Greetings!

Since our launch in November 2018, the Sean M. Healey & AMG Center for ALS at Mass General is already accelerating research towards the cure. The support of the entire ALS community will make the difference in our quest to discover life-saving therapies for people affected by ALS. I am excited to share some of our accomplishments.

  • Assembled a Science Advisory Council comprised of leading ALS researchers from around the world to dramatically rethink how treatments are developed in ALS.

  • Brought together leaders in ALS science and trial design to move quickly to design the first Platform Trial in ALS. This new paradigm, already very successful in the cancer field, will transform therapy development in ALS.

  • Met with more than two dozen pharmaceutical and biomedical companies interested in partnering with us to launch this innovative Platform Trial approach in ALS.

  • Issued our first request on March 20, 2019 for applications for treatments to bring to patients in the Platform Trial.

  • Partnered with Tim Green and Tackle ALS to support the first three therapies in the Platform Trial.

We are at a tipping point in our search for a cure for ALS. Thanks to your generosity, we will revolutionize and accelerate how therapies are developed for people with ALS. Please continue reading for a more detailed update on our progress.

Warmly,

Merit Cudkowicz, MD, MSc
Director, Healey Center for ALS at Mass General
Chief, Mass General Neurology Service

The Healey Center Brings Leading ALS Experts Together to Form Science Advisory Council
The Healey Center’s science advisory council (SAC), composed of 10 global science leaders in ALS, met in November 2018 and February 2019. Members eagerly shared their ideas on exciting science initiatives and strategies to intervene to treat ALS. The advisors enthusiastically supported the focus on accelerating therapeutics through development of the first Platform Trial for ALS. The SAC members also recommended attracting the best and brightest young investigators from around the world to ALS through a Healey Scholars Program and an annual Healey Award for Innovation. This year, we will launch all three of the initiatives. The SAC meets again in May 2019 in Boston.
Lucie Bruijn PhD, MBA joins as Senior Science Advisory
In January 2019, Dr. Lucie Bruijn joined the Healey Center as a Senior Science Advisors. Dr. Bruijn is also Senior Advisor to the Dementia Discovery Fund and Chief Development Officer for Ossianix. She has more than two decades of experience developing and managing innovative ALS research. As Chief Scientist at the ALS Association, Dr. Bruijn developed and led a global ALS research effort, Translational Research to Advance Therapies for ALS TREAT ALS™,with the goal to move treatment options from bench to bedside. She was involved in project development, encouraging partnerships with academia and biotech, and played a key role in forging collaborations amongst investigators and other foundations. 

SCIENTIFIC BREAKTHROUGHS
First Platform Trial for ALS

Only recently have major advances in ALS science led to a rich pipeline of promising therapeutics. To help accelerate the transfer of the most effective therapies out of the lab and to our patients, the Healey Center will establish the first Platform Trial initiative for ALS. Historically, clinical trials in ALS evaluate only one drug at a time, leading to lengthy start-up and execution times. Platform trials allow multiple drugs to be tested in parallel, using specialized statistical tools. New regimens (drugs) can be added as they become available, decreasing or eliminating the gap in time from identification of a therapy to testing. This new paradigm will greatly accelerate therapy development by allowing investigators to test more drugs, increase patient access to the trials and reduce the cost by quickly and efficiently evaluating the effectiveness of a therapy. Platform trials have already led to effective treatments in the cancer field. We are greatly encouraged by the interest of regulatory authorities, patients, investigators, pharmaceutical companies and foundations to bring Platform Trials to ALS.

Our primary focus since the November launch of the Healey Center has been to educate the community and prepare to implement the first Platform Trial in ALS. We held meetings with ALS clinicians, people with ALS and potential industry partners. On February 12, 2019, neuroscience drug development leaders from 18 companies gathered in Cambridge, Massachusetts with academic clinicians from the Healey Center, the Healey Center Science Advisory Council and the Northeast ALS Consortium (NEALS) to learn about the potential for these Platform Trials. Enthusiasm and interest were extremely high with all stakeholders agreeing this is the right time and right approach for ALS.

On March 20, 2018, we issued the first call for proposals for the initial drugs for the Platform Trial and we anticipate a large response. The successful partners will work closely with the Healey Center Trial Design Team to tailor the arm of the Platform Trial to the experimental treatment. Nominations will continue on an ongoing, rolling basis and we fully anticipate that in addition to these first three treatments, more treatments will be added to the platform, supported by other fundraising initiatives.

To ensure this Platform Trial meets our expectations for excellence and accuracy, we brought together global leaders in ALS science and trial design (Healey Center Trial Design Team) and hired the Berry Consultants Biostatistical Group, the world’s leaders in adaptive Platform Trial statistics.
In addition to the Healey Center support, we are partnering with Tim Green and Tackle ALS to support the first three therapies in the platform trial. We are applying for grants through the Muscular Dystrophy Association (MDA), the ALS Association and the Arthur M. Blank Family Foundation. We will continue to establish partnerships and fundraise to sustain this effort and support the cost of each additional therapy.
Researchers Identify a New Therapeutic Target for Neurodegenerative Disease
Transactive response DNA-binding protein 43 (TDP-43) is a critical nuclear protein involved in the processing over 1,500 RNAs (the part of the genetic code that is translated into proteins) in neurons. Since the discovery that TDP-43 is redistributed from the nucleus to the cytoplasm in 98% of people with ALS and half of those with frontotemporal dementia (FTD), TDP-43 has drawn interest as a key factor in the cause of these disorders. Learn more .
Targeting Inflammation in Both ALS and Alzheimer's Disease
The Science Advisory Council also enthusiastically embraced the work of Healey Center faculty Mark Albers, MD, PhD, who discovered a new mechanism that damages nerve cells and triggers an inflammatory response in people with ALS and those with Alzheimer’s disease (AD). Based on this work, Dr. Albers and the Healey Center Trial Design Team designed a trial to test a promising therapeutic, Baricitinib, with the potential to treat individuals with either ALS or Alzheimer’s disease. This will be the first time a therapy is tested in two neurodegenerative disorders at once (called a Basket Trial). Learn more .

ATTRACTING NEW INVESTIGATORS TO ALS
Research Scholars Program

Brilliant young PhD and MD graduates are interested in entering the ALS field, but are often discouraged by lack of support for their salary and supplies for the research projects. The Research Scholars Program will attract and retain outstanding early-career investigators with the potential to make important contributions to ALS research. Grants will be issued annually, including a budget of $150,000/year for 2 years. These types of grants are transformational for new investigators.

Our first Research Scholar awards will go to two young scientists who work in the laboratories of Healey Center researchers Dr. Clotilde Lagier-Tourenne at Mass General and Dr. Kevin Eggan at Harvard University’s Stem Cell Institute. The first Research Scholars will research approaches to target stathmin-2 as a promising new therapeutic approach in ALS. In the fall of 2019, the Healey Center will issue a global call for applications for Healey Scholars, ensuring a broad reach for the best scientific minds to work on the cure for ALS.

Support for these scholar opportunities will broaden the number of scientists working on ALS research and bring us more quickly to the cure. A gift of $300,000 allows us to support a young investigator or the two years of focused research and mentorship. 

 
Healey Center Award for Innovation

The annual Healey Award for Innovation will be the first global award for exceptional achievements for an individual or team in scientific advances in ALS. Nominations for the first Healey Award will be requested in June 2019. The recipient of this prestigious, first-of-its-kind award will be presented at the 30 th Annual International symposium of ALS/MND hosted by MNDA December 4-6, 2019 in Perth, Australia.

EXPANDING ACCESS TO THERAPIES
The Healey Center is committed to providing the highest quality care to our patients, including providing access to potentially beneficial therapies. This commitment includes pursuing access to investigational therapies for treatment through the Expanded Access Program (EAP) overseen by the Food and Drug Administration (FDA) when appropriate for our patients. We formed a team of Healey Center Staff to rapidly implement EAP sfor people with ALS. This allowed us to rapidly receive IRB and FDA approval for two separate protocols: EAP for RT001 and an EAP for RNS60. We are in discussions with another pharma company for a third EAP in spring of 2019. 

The Healey Center is also partnering with several other ALS-focused foundations, including Tackle ALS, a charity launched by former Atlanta Falcons linebacker Tim Green, featured on CBS’s “ 60 Minutes ” last fall.

HIGHLIGHTS FROM THE HEALEY CENTER LAUNCH
Hear Massachusetts Governor Charlie Baker express his support for the Healey Center and our battle against ALS.
DONATE TO THE HEALEY CENTER
Please join our quest to discover life-saving therapies for individuals who are affected by ALS. At the  Sean M. Healey and AMG Center for ALS at Mass General , we partner with the best scientists, our patients and friends like you to turn laboratory successes into promising therapies.