World-renowned ALS Researcher, Clinician to Receive MDA Award

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by Marta Figueiredo, PhD |

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Stanley H. Appel, a world-renowned expert in amyotrophic lateral sclerosis (ALS) and the founder of the first multidisciplinary clinic dedicated to ALS care and research in the U.S., will receive the Tribute Award from the Muscular Dystrophy Association (MDA).

The award acknowledges the neurologist’s work in pioneering ALS research and care, and celebrates the 40th anniversary of that same clinic, the Houston Methodist Neurological Institute’s MDA ALS Research and Clinical Center, where he serves as director.

“Dr. Appel helped mold our organization into the number one voluntary health organization in the US for people living with muscular dystrophy, ALS, and related neuromuscular diseases,” Donald S. Wood, PhD, MDA’s president and CEO, said in a press release.

“We celebrate Dr. Appel for his revolutionary research and his compassion for people living with ALS,” Wood said.

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MDA will recognize Appel at the MDA ALS Research and Clinical Center, among the community he’s served for 40 years, on June 2, which is designated “Lou Gehrig Day” by Major League Baseball.

The recognition will be part of the association’s Tribute Tour, celebrating local Houston champions, Care Centers, partners, volunteers, and families. The tour commenced in Tennessee before arriving in Houston and will continue to St. Louis over Labor Day.

An internationally renowned researcher and neurologist, Appel has spent more than 40 years focused on understanding the human brain and neurodegenerative diseases such as ALS, Parkinson’s disease, and Alzheimer’s disease.

He is one of the foremost ALS experts in the U.S. and was the first to demonstrate the neuroprotective role of regulatory T-cells (Tregs) in ALS progression. Tregs are a type of immune cell that typically dampens other immune responses.

Appel’s laboratory is focused on finding ways to increase the protective effects of Tregs and anti-inflammatory microglia, the brain’s immune cells, in people with neurodegenerative conditions.

Appel is also the director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist. He was a member of MDA’s board of directors from 1979 to 2019 and now serves as Emeritus board member.

The neurologist has received at least 42 individual grant awards from MDA, including a current award to evaluate a Treg-based therapy, called ALS001, in a clinical trial for ALS.

ALS001, developed by Coya Therapeutics based on Appel’s research, involves isolating Tregs from a patient, growing them in the lab, and then infusing the cells back into the person.

In an Appel-sponsored Phase 1 trial (NCT03241784) involving three ALS patients, the therapy was found to be generally well tolerated and to reduce markers of oxidative stress, a type of cellular damage, and inflammation — both implicated in ALS. Additional data suggest these reductions were accompanied by periods of disease stabilization in these patients.

These promising findings supported the launch of a Phase 2a trial (NCT04055623), which tested the therapy, given once a month for six months, against a placebo in about 12 people with ALS.

Participants completing this part could chose to enter an open-label extension phase, wherein all will receive monthly ALS001 infusions for six months. While enrollment in this trial was compromised by the pandemic, results from eight patients in the extension part showed promising safety and efficacy findings.

The company is now planning a larger clinical trial for later this year.

“For over 70 years, MDA has led the way, investing over $173 million in innovations in ALS science and care, and it is largely thanks to Dr. Appel’s invaluable service that we have made such inroads in research today,” Wood said.