CAMBRIDGE, MASS. (WHDH) - In a big step forward in the long fight against ALS, Cambridge-based ALS Therapy Development Institute announced Wednesday that it will soon be conducting the first human trial of a drug that could help slow the progression of the neurodegenerative disease.
The nonprofit biotechnology research organization got the green light from the Food and Drug Administration to begin testing AT-1501, a drug designed to block immune cell activation and protect nerves against the progression of ALS.
“For them to accept our Phase 1 application to get this drug into people is a major milestone for the community, which is exciting,” ALS Therapy Development Steven Perrin in an exclusive interview with 7News earlier this week.
The drug has already shown promising results in testing on mice, according to Perrin. Scientists hope the drug will slow down the progression of ALS by slowing the immune system’s attack on the nerves.
It was developed at the institute’s Kendall Square offices and moved through the development process thanks in part to money raised through things like the Ice Bucket Challenge.
“The research here at ALS TDI is funded by the entire ALS community in a very grassroots style approach,” Perrin said. “Everything from our 300-mile bike ride, the Tri-State Trek, to the Ice Bucket Challenge, to hundreds and hundreds of families who run events to support the research.”
ALS Therapy Development has directed $5 million in fundraising money over the last five years to AT-1501.
The initial Phase 1 test will consist of 40 people, including eight who are living with ALS. Hundreds more could potentially begin taking the drug as part of a Phase 2 trial. Perrin hopes that happens next year.
“At this point, we’re working with key opinion leaders in the ALS clinical community to design and execute a Phase 2 study, which hopefully would start sometime in 2019,” Perrin said.
The news will certainly spark hope, though big hurdles remain in the fight to combat this debilitating disease.
“This is the beginning,” Perrin said. “We wouldn’t be where we are without all of the philanthropic support that’s gotten AT-1501 out of the lab and into the clinic, but there’s a long way to go.”
The first patients with ALS to participate in the study will begin taking the drug in January. The drug is currently administered intravenously.
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