I AM ALS Grant to Support Healey Center for ALS Expanded Access to Experimental ALS Therapy

The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announces a new Expanded Access Protocol (EAP) for people with ALS, funded through a $200,000 grant from I AM ALS.

EAPs allow patients to receive an experimental treatment and contribute to research by providing safety and biomarker data, even though they are not eligible for the main clinical trial.

Pending FDA and Institutional Review Board approval, this new EAP will provide up to 35 people with ALS with access to verdiperstat, one of the drugs being tested in the HEALEY ALS Platform Trial. The EAP will be open to people with ALS who are not eligible for the platform trial and will be enrolling at up to three platform trial sites. The EAP will allow ALS patients to receive an experimental treatment and to contribute to ALS research — it is expected that their participation will provide important safety and biomarker data in a population not eligible for the Healey ALS Platform trial.

By creating a program alongside the HEALEY ALS Platform Trial and modeling successful processes and outcomes, the Healey Center expects to facilitate the growth of EAP programs that benefit patients and provide important information for ALS therapy development.

This project builds on the positive experience that the Healey & AMG Center for ALS at Mass General has accumulated since 2018 with several single-center EAPs and is a stepping stone towards creating a larger EAP program that runs in parallel to the HEALEY ALS Platform Trial. The study team, led by Mass General Researchers Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc with Duke University Researcher Richard Bedlack, MD, PhD, will also develop enduring training and advocacy materials that will be shared publicly to inform clinicians, industry and patients about the benefits of EAPs and boost EAP availability for ALS patients.

Read the I AM ALS Foundation press release.

Watch the Launch Video