ALS Clinical Trials 2023

What Are ALS Trials?

ALS (Amyotrophic lateral sclerosis) is a nervous system disease that causes loss of muscle control. The progressive condition is often called Lou Gehrig disease, after the famous baseball player who was diagnosed with it.

According to the Mayo Clinic, symptoms include:

• Weakness in limbs
• Muscle cramps and twitching
• Difficulty swallowing
• Slurred speech
• Feeling clumsy or weak

ALS trials often focus on testing medications for the condition and their dosages and investigating potential new therapies. ALS trials also test technology to help those living with the condition communicate and live more independently.

Why Is ALS Being Studied Through Clinical Trials?

According to the CDC, around 31,000 people in the US live with ALS, with approximately 5,000 new cases each year. According to ALS Canada, there are 200,000 people worldwide that have ALS.

According to the National Institute of Neurological Disorders and Strokes, the only FDA-approved drugs are Relyvrio, Riluzole, and Edaravone. Research is being done to help expand treatments and care provided to people with ALS.

For example, in 2002: BrainGate at Tufts University was in the pilot stage of trial testing BrainGate2 sensors in the motor-related cortex. Their goal is to identify ways to restore some functionality for people who have lost abilities such as speech.

What Are The Types Of Treatments Available For ALS?

According to the Mayo Clinic, treatments for ALS focus on trying to slow down the progression of the disease and help a person live as full a life as possible with the condition. However, there has yet to be a drug or therapy that can reverse the damage already caused by the disease.

Current FDA-approved drugs are:

• Relyvrio
• Riluzole (Rilutek)
• Edaravone (Radicava)

Physical therapy assists patients in using their ever-changing bodies as best as possible. There are also breathing therapies, devices, and surgeries to help a person continue to breathe. ALS care teams also assist with nutrition plans, speech therapy, and occupational therapy. Current trials are looking at new medications, dosages, therapies, and devices to help a person living with ALS.

What Are Some Recent Breakthrough Clinical Trials For ALS?

2020: Healey ALS Platform Trial – Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, in partnership with Northeast ALS Consortium (NEALS), began enrolling participants for their multicenter, double-blind, placebo-controlled, perpetual, adaptive platform trial. They are looking at experimental treatments, including Zilucoplan, Verdiperstat, and CNM-Au8 nanocrystalline gold.

2020: CENTAUR Trial: Sean M. Healey & AMG Center for ALS concluded phase 3 of the trial of AMX035. Its success led to Amylyx receiving FDA approval for Relyvrio in 2023, which is two drugs: sodium phenylbutyrate and taurursodiol. The trial found that Relyvrio would slow the decline of a person with ALS and extend their life.

Who Are Some Of The Key Opinion Leaders / Researches In ALS Clinical Trial Research?

James Berry, MD, MPH, is the Director of the Massachusetts General Hospital Multidisciplinary ALS clinic, Director of the MGH Neurological Clinical Research Institute, and Chief of the Division of ALS and Motor Neuron Diseases. He focuses on ALS identification markers in the blood and spinal fluid.

P. Hande Ozdinler, Ph.D., leads the ALS research laboratory called The Ozdinler Lab. The lab focuses on motor neurons and the cellular and molecular mechanisms that can lead to their degeneration. Ozdinler is also an Associate Professor of Neurology at Northwestern University Feinberg School of Medicine. She was the senior author of the study that identified the first compound to repair degenerating upper motor neurons impacted by diseases such as ASL.