AANEM News Express

AANEM News Express

ALS Patients Find Hope in New Study Authored by AANEM Members

There is some hope on the horizon for patients diagnosed with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.  More than a dozen members of the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) were authors on a study announcing the results showing promise in the fight against ALS that appeared in AANEM’s journal Muscle & Nerve. ALS is a fatal neurological disorder that causes a person to lose the ability to control the muscles that allow us to move, speak, breathe, and swallow. The muscles gradually weaken and waste.

The CENTAUR trial shows a drug called AMX0035 resulted in longer survival for people with ALS. Nineteen AANEM members were authors on this groundbreaking study including Drs. Sabrina Paganoni, Michael A. Elliott, Chafic Karam, James B. Caress, Adam Quick, James Wymer, Stephen A. Goutman, Daragh Heitzman, Colin Quinn, Shafeeq Ladha, Stephen N. Scelsa, Tuan H. Vu, Christina N. Fournier, Kirstin M. Johnson, Andrea Swenson, Gary L. Pattee, Suma Babu, Jeremy Shefner, and Merit E. Cudkowicz.

“This is hopeful news for the ALS community,” said Dr. Paganoni. “The results published in Muscle & Nerve suggest that treatment with AMX0035 is associated not only with functional benefits, as previously reported, but also with a long-term survival benefit. This new publication adds to the long-term safety data that we recently presented at the AANEM Annual Meeting. Altogether, this data provide substantial evidence supporting the role of AMX0035 for the treatment of ALS. The next steps will depend on ongoing conversations with regulatory authorities."

Dr. Paganoni is the principal investigator of the CENTAUR trial. She is an investigator at the Sean M. Healey & AMG Center for ALS at Mass General, and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. Her work to advance knowledge of ALS earned her the 2019 AANEM Scientific Impact Award.

Another AANEM Member, Dr. Merit E. Cudkowicz, served as senior author of the study.

“The CENTAUR study was designed to give both short-term and long-term efficacy data. We found  AMX0035 slowed progression and prolonged life. We are thrilled to design and lead this study from the HEALEY Center at Mass General and to work so closely with people with ALS, Amylyx and the Northeast ALS Consortium,” said Dr. Cudkowicz, Director of the Sean M. Healey & AMG Center for ALS at Mass General Hospital.

Dr. Cudkowicz, one of the plenary speakers at the AANEM 2020 Virtual Annual Meeting, which took place from October 7-10, pointed out the crucial role AANEM’s foundation, the American Neuromuscular Foundation (ANF), plays in research like the Centaur trial.

“Support from the ANF is critical to attracting and supporting young investigators such as Dr. Paganoni to become leaders in therapy development for people with ALS and other neuromuscular disorders,” said Dr. Cudkowicz.

The ANF is a nonprofit organization dedicated to strengthening the global effort to cure neuromuscular disease. The ANF funds important research into diseases like ALS and helps support education through awards and fellowship funding. When you donate to the ANF, 100% of your donation is used to support these initiatives. This is possible because all of the ANF’s administrative costs are paid for by the AANEM.  Learn more about the ANF at neuromuscularfoundation.org.


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