Dear Friend of the Sean M. Healey & AMG Center for ALS,

May is ALS Awareness Month ­– there remains an urgent need for new ALS treatments, and we take this month to reaffirm our commitment to developing new ALS therapies as soon as possible! 

We are making great strides in our fight against ALS. The FDA just approved Radicava ORS (edaravone) oral suspension for the treatment of adults with amyotrophic lateral sclerosis (ALS). We are also expecting an important FDA Decision for AMX0035 on June 29th.  We are hopeful that this will also be approved and look forward to updating you when we are alerted to the final decision.

We are also excited to report that our PREVENT ALS efforts are gaining momentum. We have an ambitious goal to reach a total of 750 study participants. To support that work and the cost of the testing required, The Eddie and Jo Allison Smith Family Foundation has provided the Healey Center with a $1 million matching gift opportunity.

Our researchers continue to bring us closer to understanding the molecular basis of ALS. Dr. Sadri-Vakili, a Healey & AMG Center researcher, is leading her team to discoveries about the protein tau - critical for nerve cell structure, central to Alzheimer’s disease, and now implicated in ALS. We are excited to see what her research can tell us and how it may inform new ALS treatments.

I’m also honored to share that I recently received the first annual Target ALS Rebecca Luker Courage Award. Rebecca was under my care and a huge advocate for ALS research. She was a star on Broadway who shared her courage and talent to her fight against ALS. This award means so much to me because it recognizes my passion to help those with ALS receive both great care and access to research. 
It was also a great honor to learn that The Healey Center for ALS earned a Morris ALS/MND Principles accolades certificate for introducing the first platform trial in ALS, including low placebo exposure and broad inclusion criteria.

Creating opportunities for people with ALS is really what the Healey Center is about. Generous gifts from Sean Healey, AMG and many others allowed our team to develop the first-ever platform trial in ALS and expanded access to promising therapies so that soon ALS will be a thing of the past.

I hope you enjoy reading all our latest updates and news at the Sean M. Healey & AMG Center for ALS. Please reach out to me with any questions.

With gratitude,

Merit Cudkowicz, MD, MSc and the ALS Clinic Team
IMPORTANT FDA DECISION FOR NEW DRUG APPLICATION FOR ALS
On March 29th, the FDA’s Advisory Committee voted 4 (yes) and 6 (no) that AMX0035 is effective in the treatment of ALS based on the data from the Phase 2 CENTAUR clinical trial. The committee’s recommendations are non-binding, and we continue to advocate for a timely approval given the acknowledged safety profile for AMX0035. Read more in our recently published editorial in the Annals of Neurology.

We look forward to the FDA’s final decision, which is expected on June 29.
PREVENT ALS: STOPPING THE DISEASE BEFORE IT STARTS
Our global research efforts and philanthropic support are allowing us to envision a world in which ALS is both treatable and preventable. The PREVENT ALS initiative builds on the success of the Dominantly Inherited ALS (DIALS) study led by Dr. James D. Berry at Mass General Hospital and Dr. Timothy Miller at Washington University. By studying people with an ALS-causative gene mutation that do not yet have symptoms we are able to identify the earliest symptoms of ALS and molecular changes that start even before symptoms. This will hasten diagnosis, support earlier treatment, and teach us about the causes of ALS – in those with genetic and sporadic (non-genetic) forms of ALS.
 
The PREVENT ALS initiative engages geneticists, genetic counsellors, laboratory researchers, clinical trialists and families at risk for ALS who work together to identify biological changes preceding ALS and novel therapeutic targets. And our efforts are gaining steam. Currently, 169 participants are enrolled in the DIALS study. Our goal is to expand our network of PREVENT ALS sites to enroll at least 750 participants.

Expanding the PREVENT ALS study will drive us to answers more quickly than ever. To support this critical expansion and the cost of the testing and staff to run the study, The Eddie and Jo Allison Smith Family Foundation has provided the Healey Center with a $1 million matching gift opportunity, meaning that each dollar we raise for PREVENT ALS goes twice as far. Please consider making a gift to double your impact today! Learn more.
UPDATES FROM THE LABORATORY OF GHAZELAH SADRI-VAKILI, PHD
Dr. Sadri-Vakili, director of the NeuroEpigenetics Laboratory at the MassGeneral Institute for Neurodegenerative Disease and the Healey & AMG Center for ALS at Mass General, and her team are studying tau, a protein that is critical for stabilizing the structure of nerve cells and has been implicated in Alzheimer's disease, to understand the role it plays in ALS pathogenesis.
 
To determine if tau protein is a viable biomarker for ALS, the team measured tau and its phosphorylated form in cerebrospinal fluid from people living with ALS. This work,Novel genetic variants in MAPT and alterations in tau phosphorylation in amyotrophic lateral sclerosis post-mortem motor cortex and cerebrospinal fluid,” was published in Brain Pathology, demonstrated that increases in tau protein in cerebrospinal fluid from people with ALS correlated with disease progression as well as levels in post-mortem brain. Therefore, tau levels -- and specifically the ratio between tau and the phosphorylated form of the tau protein -- might help clinicians predict the rate of disease progression in people with ALS.

Dr. Sadri-Vakili and researchers in her lab are also studying how the abnormal version of tau protein may contribute to the progression of ALS by damaging mitochondria in neurons, according to the study “Targeting Tau Mitigates Mitochondrial Fragmentation and Oxidative Stress in Amyotrophic Lateral Sclerosis,” that was published in the journal Molecular Neurobiology. The findings suggest that decreasing tau levels may provide a novel therapeutic strategy to lessen mitochondrial dysfunction in ALS.
FIRST ANNUAL REBECCA LUKER COURAGE AWARD WINNER:
MERIT E. CUDKOWICZ
Merit E. Cudkowicz, MD, MSc, Chief of the Neurology Department at Massachusetts General Hospital and Director of the Healey & AMG Center for ALS was named the winner of the first annual Target ALS Rebecca Luker Courage Award. The award was given to those who have demonstrated a courageous spirit and made a positive impact on the ALS community in honor of the life and legacy of Broadway star Rebecca Luker. The award was presented at Target ALS Annual Meeting May 3-5, 2022 in partnership with Mitsubishi Tanabe Pharma America, Inc. (MTPA).
HEALEY & AMG CENTER FOR ALS EARN MORRIS ALS/MND PRINCIPLES ACCOLADES
The Healey & AMG Center for ALS earned a Morris ALS/MND Principles accolades certificate for introducing the first platform trial in ALS, including low placebo exposure and broad inclusion criteria. The accolades certificate also honors the Healey & AMG Center for ALS’ incorporation of the ideas of people with lived experience of ALS (PLEx) into the HEALEY ALS Platform trial. Enthusiastically keeping consistent and transparent communication with people living with ALS (PLWALS) through weekly webinars also contributed to the award.

The Morris ALS/MND Principles are a group advocates who sought to create the first guidance document to outline people living with ALS’/MND’s expectations regarding their inclusion in matters directly affecting them.

The Morris ALS Principles were created with the intent of breaking down silos, avoiding expensive duplication, creating a human-centric ALS landscape and encouraging valuable collaboration between ALS stakeholders. ALS/MND advocates sought to create the first guidance document to outline people living with ALS’/MND’s expectations regarding their inclusion in matters directly affecting them. The Morris Principles serve as a social contract solidifying the necessary involvement of people living with ALS/MND. 
COMMUNITY FUNDRAISING

PREVENT ALS Matching Gift Opportunity
PREVENT ALS is gaining momentum as more and more people and families at risk for developing ALS join us to search for the triggers that initiate the disease, so that we can stop it before it starts. We received a generous matching gift opportunity for $1 million from the Eddie and Jo Allison Smith Family Foundation to inspire raising an additional $1 million for the expansion of the PREVENT ALS initiative. Please consider supporting our PREVENT ALS initiative and double your impact through making a gift today

Annual Lou Gehrig’s Day June 2, 2022
We are thrilled to announce that the Sean M. Healey & AMG Center for ALS will be working with Major League Baseball for this year’s Lou Gehrig’s Day. For the second year, the MLB and the Lou Gehrig Day Committee have selected the Expanded Access Protocol (EAP) Program at the Healey Center for ALS as the beneficiary for all charitable fundraising efforts centralized out of the Commissioner’s Office. We look forward to sharing more details about how you can get involved and support this effort in the coming weeks.

Tackle ALS
Tim Green - fundraising champion and advocate for ALS research at the Healey Center - was recently featured in an article on the sports news website The Athletic. The interview encompasses his numerous successful careers, the importance of family, and how his ALS diagnosis continues to motivate him. Tim launched the Tackle ALS fundraising campaign in 2018 and has raised over $5.5M in support of the HEALEY ALS Platform Trial.
JOIN US
All our work at the Sean M. Healey & AMG Center for ALS combines outstanding, multidisciplinary care with clinical and basic research informed by the needs of our patients. Philanthropy allows us to accelerate the pace of discovery and make a meaningful difference to our patients. We are determined to find the cures. Your partnership will make this possible.

If you have any questions or would like to learn more about how you can support the Sean M. Healey & AMG Center for ALS, please contact Emily Monteiro at emonteiro7@mgh.harvard.edu or visit https://www.massgeneral.org/neurology/als/support/