On February 4, a crowd of 200 friends and supporters celebrated Massachusetts General Hospital’s promising new approach to finding a cure for ALS.
At a dinner in Palm Beach, Florida at the home of Darlene and Jerry Jordan, Merit Cudkowicz, MD, Chief of Neurology, outlined the progress already underway at the new Sean M. Healey and AMG Center for ALS, an initiative made possible by the generosity and partnership of Mr. Healey and his associates at Affiliated Mangers Group, the organization he founded 20 years ago and where now serves as executive chairman.
“The Healey Center builds on a strong research foundation and will serve as the bridge to the cure for ALS,” said Dr. Cudkowicz, who serves as director of the Healey Center. “We have already taken the first steps on the bridge, and Sean’s courage, generosity and leadership is helping to take us to the other side.”
Mr. Healey was diagnosed with ALS last May and went to work immediately, advocating for accelerated therapy development, expanding access for more patients, and increasing visibility for this disease to a broader public. His visionary leadership has already helped to raise $40 million toward Dr. Cudkowicz’s research efforts.
“I am so happy to be working with Dr. Cudkowicz, as her patient and her partner in the effort to find a cure for ALS,” Mr. Healey said after the dinner. “I am confident she has created a strong, international network of researchers and has an impressive plan of action in place that will find effective treatments.”
“Advances in the laboratory have created a pipeline of promising therapeutics,” Dr. Cudkowicz said. “The Healey Center is allowing us to, fast-track the most promising therapies and reduce the length of trials from years to months.” Resources from the Healey Center are directed to attracting the best researchers from around the world; supporting senior scientists, allowing them the time to focus on their research; awarding fellowships to the most courageous, forward-thinking innovators who will work to find solutions for people with ALS today and tomorrow; and move therapies out of the laboratory and get them to patients.
One of the most effective tools Dr. Cudkowicz’s team will employ is the Master Trial Platform, which allows researchers to test several treatments at once and increases access for individuals with ALS. The Master Trial Platform approach has been used successfully in cancer research and is strongly supported by the US FDA and now also European regulatory authorities.
“By borrowing a page from our colleagues in cancer research,” Dr. Cudkowicz said, “we are confident we can make a great leap forward in our efforts to cure ALS.”
Since opening in November 2018, the Healey Center has already begun two new clinical trials and hosted a meeting of 18 pharmaceutical companies who are interested participating in the Master Trial Platform initiative. In addition, two of the researchers affiliated with the Healey Center recently identified critical pathway in the disease progression, and a mechanism to fix it. This pathway is also used by other neurological diseases, including Alzheimer’s, Parkinson’s, and other forms of dementia. This discovery may open the door to promising treatments for a wide range of neurodegenerative diseases.
“Our efforts are global and collaborative,” said Dr. Cudkowicz. “We have our work cut out for us but have taken the first significant steps toward the cure.”