Dominant Inherited ALS (DIALS) Network, A New Approach to Prevention
At this critical time, marked by the emergence of amazing gene therapy strategies targeting familial ALS, the DIALS Network has the potential to profoundly accelerate our ability to accurately predict disease onset to halt ALS before the onset of symptoms. Within the last two years we launched the DIALS Network as a multicenter study to follow people at risk for familial ALS so that we can effectively design preventive strategies in familial ALS. Co-led by Dr. Katie Nicholson at the Healey Center at Mass General and Dr. Timothy Miller at Washington University, the goal of the DIALS Network is to identify the earliest biological and clinical markers of disease among individuals with dominantly-inherited ALS, in order to rapidly move the ALS field towards disease prevention. These components are essential in the design of the first prevention trials in those who are asymptomatic and at risk. We are aiming to expand the DIALS network to more families and accelerate our ability to conduct prevention trials with digital phenotyping.
Changing the Face of Familial ALS Through Innovative Clinical Trials
The Healey Center for ALS is spearheading an initiative focused on innovation in familial ALS trials for both people who are affected by neurodegenerative disease and those who are pre-symptomatic and at risk. Fast-paced lab discoveries in familial ALS have led to a growing list of ideas for translation into therapy, including genetic approaches and small molecules. A major challenge in ALS drug development is the conventional “parallel group” trial design requiring large patient numbers and long trial duration. Part of the mission would promote “N-of-1” trials, in which a therapy is tested in just one patient or a small group of targeted patients, to determine potential impact. An experienced team at the Healey Center would lead the design of these small trials conducted in a new familial ALS-focused trial network.
Parenting at a Challenging Time (PACT-ALS) Program
PACT-ALS provides parent guidance to support parents in addressing the illness-related challenges faced by their children and adolescents. We work collaboratively with parents and the ALS care team to help them anticipate what to expect in terms of their children’s ongoing adjustment, how to plan communication around a parent’s medical condition, when to feel comfortable with a child’s coping, and when to consider additional resources or interventions. This service is available at no charge to patients receiving care at the Healey Center for ALS. This program is made possible through philanthropic support.
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.