In 2020, the ALS Association, in partnership with Massachusetts General Hospital, launched an innovative approach to research, the HEALEY ALS Platform Trial. In most clinical trials, a single drug is tested, and participants cannot be enrolled in more than one trial at a time. The HEALEY ALS Platform Trial enables us to test multiple proposed drug treatments at once. This is a model that has been successful in cancer research. It accelerates our search for effective treatments for people living with ALS by allowing investigators to test more drugs, increase patient access to trials, and reduce costs by quickly and efficiently evaluating the effectiveness of multiple therapies. There are now 54 test sites nationwide, three of which are in Texas.
Texas Neurology was one of the first five initial sites for the new model. “This is big. I’ve been doing trials since the early nineties. The average trial takes 8 years. Going through platform methodology shortens it to 2-3 years,” states Dr. Daragh Heitzman, clinical director at Texas Neurology. Dr. Heitzman was recently appointed to the HEALEY ALS Platform Trial Executive Committee, as the first physician investigator to join an elite group of scientists.
“What’s different about this trial is that the placebo group is being shared for all five drugs, such that the number of participants who actually get a drug is dramatically increased. So, it’s a lot quicker to get to an answer, more patients are getting drugs, and less getting a placebo.”
Trial participants will have the option to continue treatment for at least a year, and if the drug shows promise during the six-month trial, the FDA could potentially fast-track its approval, getting a crucial treatment to people living with ALS.
Texas HEALEY ALS Platform Trial Sites
- The ALS Clinic at Texas Neurology in Dallas
- Houston Methodist ALS Clinic
- The ALS Clinic at UT Health San Antonio
Read more about Dr. Heitzman and the clinic at Texas Neurology
John Robinson from Parish, FL, a member of the board of trustees of the ALS Association and retired military veteran of nearly 32 years, was diagnosed with ALS in 2019. ALS not only affects the person with the disease but also their family caregivers. Both he and his wife can no longer serve their communities in the same way because of ALS. “The loss to society of their skills and humanity leaves holes never filled the same way again.”
Sunny Brous, a Texan with ALS, joined the call to share her perspective. Sunny was diagnosed with ALS in 2015 at the young age of 27. For Sunny, access to experimental treatments go beyond extending her own life. “Expanded access is how I can contribute to finding a cure for ALS. It takes my love for others and makes it a quantifiable action combining research and science and my dedication to the greatest good of people living with ALS.”
Sandy Morris, from Truckee, CA played a recorded message. She tearfully shared how many of her friends with ALS died waiting for access to life saving drug treatments. “Think about how much better it would be if we were able to die trying rather than die waiting,” she shared, urging the FDA to consider how their delays harm those living with ALS. “It’s not within my power to save my life—it’s within yours.”
Troy Fields from Champa Bay, FL was diagnosed with ALS in 2018. He noted how the considerations of benefits versus risk should be put in the hands of those living with ALS. “Because unless it is your own life on the line, you simply do not get it.”
Cari Meystrik from Nashville Tennessee was diagnosed with ALS in 1998 when she was only 28 years old. She has watched hundreds of friends die from this awful disease. “Would I jump to try a drug that might help me live longer of a disease that I am dying from if it has already proven to be sage and well tolerated but isn’t approved yet? Absolutely. Give me that chance.”
Jay Quinlan from New Orleans joined with his wife and caregiver Stephanie. “You have shown through your recent handling approvals for COVID vaccines that flexibility and expediting processes are possible.” He asked the FDA to use the same urgency when it comes to approving drug treatments for those living with ALS.
Tommy May from Pine Bluff, AR was diagnosed with ALS in 2005. Due to slow progressions, he was both unable to participate in clinical tries and frustrated with the lack of access to treatments. He discussed how those living with ALS are desperate for HOPE on their path. “We are not interested in the long-term. We are desperate for the here now.”
Larry Falivena of Apex, NC, has ALS caused by a mutation of the SOD1 gene. The fight for treatment access is not just for himself—it is also for his children. “I’m asking the FDA to help make sure my boys don’t have to worry about ALS.” He shares that even if the opportunity exists for just a small number of patients to have extended time on this earth that they need that chance.
