The ALS Association, ALS Finding a Cure (ALSFAC), and MDA have jointly awarded a $2.5 million clinical trial grant to Dr. Stanley Appel and his team of researchers at the Houston Methodist Neurological Institute, and Dr. Merit Cudkowicz and his team at Massachusetts General Hospital.
The funding award will allow Dr. Appel to expand the first-in-human study that demonstrated promising results leveraging patients’ own immune cells to treat ALS.
“We are proud to support a dedicated community of scientists and clinicians who are refining promising research approaches in the exciting new field of neuro-immunology to further define the causes of ALS and develop strategies to treat or block the disease process.”
-Peter Foss, president of ALSFAC
“Dr. Appel’s work gives us great hope. We are grateful to Dr. Appel and Dr. Jason Thonhoff [at Houston Methodist] as well as all the scientists in the lab, and especially grateful to the volunteers in the studies. These volunteers are putting themselves on the front line of science to fight ALS.”
-Neil Thakur, executive vice president of mission strategy at The ALS Association
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. Because Tregs are reduced in people with ALS, harmful immune processes can occur. In fact, people with more rapid ALS progression have even lower Treg function.
Building on positive preclinical results showing that it was possible to restore Treg function outside of 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. In the three people who received this treatment with “expanded Tregs” in the first trial, a significant improvement in their Treg suppressive function was observed, as well as an apparent slowing of disease progression.
“Now is a time of unprecedented promise for the field of ALS research and drug development,” Dr. Cudkowicz says. “This collaboration among the three foundations has made possible the support needed to conduct a trial of this nature. It is only with this support that we will be able to continue to explore the possibility of harnessing the immune system to slow disease progression in ALS — possibly bringing us closer to a breakthrough that may one day lead to a cure.”