Welcome to our September 2024 newsletter.
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FDA approves first engineered TCR therapy for cancer.
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The U.S. Food and Drug Administration (FDA) made history last month by approving the first engineered T-cell receptor (TCR) therapy for cancer. TCR therapy is a type of cell and gene therapy that engineers a patient’s own T cells to attack cancer more effectively.
Tecelra (afamitresgene autoleucel), developed by biotech company Adaptimmune, is approved to treat adults with unresectable or metastatic synovial sarcoma. This type of cancer forms in soft tissues and affects approximately 1,000 people in the United States each year.
The authorization of Tecelra marks two additional cell therapies approved in 2024 to treat solid tumors. The FDA approved the T-cell therapy Amtagvi (lifileucel) in February to treat advanced melanoma, a skin cancer diagnosed in approximately 100,000 people in the U.S. each year. Amtagvi, a tumor-infiltrating lymphocytes (TIL) therapy, expands the number of immune cells capable of attacking the patient’s tumor.
There are now 11 cancer cell and gene therapies approved by the FDA.
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Childhood Cancer Awareness Month: ACGT’s investment in pediatric cancer research.
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Since 1970, the cancer death rate for children and adolescents has declined by more than 50%. This progress is thanks to advancements in detection and treatment.
Despite this improvement, work still needs to be done to better the odds that all children survive a diagnosis. ACGT is committed to funding cell and gene therapy research that leads to cures for all.
ACGT recently awarded a multi-year research grant to Hideho Okada, MD, PhD (University of California, San Francisco), who is testing a promising cell and gene therapy in a phase 1 clinical trial for children with a rare brain tumor. The therapy, called an engineered T-cell receptor, genetically modifies a patient’s own T cells to improve the immune system’s ability to fight cancer.
If Dr. Okada’s therapy is successful, it could translate to the development of similar cell and gene therapies for other pediatric cancers.
Read more about Dr. Okada’s pediatric cancer research.
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Highlighting other pediatric cancer grants from ACGT.
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ACGT has a proven track record of funding pediatric cancer research. Below are two recent ACGT research grants awarded to focus specifically on childhood cancers:
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Stephen Gottschalk, MD (St. Jude Children’s Research Hospital), developed CAR T cells for pediatric sarcomas, a class of cancers forming in bones and connective tissues. CAR T cells are immune cells engineered to target cancer cells carrying a specific protein. Dr. Gottschalk is planning a clinical trial to test the therapy.
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Crystal Mackall, MD (Stanford University), developed CAR T cells for patients with the bone cancer osteosarcoma and the nerve cell cancer neuroblastoma. This ACGT-funded research translated into a CAR T-cell therapy for children with diffuse intrinsic pontine glioma, a rare brain tumor with no available treatments. The therapies are currently being tested in clinical trials.
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A new approach proposed to advance cell and gene therapies for children.
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The medical field has witnessed the development of several exciting new pediatric cell and gene therapies, but there is broad consensus that the standard model for drug development via the biopharmaceutical industry will not deliver the breadth of new pediatric cell and gene therapies that scientific progress is making possible.
In a recent Nature Medicine article titled “Enhancing pediatric access to cell and gene therapies,” ACGT Research Fellow and Scientific Advisory Council member Crystal Mackall, MD (Stanford University), outlines a new approach to advancing cell and gene therapies for children.
Dr. Mackall and others propose the creation of a new entity, the Pediatric Advanced Medicines Biotech, to lead late-stage development and commercialize pediatric cell and gene therapies – no longer depending on the biopharmaceutical model in the United States.
Read the Nature Medicine article.
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Leukemia and Lymphoma Awareness Month: The impact of ACGT funding.
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Today, there are four cell and gene therapies approved by the FDA to treat certain types of leukemia or lymphoma, giving patients new hope that their blood cancer can be defeated. These approvals trace back to cancer research breakthroughs sparked by ACGT funding.
In 2004, the foundation awarded research grants to Carl June, MD (University of Pennsylvania), and Michel Sadelain, MD, PhD (Memorial Sloan Kettering Cancer Center), whose work led to the first two FDA-approved CAR T-cell therapies for cancer: Kymriah and Yescarta.
CAR T-cell therapy, a type of cell and gene therapy, engineers a patient’s own T cells to attack cancer more effectively. The therapy has been curative for certain patients and is seen as a transformative breakthrough in cancer medicine.
This monumental leap forward in transforming cancer treatment was made possible through funding from ACGT to conduct the first ever clinical trials with CAR T cells in leukemia patients.
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Be a part of the momentum: Support critical next steps in cancer research.
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The generosity of ACGT’s donors is vital in addressing the funding gap between groundbreaking scientific discoveries and the clinical trials necessary to bring new life-saving therapies to patients. This gap often delays the development of transformative cancer treatments.
By supporting ACGT today, you enable pioneering researchers to advance therapies from pre-clinical studies to early-phase clinical trials, accelerating the journey from the lab to patients. These efforts are crucial in the fight against cancers that resist traditional treatments, and your gift can make a tangible difference.
As we celebrate recent FDA approvals of cell and gene therapies and highlight new initiatives in pediatric brain cancers, we urge you to help us continue this vital work. Your support today could lead to tomorrow’s cures.
Make a gift to ACGT with confidence and ease. Backed by the highest charitable ratings and knowledge that 100% of your gift directly supports ACGT’s programs and research, you can donate using cash, stocks, donor-advised funds, IRA rollovers and more to make supporting life-saving research straightforward and impactful.
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ACGT stakeholder spotlight: Board member Chau Q. Khuong.
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Chau Q. Khuong, a biotech entrepreneur and venture capital investor, joined the ACGT Board of Directors in 2016. Since then, he has provided governance, advocacy and stewardship to help the philanthropic foundation advance novel cancer research.
From 2003-2021, Mr. Khuong was a Private Equity Partner at OrbiMed Advisors, a global healthcare-dedicated investment firm, where he was a partner and member of the investment committee for the OrbiMed venture capital funds. During the past two decades, he has been an active lead investor in innovative drug development and medical device enterprises across all stages and therapeutic areas including oncology, infectious diseases, ophthalmology, gene therapy and gene editing.
Mr. Khuong earned a bachelor’s degree in molecular, cellular and developmental biology with a concentration in biotechnology and a Master of Public Health degree with a concentration in infectious diseases, both from Yale University. He currently serves or has served on the Board of Directors for numerous public and private companies, including Arius Research (acquired by Roche), BELLUS Health, Durata (acquired by Actavis), Fusion Pharma, Glaukos Medical, Intellia, Intercept, Inspire Medical Systems, NextCure, Otonomy, Pieris, Rempex and ReViral.
ACGT is grateful for Mr. Khuong’s continued service as an integral member of its Board of Directors.
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Cell and gene therapy guide published for health care providers.
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The International Society for Cell & Gene Therapy (ISCT) recently published “Unproven Cell & Gene Therapies: Guidelines and Resources for Healthcare Providers.” The guide provides information for health care practitioners on how to identify and distinguish safe and approved cell and gene therapy products from those products that do not have a proven record of safety and efficacy or have not received approval from appropriate regulatory organizations.
“As the cell and gene therapy sector continues to grow and these therapies become more common, this resource will be instrumental in ensuring the growth of the sector continues to meet research and clinical standards,” said ACGT Board of Directors member Bruce Levine, PhD (University of Pennsylvania), Chair of the ISCT Committee on the Ethics of Cell and Gene Therapy.
Learn more about this important guide here.
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The latest on cancer cell and gene therapy from around the world.
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