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 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.

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

CNM-Au8, by Clene Nanomedicine: Geli Kane, gckane@mgh.harvard.edu or 617-726-1531 and Dario Gelevski, dgelevski@mgh.harvard.edu or 617-726-0563

IC14, by Implicit Bioscience: Dario Gelevski, dgelevski@mgh.harvard.edu or 617-726-0563 and Caroline Cohen, ccohen0@mgh.harvard.edu or 617-643-7828

NurOwn (MSC-NTF cells), by Brainstorm Cell Therapeutics: Mary Kay Turner, mkt@brainstorm-cell.com or 201-488-0460 ext 105 and Yael Gothelf, PhD, ygothelf@brainstorm-cell.com or 646-666-3188 ext 111

Pridopidine, by Prilenia Therapeutics: Vishni Arulanandam, mghsitehealeyplatform@mgh.harvard.edu or 617-643-2499

RNS60, by Revalesio: Grace Addy, gaddy@mgh.harvard.edu or 617-726-4282 and Dario Gelevski, dgelevski@mgh.harvard.edu or 617-726-0563

Tofersen, by Biogen: Kush Mehta, kmehta9@mgh.harvard.edu or 617-643-5376 and Gabe Jacobs, gjacobs@mgh.harvard.edu or 617-726-3015

Verdiperstat, by Biohaven: Vishni Arulanandam, mghsitehealeyplatform@mgh.harvard.edu or

HEALEY ALS Platform Trial Multicenter Companion Expanded Access

CNM-Au8, by Clene Nanomedicine: Multiple Locations

Barrow Neurological Institute, AZ: Jessie Duncan, jessie.duncan@dignityhealth.org or 602-406-1466

Hospital for Special Care, CT: Honora Dalamagas, RN, MSN, HDalamagas@hfsc.org or 860-612-6356

Holy Cross Hospital, FL: Ashley Stepler, Ashley.Stepler@holy-cross.com

Pridopidine, by Prilenia Therapeutics: Multiple Locations
California Pacific Medical Center (CPMC), CA: Marnina Murez, MurezMO@sutterhealth.org or 415-600-3758
Mass General, MA: Vishni Arulanandam, mghsitehealeyplatform@mgh.harvard.edu or 617-643-2499
Texas Neurology, TX: Mohamad Asaad Nasri, mnasri@texasneurology.com or 214-827-3610, ext 251
Verdiperstat, by Biohaven: Multiple Locations

Northwestern University, IL: Emma Schmidt, 312-503-4362

Mass General, MA: Vishni Arulanandam, mghsitehealeyplatform@mgh.harvard.edu or 617-643-2499

Duke University, NC: Sarah Parker, 919-681-4191

 

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Ellen Corindia with a member of her care team
Ellen Corindia (left) has been an ALS patient since 2001.

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."