HEALEY ALS Platform

When it comes to a degenerative, rapidly progressing disease like ALS, time is of the essence. Reducing the cost of research by 30%, decreasing trial time by 50%, and increasing patient participation by 67%, the HEALEY ALS Platform Trial is a vital step toward finding new ALS therapies.

The first-ever ALS platform trial, the HEALEY Trial, tests multiple investigational products for ALS simultaneously or sequentially, evaluating the safety and efficacy of medical interventions for the treatment of ALS. The study, sponsored by Massachusetts General Hospital, comprises 54 study locations across the United States, including Saint Louis University.

Each investigational product is tested in a regimen, which consists of a placebo-controlled trial. Once randomized to a regimen, participants are randomized in a 3:1 ratio to either study drug or placebo. The medications tested in the trial include:

• Zilucoplan — an oral myeloperoxidase inhibitor
• Verdiperstat — an oral myeloperoxidase inhibitor
• CNM-Au8 — a new class of medicine that provides an energetic assist to impaired motor neurons, helping them improve their ability to function more normally (motor neurons consume significant energy in order to function normally, but in ALS, corrupted energy metabolism together with increased cellular stress lead to motor neuron degeneration).
• Pridopidine — a highly selective Sigma-1 receptor (S1R), which regulates key cellular pathways, commonly impaired in neurodegeneration

New medications are continuously added as they become available. 

The HEALEY Trial is probably the best trial I’ve ever come across. Everyone is collaborating with each other, having bi-monthly meetings to discuss progress, challenges, and how to increase patient enrollment.

Dr. Ghazala Hayat

“The HEALEY Trial is probably the best trial I’ve ever come across,” says Dr. Ghazala Hayat, director of the SLUCare ALS Clinic at Saint Louis University. “Everyone is collaborating with each other, having bi-monthly meetings to discuss progress, challenges, and how to increase patient enrollment.”

The primary outcome measure for the trial is change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Secondary outcome measures include change in respiratory function over time as measured by Slow Vital Capacity (SVC), change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD) and survival as measured by the comparison of rate of occurrence between groups. The platform will remain open until successful cures are found. 

“There are 500,000 people suffering at this moment from ALS, and we have only two medications to help them,” says the ALS Clinic’s Dr. Jafar Kafaie. “We have a big hope that by speeding the clinical trials through this HEALEY platform, we will find some new, effective interventions.”