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Cargo Dragon Docks to Station with Brand New Science – NASA

Posted: December 24, 2021 at 1:42 am

The space station is viewed from the SpaceX Cargo Dragon during its automated approach before docking. Credit: NASA TV

While the International Space Station was traveling more than 260 miles over the South Pacific Ocean, a SpaceX Dragon cargo spacecraft autonomously docked to the space-facing side of the orbiting laboratorys Harmony module at 3:41 a.m. EST, Wednesday, Dec. 22. NASA astronauts Raja Chari and Thomas Marshburn were monitoring docking operations for Dragon.

The Dragon launched on SpaceXs 24th contracted commercial resupply mission at 5:07 a.m. EST, Tuesday, Dec. 21 from Launch Complex 39A at NASAs Kennedy Space Center in Florida. After Dragon spends about one month attached to the space station, the spacecraft will return to Earth with cargo and research.

Among the science experiments Dragon is delivering to the space station are:

Bioprinting bandagesBioprinting uses viable cells and biological molecules to print tissue structures. The German Aerospace Center studyBioprint FirstAiddemonstrates a portable, handheld bioprinter that uses a patients own skin cells to create a tissue-forming patch to cover a wound and accelerate the healing process. On future missions to the Moon and Mars, bioprinting such customized patches could help address changes in wound healing that can occur in space and complicate treatment. Personalized healing patches also have potential benefits on Earth, providing safer and more flexible treatment anywhere needed.

Improving delivery of cancer drugsMonoclonal antibodies, used to treat a wide range of human diseases, do not dissolve easily in liquid and so typically must be given intravenously in a clinical setting.The Center for the Advancement of Science in Space Protein Crystal Growth 20 (CASIS PCG 20) experimentcontinues work on crystallizing a monoclonal antibody, pembrolizumab, that Merck Research Labs developed. It is the active ingredient in Keytruda, a drug that targets multiple cancers. Scientists analyze these crystals to learn more about the structure and behavior of the component to create drug formulations that can be administered at a doctors office or even at home.

Assessing infection riskScientists have observed that spaceflight sometimes increases the virulence of potentially harmful microbes and reduces human immune function, increasing the risk for infectious disease.Host-Pathogenassesses space-induced changes in immune status by culturing cells collected from crew members before, during, and after spaceflight with both normal bacteria and bacteria grown under simulated spaceflight conditions. Results could help assess the potential risk infectious microbes may pose and may support development of countermeasures. This could improve care for those with compromised immune systems on Earth.

Roots, shoots, and leavesMulti Variable Platform (MVP) Plant-01profiles and monitors the development of the shoots and roots of plants in microgravity. Plants could serve as a vital part of human life support systems for long-duration spaceflight and habitation of the Moon and Mars. However, space-grown plants experience stress from various factors and recent studies indicate changes in plant gene expression in response to those stressors. Improved understanding of these changes could enable the design of plants that are better suited for growth in spaceflight environments.

Toward lunar laundromatsAstronauts on the space station wear items of clothing several times, then replace them with new clothes delivered on resupply missions. Limited cargo capacity makes this a challenge, and resupply is not an option for longer missions, such as those to the Moon and Mars. In a collaboration with NASA, Procter & Gamble has developed Tide Infinity, a fully degradable detergent specifically designed for use in space, and theP&G Telescience Investigation of Detergent Experiments (PGTIDE) study the performance of its stain removal ingredients and the formulations stability in microgravity. Once proven in space, Tide plans to use the new cleaning methods and detergent to advance sustainable, low-resource-use laundry solutions on Earth.

Parts made in spaceTurbine Superalloy Casting Module (SCM)tests a commercial manufacturing device that processes heat-resistant alloy parts in microgravity. Alloys are materials made up of at least two different chemical elements, one of which is a metal. Researchers expect more uniform microstructures and improved mechanical properties in superalloy parts processed in microgravity compared to those processed on Earth. These superior materials could improve the performance of turbine engines in industries such as aerospace and power generation on Earth.

Students and citizens as space scientistsStudents enrolled in institutions of higher learning can design and build microgravity experiments as part of NASAs Student Payload Opportunity with Citizen Science (SPOCS). As part of their experiments, selected teams include students in kindergarten through 12th grade as citizen scientists. Citizen science allows individuals who are not professional scientists to contribute to real-world research. TheNASA STEM on Stationproject is funding experiments flying on this SpaceX resupply mission, including a study on antibiotic resistance in microgravity from Columbia University in New York and one on how microgravity affects bacteria-resistant polymers from the University of Idaho in Moscow, Idaho.

These are just a few of the hundreds of investigations currently being conducted aboard the orbiting laboratory in the areas of biology and biotechnology, physical sciences, and Earth and space science. Advances in these areas will help keep astronauts healthy during NASAsArtemismissions to the Moon and long-duration space travel and demonstrate technologies for future human and robotic exploration beyond low-Earth orbit to theMoon and Mars.

Learn more about station activities by following thespace station blog,@space_stationand@ISS_Researchon Twitter, as well as theISS FacebookandISS Instagramaccounts.

Get weekly video highlights at:http://jscfeatures.jsc.nasa.gov/videoupdate/

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Cargo Dragon Docks to Station with Brand New Science - NASA

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Use of CAR T-Cell Therapy in LBCL Could Come Earlier After ASH 2021 – AJMC.com Managed Markets Network

Posted: December 10, 2021 at 2:08 am

Some of the most anticipated presentations at the 63rd Annual American Society of Hematology (ASH) Meeting and Exposition involve phase 3 results for chimeric antigen receptor (CAR) T-cell therapy in second-line treatment.

In 2017, FDA approved the first chimeric antigen receptor (CAR) T-cell therapies, tisangenlecleucel (tisa-cel), sold as Kymriah, and axicabtagene cilocleucel (axi-cel), sold as Yescarta, for patients with B-cell lymphomas treated with at least 2 prior therapies.

Not long after the question arose: would patients have better responses if they received CAR T-cell therapy earlier?

Its a question that will get some answers during the 63rd Annual American Society of Hematology Meeting and Exposition, which takes place December 11-14 in Atlanta and online. Some of the most anticipated presentations at ASH involve phase 3 results for CAR T-cell therapy in second-line treatment, and there are also phase 2 results for axi-cel in the first-line setting coming Monday.

Given the cost of CAR T-cell therapy, which lists for $373,000 to $475,000, depending on the indication, the answer is important to payers, who want to know if these expensive processes are more likely to succeed if patients are treated when their immune systems are less depleted. CAR T-cell therapy works by harnessing the patients own immune system to fight cancer cells.

Theres also the issue of avoiding the cost of prior treatments. However, Peter Marks, MD, PhD, director of FDAs Center for Biologics Evaluation and Research,said during a keynote address in October that widespread use of CAR T-cell therapy at earlier stages might only come if the cost of these treatments came down significantly.

Second-line treatment in LBCL. Two presentations will address this, led by the primary analysis of ZUMA-7, the phase 3 randomized trial that examines the use of axi-cel vs second-line standard of care in relapsed/refractory large B-cell lymphoma (LBCL). Gilead Sciences, which sells axi-cel as Yescarta, announced in June that results showed axi-cel offered a 62% event-free survival benefit over chemotherapy and stem-cell transplant. Results will be presented in Sundays plenary session by Frederick Locke, MD, of Moffitt Cancer Center. Axi-cel is currently approved for LBCL or follicular lymphoma when patients have received at least 2 other therapies.

The race is already on for use of axi-cel in the first-line stage, with phase 2 results from ZUMA-12 to be presented by Sattva S. Neelapu, MD, of MD Anderson Cancer Center.

Getting a jump on ZUMA-7 will be a Saturday morning presentation on interim findings from the phase 3 TRANSFORM study involving lisocabtagene maraleucel (liso-cel), which was approved earlier this year for patients with certain types of LBCL who have received at least 2 treatments. Sold as Breyanzi, liso-cel has a different manufacturing process that investigators say results in less toxicity than earlier CAR T-cell therapies. Officials from Bristol Myers Squibb also announced a significant benefit based on early results; the prespecified interim analysis to be presented at ASH shows event-free survival of 10.1 months, while overall survival data are immature.

Results for tisa-cel in the second-line setting will be presented in the late-breaking session Tuesday, but Novartis announced in August that the BELINDA study did not meet its end point.

Biomarkers and CAR T-cell therapy. Payers will be interested in the effort by 12 academic institutions to track molecular features for 121 patients with diffuse LBCL and assess which biomarkers had effects on clinical outcomes after the patients were later treated with tisa-cel or axi-cel. This study promises to identify possible targetable pathways to bring better responses to CAR T-cell therapy and identify which patients will have the best outcomes.

MCL in real-world settings. FDA approved brexucabtagene autoleucel (brexu-cel) in mantle cell lymphoma (MCL) in July 2020, based on results from the ZUMA-2 trial that showed a 90% objective response rate (ORR) and a 67% complete response rate (CR). A study to be presented by the US Lymphoma Consortium leverages the use of real-world data to show that patients with MCL who fell outside the ZUMA-2 criteria have reported high safety and efficacy rates when treated with brexu-cel.

Beyond R-CHOP. For patients with previously untreated LBCL, there will still be choices beyond CAR T-cell therapy. Results from Tuesday mornings late-breaking abstracts will start with the phase 3 POLARIX study, which examines the use of polatuzumab vedotin with rituximab, cyclophosphamide, doxorubicin, and prednisone, or pola-R-CHP, vs the well-known combination of rituxiumab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) in patients with previously untreated diffuse LBCL.

A discussion, Novel Findings in CAR T-Cell Therapies for Hematologic Malignancies, with a live question and answer session, will take place Monday from 4:30 to 5:15 pm. The session chair will be Marcela V. Maus, MD, Massachusetts General Hospital and Harvard Medical School; panelists will be Maus; David Baker, PhD, University of Washington; and Marco Ruella, MD, University of Pennsylvania.

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Use of CAR T-Cell Therapy in LBCL Could Come Earlier After ASH 2021 - AJMC.com Managed Markets Network

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CAR T-cell therapy: Hear from a Canadian patient – CTV News

Posted: November 8, 2021 at 2:01 am

TORONTO -- Owen Snider was given as little as three months to live. His blood cancer had returned and the prognosis was not good. The news, delivered over the phone during the height of the early pandemic lockdown in spring 2020, was devastating.

The Ottawa-area retiree began putting his affairs in order, preparing for what appeared to be inevitable.

It was terrible, his wife Judith Snider told CTV News. But we finally decided that what we had to do was to live each day not to look forward to the end, but to look forward to tomorrow.

And yet, a year later, Snider is alive -- transformed, even -- and his non-Hodgkins lymphoma is in remission. His second chance is all thanks to a promising, Canadian-run program for cancer treatment called CAR T-cell therapy.

Snider became one of the first patients to participate in a national research program that is assessing whether this experimental treatment can be done safely in Canada and cheaper than in the U.S., where costs can run upwards of half a million dollars per patient.

I think I am a pretty successful lab rat, Snider, who previously endured chemotherapy treatments and a stem cell transplant, said in an interview.

Thirty days after treatment, the lymphoma was gone. So how can you not be happy about that?

CAR T-cell therapy is a type of gene therapy that trains or engineers a patients own immune system to recognize cancerous cells. A type of white blood cell, called a T-cell, is a key component of a bodys immune system. They are developed from stem cells in the bone marrow and help fight infection and disease by searching and targeting specific foreign substances, known as antigens, in the body.

The protein receptors on T cells bind to the protein antigens on the surfaces of foreign particles that fit those receptors, like a lock and key. The foreign substance is eradicated once their antigens are bound to a T-cell. But blood cancer cells are normal cells that undergo mutations, so they are not recognized as a foreign threat to the body. In other words, T-cells generally do not have the right receptor key to fit with the antigens of a cancer cell.

CAR T-cell therapy modifies the cells so they are able to identify the cancer cells and destroy them. Its a labour-intensive process that involves taking blood from a patient and separating the T-cells. Then scientists add a gene to the cells that gives them instructions to develop an artificial receptor called a chimeric antigen receptor, or CAR.

We actually take the T-cells out and we modify them in the lab and put them back into the patient. So now they're able to recognize the cancer and kill it off, explained Dr. Kevin Hay, Medical Director for Clinical Cell Therapy with BC Cancer.

I think we're just at the cusp of really understanding what this is going to do for patients in the future.

The therapy is a labour-intensive process -- Snider's cells were shipped to Victoria, B.C to be processed in a special lab facility, then shipped back to Ottawa about a week later, where they were infused back into his body.

The treatment is still being studied, but is already available for some cancers in the U.S. and Canada at a steep price.

Researchers began trials in Canada in 2019 to see if it could be done domestically at a lower cost, highlighting the importance of having key medical production and therapies available in Canada.

We knew we had to do domestic manufacturing and if we've learned anything from COVID-19, it's that domestic capability is really important when it comes to science and medicine, and this is a perfect example of that, said Dr. Natasha Kekre, a hematologist and lead researcher on the trail based at the Ottawa Hospital.

Progress was impacted slightly by the pandemic, but Snider was fortunate enough to participate and is the first patient to come forward to discuss their experience and why he hopes the program will expand across Canada to help others dealing with otherwise untreatable forms of cancer.

Scientists are hoping to release more data in the coming months -- more than 20 patients have been treated so far, according to Dr. Kekre.

This is hopefully just the beginning for us. So this first trial was a foundation to prove that we could actually manufacture T cells, that we could do this in a clinical trial. And so this trial will remain open for patients who are in need, she said.

So absolutely we feel like were opening a door.

Snider's first experience with cancer treatment was more than a decade ago, in 2010, when he underwent a powerful and aggressive chemotherapy regimen that helped him stay cancer-free for six years.

But the treatment was so harsh that when his cancer came back in 2016, doctors told him he could not go through that kind of chemotherapy again. Instead, Snider underwent a stem cell transplant, which gave him another four years without cancer, until April 2020.

This time the outlook was grim, so doctors decided to try and get him into the CAR T-cell trials that started just before the pandemic hit. The study was specifically for patients with acute lymphoblastic leukemia and non-Hodgkins lymphoma who were not responding to other treatments.

Snider said the entire process was a walk in the park compared to what he had gone through before. He was given a mild chemotherapy treatment for three days while his T-cells were being modified in a lab on the other side of the country.

[The T-cells] went to work right away. There's a period of time where there's a lot going on inside fighting each other and that sort of thing. You don't feel great or you don't really know how you feel, Snider described. The treatment was met with outstanding success.

And in 30 days, there was no lymphoma. I couldn't believe it.

For Dr. Kekre, the results bring hope. Snider has done quite well and does not have any evidence of lymphoma at the moment, she said.

I'm unfortunately in a business where I often have to give bad news, and it is really motivating and exciting to be able to offer therapies to patients who didn't have options and to make them better, she said.

The trial is currently at the stage where scientists are making sure the product remains safe. Side-effects can include neurotoxicity, which harms the nervous system, and cytokine release syndrome, which triggers an acute system-wide inflammatory response that can result in organs not functioning properly. But so far researchers have, for the most part, been able to manage and reverse any side effects.

With such promising outcomes for patients who otherwise had no options left, researchers are talking about expanding these studies across Canada and to other forms of cancer. For now, the lab in Victoria is the only facility equipped to make these cell modifications.

I think its really going to be revolutionary with how we treat cancer in the future, not just blood cancers, but all cancers, said Dr. Hay.

Today, Snider is healthy and strong, even able to chop wood at his home near Ottawa. He and his wife Judith, a retired federal judge, are enjoying life anew.

It certainly has given us a future that we didnt know we had, she said.

The treatment not only bought Snider extra time, but also significantly improved his quality of life.

What was given to me is practically a normal life, he added.

It's really just transformed, not just extended, but transformed my life.

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GABAeron Presents Promising Preclinical Data on Stem Cell-Based Therapy for Alzheimer’s Disease – WJHG-TV

Posted: October 28, 2021 at 2:37 am

Human interneuron progenitors derived from induced pluripotent stem cells (iPSCs) can be successfully transplanted and integrate into mouse brains, mature, and reverse signs of hippocampal network dysfunction associated with Alzheimer's disease, GABAeron scientists reported at ISSCR/JSRM.

Published: Oct. 27, 2021 at 7:00 AM CDT|Updated: 18 hours ago

SAN FRANCISCO, Oct. 27, 2021 /PRNewswire/ -- GABAeron, Inc. today presented promising preclinical data on their first-in-class, iPSC-based cell therapy product for Alzheimer's disease at the International Society for Stem Cell Research (ISSCR) and Japanese Society for Regenerative Medicine (JSRM) international meeting "Stem Cells: From Basic Science to Clinical Translation". The data, the first to be publicly shared since the company was founded in 2017, highlight the potential of transplanted, human iPSC-derived interneuron progenitors in treating Alzheimer's disease as well as other neurological disorders with interneuron deficit or loss.

GABAeron scientists successfully differentiated GABAergic interneuron progenitors from human iPSCs, and showed that when transplanted into the brains of an Alzheimer's disease mouse model carrying the major genetic risk factor apolipoprotein E4 (APOE4) the cells could mature, integrate into the hippocampus, and reverse signs of the hippocampal network dysfunction associated with Alzheimer's disease.

"We are incredibly excited by these data, which show the safety and efficacy of our novel iPSC-based approach in an Alzheimer's disease mouse model," said Robert W. Mahley, MD, PhD, chief executive officer and chief scientific officer of GABAeron. "Based on these results, we plan to continue our work to develop a cell replacement therapy to treat patients with APOE4-positive Alzheimer's disease."

Over the course of his career, Mahley president emeritus of the Gladstone Institutes and professor of pathology and medicine at the University of California, San Francisco has illuminated the importance and molecular details of the protein APOE. The gene for APOE comes in several versions and we now know that people with the APOE4 version of the gene have an increased risk of Alzheimer's disease and an earlier age of disease onset compared to people with the more common APOE3 version. Strikingly, APOE4 is associated with 6075% of all Alzheimer's disease cases.

GABAeron scientific co-founder Yadong Huang, MD, PhD, director of the Center for Translational Advancement at the Gladstone Institutes, San Francisco, discovered one important reason for the association between APOE4 and Alzheimer's disease. APOE4, his lab demonstrated, leads to the impairment and loss of hippocampal GABAergic interneurons cells critical for maintaining normal hippocampal activity, required for normal learning and memory, and damaged or lost in Alzheimer's disease brains.

"GABAeron was founded on the premise that if we can replace those interneurons via cell-based therapy, we can restore normal hippocampal activity and thus slow or reverse many of the memory and cognitive impairments associated with Alzheimer's disease," said Huang. "If this approach works, it will be a single treatment with long-lasting impact for Alzheimer'spatients."

In the new study, researchers led by Wen-Chin (Danny) Huang, PhD, and Iris Avellano, developed a novel effective method of coaxing human iPSCs carrying the APOE3 gene to differentiate into GABAergic interneuron progenitors. The resulting cells showed high viability, purity, and robust functionality with more than 90% committed to the correct developmental lineage.

A team led by Wan-Ying Hsieh, PhD, transplanted these interneuron progenitors into the hippocampus of 10-month-old mice carrying the human APOE4 gene; the interneuron progenitors showed robust survival and matured into functional GABAergic interneurons. At 7 months post-transplantation, more than half of the surviving cells had migrated out of the local area, populated the hippocampal subregions, and established connections with other existing neurons throughout the hippocampus.

"These exciting data reveal the high quality of human iPSC-derived interneuron progenitors generated at GABAeron and highlight the feasibility of their long-term survival and integration into mouse brains," said Hsieh. "Importantly, there was no tumor formation from the transplanted cells in over a hundred mice."

They next carried out electrophysiological recordings to study hippocampal network activity in the mice. As expected, the APOE4 mice had deficits in hippocampal activity that can underlie the memory impairments associated with Alzheimer's disease. Specifically, the mice had fewer sharp-wave ripples and their associated slow gamma power in the hippocampus both of which are critical for memory formation and retrieval. When each mouse was transplanted with approximately 120,000 iPSC-derived human interneuron progenitors carrying APOE3, these measurements of hippocampal activity both improved to the levels seen in healthy mice 7 months later.

"These results as a whole represent a critical step toward a potential interneuron-based therapy for APOE4-related Alzheimer's disease," said Qin Xu, PhD, senior director at GABAeron. "This builds up a solid foundation for our further work with clinical-grade human iPSCs."

GABAeron scientists are now adapting their culture techniques for the mass production of clinical-grade human iPSC-derived GABAergic interneuron progenitors. They are also working to identify the molecular characteristics of the mature GABAergic interneurons, which become successfully integrated into the hippocampus in the Alzheimer's disease mouse model.

"With this critical milestone reached, GABAeron will move forward, with great confidence, toward IND-enabling studies and future trials with clinical-grade human iPSC-derived interneuron progenitors for treating APOE4-related Alzheimer's disease," said Sheng Ding, PhD, scientific co-founder of GABAeron and a serial entrepreneur who co-founded two leading public companies, Fate Therapeutics (FATE) and Tenaya Therapeutics (TNYA). "We also plan to explore the usefulness of such a cell-based therapy for other neurological diseases with interneuron deficits or loss."

The ISSCR/JSRM international meeting "Stem Cells: From Basic Science to Clinical Translation" runs from October 2729, 2021 and is being held virtually this year.

About GABAeron

GABAeron, Inc. is a biopharmaceutical company founded in 2017, based on pioneer work initiated at the Gladstone Institutes, to build on the promise of combining precision medicine, regenerative medicine, and pharmaceutical intervention. The company is exploring a new first-in-class IND candidate to replace or restore neurons injured or lost in the brains of patients suffering from neurodegenerative and neurodevelopmental disorders.

For more information about GABAeron, please visit http://www.GABAeron.com.

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SOURCE GABAeron

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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GABAeron Presents Promising Preclinical Data on Stem Cell-Based Therapy for Alzheimer's Disease - WJHG-TV

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Deterioration of brain cells in Parkinson’s disease is slowed by blocking the Bach1 protein, preclinical study shows – Medical University of South…

Posted: October 28, 2021 at 2:37 am

Parkinsons disease (PD) is the most common neurodegenerative movement disorder, afflicting more than 10 million people worldwide and more than one million Americans. While there is no cure for PD, current therapies focus on treating motor symptoms and fail to reverse, or even address, the underlying neurological damage. In a new study, researchers at the Medical University of South Carolina (MUSC) have identified a novel role for the regulatory protein Bach1 in PD. Their results, published on Oct. 25 in the Proceedings of the National Academy of Sciences, showed that levels of Bach1 were increased in postmortem PD-affected brains, and that cells without Bach1 were protected from the damages that accumulate in PD. In collaboration with vTv Therapeutics, they identified a potent inhibitor of Bach1, called HPPE, that protected cells from inflammation and the buildup of toxic oxidative stress when administered either before or after the onset of disease symptoms.

This is the first evidence that Bach1 is dysregulated in Parkinsons disease, said Bobby Thomas, Ph.D., professor of Pediatrics in the College of Medicine and the SmartState COEE Endowed Chair in Pediatric Neurotherapeutics.

In PD, brain cells that produce the chemical messenger dopamine begin to die as the disease progresses, resulting in tremors and other disruptions to motor function. Additionally, as we age, neurons accumulate damage through inflammation and the buildup of toxic oxidative stress.

There are many genes that combat these destructive pathways, many of which are controlled by two key proteins: Nrf2 and Bach1. Nrf2 functions to turn on the expression of over 250 genes that are involved in protecting the cell from these stressors. Conversely, Bach1 prevents these genes from being activated.

Thomas lab found that levels of Bach1 are increased in autopsied brains of patients with PD, as well as toxin-based preclinical PD models, suggesting that high levels of Bach1 may contribute to PD pathophysiology. To confirm this, the researchers depleted Bach1 in a PD mouse model and showed that dopamine-producing neurons were protected from some of the destructive stress pathways.

To determine how the loss of Bach1 protected neurons from accumulated stress, they analyzed the entire genome of brains from Bach1-depleted mice and looked at which genes were activated.

What we found was that Bach1 not only represses the expression of protective genes that are under the control of Nrf2, but it also regulates the expression of many other genes not directly regulated by Nrf2, said Thomas. So there are additional advantages to inhibiting Bach1 besides just activating Nrf2. Ideally you would want a drug that inhibits Bach1 and also activates Nrf2.

To that end, Thomas partnered with the North Carolina-based company vTv Therapeutics to develop Bach1 inhibitors. Using its proprietary TTP Translational Technology platform, vTv discovered several potential candidates that were validated by Thomas. The top candidate, HPPE, functioned as a superior Bach1 inhibitor in in vitro models. Importantly, HPPE was also a potent activator of Nrf2.

Therefore, pharmacological intervention using HPPE provided the dual benefit of stabilizing Nrf2 and inhibiting Bach1. But how would HPPE work in a preclinical PD mouse model?

The effectiveness of HPPE was tested in a neurotoxin-based PD mouse model. HPPE alleviated toxin-induced PD symptoms when given either before the induction of disease or after the onset of disease symptoms. Further analyses showed that HPPE protects neurons from destructive pathways by turning on antioxidant genes and turning off pro-inflammatory genes.

Interestingly, HPPE worked better at protecting neurons than current FDA-approved Nrf2 activators, such as Tecfidera (dimethyl fumarate). Current activators function as electrophiles they permanently bind to and modify proteins which can lead to cellular toxicity or activation of the immune system and have many side effects.

The most interesting aspect of the study is that the Bach1 inhibitor is a non-electrophile, so it doesnt work like the FDA-approved Nrf2 activators, said Thomas. As a result of this difference, hopefully, HPPE will not demonstrate as many side effects.

Disruption of Bach1 and the simultaneous activation of Nrf2 clearly provide a strong basis for using HPPE as a potential therapeutic in PD. But several questions remain unanswered. While there were no side effects observed with acute treatment using HPPE in the PD mouse model, one key goal moving forward is to determine what impacts, if any, long-term use of HPPE might have. Another key question centers on the benefits of modulating this pathway in more chronic models of PD, other cell types in the brain and potentially other dementias.

Thispathway may be beneficial whenever you have impairments in anti-inflammatorypathways or mitochondrial dysfunctions, said Thomas. I think any disease thathas these kinds of etiologies would benefit from modulating this pathway.

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GABAeron Presents Promising Preclinical Data on Stem Cell-Based Therapy for Alzheimer’s Disease – KFYR-TV

Posted: October 28, 2021 at 2:05 am

Human interneuron progenitors derived from induced pluripotent stem cells (iPSCs) can be successfully transplanted and integrate into mouse brains, mature, and reverse signs of hippocampal network dysfunction associated with Alzheimer's disease, GABAeron scientists reported at ISSCR/JSRM.

Published: Oct. 27, 2021 at 7:00 AM CDT|Updated: 18 hours ago

SAN FRANCISCO, Oct. 27, 2021 /PRNewswire/ -- GABAeron, Inc. today presented promising preclinical data on their first-in-class, iPSC-based cell therapy product for Alzheimer's disease at the International Society for Stem Cell Research (ISSCR) and Japanese Society for Regenerative Medicine (JSRM) international meeting "Stem Cells: From Basic Science to Clinical Translation". The data, the first to be publicly shared since the company was founded in 2017, highlight the potential of transplanted, human iPSC-derived interneuron progenitors in treating Alzheimer's disease as well as other neurological disorders with interneuron deficit or loss.

GABAeron scientists successfully differentiated GABAergic interneuron progenitors from human iPSCs, and showed that when transplanted into the brains of an Alzheimer's disease mouse model carrying the major genetic risk factor apolipoprotein E4 (APOE4) the cells could mature, integrate into the hippocampus, and reverse signs of the hippocampal network dysfunction associated with Alzheimer's disease.

"We are incredibly excited by these data, which show the safety and efficacy of our novel iPSC-based approach in an Alzheimer's disease mouse model," said Robert W. Mahley, MD, PhD, chief executive officer and chief scientific officer of GABAeron. "Based on these results, we plan to continue our work to develop a cell replacement therapy to treat patients with APOE4-positive Alzheimer's disease."

Over the course of his career, Mahley president emeritus of the Gladstone Institutes and professor of pathology and medicine at the University of California, San Francisco has illuminated the importance and molecular details of the protein APOE. The gene for APOE comes in several versions and we now know that people with the APOE4 version of the gene have an increased risk of Alzheimer's disease and an earlier age of disease onset compared to people with the more common APOE3 version. Strikingly, APOE4 is associated with 6075% of all Alzheimer's disease cases.

GABAeron scientific co-founder Yadong Huang, MD, PhD, director of the Center for Translational Advancement at the Gladstone Institutes, San Francisco, discovered one important reason for the association between APOE4 and Alzheimer's disease. APOE4, his lab demonstrated, leads to the impairment and loss of hippocampal GABAergic interneurons cells critical for maintaining normal hippocampal activity, required for normal learning and memory, and damaged or lost in Alzheimer's disease brains.

"GABAeron was founded on the premise that if we can replace those interneurons via cell-based therapy, we can restore normal hippocampal activity and thus slow or reverse many of the memory and cognitive impairments associated with Alzheimer's disease," said Huang. "If this approach works, it will be a single treatment with long-lasting impact for Alzheimer'spatients."

In the new study, researchers led by Wen-Chin (Danny) Huang, PhD, and Iris Avellano, developed a novel effective method of coaxing human iPSCs carrying the APOE3 gene to differentiate into GABAergic interneuron progenitors. The resulting cells showed high viability, purity, and robust functionality with more than 90% committed to the correct developmental lineage.

A team led by Wan-Ying Hsieh, PhD, transplanted these interneuron progenitors into the hippocampus of 10-month-old mice carrying the human APOE4 gene; the interneuron progenitors showed robust survival and matured into functional GABAergic interneurons. At 7 months post-transplantation, more than half of the surviving cells had migrated out of the local area, populated the hippocampal subregions, and established connections with other existing neurons throughout the hippocampus.

"These exciting data reveal the high quality of human iPSC-derived interneuron progenitors generated at GABAeron and highlight the feasibility of their long-term survival and integration into mouse brains," said Hsieh. "Importantly, there was no tumor formation from the transplanted cells in over a hundred mice."

They next carried out electrophysiological recordings to study hippocampal network activity in the mice. As expected, the APOE4 mice had deficits in hippocampal activity that can underlie the memory impairments associated with Alzheimer's disease. Specifically, the mice had fewer sharp-wave ripples and their associated slow gamma power in the hippocampus both of which are critical for memory formation and retrieval. When each mouse was transplanted with approximately 120,000 iPSC-derived human interneuron progenitors carrying APOE3, these measurements of hippocampal activity both improved to the levels seen in healthy mice 7 months later.

"These results as a whole represent a critical step toward a potential interneuron-based therapy for APOE4-related Alzheimer's disease," said Qin Xu, PhD, senior director at GABAeron. "This builds up a solid foundation for our further work with clinical-grade human iPSCs."

GABAeron scientists are now adapting their culture techniques for the mass production of clinical-grade human iPSC-derived GABAergic interneuron progenitors. They are also working to identify the molecular characteristics of the mature GABAergic interneurons, which become successfully integrated into the hippocampus in the Alzheimer's disease mouse model.

"With this critical milestone reached, GABAeron will move forward, with great confidence, toward IND-enabling studies and future trials with clinical-grade human iPSC-derived interneuron progenitors for treating APOE4-related Alzheimer's disease," said Sheng Ding, PhD, scientific co-founder of GABAeron and a serial entrepreneur who co-founded two leading public companies, Fate Therapeutics (FATE) and Tenaya Therapeutics (TNYA). "We also plan to explore the usefulness of such a cell-based therapy for other neurological diseases with interneuron deficits or loss."

The ISSCR/JSRM international meeting "Stem Cells: From Basic Science to Clinical Translation" runs from October 2729, 2021 and is being held virtually this year.

About GABAeron

GABAeron, Inc. is a biopharmaceutical company founded in 2017, based on pioneer work initiated at the Gladstone Institutes, to build on the promise of combining precision medicine, regenerative medicine, and pharmaceutical intervention. The company is exploring a new first-in-class IND candidate to replace or restore neurons injured or lost in the brains of patients suffering from neurodegenerative and neurodevelopmental disorders.

For more information about GABAeron, please visit http://www.GABAeron.com.

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The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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Cancer Stem Cells (CSCs) Market Analytical Overview, Growth Factors, Demand and Trends Forecast to 2027 The Manomet Current – The Manomet Current

Posted: July 21, 2021 at 2:24 am

A thorough overview of the global Cancer Stem Cells (CSCs) market has been presented in this market intelligence study in order to provide a strong understanding of the market. The research report offers a comprehensive analysis of the lucrative opportunities and latest trends within the global Cancer Stem Cells (CSCs) market. In addition, the study comprises strategic business policies that have been adopted by the leading players operating in the global Cancer Stem Cells (CSCs) market, along with the key growth drivers, limitations, challenges, regulatory ecosystem, and future estimates of the global Cancer Stem Cells (CSCs) market throughout the forecast period.

The research study talks about the competitive landscape of the global Cancer Stem Cells (CSCs) market and offers a detailed analysis for the same. In order to provide a clear picture of the market, the research study has provided a list of all the leading players operating in the market across the globe and also provide coronavirus pandemic impacts analysis. Additionally, the product segmentation, SWOT analysis, and financial overview of these players have been mentioned in the research study.

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Market Segmented are as Follows:

Segmented by Category:

CellSearch Others

Segmented by End User/Segment:

Breast Cancer Diagnosis and Treatment Prostate Cancer Diagnosis and Treatment Colorectal Cancer Diagnosis and Treatment Lung Cancer Diagnosis and Treatment Other Cancers Diagnosis and Treatment

Key manufacturers included in this survey:

YZY Bio Silicon Biosystems ScreenCell Qiagen On-chip Miltenyi Biotec Janssen IVDiagnostics Ikonisys Gilupi Fluxion Fluidigm CytoTrack Cynvenio Creatv MicroTech Clearbridge Biomedics Celsee BioView Biofluidica Aviva Biosciences Corporation ApoCell Advanced Cell Diagnostics AdnaGen

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Cancer Stem Cells (CSCs) Market Regional Analysis Includes:

Table of Contents:

Chapter 1: Product Introduction and Overview

1.1 Product Definition

1.2 Product Specification

1.3 Global Market Overview

1.4 Market Drivers, Inhibitors

Chapter 2: Global Cancer Stem Cells (CSCs) Supply by Company

2.1 Global Cancer Stem Cells (CSCs) Sales Volume by Company

2.2 Global Cancer Stem Cells (CSCs) Sales Value by Company

2.3 Global Cancer Stem Cells (CSCs) Price by Company

2.4 Cancer Stem Cells (CSCs) Production Location and Sales Area of Main Manufacturers

2.5 Trend of Concentration Rate

Chapter 3: Global and Regional Cancer Stem Cells (CSCs) Market Status by Category

Chapter 4: Global and Regional Cancer Stem Cells (CSCs) Market Status by End User/Segment

Chapter 5: Global Cancer Stem Cells (CSCs) Market Status by Region

Chapter 6: North America Cancer Stem Cells (CSCs) Market Status

Chapter 7: Europe Cancer Stem Cells (CSCs) Market Status

Chapter 8: Asia Pacific Cancer Stem Cells (CSCs) Market Status

Chapter 9: Central & South America Cancer Stem Cells (CSCs) Market Status

Chapter 10: Middle East & Africa Cancer Stem Cells (CSCs) Market Status

Chapter 11: Supply Chain and Manufacturing Cost Analysis

11.1 Supply Chain Analysis

11.2 Production Process Chart Analysis

11.3 Raw Materials and Key Suppliers Analysis

11.4 Cancer Stem Cells (CSCs) Manufacturing Cost Analysis

11.5 Cancer Stem Cells (CSCs) Sales Channel and Distributors Analysis

11.6 Cancer Stem Cells (CSCs) Downstream Major Buyers

Chapter 12: Global Cancer Stem Cells (CSCs) Market Forecast by Category and by End User/Segment

12.1 Global Cancer Stem Cells (CSCs) Sales Volume and Sales Value Forecast (2022-2027)

12.2 Global Cancer Stem Cells (CSCs) Forecast by Category

12.3 Global Cancer Stem Cells (CSCs) Forecast by End User/Segment

Chapter 13: Global Cancer Stem Cells (CSCs) Market Forecast by Region/Country

14 Key Participants Company Information

15 Conclusion

16 Methodology

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The research report has addresses several questions related to the growth of the global Cancer Stem Cells (CSCs) market. Some of them are mentioned as below:

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Cancer Stem Cells (CSCs) Market Analytical Overview, Growth Factors, Demand and Trends Forecast to 2027 The Manomet Current - The Manomet Current

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A $50,000 Helmet Can Read User’s Mind. And It’s Ready – NDTV

Posted: June 23, 2021 at 2:01 am

This helmet measures changes in blood oxygenation levels.

Over the next few weeks, a company called Kernel will begin sending dozens of customers across the U.S. a $50,000 helmet that can, crudely speaking, read their mind. Weighing a couple of pounds each, the helmets contain nests of sensors and other electronics that measure and analyze a brain's electrical impulses and blood flow at the speed of thought, providing a window into how the organ responds to the world. The basic technology has been around for years, but it's usually found in room-size machines that can cost millions of dollars and require patients to sit still in a clinical setting.

The promise of a leagues-more-affordable technology that anyone can wear and walk around with is, well, mind-bending. Excited researchers anticipate using the helmets to gain insight into brain aging, mental disorders, concussions, strokes, and the mechanics behind previously metaphysical experiences such as meditation and psychedelic trips. "To make progress on all the fronts that we need to as a society, we have to bring the brain online," says Bryan Johnson, who's spent more than five years and raised about $110 million-half of it his own money-to develop the helmets.

Johnson with one of his helmets in a lab at Kernel's offices.

Johnson is the chief executive officer of Kernel, a startup that's trying to build and sell thousands, or even millions, of lightweight, relatively inexpensive helmets that have the oomph and precision needed for what neuroscientists, computer scientists, and electrical engineers have been trying to do for years: peer through the human skull outside of university or government labs. In what must be some kind of record for rejection, 228 investors passed on Johnson's sales pitch, and the CEO, who made a fortune from his previous company in the payments industry, almost zeroed out his bank account last year to keep Kernel running. "We were two weeks away from missing payroll," he says. Although Kernel's tech still has much to prove, successful demonstrations, conducted shortly before Covid-19 spilled across the globe, convinced some of Johnson's doubters that he has a shot at fulfilling his ambitions.

A core element of Johnson's pitch is "Know thyself," a phrase that harks back to ancient Greece, underscoring how little we've learned about our head since Plato. Scientists have built all manner of tests and machines to measure our heart, blood, and even DNA, but brain tests remain rare and expensive, sharply limiting our data on the organ that most defines us. "If you went to a cardiologist and they asked you how your heart feels, you would think they are crazy," Johnson says. "You would ask them to measure your blood pressure and your cholesterol and all of that."

The first Kernel helmets are headed to brain research institutions and, perhaps less nobly, companies that want to harness insights about how people think to shape their products. (Christof Koch, chief scientist at the Allen Institute for Brain Science in Seattle, calls Kernel's devices "revolutionary.") By 2030, Johnson says, he wants to bring down the price to the smartphone range and put a helmet in every American household-which starts to sound as if he's pitching a panacea. The helmets, he says, will allow people to finally take their mental health seriously, to get along better, to examine the mental effects of the pandemic and even the root causes of American political polarization. If the Biden administration wanted to fund such research, Johnson says, he'd be more than happy to sell the feds a million helmets and get started: "Let's do the largest brain study in history and try to unify ourselves and get back to a steady state."

Johnson is something of a measurement obsessive. He's at the forefront of what's known as the quantified-self movement. Just about every cell in his body has been repeatedly analyzed and attended to by a team of doctors, and their tests now cast him as a full decade younger than his 43 years. Along those lines, he wants to let everyone else analyze, modify, and perfect their minds. No one knows what the results will be, or even if this is a good idea, but Johnson has taken it upon himself to find out.

After selling his payments startup, Johnson radically changed his life.

Unlike many of his tech-millionaire peers, Johnson grew up relatively poor. Born in 1977, he was raised in Springville, Utah, the third of five children. "We had very little and lived a very simple life," says his mother, Ellen Huff. A devout Mormon, she stayed home with the kids as much as possible and earned a modest income from a rental unit on the other side of the family's duplex.

Johnson remembers his mother knitting his clothes and grinding wholesale batches of wheat to make bread. "We were not like my friends," he says. "They would buy things from stores, and we just did not do that." His dad, a trash collector turned lawyer, had a drug problem and an affair, which led to his divorce from Huff. Later, delinquent child support payments, missed pickups on the weekends, and legal troubles contributed to his disbarment. "After some time of challenge, my father successfully overhauled his life 20 years ago," Johnson says. "Throughout his struggles, we remained close and without conflict. He has been a unique source of wisdom, counsel, and stability in my life."

Johnson had little idea what to do with his life until he served a two-year church mission in Ecuador, where he interacted with people living in huts with dirt floors and walls made of mud and hay. "When I came back, the only thing I cared about was how to do the most good for the most people," he says. "Since I didn't have any skills, I decided to become an entrepreneur."

While at Brigham Young University, he started his own business selling cellphones and service plans, making enough money to hire a team of salespeople. After that, he invested in a real estate development company that collapsed and left him $250,000 in debt. To get out of the hole, he took a job selling credit card processing services to small businesses door to door. Soon he was the company's top salesman.

This was the mid-2000s, and Johnson's customers kept complaining about the hassle of setting up and maintaining credit card payment systems on their websites. In 2007 he started Braintree, a software company focused on easing the process with slick interfaces. It succeeded-and had good timing. After signing up a slew of restaurants, retailers, and other small businesses, Braintree became the middleman of choice for a profusion of startups premised on ordering services online, including Airbnb, OpenTable, and Uber. The company also made a great bet on mobile payments, acquiring Venmo for only $26 million in 2012. The next year, EBay bought Braintree for $800 million in cash, a little less than half of which went to Johnson.

Despite his newfound fortune, Johnson felt miserable. He was stressed out and overweight. He'd gotten married and had kids at a young age, but his marriage was falling apart, and he was questioning his life, religion, and identity. He says he entered a deep depressive spiral that included suicidal thoughts.

The decision to sell Braintree well before it peaked in value had been motivated in part by Johnson's need to change those patterns. "Once I had money, it was the first time in my life that I could eliminate all permission structures," he says. "I could do whatever I wanted." He broke with the Mormon church, got divorced, and moved from Chicago, where Braintree was headquartered, to Los Angeles to start over.

Arriving in California, Johnson consulted with all manner of doctors and mental health specialists. His bodily health improved with huge changes to his diet, exercise, and sleep routines. His mind proved a tougher puzzle. He meditated and studied cognitive science, particularly the ways people develop biases, in an effort to train himself to think more rationally. By late 2014 he was convinced his wealth would be best spent advancing humanity's understanding of the brain. He took a large portion of his windfall and started OS Fund, a venture firm that has invested in several artificial intelligence and biotech companies. These include Ginkgo Bioworks, Pivot Bio, Synthego, and Vicarious, some of the most promising startups trying to manipulate DNA and other molecules.

Mostly, though, Johnson staked his fortune on Kernel. When he founded the company, in 2015, his plan was to develop surgical implants that could send information back and forth between humans and computers, the way Keanu Reeves downloads kung fu into his brain in The Matrix. (In the early days, Johnson discussed a potential partnership with Elon Musk, whose company Neuralink Corp. has put implants in pigs and monkeys, but nothing came of it.) The idea was, in part, to transfer thoughts and feelings directly from one consciousness to another, to convey emotions and ideas to other people more richly than human language allows.

Perhaps more important, Johnson reckoned, AI technology was getting so powerful that for human intelligence to remain relevant, the brain's processing power would need to keep pace.

Johnson and I began discussing brains in mid-2018, when I was working on a story about the overlap between neuroscience and AI software. During an initial interview at his company's headquarters in L.A.'s Venice neighborhood, Johnson was cordial but somewhat vague about his aims. But at the end of the visit, I happened to mention the time I underwent a mental healing ritual that involved a Chilean shaman burning holes in my arm and pouring poisonous frog secretions into the wounds. (I do mention this a lot.) Excited, Johnson replied that he had a personal shaman in Mexico and doctors in California who guided him on drug-induced mind journeys. Based on this common ground, he decided to tell me more about Kernel's work and his own adventurous health practices.

By then, Johnson had abandoned neural implants in favor of helmets. The technology needed to make implants work is difficult to perfect-among other things, the human body tends to muddy the devices' signals over time, or to reject them outright-and the surgery seemed unlikely to go mainstream. With the helmets, the basic principle remained the same: put tiny electrodes and sensors as close as possible to someone's neurons, then use the electrodes to detect when neurons fire and relay that information to a computer. Watch enough of these neurons fire in enough people, and we may well begin to solve the mysteries of the brain's fine mechanics and how ideas and memories form.

A wider shot of the Flux helmet's enclosure at Kernel's lab. The booth shields against electromagnetic interference so the instrument can measure very sensitive brain output.

On and off for almost three years, I've watched as Kernel has brought its helmets into reality. During an early visit to the company's two-story headquarters in a residential part of Venice, I saw that Johnson's team had converted the garage into an optics lab full of mirrors and high-end lasers. Near the entryway sat a shed-size metallic cube designed to shield its contents from electromagnetic interference. On the second floor, dozens of the world's top neuroscientists, computer scientists, and materials experts were tinkering with early versions of the helmets alongside piles of other electrical instruments. At that point the helmets looked less like 21st century gadgets and more like something a medieval knight might wear into battle, if he had access to wires and duct tape.

Despite the caliber of his team, Johnson and his odd devices were considered toys by outsiders. "The usual Silicon Valley people and investors would not even talk to us or poke around at all," he says. "It became clear that we would have to spend the time, and I would have to spend the money, to show people something and demonstrate it working."

A hospital or research center will typically employ a range of instruments to analyze brains. The list is a smorgasbord of acronyms: fMRI (functional magnetic resonance imaging), fNIRS (functional near-infrared spectroscopy), EEG (electroencephalography), MEG (magnetoencephalography), PET (positron emission tomography), etc. (et cetera). These machines measure a variety of things, from electrical activity to blood flow, and they do their jobs quite well. They're also enormous, expensive, and not easily condensed into helmet form.

In some cases the machines' size owes in part to components that shield the patient's head from the cacophony of electrical interference present in the world. This allows the sensors to avoid distracting signals and capture only what's happening in the brain. Conversely, signals from the machines need to penetrate the human skull, which happens to be well-evolved to prevent penetration. That's part of the argument for implants: They nestle sensors right up against our neurons, where the signals come in loud and clear.

It's unlikely a helmet will ever gain the level of information an implant can, but Kernel has striven to close the gap by shrinking its sensors and finding artful ways to block electromagnetic interference. Among its breakthroughs, Johnson's team designed lasers and computer chips that were able to see and record more brain activity than any previous technology. Month after month, the helmet became more refined, polished, and lightweight as the team made and remade dozens of prototypes. The only trick was that, to suit the different applications Johnson envisioned for the helmet, Kernel wound up needing to develop two separate devices to mimic all the key functions of more traditional machines.

One of the devices, called Flow, looks like a high-tech bike helmet, with several brushed aluminum panels that wrap around the head and have small gaps between them. Flip it over, and you'll see a ring of sensors inside. A wire at the back can be connected to a computer system.

This helmet measures changes in blood oxygenation levels. As parts of the brain activate and neurons fire, blood rushes in to provide oxygen. The blood also carries proteins in the form of hemoglobin, which absorbs infrared light differently when transporting oxygen. (This is why veins are blue, but we bleed red.) Flow takes advantage of this phenomenon by firing laser pulses into the brain and measuring the reflected photons to identify where a change in blood oxygenation has occurred. Critically, the device also measures how long the pulse takes to come back. The longer the trip, the deeper the photons have gone into the brain. "It's a really nice way to distill out the photons that have gone into the brain vs. ones that only hit the skull or scalp and bounced away," says David Boas, a professor of biomechanical engineering and director of the Neurophotonics Center at Boston University.

The other Kernel helmet, Flux, measures electromagnetic activity. As neurons fire and alter their electrical potential, ions flow in and out of the cells. This process produces a magnetic field, if one that's very weak and changes its behavior in milliseconds, making it extremely difficult to detect. Kernel's technology can discover these fields all across the brain via tiny magnetometers, which gives it another way to see what parts of the organ light up during different activities.

The helmets are not only smaller than the devices they seek to replace, but they also have better bandwidth, meaning researchers will receive more data about the brain's functions. According to the best current research, the Flow device should help quantify tasks related to attention, problem-solving, and emotional states, while Flux should be better suited to evaluating brain performance, learning, and information flow. Perhaps the No.1 thing that has scientists gushing about Kernel's machines is their mobility-patients' ability to move around wearing them in day-to-day settings. "This unlocks a whole new universe of research," Boas says. "What makes us human is how we interact with the world around us." The helmets also give a picture of the whole brain, as opposed to implants, which look solely at particular areas to answer more specific questions, according to Boas.

Once their Kernel helmets arrive, Boas and his colleagues plan to observe the brains of people who've had strokes or suffer from diseases such as Parkinson's. They want to watch what the brain does as individuals try to relearn how to walk and speak and cope with their conditions. The hope is that this type of research could improve therapy techniques. Instead of performing one brain scan before the therapy sessions start and another only after months of work, as is the practice today, researchers could scan the brain each day and see which exercises make the most difference.

Devices are also going out to Harvard Medical School, the University of Texas, and the Institute for Advanced Consciousness Studies (a California lab focused on researching altered states) to study such things as Alzheimer's and the effect of obesity on brain aging, and to refine meditation techniques. Cybin Inc., a startup aiming to develop therapeutic mental health treatments based on psychedelics, will use the helmets to measure what happens when people trip.

All of this thrills Johnson, who continues to harbor the grandest of ambitions for Kernel. He may have given up on computer-interfacing implants, but he still wants his company to help people become something more than human.

A couple years ago, Johnson and I boarded his private jet and flew from California to Golden, Colo. Johnson, who has a pilot's license, handled the takeoffs and landings but left the rest to a pro. We were in Colorado to visit a health and wellness clinic run by physician-guru Terry Grossman and have a few procedures done to improve our bodies and minds.

The Grossman Wellness Center looked like a cross between a medical clinic and the set of Cocoon. Most of the other guests were elderly. In a large central room, about 10 black leather chairs and matching footrests were arranged in a loose circle. Each chair held a couple of fluffy white pillows, with a metal pole on the side for our IV drips. A few of the ceiling tiles had been replaced and fitted with pictures of clouds and palm trees. In rooms off to the side, medical personnel performed consultations and procedures.

Our morning began with an IV infusion of two anti-aging fluids: Myers' Cocktail-a blend of magnesium, calcium, B vitamins, vitamin C, and other good stuff-followed by a helping of nicotinamide adenine dinucleotide. Some of the IV fluids can trigger nausea, but Johnson set the drip to maximum and complemented the IV by having a fiber-optic cable fed into his veins to pepper his blood with red, green, blue, and yellow wavelengths of light for added rejuvenation. "I have to experience pain when I exercise or work," he said, adding that the suffering makes him feel alive.

A few hours later, Johnson went into one of the treatment rooms with Grossman to get a stem cell injection straight into his brain. Earlier he'd provided 5 ounces of his blood, which had then been spun in a centrifuge so Grossman could separate out the plasma and put it through a secret process to "activate the stem cells." Now, Johnson hopped onto a reclined exam table, lying on his back with his head angled toward the floor. Grossman pulled out a liquid-filled syringe. Instead of a needle at the end, it had a 4inchlong, curved plastic tube, which the doctor coated with some lubricating jelly. He pushed the tube into one of Johnson's nostrils, told the patient to take a big sniff, then pinched Johnson's nose shut. They repeated the process for the other nostril. The procedure looked incredibly uncomfortable, but again, Johnson was unfazed, pulling in the stem cells with determination and excitement.

This snorting procedure-designed to improve mood, energy, and memory-was just a small part of Johnson's overall health regimen. Each morning the CEO took 40 pills to boost his glands, cell membranes, and microbiome. He also used protein patches and nasal sprays for other jobs. After all this, he did 30 minutes of cardio and 15 minutes of weights. At lunch he'd have some bone broth and vegetables foraged by his chef from the yards of houses in Venice. He might have a light dinner later, but he never consumed anything after 5 p.m. He went to bed early and measured his sleep performance overnight. Every now and then, a shaman or doctor would juice him up with some drugs such as ketamine or psilocybin. He'd taken strongly enough to these practices to tattoo his arm with "5-MeO-DMT," the molecular formula for the psychoactive compound famously secreted by the Sonoran Desert toad.

To make sure all his efforts were doing some good, Johnson had a lab measure his telomeres. These are the protective bits at the end of DNA strands, which some Nobel Prize-winning science has shown can be good indicators of how your body is aging. The longer the telomeres, the better you're doing. Johnson used to register as 0.4 years older internally than his chronological age, but a couple of years into his regimen under Grossman, when he was in his early 40s, his doctors were telling him he was testing like a man in his late 30s.

During one of our most recent conversations, Johnson tells me he's stopped snorting stem cells and experimenting with hallucinogens. "I got what I wanted from that and don't need to mess with it right now," he says. After many tests and much analysis, he's discovered he operates best if he wakes up at 4 a.m., consumes 2,250 calories of carefully selected food over the course of 90 minutes, and then doesn't eat again for the rest of the day. Every 90 days he goes through another battery of tests and adjusts his diet to counteract any signs of inflammation in his body. He goes to bed each night between 8 and 8:30 p.m. and continues to measure his sleep metrics. "I have done tremendous amounts of trial and error to figure out what works best for my health," he says. "I have worked very hard to figure these algorithms out."

Johnson eats once a day, first thing. Yes, including the wine.

In terms of what our birth certificates say, Johnson and I are the same age. He'll turn 44 in August, a month before I do. To someone like me, who prizes late nights with friends, food, and drink, Johnson's rigid lifestyle doesn't exactly sound romantic. But it does seem to be paying off: When he last got tested, he had the exercise capacity of someone in his late teens or early 20s, and a set of DNA and other health markers pegged his age at somewhere around 30. As for me, I lack the courage to ask science what it makes of my innards and will go on celebrating my dad bod.

As Johnson sees it, had he not changed his lifestyle, he'd have remained depressed and possibly died far too young. Now he does what the data say and nothing else. "I did a lot of damage to myself working 18-hour days and sleeping under a desk," he says. "You might earn the praise of your peers, but I think that sort of lifestyle will very quickly be viewed as primitive." He says he's at war with his brain and its tendencies to lead him astray. "I used to binge-eat at night and could not stop myself," he says. "It filled me with shame and guilt and wrecked my sleep, which crushed my willpower. My mind was a terrible actor for all those years. I wanted to remove my mind from the decision-making process."

The nuance in his perspective can be tricky to navigate. Johnson wants to both master the mind and push it to the side. He maintains, however, that our brain is flawed only because we don't understand how it works. Put enough Kernel devices on enough people, and we'll find out why our brain allows us to pursue addictive, debilitating behaviors-to make reckless decisions and to deceive ourselves. "When you start quantifying the mind, you make thought and emotion an engineering discipline," he says. "These abstract thoughts can be reduced to numbers. As you measure, you move forward in a positive way, and the quantification leads to interventions."

Of course, not everyone will want to make decisions based on what a helmet says their brain activity means. Taking the decisions out of thought patterns-or analyzing them for the purposes of market research and product design-poses its own, perhaps scarier, questions about the future of human agency. And that's if the Kernel devices can fulfill the company's broader ambitions. While the big, expensive machines in hospitals have been teaching us about the brain for decades, our understanding of our most prized organ has remained, in many ways, pretty basic. It's possible Kernel's mountain of fresh data won't be of the kind that translates into major breakthroughs. The brain researchers who are more skeptical of efforts such as Johnson's generally argue that novel insights about how the brain works-and, eventually, major leaps in brain-machine interfaces-will require implants.

Yet scientists who have watched Kernel's journey remark on how the company has evolved alongside Johnson, a complete outsider to the field. "Everybody he's recruited to Kernel is amazing, and he's been able to listen to them and motivate them," says MIT neuroscientist Edward Boyden. "He didn't have scientific training, but he asked really good questions." The test now will be to see how the company's devices perform in the field and if they really can create a whole new market where consumers buy Flow and Flux helmets alongside their Fitbits and Oura rings. "There's a lot of opportunity here," Boyden says. "It's a high-risk, high-payoff situation."

If Johnson's theories are correct and the Kernel devices prove to be as powerful as he hopes, he'll be, in a sense, the first person to spark a broader sort of enlightened data awakening. He recently started a program meant to quantify the performance of his organs to an unprecedented degree. Meanwhile, he's taking part in several experiments with the Kernel helmets and is still looking for ways to merge AI with flesh. "We are the first generation in the history of Homo sapiens who could look out over our lifetimes and imagine evolving into an entirely novel form of conscious existence," Johnson says. "The things I am doing can create a bridge for humans to use where our technology will become part of our self."

(Except for the headline, this story has not been edited by NDTV staff and is published from a syndicated feed.)

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A $50,000 Helmet Can Read User's Mind. And It's Ready - NDTV

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Global Cell and Gene Therapy Manufacturing Market Report 2021-2025: Expected Capacity Crunch, Investments for CGT Capacity Expansion, Events Driving…

Posted: June 23, 2021 at 1:54 am

DUBLIN, June 21, 2021 /PRNewswire/ -- The "The Market for Cell and Gene Therapy Manufacturing - The Rise of CMOs & CDMOs (2021)" report has been added to ResearchAndMarkets.com's offering.

Funding the Cell and Gene Therapy Boom

The rapid ascension of cell and gene therapies (CGT) has created a crucial turning point in the history of modern medicine. Not only have new therapies such as CAR-T produced a shift towards highly individualized medicine and a powerful new front in the war against cancer, but the rapidity of change with the CGT sector has taken the world by storm.

By 2025, the FDA has indicated that it will be reviewing an estimated 10 to 20 of CGTs per year, while the European Medicines Agency (EMA), has estimated that it will soon be approving as many as a dozen such therapies per year.

With the hard-earned marketing approvals gained by cell and gene therapy companies in recent years, biopharmaceutical companies have substantially increased their interest in the CGT sector. Some biopharma companies are resorting to increasing their manufacturing capacity by investing in expansion and new facilities, while others are partnering with third-party contract manufacturing organizations to outsource this work.

With the future in mind, contract manufacturing organizations (CMOs) and contract development and manufacturing organizations (CDMOs) are also investing heavily in expanding their manufacturing capacity to be able to take on new clients and larger, later-stage projects as the market expands.

Currently, the cell and gene therapy field is currently on track for a massive capacity shortage. In particular, there is likely to be a shortage of manufacturing capacity at the commercial scale. Although approximately 90% of cell and gene therapy developers would prefer to use CMOs, related CMO capacity is not available within the industry.

Moreover, the lead time for CMOs to begin cell and gene therapy projects averages over 18 months. Thus, CGT developers are often forced to expand their in-house capacity. Of course, CMOs are also investing heavily into the expansion of their cell and gene therapy manufacturing capabilities, but these large build-outs take time.

This report is tailed to the strategic needs of cell and gene therapy (CGT) companies, as well as the CMOs and CDMOs who are handling third-party manufacturing services for these clients. With the competitive nature of this global market, you don't have the time to do the research. Claim this report to become immediately informed, without sacrificing hours of unnecessary research or missing critical opportunities.

In compiling this report, the research analysts leveraged nearly a decade of historical data on the cell and gene therapy manufacturing industry. In addition to conducting extensive secondary research, our analysts interviewed dozens of highly regarded industry leaders.

Key Topics Covered:

1. Funding the Cell and Gene Therapy Boom1.1 Recent CMO/CDMO Expansions in Cell and Gene Therapy Sector1.1.1 Expected Capacity Crunch1.1.2 Investments for CGT Capacity Expansion1.1.3 Other CGT Manufacturing Investments1.1.4 Events Driving Expansion for CGT Facilities and Infrastructure

2. Recent Merger and Acquisition (M&A) Deals within the Cell and Gene Therapy Sector2.1 Charles River Laboratories International/Retrogenix2.2 Charles River Laboratories International/Cognate BioServices2.3 Beam Therapeutics/Guide Therapeutics2.4 Thermo Fisher Scientific/Henogen2.5 Eli Lilly/Prevail Therapeutics2.6 Novartis/Vedere Bio2.7 Bayer/Asklepios2.8 AGC Biologics/MolMed2.9 Sorrento Therapeutics/SmartPharm2.10 Catalent/MaSTherCell2.11 ElevateBio2.12 Ixaka

3. Financing Rounds3.1 Major Private Placements3.1.1 Orchard Therapeutics3.1.2 Amryt3.1.3 BioNTech3.1.4 Kiadis Pharma3.2 Major Venture Capital Financings3.2.1 ElevateBio3.2.2 Century Therapeutics3.2.3 Artiva Biotherapeutics, Inc.3.2.4 Vineti, Inc.3.2.5 Ori Biotech Ltd.3.2.6 Neogene Therapeutics3.2.7 Forge Biologics3.2.8 Sana Biotechnology3.2.9 Orca Bio3.2.10 Freeline Therapeutics3.2.11 Poseida Therapeutics3.2.12 Kriya Therapeutics3.2.13 Legend Biotech Corporation3.2.14 Lyell Immunopharma3.2.15 Mammoth Biosciences3.2.16 Kyverna Therapeutics3.3 Major Corporate Partnerships within the CGT Market3.3.1 Bayer/Atara Biotherapeutics3.3.2 Novartis/Mesoblast3.3.3 Novartis/Sangamo3.3.4 Janssen Biotech/Fate Therapeutics3.3.5 Biogen/Sangamo3.4 Follow-on Public Offerings by CGT Companies3.4.1 Rocket Pharmaceuticals3.4.2 Adaptimmune Therapeutics3.4.3 Allogene Therapeutics3.4.4 Iovance Biotherapeutics, Inc.3.4.5 Bluebird bio3.5 Initial Public Offerings (IPO)3.5.1 Legend Biotech3.5.2 Decibel Therapeutics3.5.3 Sana Biotechnology3.5.4 Rocket Pharmaceuticals3.5.5 JW Therapeutics3.5.6 AlloVir3.5.7 Akouos3.5.8 Generation Bio3.5.9 Passage Bio3.5.10 Beam Therapeutics

4. Market Size for Cell and Gene Therapy CMO/CDMOs

5. Profiles of Cell and Gene Therapy CMOs and CDMOs5.1 3P Biopharmaceuticals5.2 ABL, Inc.5.3 AGC Biologics5.4 Advent BioServices Ltd.5.5 Akron Biotech5.6 Aldevron5.7 Anemocyte S.r.l5.8 Applied Viromics5.9 ATVIO Biotech, Ltd./Orgenesis Biotech Israel, Ltd.5.10 Austrianova5.11 Avid Bioservices, Inc.5.12 Batavia Biosciences B.V.5.13 Bio Elpida5.14 BioCentriq5.15 BioNTech IMFS GmbH5.16 BioReliance Corporation/Merck Millipore5.17 Bio-Techne5.18 Biovian Oy5.19 Boehringer Ingelheim BioXcellence5.20 Brammer Bio/Thermo Fisher Scientific5.21 C3i5.22 Catalent Biologics5.23 CATAPULT5.24 CCRM5.25 Cell Therapies Pty Ltd.5.26 CELLforCURE (Novartis)5.27 Celonic AG5.28 Cellular Therapeutics Ltd.5.29 Center for Breakthrough Medicines5.30 Charles River Laboratories International, Inc.5.31 Cobra Biologics5.32 Cognate BioServices5.33 Delphi Genetics S.A.5.34 ElevateBio5.35 Emergent BioSolutions5.36 Eurogentec5.37 Exothera5.38 FinVector5.39 Flash Therapeutics5.40 Fraunhofer Institute for Cell Therapy and Immunology5.41 FUJIFILM Cellular Dynamics, Inc.5.42 FUJIFILM Diosynth Biotechnologies5.43 GenScript Biotech Corporation5.44 KBI Biopharma5.45 Lonza Group Ltd.5.45.2 Services5.47 Matica Biotechnology, Inc.5.48 Medinet Co., Ltd.5.49 Minaris Regenerative Medicine, LLC5.50 MolMed S.p.A5.51 NECSTGEN5.52 NEOBIOSIS, LLC5.53 NIKON CeLL Innovation Co., Ltd.5.54 Ology Bioservices, Inc.5.55 OrganaBio5.56 Orgenesis5.57 OxfordBiomedica plc5.58 Patheon/Thermo Fisher Scientific5.59 Performance Cell Manufacturing5.60 Q-Gen Cell Therapeutics5.61 RoslinCT5.62 Samsung Biologics5.63 SK biotek5.64 Stanford Laboratory for Cell and Gene Medicine5.65 Stemmatters, Biotechnologia e Medicina Regenerativa S.A.5.66 Takara Bio Europe SAS5.67 TUM Cells5.68 Interdisciplinary Stem Cell Institute/Miami University5.69 Upstate Stem Cell cGMP Facility5.70 VGXI, Inc.5.71 Vigene Biosciences, Inc.5.72 Waisman Biomanufacturing5.73 Yposkesi

For more information about this report visit https://www.researchandmarkets.com/r/3ef6qt

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Research and Markets Laura Wood, Senior Manager [emailprotected]

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Global Cell and Gene Therapy Manufacturing Market Report 2021-2025: Expected Capacity Crunch, Investments for CGT Capacity Expansion, Events Driving...

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Rare procedure to treat pancreatitis creates ‘super liver’ by transplanting pancreatic cells – CTV News

Posted: June 6, 2021 at 1:50 am

TORONTO -- Amid a rise in people developing a painful condition called pancreatitis, doctors are looking to a rare procedure to help patients with this chronic pain creating a super liver using the patients own cells.

The pancreas, located behind the stomach, produces enzymes that assist with digestion, as well as hormones that help you to process sugars. Pancreatitis is a condition that interferes with the functioning of the pancreas, and it can come on suddenly and intensely or can be a chronic problem.

For 27-year-old Kaitlin Saari, who was diagnosed with pancreatitis last summer in the middle of the pandemic, the condition was debilitating.

I just started to get sicker and sicker, she told CTV News. I was in much more excruciating pain after anything would go into my mouth, like even drinking water.

While some people with mild cases of pancreatitis get better on their own, severe cases can be life-threatening.

Saari was experiencing nausea and vomiting regularly. She had no energy, and was unable to go for walks or see friends.

I couldn't even get into the bed, some days, she said. I don't have life with it.

Her condition worsened until she wound up in hospital, during COVID-19 lockdowns. She was dealing with serious pain and was in hospital for weeks on end, only able to leave for a few days before she was hospitalized again, relying on heavy narcotics to manage the pain.

I lived in the hospital, Saari said.

It was clear that something needed to done.

The answer came in a rare procedure called total pancreatectomy and islet autotransplantation.

Saari was the first of two patients treated at Toronto General Hospital, and one of the first in Ontario, to receive the procedure, the first step of which involved removing her inflamed pancreas in a laborious, 12-hour operation.

But removing the pancreas creates another problem without a pancreas, patients become diabetic, requiring insulin for life.

Thats why, in this new procedure, surgery to remove the pancreas is only the first step.

The lab technicians isolate the pancreatic cells, and then return them to the operating room, where the surgeon injects them into the patients liver.

Within the liver, these cells take root and grow.

Dr. James Shapiro, Canada Research Chair in Transplant Surgery and Regenerative Medicine, told CTV News that these islet autotransplants are a very unique situation for surgery.

Because we're taking out somebody's pancreas that is diseased, we're extracting the cells that make insulin, which is about one to two per cent of the pancreas, and we're putting those cells back into the liver, he said.

And because they're the patient's own cells, the body will accept them without need for any anti-rejection drugs, so it's a very unique situation. They're very precious cells.

Saari is a success story, among the 70 per cent of cases where the new "super liver" starts to also produce some or all of the insulin her body requires.

I'm completely insulin independent now, Saari said. It's fantastic. My blood sugar stayed [] normal every time that was tested.

Hospital officials say the second patient is also doing well though still requires some daily insulin injections

The incidence of pancreatitis has been increasing worldwide, and doctors arent sure why. There are over 116,500 new cases of pancreatitis in Canada now, representing a 75-per-cent increase in its incidence since 1990.

In 2016, there were 474 deaths from this condition, making the development of treatments all the more vital.

This super liver procedure was first offered in Alberta, and Shapiro said patients would fly in from across the country to receive this unique operation. They operate on around 10-15 people per year, including small children.

We have a two-year-old child who remains off insulin today with excellent sugar control, Shapiro said.

The procedure is also being done in Toronto now, something that Dr. Trevor Reichman, a Toronto surgeon, calls exciting.

There's a group of patients that don't have a lot of options as far as long-term durable treatment outside of basically being dependent on narcotics, he told CTV News. "So this is a real opportunity to take care of a very challenging group of patients and really offer them a long-term durable treatment in a way to sort of get their life back after.

Patients dont have any pain afterwards and can continue with their normal lives, he said, making it a life-changing procedure.

This method also has implications for the broader medical world, potentially opening up avenues for treating diabetes itself, or for using a patients own cells to repair organs.

It's an exciting, very exciting area, Dr. Bradly Wouters, executive vice president of science and research at the University Health Network, told CTV News.

It's in the larger area of what we call cell and regenerative medicine, its the idea of being able to repair or regenerate tissues.

Right now, this operation is only available to those who are suffering from chronic pain as a result of pancreatitis. But Wouters explained that if we could transplant these pancreatic islet cells into those with diabetes, it could potentially allow those patients to make their own insulin.

Its something researchers are looking into in Alberta, exploring how a patients stem cells might be extracted, grown into these pancreatic islet cells and re-injected to help the body make insulin.

In theory, if we make enough cells, you could potentially cure Type 1 and Type 2 diabetes, he said. And the process now of simplifying that process and scaling it up is the challenge that we're working on in the lab.

While receiving a super liver may not be the answer for everyone suffering from pancreatitis, its undeniably a step towards a world where our own cells could help replace the function of failing or diseased organs.

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Rare procedure to treat pancreatitis creates 'super liver' by transplanting pancreatic cells - CTV News

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