Introducing the First-Ever ALS Platform Trial

The Sean M. Healey & AMG Center at Massachusetts General Hospital is launching the first-ever ALS platform trial aimed at advancing the development of ALS treatments.

Our Lois Insolia ALS Clinic at the Les Turner ALS Center at Northwestern Medicine has been selected as a participating site, with enrollment of patients to begin in 2020.

A “platform trial” is a clinical trial that involves the testing and evaluating of multiple treatments for a disease simultaneously. In addition, because different treatments are tested using shared infrastructure and a common master protocol, data from participants in all placebo groups can be pooled. This increases the chance for each individual participant of being on active treatment versus placebo, unlike in traditional trials. Furthermore, new treatments will be added to the platform as they are made available.

The benefits of the “platform trial” approach to drug development have already been tested and proven successful in the cancer field. “This design optimizes chances for people to receive active treatment, provides answers faster and ensures we keep learning about the disease,” shares Merit Cudkowicz, MD, MSc, Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.

As one of just fifty-four selected sites across the country, Senda Ajroud-Driss, MD, Director of the Lois Insolia ALS Clinic, explains what this means for our Chicagoland ALS community, “We are really excited to participate in this innovative design for Phase 2 clinical trials in ALS. We are hoping that this new initiative will shorten the time that it takes to do traditional clinical trials, broaden patient access to trials and help us better understand this disease.”

Learn more about the comprehensive care at our Lois Insolia ALS Clinic here.

The Healey Center issued a call for the best therapeutic ideas ready to enter the HEALEY ALS Platform Trial and received an outstanding response with almost 30 applicants from 10 countries. A group of expert ALS scientists chose the top five therapeutic ideas ready to enter the platform trial. The platform trial will launch with three therapies first, with plans to add two more shortly afterward.

“We are thrilled to partner with leading organizations around the country to bring this innovative and potentially disease-changing, trial model to our Center at Northwestern Medicine and to our Chicagoland area families. Our vision is to see a future without ALS. This trial takes a giant leap toward making that vision a reality,” shares Les Turner ALS Foundation CEO, Andrea Pauls Backman.

You can read more about the HEALEY ALS Platform Trial from Mass General News here.

Dr. Cudkowicz recently hosted a NEALS webinar where she discussed accelerating therapy development and expanding access for ALS treatments as well as information about the upcoming HEALEY ALS Platform Trial.

General questions about clinical trials at Northwestern Medicine can be directed to ALSresearch@nm.org.