Dr. Stanley Appel, neurologist and co-director of the Houston Methodist Hospital Neurology Institute, has been leading the ALS Clinic at Houston Methodist Hospital since its founding in 1982. This was the first multi-disciplinary clinic dedicated to the treatment of and research on ALS. More than 35 years later, he continues to innovate in the field.
His latest research? An immunotherapy treatment that could stop the progression of ALS in its tracks.
After years of research and studies related to the immune system, Dr. Appel and his team discovered a key component accelerating ALS progression- regulatory T cells. Also known as Tregs, regulatory T cells are immune cells that help protect the body from harmful inflammation that accelerates the progression of ALS.
“We found that many of our ALS patients not only had low levels of Tregs, but also that their Tregs were not functioning properly,” said Appel. “We believed that improving the number and function of Tregs in these patients would affect how their disease progressed.”
The research team discovered that after removing blood from patients, the Tregs returned to normal once outside the body. The phase 1 study included three patients who underwent a process called leukapheresis, a procedure in which blood is removed, and white blood cells are separated from red blood cells to increase the number of each patient’s own Tregs before administering the red blood cells intravenously back into the patients.
The phase 1 study, funded in part by the ALS Association, has shown to stop the progression of ALS in patients, a tremendous feat for a disease with only a few treatments available to slow progression. Phase 2 trials will further evaluate the safety and effectiveness of the treatment, and, hopefully, turn this research into an off-the-shelf cellular therapy for ALS patients.
“Our goal is to give a patient hope for the future. We may not cure a patient’s disease, but we can make a difference.” – Dr. Stanley Appel