One of the greatest strengths of the Healey Center is the international expertise of the Scientific Advisory Council (SAC). Our meetings are opportunities to brainstorm about ALS science, identify research gaps and pool our contacts to encourage researchers from other scientific specialties to join our cause. We made extraordinary progress this year thanks to the resources the Healey Center and our partners have made available, but also thanks to the dedication of the SAC to direct those resources where they can make the largest impact.

Mark Poznansky
Mark Poznansky, MD, PhD

At the May, 2019 meeting of the Healey SAC, we invited several new investigators from other fields to learn about therapeutic approaches in other disease areas that might be effective in ALS. At the meeting, we heard from Mark Poznansky, MD, PhD, who is the Director of the Vaccine and Immunotherapy Center (VIC) at Mass General and an Attending Physician in Infectious Diseases medicine. Dr. Poznansky spoke about how the progress in our understanding of immunotherapy has revolutionized treatment of cancers and autoimmune diseases. 

This led directly to the launch of an exciting collaboration with Dr. Poznansky’s team at VIC: a research effort to determine if there is a defect in B cells associated with ALS. If the hypothesis is proven, there is the potential that ALS could be treated using B cell therapy.

Progress in Personalized Medicine

The Healey SAC also invited leading stem cell scientist Clive Svendsen (Cedars Sinai) and computational biologist and Big Data and Artificial Intelligence expert Ernest Frankel (MIT) to discuss recent advances in personalized medicine. Drs. Svendsen and Frankel have developed tools to model individual people’s illness using their own stem cells as part of the Answer ALS project. It is now possible to create personalized induced pluripotent stem cells (iPSCs) by doing a simple blood draw. Blood cells can be used to create stem cells which can then be differentiated into motor neurons that have the unique genetic make-up of the individual they are from. This breakthrough technology allows scientists to do something that was unimaginable even just 3-5 years ago: we are getting close to be able to study people’s own stem cells to predict which treatment they are more likely respond to. In the future, this could lead to a monumental paradigm shift in the way we treat ALS and could enable truly personalized medicine for this disease.

Based on this opportunity, the HEALEY ALS Platform Trial Design team will collect and store blood cells from all participants in the platform trial. The goal is to discover more about ALS and how to find the best treatment for individual participants. In addition, this project represents a phenomenal example of synergies and collaboration between the HEALEY ALS Platform Trial, Answer ALS and top scientists from all over the country.

The ideas and projects that arise at the SAC meetings are exciting and often unprecedented. We look forward to reporting back on the possibilities that will emerge from our November meeting.

Expanded Access Programs

Expanded Access Programs (EAPs) provide investigational therapies to individuals with life-threatening diseases who are not eligible or able to participate in clinical trials, which includes most ALS patients. Thanks to the support of the Healey Center, ALS patients now have access to four new investigational treatments.

Healey Center support allowed Alex Sherman, Director, Center for Innovation and Bioinformatics at the Neurological Clinical Research Institute (NCRI) to develop NeuroREACH, a data platform that collects patient data to quickly and efficiently manage simultaneously-running EAP trials in ALS in a regulatory-compliant, secure and standardized manner. NeuroREACH allows investigators and sponsors around the world to match investigational therapies with patients and track results. Our current EAPs include:

RT001 – RT001 is a highly stabilized version of linoleic acid, also known as Omega-6 fatty acid, that has been shown in the lab to block the chemical reactions that damage cellular membranes and mitochondria. Impaired function of mitochondria is increasingly recognized as an initial event in ALS, and the EAP is allowing individuals with ALS to participate in a trial to see if RT001 can restore membrane functions and cellular health.

RNS60 – RNS60 is an experimental compound that acts on the immune and inflammatory mechanisms implicated in ALS. Preclinical studies showed the drug protects the integrity of neurons and other supporting cells in the brain and spinal cord by specifically modifying inflammatory pathways, preventing cell damage or death.

IC14 – IC14 is an antibody platform drug that modulates CD14, a master regulator of the immune response. IC14 administered through infusion improves T regulatory cell function and decreases the inflammatory response.

CNM-Au8 – This is an orally administered, clean-surface gold nanocrystal suspension drug that shows promise for improving cellular energetics and increasing a cell’s ability to function efficiently.

We receive many accolades and positive feedback from participants and their families for the chance to be part of research and to have access to therapies through this program.

No two patients are the same, and no one knows what the future holds.  But the people along the journey make all the difference in the world, and you people do it so well and warmly.

EAP participant