When a friend asked Patrick Flynn what he could do to help him cope with an ALS diagnosis, Mr. Flynn suggested supporting research that could help others.
Sabrina Paganoni, MD, PhD, a Mass General ALS specialist and a researcher
Sabrina Paganoni, MD, PhD, a Mass General ALS specialist and a researcher

The story begins with Patrick Flynn, a Florida man who is being treated at Massachusetts General Hospital for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Lawyer George Taylor, a good friend, asked what he could do after hearing about the devastating diagnosis. At first, Mr. Flynn simply said that he was fine and felt blessed to have had such a great life.

Moved by the answer, Mr. Taylor, who is a board member of his family’s Salah Foundation, kept pressing. Eventually, his friend offered a suggestion.

“I told George that doing something to advance the field is what would most help me,” recalls Mr. Flynn, who went on to play matchmaker between the Salah Foundation and Sabrina Paganoni, MD, PhD, his Mass General ALS specialist and a researcher. The result is a generous donation that will enable Dr. Paganoni to start testing a promising potential therapy for this disease that has few treatment options.

Living Longer with ALS

The recent Ice Bucket Challenge spotlighted this relatively rare disease, which afflicts about 30,000 Americans at any given time. Awareness and greater research funding, both Dr. Paganoni and Mr. Flynn hope, will quicken the pace and depth of ALS research possible. “If we can catalyze this attention, big changes can happen,” Dr. Paganoni predicts.

“We are very excited by some of the treatment possibilities being studied now. But the clock is ticking so we need to move fast.”

Time is of the essence for people with ALS, which is often fatal within a few years. By that time, those with ALS have lost so many motor neurons—brain cells necessary to control muscle movements— that they need assistance to move, breathe, swallow or speak.

People are living longer with ALS, however, and there is hope on the horizon.  “We’re very excited about some treatment possibilities being studied now,” Dr. Paganoni says.  “But the clock is ticking so we need to move fast.”

The Promise of Inosine

There is only one FDA-approved drug treatment for ALS, but it only slows disease progression for a few months. One very early, yet promising treatment is an over-the-counter supplement called inosine. It has already been shown by Mass General researcher Michael Schwarzschild, MD, PhD, to be a promising drug for those with Parkinson’s disease. Inosine increases the body’s level of uric acid. Dr. Schwarzschild and his colleagues have also shown that people with higher levels of uric acid have a reduced risk of developing Parkinson’s, and among people diagnosed with Parkinson’s disease having a higher level is predictive of slower disease progression.

“This pilot funding is crucial for us to quickly explore the applicability of inosine to ALS,” Dr. Paganoni says.

Both Parkinson’s and ALS involve a cell-death mechanism called oxidative stress. Inosine appears to operate as an anti-oxidant, slowing or even stopping cell death.

“This pilot funding is crucial for us to quickly explore the applicability of inosine to ALS,” Dr. Paganoni says. “It would take us much longer if we had to wait for funding from industry or the National Institutes of Health.”

A Hub for ALS Research

Every three months Mr. Flynn flies up from Florida to see Dr. Paganoni, whom he calls one of his favorite people. “As long as I have to get medical care, I thought I might as well get the best,” he explains. People with ALS come to Mass General from all over the country for care focused on maintaining the best quality of life possible. They also come for opportunities to participate in clinical trials of promising new drugs or treatment approaches, such as the inosine study.

Mass General— through the leadership of Neurology Department Chief Merit Cudkowicz, MD— is a hub for ALS research to find better treatments. Mass General researchers are currently leading or participating in a number of ALS clinical trials testing different disease targets and drug strategies.

Help for a Friend

“But I’m not dwelling on what I can’t do anymore,” he says. “I think about what I can do to help others.”

The Salah Foundation most typically supports nonprofit organizations working to strengthen families and communities. “We’ve never invested in anything cutting edge like this before,” says Mr. Taylor. Presented with research options to support at Mass General, they decided on the inosine project, “because it looks at a conventional problem differently and transfers benefits from one disease to another,” he says. “But also, to help Patrick in this way makes me feel so good.”

“I knew he was an amazing man before,” Mr. Taylor adds, “but I’m even more impressed now.”

Last year Mr. Flynn could travel to Boston on his own, but this year he needs his wife’s assistance. “But I’m not dwelling on what I can’t do anymore,” he says. “I think about what I can do to help others.”

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