ALS specialist Sabrina Paganoni, MD, PhD, is beloved by her patients at Massachusetts General Hospital for her thoughtful care and encouragement. At the same time, Dr. Paganoni’s patients, many of whom suffer from Amyotrophic Lateral Sclerosis (ALS), provide her with continuous inspiration.
“Our patients are our partners in our search for a cure for this disease. With their help, we can make a big impact.”
“Our patients are our partners in our search for a cure for this disease,” Dr. Paganoni says. “With their help, I know we can make a big impact.”
ALS is a progressive disease that affects nerve cells in the brain and spinal cord. When the motor neurons die, the brain is no longer able to control muscle movement. While researchers like Dr. Paganoni have learned a great deal about the physiology of the disease in recent years, there is still no cure. Dr. Paganoni is among the ALS specialists who are exploring drugs with the potential to slow the disease’s progression by testing the drug’s effectiveness in patients.
Providing a Direct Connection
Mass General is one of the few academic medical centers able to provide a direct connection between the laboratory research and patient care. That’s because patients in the MGH ALS program are able to participate in drug trials while working collaboratively with an ALS specialist.
“Many of us in the Mass General ALS program divide our time between the lab and the clinic,” Dr. Paganoni says. She is board-certified in physical medicine and rehabilitation, neuromuscular medicine, and electrodiagnostic medicine and sees patients at Spaulding Rehabilitation Hospital and at the MGH Neurological Clinical Research Institute.
Through her work with patients and in the lab, Dr. Paganoni has become encouraged about the potential of the over-the-counter supplement inosine as a neuroprotective agent. She is currently leading a phase II trial to test the safety of the supplement and its ability to reduce inflammation in the neurons of ALS patients.
Collaborations for ALS Therapy
In addition to collaborating with her patients, Dr. Paganoni also works closely with scientists and clinicians across neurological specialties. The MassGeneral Institute for Neurodegenerative Diseases (MIND) in particular, offers the opportunity to share knowledge and expertise around the basic science of Huntington’s disease, Parkinson’s disease, Alzheimer’s disease and ALS. That access and collaboration, Dr. Paganoni says, allow the research to advance in all areas at once.
“We meet monthly to brainstorm and share our progress and our challenges,” Dr. Paganoni says. “Getting different perspectives on a problem is hugely helpful.”
She learned about the possibilities of inosine from MIND colleague and mentor Michael Schwarzschild, MD, PhD, who was studying the impact of the compound on patients with Parkinson’s disease. “Thanks to Dr. Schwarzschild’s positive research results, I was able to move forward more quickly with testing this treatment for ALS patients,” Dr. Paganoni says.
The Impact of Philanthropy
The challenge is that many early phase clinical trials must rely primarily on private philanthropy. Since inosine is a commercially available supplement, there is little incentive for pharmaceutical companies to support the research. Fortunately, the Salah Foundation, based in Florida, stepped up to support a pilot study that was recently completed and the ongoing phase II study.
The Salah Foundation supports non-profit organizations with a special interest in education, medical research, community development and self-sufficiency, as well as programs aimed at the economically disadvantaged, the young, the elderly and the disabled.
“We were inspired by a longtime friend, Patrick Flynn, who endured his diagnosis of ALS with courage and grace,” says George Taylor, a board member of the Salah Foundation. “We are also impressed by the collaborative approach MGH is making in their determination to find a cure.”
The Salah Foundation is a private foundation that supports non-profit organizations in the United States that strengthen families and communities and advances individuals to become productive and responsible citizens. There is a special interest in education, medical research, community development and self-sufficiency programs aimed at the economically disadvantaged, the young, the elderly and the disabled.
Mass General is spearheading a series of trials for inosine as a potential treatment for both Parkinson’s disease and ALS and Dr. Paganoni says she is eager to make progress. Private philanthropy is critically important to continue to test inosine and other drugs that can help relieve the suffering of ALS patients.
ALS Specialist Finds Inspiration
“I found particular inspiration from the extraordinary bravery, grace and passion with which my patient, Patrick Flynn, faced his disease,” Dr. Paganoni says. “I am dedicating this work to his memory.”
Mr. Flynn, who was active in the arts scene in South Florida for three decades, died in May at age 65, after a three-year battle with ALS.
“Our goal is to slow the progression of the disease,” Dr. Paganoni says. “If we can achieve that for more patients suffering from ALS, that will be an excellent outcome.”
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