Washington, D.C. (March 19, 2021) –The ALS Association, Muscular Dystrophy Association (MDA) and ALS Finding a Cure® (ALSFAC), provided an additional $1.1 million in supplementary funding to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital for promising research into cell therapy that could slow the progression of amyotrophic lateral sclerosis (ALS). These organizations originally provided $2.4 million in initial funding for this research.
“This additional support underscores just how promising this research is,” said Dr. Kuldip Dave, Vice President of Research at The ALS Association. “While the COVID-19 pandemic has created new challenges, it is critical that we ensure this research can continue.”
The research study is led by Dr. Stan Appel at Houston Methodist and Dr. James Berry at the Sean M. Healey and AMG Center for ALS at Massachusetts General and focuses on regulatory T-lymphocytes, or Tregs, a type of white blood cell that functions to suppress the body’s immune response. Early research had shown that Tregs are reduced in ALS and that lower levels are correlated with a faster progression of the disease. The concept of this research is to extract and isolate Tregs from people with ALS, and to expand them and safely inject them back into the patient.
“Isolation, expansion, and long-term storage of T-regs is cutting edge technology,” said Dr. Merit Cudkowicz, Chief Medical Officer for ALSFAC. “When we learned it was more expensive than researchers initially envisioned, we came together to ensure the work could continue.”
The current study is a phase 2a trial testing the biological activity, measuring biomarkers, and assessing the safety and tolerability of the infusions in eight people with ALS over twelve months. An earlier pilot phase 1 study approved by the FDA tested the therapy on three patients and found the infusions to be safe and well tolerated over a shorter period of time. If the current trial shows Treg infusion to be safe and tolerable, researchers would conduct additional safety and efficacy testing in more people with ALS across multiple clinical sites.
“The early results are incredibly promising, which is why it was so critical to make sure we continue testing to learn whether this therapy can be put on a path to bring it to the patient community,” said Sharon Hesterlee, Ph.D., Executive Vice President and Chief Research Officer for MDA.
The early results have attracted private sector interest in developing the technology. Coya Therapeutics, a clinical stage biotechnology company, recently announced that it had raised $10 million in Series A financing to partner with Dr. Appel and advance his efforts to develop Treg therapy for the treatment of ALS. The ALS Association, MDA and ALSFAC are gratified that our partnership to provide early-stage funding for initial proof of concept clinical testing has led to a drug discovery program focused on bringing this novel therapy forward.
About The ALS Association
The ALS Association is the largest philanthropic funder of ALS research in the world. The Association funds global research collaborations, assists people with ALS and their families through its nationwide network of chapters and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association builds hope and enhances quality of life while urgently searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.als.org.
About the Muscular Dystrophy Association
For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best in class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational opportunities for families and healthcare providers by providing conferences, events, and materials. MDA Advocacy supports equal access for our community, and each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA’s COVID-19 guidelines and virtual events are posted at mda.org/COVID19. For more information, visit mda.org.
About ALS Finding a Cure
ALS Finding a Cure® is a research organization dedicated to being a game changer in discovering a cure for this fatal disease. Founded in 2014 by noted philanthropist and Conair Founder and Chairman, Leandro (Lee) Rizzuto, as a tribute to Christie Rizzuto, Lee’s daughter-in-law who was diagnosed with ALS in 2009 at the age of 41, and under the joint leadership of Denis Rizzuto and Peter N. Foss, ALS Finding a Cure® is focused on identifying the gaps in the scientific understanding of ALS that are preventing the development of a cure. The organization, a program of The Leandro P. Rizzuto Foundation, collaborates with a wide range of companies, ALS organizations, other disease non-profits, and ALS patients and families to ensure research efforts are non-duplicative, synergistic, and focused on the ultimate goal: getting to a cure. For more information about ALS Finding a Cure®, visit www.alsfindingacure.org.