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Grant - Additional Grants 2019 - ALS - Stanley Appel, MD

Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of the Stanley H. Appel Department of Neurology and the Peggy and Gary Edwards Distinguished Chair in ALS at Houston Methodist Hospital, and professor of Neurology at Weill Cornell Medical College, was awarded an MDA human clinical trial grant totaling $558,800 over two-and-a-half years to expand the first-in-human study that demonstrated promising results leveraging patients’ own immune cells to treat amyotrophic lateral sclerosis (ALS). In ALS, muscle-controlling nerve cells called motor neurons are progressively lost, eventually causing muscles to become nonfunctional.
The award is part of a larger grant from The ALS Association, ALS Finding a Cure (ALSFAC), and MDA, which jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The clinical trial grant is a collaborative study between the ALS team at Houston Methodist Hospital led by principal investigator Dr. Appel and the ALS team at Mass General Hospital led by Merit Cudkowicz, MD, chief of Neurology at Mass General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School.
Regulatory T-lymphocytes (Tregs) are a type of T-lymphocyte (a white blood cell that plays a central role in the immune response) that reduces immune system activation. Tregs are decreased in both numbers and function in the blood of people with ALS. Building on positive preclinical results showing that it was possible to restore Treg function outside the body, the first clinical trial in humans demonstrated that Tregs could be extracted from people living with ALS, expanded in vitro, and then safely infused back into the same patients, with three treated patients showing improved Treg suppressive function and slowed disease progression. The phase 2a clinical trial led by Dr. Appel will expand on the results of the first trial with participants receiving infusions of expanded regulatory (EPAR) T-cells followed by an ascending-dose, open-label extension trial.
https://doi.org/10.55762/pc.gr.81882
Grantee: ALS - Stanley Appel, MD
Grant type: Human Clinical Trial Grant
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