Expanding Access to Therapies

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 Programs (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 drugs even for those who don’t qualify for a clinical trial. We are already working with several companies and are in discussions with more. Each EAP may enroll only a limited number of participants, and our goal is to expand to numerous EAPs to best meet the needs of people with ALS.

Since our EAP launch at the Healey Center in September 2018, we have offered 8 investigational therapies to a number of people with ALS. Some of the therapies we have provided this way include:

• IC14, an infusion medication provided through Implicit Bioscience
• CNM-Au8, a liquid suspension medication provided through Clene Nanomedicine, Inc.
• RNS60, a nebulized medication provided through Revalesio Corporation

Our goal is to continually grow our EAP to hasten drug discovery and meet the needs of people with ALS through this kind of compassionate research. We are adding EAPs as a companion to the HEALEY ALS Platform Trial to connect more people with ALS to the selected therapies. The HEALEY ALS Platform Trial EAP will represent a new way of approaching expanded access research for people with ALS and will be a powerful tool for hope.

To make our vision of ever-growing EAPs a reality we will need dedication, innovation and funding. We are writing grant applications and raising philanthropic support so we can provide several EAPs each year.

Your support makes this work possible.

Thank you for partnering with us to accelerate the drive to the cure for ALS.

Follow us on Twitter with #EAP4U

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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."