Category Archives: Cell Medicine

UC San Francisco has launched the Baszucki Lymphoma Therapeutics Initiative to increase the effectiveness and availability of chimeric antigen receptor T-cell (CAR T) therapy for lymphoma patients. Jan Ellison Baszucki and her husband, Roblox CEO David Baszucki, gifted $6 million over five years to support the initiative. After witnessing Jans father recover from a near-fatal lymphoma thanks to treatment with CAR T therapy, the couple wanted to help accelerate progress in the field so more families can benefit.

An emerging approach to immunotherapy, CAR T therapy has dramatically increased survival rates for patients with certain hard-to-treat blood cancers. It also shows promise for treating some solid tumors as well as diseases beyond cancer, including autoimmunity and neurodegeneration. The therapy involves harvesting T cells from a patients blood and genetically engineering them to kill cancer cells by detecting specific molecular targets, called antigens, on the cancer cells surface. The engineered T cells are then cultured to increase their numbers and infused back into the patients bloodstream, where they act as a living therapeutic against the cancer cells.

In cancer, we dont often use the big C word cure but CAR T therapy really does seem to lead to miracle cures after no other therapies have worked, says Babis Andreadis, MD, MSCE, a lymphoma specialist and professor of clinical medicine at UCSF who will direct the new initiative.

Since 2017, the US Food and Drug Administration (FDA) has approved five CAR T therapies for acute lymphoblastic leukemia (ALL), multiple myeloma, and several lymphomas. Andreadis ran some of the first clinical trials of CAR T therapies for non-Hodgkins lymphoma, which have shown complete remission rates of around 50 percent in patients who have failed multiple prior treatments.

In cancer, we dont often use the big C word cure but CAR-T therapy really does seem to lead to miracle cures after no other therapies have worked.

Babis Andreadis, MD, MSCE

The Baszucki Lymphoma Therapeutics Initiative will expand research underway at UCSF aimed at understanding who will benefit from CAR T therapy and developing new therapeutic targets for lymphoma patients who dont respond to current CAR T therapies.

Our goal is to reach a point when cancer medicine is completely personalized and every patient can get a therapy designed specifically for their own disease, says Alan Ashworth, PhD, FRS, president of the UCSF Helen Diller Family Comprehensive Cancer Center and the E. Dixon Heise Distinguished Professor of Oncology. Jan and Davids commitment to helping realize this vision exemplifies their remarkable foresight and generosity.

The Baszuckis, who live in San Francisco with their four children, are veterans of the Bay Area technology industry. Jan was the director of marketing at Infinity Financial Technology before becoming an award-winning writer and novelist. David is a software engineer and co-founder of Roblox Corporation, which develops Roblox, an online gaming platform that allows users to create and play their own games.

After the company went public, in March 2021, the couple launched the Baszucki Group to create foundational change through philanthropy, impact investing, community building, and other initiatives. One of the organizations first projects was establishing the Baszucki Brain Research Fund, which supports scientists and innovators pursuing breakthrough treatments that promote vitality, stability, and well-being in people at risk for or living with bipolar disorder. This year, the fund has invested more than $13 million in research grants, including a competitive program that will fund 45 grants to support pilot research in therapeutic discovery and translational research for bipolar disorder.

Our goal with Baszucki Group is to direct resources toward the greater good, Jan Baszucki says. Were interested in big, bold moves that will leapfrog current treatments rather than make incremental progress. Its obvious that CAR T therapy fits into that category.

One of the exciting things about CAR T is its not just one product, David Baszucki adds. Its a revolutionary therapeutic platform that offers infinite avenues for innovation, and Dr. Andreadis is one of those brilliant researchers who can use it to make CAR T therapy a reality for more families like ours.

The new initiative will also further the goals of the UCSF Living Therapeutics Initiative (LTI), which launched in June. By uniting many established UCSF programs, the LTI seeks to propel research in living therapeutics a broad category of medicines that use human or microbial cells to treat disease and speed promising therapies to clinical trials for patients who lack effective treatment options.

UCSF is laying the groundwork for the next frontier in pharmaceutical medicine, says Sam Hawgood, MBBS, UCSF chancellor and the Arthur and Toni Rembe Rock Distinguished Professor. We are deeply grateful to the Baszucki family and other philanthropic partners who inspire everyone engaged in our mission to treat patients compassionately and equitably with the best therapies they can get anywhere in the world.

The Baszuckis first met Andreadis in October 2020, after Jans father, Todd Ellison also an award-winning novelist, who writes under the pen name E.T. Ellison had endured three years of chemotherapy while only growing sicker. I was told I had no options left, Ellison says. Thats when his oncologist, who had trained at UCSF, referred him to Andreadis.

Earlier that fall, Andreadis had launched a study to test the viability of administering CAR T therapies manufactured in a university setting. Clinical researchers have previously partnered with pharmaceutical companies to use CAR T products already on the market or in development for commercialization. These products typically are manufactured by research contractors or by the companies themselves because they require specialized clean rooms, called current good manufacturing practice (cGMP) facilities, to ensure that the drugs are safe and of the highest quality.

However, some universities recently have begun making CAR T and other cell-based therapies in house. In June of this year, as part of the LTI, UCSF announced a strategic alliance with Thermo Fisher Scientific Inc. to build and manage a cGMP facility in leased space on UCSFs Mission Bay campus. The new facility will allow clinicians to offer cell therapies to more patients, including those who may be ineligible for or cant afford commercial products. In-house manufacturing also will allow researchers to optimize production specifications and doses and develop new therapeutic targets that drug companies might not pursue.

The CAR T products available today are designed for generic targets, Andreadis says, adding that while this approach maximizes the number of patients who are likely to respond to a particular CAR T product, it also means that patients whose cancers express low levels of these targets might not benefit.

Andreadiss study is a phase I trial to test the safety and appropriate dose of CAR T cells manufactured under custom protocols to target a common FDA-approved antigen known as CD19. (Until the cGMP facility at UCSF is operational, Andreadiss team is making the cells at a facility at UC Davis.) Ellison was the first patient to enroll. When he arrived here, he was seriously ill, Andreadis says.

Within a week of starting the treatment, however, Ellison began to improve. We kept waiting for him to get worse, but he just got better and better, Jan Baszucki recalls. And there were zero side effects, none at all, Ellison adds. By December 2021, his lymphoma was gone, and he remains cancer-free. (Not all CAR T patients have the same experience; some patients may experience short- or long-term side effects, including cytokine release syndrome and neurotoxicity.)

We are beyond grateful to Dr. Andreadis and his staff for the incredible care given to my father, Jan Baszucki says.

Andreadiss team has since treated five more patients in the phase I trial, several of whom had lymphomas that couldnt be treated with commercial CAR T products or who would not have qualified for insurance coverage because they were too sick. With the Baszuckis support, the team can now expand the study to enroll more patients with various lymphomas, including rare subtypes. They also plan to use patients blood samples to begin investigating new disease targets for the next generation of CAR T therapies.

Beyond improving treatment options for lymphoma patients, Andreadis predicts, this pioneering work will help set the stage for the development of in-house CAR T products for other cancers and diseases. Clinicians and scientists around the world will be able to use the infrastructure we are creating here at UCSF to customize CAR T therapies for a broad range of applications, he says.

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$6M Gift from Family of Roblox Creator Launches New Initiative to Advance CAR T Therapy - UCSF News Services

NEW YORK and SOMERSET, N.J., Dec. 7, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, and Catalent (NYSE: CTLT), a global leader in enabling biopharma, cell, gene and consumer health partners to optimize development, launch, and supply of better patient treatments across multiple modalities, today announced that the technology transfer for NurOwn manufacturing at Catalent's facility has been finalized. NurOwn is BrainStorm's autologous cellular therapy being developed for the treatment of amyotrophic lateral sclerosis (ALS), progressive multiple sclerosis (PMS) and other neurodegenerative diseases.

Catalent entered into a partnership with Brainstorm in 2020 to provide CGMP clinical supply of NurOwn, in anticipation of the product candidate's potential regulatory approval. NurOwn will be manufactured at Catalent's world-class 32,000 square-foot cell therapy manufacturing facility in Houston, Texas.

"The successful completion of this technology transfer with Catalent is an important step in establishing manufacturing preparedness for NurOwn," said Chaim Lebovits, Chief Executive Officer, Brainstorm Cell Therapeutics. "The manufacturing of cellular therapies such as NurOwn is complex, and requires careful planning and very specific expertise. We are very pleased with the progress we have made with our partner Catalent, which has industry-leading capabilities in this area."

Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy, said, "Our extensive experience in cell therapy development and scale-up was key to the completion of this technology transfer to our state-of-the-art cell therapy facility in Houston, Texas. We look forward to continuing our partnership with BrainStorm and are committed to enabling the advancement of their autologous stem cell therapy product candidate toward a potential future commercial launch."

About NurOwn

The NurOwn technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About Catalent

Catalent is the global leader in enabling pharma, biotech, and consumer health partners to optimize product development, launch, and full life-cycle supply for patients around the world.

With broad and deep scale and expertise in development sciences, delivery technologies, and multi-modality manufacturing, Catalent is a preferred industry partner for personalized medicines, consumer health brand extensions, and blockbuster drugs. Catalent helps accelerate over 1,000 partner programs and launch over 150 new products every year. Its flexible manufacturing platforms at over 50 global sites supply over 70 billion doses of more than 7,000 products to over 1,000 customers annually.

Catalent's expert workforce exceeds 17,000, including more than 2,500 scientists and technicians. Headquartered in Somerset, New Jersey, the company generated $4 billion in revenue in its 2021 fiscal year. For more information, visitwww.catalent.com.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Contacts:

Brainstorm Contacts

Investor Relations:John MullalyLifeSci Advisors, LLCPhone: +1 617-429-3548jmullaly@lifesciadvisors.com

Media:Mariesa Kemblekemblem@mac.com

Catalent Media Contact:Chris HallingPhone: +44 (0)7580 041073 chris.halling@catalent.com

View original content:https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-and-catalent-announce-completion-of-technology-transfer-for-nurown-manufacturing-301438701.html

SOURCE Brainstorm Cell Therapeutics Inc

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BrainStorm Cell Therapeutics and Catalent Announce Completion of Technology Transfer for NurOwn Manufacturing - BioSpace

Nobel laureate Shinya Yamanaka will step down as director of Kyoto Universitys Center for iPS Cell Research and Application (CiRA) by the end of March to focus on my own research.

The university announced on Dec. 8 that Jun Takahashi, a CiRA professor who studies Parkinsons disease treatments using induced pluripotent stem (iPS) cells, will become the next CiRA director.

Yamanaka, who won the Nobel Prize in Physiology or Medicine in 2012 for his groundbreaking research on iPS cells, will continue his studies as a professor at CiRA.

In the past few years, I have wanted more and more to focus on my own research, Yamanaka said in a statement released on Dec. 8. As a basic researcher, I will do my best to contribute to the development of iPS cell research, medicine and biology.

According to the university, Yamanaka was considering retiring at the end of his current term and recommended Takahashi as his successor at a meeting of the CiRA Faculty Council on Dec. 2.

Takahashi was elected the next director by a majority vote at the meeting.

Using this (iPS cell) technology and other technologies, I will promote research and medical applications with CiRA faculty, staff and students to contribute to future medicine and life sciences, Takahashi said in a statement issued on Dec. 8.

Yamanaka, now in his sixth term, has served as director of CiRA since it was established in 2010.

Takahashis term as director will start in April and continue until the end of March 2024.

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Nobel laureate Yamanaka to retire as director of iPS cell center | The Asahi Shimbun: Breaking News, Japan News and Analysis - Asahi Shimbun

Passage Of California Stem Cell Proposition Boosts Research

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

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

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

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

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

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

Drapeau explains the concept for us;

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

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

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

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

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

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

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

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

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

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

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

Drapeau explains why this is so exciting;

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

Christian Drapeau

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

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

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

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

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

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

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

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

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

-Initiation of patient enrollment expected by year-end-

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

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

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

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

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

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

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

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

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

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

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

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

Dr Pengyi Yang uses computational expertise to build virtual cells.

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

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

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

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

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

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

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

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

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

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

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

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Dr Pengyi Yang wins National Stem Cell Foundation Metcalf Prize - News - The University of Sydney

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Its like a little smart bomb, says Sartor.

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

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

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

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

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

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

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

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

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

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

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

But providers now have another option.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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SC21- 21st century cellular medicines specialists - The Thaiger

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

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

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

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

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

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

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

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

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

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

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

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

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

The National Institutes of Health funded this research.

Media contact: Mark Derewicz, 919-923-0959

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