cell therapies are at the vanguard of many precision medicine initiatives, from off-the-shelf products designed to treat a broad range of patients, to personalized therapeutics that have been engineered to compliment patients genetics. Yet, with all their success comes the challenges that researchers face dailydonor access, scaling manufacturing operations, and safety, to name a few. Understanding the current cell therapy landscape and incorporating the lessons learned from previous successes and failures will allow the industry to keep pushing the limits of whats possible scientifically while helping speed up new safe, efficacious therapies to market.

ThisGENSummit has amassed an extraordinary selection of thought leaders to discuss the latest trends, newest technologies, and practical solutions to common challenges that face organizations devoting their time to cell therapy research. Our summit will kick off with an exciting keynote address fromWilliam Ho,President, CEO, and Co-Founder of IN8bio, which is focused on delivering a novel off-the-shelf cell therapy for the treatment of cancer. Together, during this half-day conference, we will discover the current state of the cell therapy space and hear about the amazing potential that many novel cell therapies have to truly impact patients lives.

Register now and reserve your spot for what is sure to be an exciting and immensely informative event!

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From Donor to Patient: Advancing the Future of Cell Therapies - Genetic Engineering & Biotechnology News

An ad hoc committee has been appointed to undertake a routine review of the Biomedical Research Imaging Center and the leadership of its Director, Weili Lin, PhD, Dixie Lee Boney Soo Distinguished Professor of Neurological Medicine. The review is a standard procedure of the University of North Carolina at Chapel Hill and will take place on September 13.

The review committee invites your participation and input:

The deadline to request time on the review committee agenda, or to share written comments, is Sept. 2, 2022.

Note that North Carolina law requires that any written materials developed or received by the committee during the review may be made available to the person reviewed upon request.

All requests from the person reviewed will be handled by the Legal Department and any identifying information will be redacted prior to release of the material.

Members of the Review Committee

Henrik Dohlman, PhD Review Committee Chair, Distinguished Professor, Pharmacology

Jan Busby-Whitehead, MD Distinguished Professor,Geriatric Medicine

Leon Coleman, MD,PhD Assistant Professor, Pharmacology

Felicia Williams, MD,FACS Associate Professor, Surgery, Burn Center

Mark Shen, PhD Assistant Professor, Psychiatry, Carolina Institute for Developmental Disabilities

Benjamin Philpot, PhD Distinguished Professor, Cell Biology and Physiology

Dirk Dittmer, PhD-Professor, Microbiology and Immunology

Joyce Besheer, PhD Professor, Psychiatry, Bowles Center for Alcohol Studies

Morika Williams, DVM, PhD, DACLAM Assistant Professor, Pathology and Lab Medicine

Aysenil Belger, PhDProfessor, Psychiatry

Original post:
Five-Year Review of Biomedical Research Imaging Center, Center Director | Newsroom - UNC Health and UNC School of Medicine

Patients with high-grade ovarian cancer and uterine serous cancer (USC) often respond well to surgery and chemotherapy. At first.

But these can be highly aggressive tumors that often spread into the space within the abdomen known as the peritoneal cavity. According to a recent study, one rare but aggressive type of uterine cancer is propelling an increase in deaths from the disease in the United States, particularly among Black women.

Moreover, resistance to chemotherapy often develops, and the disease recurs. This results in ovarian cancer causing more deaths than any other cancer of the female reproductive system.

For one possible treatment, clinical trials demonstrated the effectiveness of injecting a drug known as epothilone B (EB) into the abdominal cavity, targeting tumor cells that have grown resistant to standard chemotherapy medications. However, the drugs high toxicity when delivered this way causes severe side effects, preventing further use.

Now, thanks in part to research begun more than a decade ago with funding from Womens Health Research at Yale, our colleagues are closing in on a way to deploy effective cancer-fighting medication safely with the help of ultra-tiny non-toxic biodegradable objects known as nanoparticles. Developed by Dr. W. Mark Saltzman, the Goizueta Foundation Professor of Biomedical and Chemical Engineering, these nanoparticles have organic chemicals on their surface that allow them to stick to cells in the abdominal cavity so they are not cleared from the area before they can do their job.

With bioadhesive nanoparticles, we can safely entrap a drug and deliver it so it slowly releases in a high concentration, directly to our target, over a long time, Saltzman said. By localizing the delivery of the drug, we are decreasing toxicity and increasing effectiveness.

With data funded through WHRYs grant, Drs. Saltzman and Alessandro Santin, professor of obstetrics, gynecology, and reproductive sciences, secured funding from the National Institutes of Health to demonstrate the safety and efficacy of this technique in a model system, publishing their results in 2016.

Saltzman then partnered with Dr. Michael Girardi, Evans Professor of Dermatology, to develop a non-surgical treatment for skin cancer using injections of nanoparticles carrying a chemotherapy agent. In a paper published last year, they demonstrated the capacity for this method to bind to the tumors and kill a significant number of cancer cells. In addition, the treatment involves triggering an immune response to rid the body of cancer cell waste and respond against any remaining cancer cells.

Drs. Saltzman and Girardi founded a company called Stradefy Biosciences, which has licensed patents to this technology from Yale, while continuing to develop these techniques for clinical use.Dr. Nita Ahuja, William H. Carmalt Professor of Surgery and chair of surgery, serves as an advisor for abdominal cancer applications.

We are thrilled that the work we sponsored many years ago continues to produce such varied applications for serious health concerns, said WHRY Director Carolyn M. Mazure, PhD. This is the model for how investing in Yales most innovative and collaborative individuals can produce steady progress that will improve and even save lives.

Dr. Saltzman also used a WHRY grant to create a vaginal ring that provides contraception while protecting against sexually transmitted infections. Yale has filed a patent application on this unique ring design, and Saltzman continues to seek funding to further develop the product and possibly adapt it to treat endometriosis.

The type of funding WHRY provides is critical for the innovation-based work I do, Saltzman said. I could say, We are going to make these particles with this unique property. But to get substantial buy-in from a company or the NIH, you need to have the data to demonstrate that this works. Early funding, particularly for collaborative projects with unproven technologies, is critical.

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Using Particles That Are Smaller Than the Head of a Pin to Treat Cancer - Yale School of Medicine

DUBLIN--(BUSINESS WIRE)--The "Stem Cell Assays Market by Type (Viability, Proliferation, Differentiation, Apoptosis), Cell Type (Mesenchymal, iPSCs, HSCs, hESCs), Product & Service (Instrument), Application (Regenerative Medicine, Clinical Research), End User - Global Forecast to 2027" report has been added to ResearchAndMarkets.com's offering.

The stem cell assay market is projected to reach USD 4.5 Billion by 2027 from USD 1.9 Billion in 2022, at a CAGR of 17.7% during the forecast period.

The growth of the market is projected to be driven by collaborations and agreements among market players for stem cell assay products & services, the launch of new stem cell analysis systems such as flow cytometers, and increase in R&D expenditure by biopharmaceutical and biotechnology companies.

The viability/cytotoxicity assays accounted for the largest share of the type segment in the stem cell assays market in 2021

Cell viability assays help to determine the number of live and dead cells in a culture medium. The viability/cytotoxicity assays include various types such as tetrazolium reduction assays, resazurin cell viability assays, calcein-AM cell viability assays, and other viability/cytotoxicity assays. The cell viability/cytotoxicity market is likely to be driven by rising R&D spending on stem cell research, an increase in demand for stem cell assays in drug discovery, and development of new stem cell therapies..

The adult stem cells segment accounted for the largest share of the cell type segment in the stem cell assays market in 2021.

Adult stem cells account for the largest share of the stem cell assay market. The adult stem cells include mesenchymal stem cells, induced pluripotent stem cells, hematopoietic stem cells, umbilical cord stem cells, and neural stem cells. The growth of the adult stems cells segment is driven by the increasing usage of adult stem cells in regenerative medicine and the development of advanced therapies.

Asia Pacific: The fastest-growing region in the stem cell assays market

The Asia Pacific is estimated to be the fastest-growing segment of the market, owing to the rising prevalence of cancer & other diseases, increasing R&D spending on biopharmaceutical projects, and focus on developing stem cell-based therapies. In this region, China and Japan are the largest markets.

Key Topics Covered:

1 Introduction

2 Research Methodology

3 Executive Summary

4 Premium Insights

4.1 Stem Cell Assays Market Overview

4.2 North America: Stem Cell Assays Market, by Product & Service and Country (2021)

4.3 Stem Cell Assays Market Share, by Type, 2022 Vs. 2027

4.4 Stem Cell Assays Market Share, by Application, 2021

4.5 Stem Cell Assays Market: Geographic Growth Opportunities

5 Market Overview

5.1 Introduction

5.2 Market Dynamics

5.2.1 Drivers

5.2.1.1 Increasing Awareness About Therapeutic Potency of Stem Cells

5.2.1.2 Increasing Funding for Stem Cell Research

5.2.1.3 Rising Demand for Cell-Based Assays in Drug Discovery

5.2.1.4 Collaborations and Agreements Among Market Players for Stem Cell Assay Products & Services

5.2.1.5 Rising Incidence of Cancer

5.2.2 Restraints

5.2.2.1 Issues in Embryonic Stem Cell Research

5.2.2.2 High Cost of Stem Cell Analysis Instruments

5.2.3 Opportunities

5.2.3.1 Emerging Economies

5.2.3.2 Government Initiatives to Boost Stem Cell Research

5.2.4 Challenges

5.2.4.1 Lack of Infrastructure for Stem Cell Research in Emerging Economies

5.2.4.2 Dearth of Trained and Skilled Professionals

5.3 Ranges/Scenarios

5.4 Impact of COVID-19 on Stem Cell Assays Market

5.5 Trends/Disruptions Impacting Customers' Business

5.6 Pricing Analysis

5.6.1 Average Selling Prices of Products Offered by Key Players

5.6.2 Average Selling Price Trend

5.7 Technology Analysis

6 Stem Cell Assays Market, by Type

6.1 Introduction

6.2 Viability/Cytotoxicity Assays

6.3 Isolation & Purification Assays

6.4 Cell Identification Assays

6.5 Proliferation Assays

6.6 Differentiation Assays

6.7 Function Assays

6.8 Apoptosis Assays

7 Stem Cell Assays Market, by Cell Type

7.1 Introduction

7.2 Adult Stem Cells

7.3 Human Embryonic Stem Cells

8 Stem Cell Assays Market, by Product & Service

8.1 Introduction

8.2 Instruments

8.3 Kits

8.4 Services

9 Stem Cell Assays Market, by Application

9.1 Introduction

9.2 Regenerative Medicine & Therapy Development

9.3 Drug Discovery & Development

9.4 Clinical Research

10 Stem Cell Assays Market, by End-User

11 Stem Cell Assays Market, by Region

12 Competitive Landscape

13 Company Profiles

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/hvgp4o

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Stem Cell Assays Market Report 2022-2027: Increasing Awareness About Therapeutic Potency of Stem Cells Driving Growth - ResearchAndMarkets.com -...

A common plant from west Africa works as a treatment for sickle cell disease, new research has found.

Scientists at Aberystwyth University isolated a chemical in the Alchornea cordifolia plant, also known as the Christmas Bush, which could help relieve the symptoms of the life-threatening and painful disease suffered by 15,000 people in the UK.

In sufferers of sickle cell anaemia, red blood cells change from their usual soft disc shape to a curved "sickle" shape and also become sticky and hard, which means they don't move properly around the body.

Blood is blocked from flowing, particularly to joints, the chest and abdomen, leading to severe pain, swelling of hands and feet, delayed growth and problems with eyesight, among other symptoms. It can lead to stillbirth and is also fatal in some cases.

A genetic disease, it is more common among people of African, Mediterranean and Middle Eastern descent, with over 20m people around the world affected.

In Nigeria around 150,000 children are born with sickle cell anaemia each year. Half of them are likely to die before their 10th birthday.

Juice from the plant, which grows widely across the tropical regions of Africa, has been used in a "blood tonic" as a traditional remedy for generations, but it has never been scientifically proven to work until now.

Dr Olayemi Adeniyi, a researcher at the university who suffers from the condition herself, interviewed traditional healers from south western Nigeria, who said the plant had been used for years as a treatment.

Leaves are crushed manually or blended, and can also be brewed into a tea.

Dr Adeniyi said the research had shown that quercitrin, the active ingredient in the plant, could both prevent and reverse the "sickling" caused by the disease.

She said: "Until now there has been no scientific proof of the plants effectiveness.

The research is particularly important because so many people affected by sickle cell disease live below the poverty line and have no access to medicine. The plant grows in bushes and is relatively easy to grow on fertile land - all you need are seeds.

Its crucial that people in the countries affected, Africa in particular, hear that this plants benefits have now been scientifically proven. Our findings show that this is a treatment that has firm scientific foundations, not just psychological ones.

Existing treatments are expensive, and some involve blood transfusions. It can only be cured with a stem cell or bone marrow transplant, but this is rarely done because of the risks involved.

The finding "could inform efforts directed to the development of an anti-sickling drug", the paper concluded.

The study formed part of an Aberystwyth University project looking at the scientific effectiveness of traditional and herbal remedies, which has also focused on developing new antibiotics to counter the growing problem of antimicrobial resistance.

Professor Luis Mur, who led the research, said: "We're running out of drug leads. There is a recognition, especially with diseases, that they are evolving, and they're evolving through misuse of antimicrobials for example, and so we need to look at new sources.

"So let's go back to where traditional practices have actually given a hint that this particular plant or fungus has some efficacy."

The results were published in the Journal of Clinical Medicine.

See the article here:
Sickle cell disease could be treated with common plant, study finds - The Telegraph