At the Healey Center, we are dedicated to developing effective treatments for ALS and to providing people with ALS access to potentially beneficial therapies.
Healey & AMG Center Director Merit Cudkowicz, MD, MSc and Co-Director of the NCRI Sabrina Paganoni, MD, PhD explains how the Expanded Access Program increases access to experimental treatments for patients with ALS, while participant Shawn Sarbacker of TechVsALS shares what the EAP program means to him.
In addition to clinical trials, our commitment extends to Food and Drug Administration (FDA)-regulated Expanded Access Protocols (EAPs). Drugs available through EAPs hold potential for benefit but have not yet been proven to be effective therapies for ALS. EAPs allow people with ALS who are not eligible for clinical trials to access these promising therapies. EAPs also generate data that can be useful, alongside traditional trial data, to bring new therapies to market for everyone.
Being part of the EAP has been wonderful, mainly because I know I am helping advance the field toward an effective treatment for ALS.
— Ellen Corindia, ALS patient since 2001
We have built a dedicated team at the Healey Center to rapidly implement EAPs for people with ALS at Mass General. This allows us to work efficiently with drug companies and regulatory agencies to provide access to investigational products even for those who don’t qualify for a clinical trial. We are also coordinating with HEALEY ALS Platform Trial sites to offer a companion expanded access program at multiple locations.Expanded Access FAQ
If you are interested in participating in EAPs, please contact your doctor or one of the sites offering expanded access through the HEALEY ALS Platform Trial companion program. To learn about other expanded access opportunities, visit the NEALS website.
Opportunities at the Healey Center for ALS
Expanded Access Protocols Available at Mass General
Verdiperstat, by Biohaven: Vishni Arulanandam, firstname.lastname@example.org or
HEALEY ALS Platform Trial Multicenter Companion Expanded Access
CNM-Au8, by Clene Nanomedicine: Multiple Locations
Pridopidine, by Prilenia Therapeutics: Multiple Locations
Verdiperstat, by Biohaven: Multiple Locations
Follow us on Twitter with #EAP4U
Ellen Corindia, a long-time ALS patient and a participant in the EAP, says, "Being part of the EAP has been wonderful, mainly because I know I am helping advance the field toward an effective treatment for ALS. Everyone is in agreement that there are many different forms of ALS. These groups respond differently to therapies based on genetics and the unknown tipping point that sent us spiraling toward the complex damage signature of ALS. Those of us who do not qualify for clinical trials have untapped data to contribute to the overall field. Since EAP is a regulated process, my safety testing and biomarker data can be fed back to the drug companies and FDA. Also, one drug might prove to help my particular form of ALS. That is important information we can’t afford to miss. I have given blood, spinal fluid, skin biopsies to make stem cells and various other body fluids to anyone who wants it for research. However, taking a new drug and providing data feels way more hopeful. My personal belief is that EAPs should become a standard arm of all new clinical trials moving forward."
Healey Center Director Merit Cudkowicz, MD, MSc explains how the Expanded Access Program increases access to experimental treatments for patients with ALS, while participant Bruce Rosenblum shares what the EAP means to him.
Transforming Medicine Through Innovation
Expanded Access Programs at the Sean M. Healey and AMG Center for ALS are funded by multiple sources, including contributions from donors like you. Thank you for partnering with us in the quest to discover life-saving therapies for individuals affected by ALS.