John’s daughter, Anne, who’s living with ALS, is an outlier. Though the average life expectancy of someone living with ALS is just 2-4 years, Anne has a slowly progressing type of ALS and has been living with the disease for almost 12 years, since October 2013. She was in her mid-20s when she was diagnosed. At that time, Anne was living and working in Washington, D.C. and was able to start a clinical trial at nearby Georgetown University soon after her diagnosis. But, John says, after Anne aged out of that trial (once she was two years post-diagnosis), she needed more options for treatment to further slow the progression of the disease. 

One year post-diagnosis, after Anne moved back home to New York to live with her parents, they began working with a neurologist at Columbia, investigating the best pathways for treatments for her. Anne was able to take an off-label prescription for a glutamate antagonist, which treats one element of ALS: the excitotoxicity. But other than that single treatment, options were very limited. “There was a period of time when she’d aged out of clinical trials, and there were no other [access programs or pathways], so there was nothing new that she could try,” John said.

That was before ACT for ALS was passed into law in 2021. One of the things ACT for ALS did was to create government-funded Expanded Access Programs (EAPs) that provide additional treatment pathways to patients living with ALS. EAPs have existed previously, but patients need to find a neurologist willing to create and run an EAP and then find a way to pay for the therapy. These two obstacles  made EAPSthem out of reach for almost all people living with ALS.

As a volunteer advocate with I AM ALS, John is deeply connected to and works closely on the community’s initiatives around clinical trials and treatments, including projects like the patient-centric trial design rating system (PaCTD), an I AM ALS community-led initiative  in which John was involved. (“One of the criteria for a clinical trial getting the highest rating in the PaCTD is whether or not they’re doing an EAP, or open-label extension,” John says proudly.)

Working with her neurologist—and having the benefit of being entrenched in the ALS community—John learned about a new EAP in 2023 that was offered at a hospital near his home. The family currently traveled across state lines to see her neurologist at Columbia, so having to travel shorter distances to receive treatment was a huge potential benefit.  

In January 2024, John began making inquiries about enrolling in an EAP funded by ACT for ALS. He was put off again and again before learning that bureaucratic red tape was the cause for the delays. In July, he wrote a letter to the hospital site’s CEO, copying the chairman and vice chairman of their board. “I wrote a very nicely worded but pointed letter and said that their bureaucracy needed to move more quickly,” John recalls. Within two weeks, they were “ready to go” and Anne was the first person to enroll in that EAP.

The initial, invasive medical procedures that are in place to collect her data were grueling, but it was worth it. After just five months of treatments, Anne’s breathing capacity has increased by 40% (from 36% pre-EAP to 51%). “Obviously, that’s one of the biggest indicators of survival going forward, since people with ALS die due to respiratory failure,” John said. Other patients have had the similar results on his daughter’s EAP, he said. 

Ideally, John would like the EAP to extend treatment to participants who are currently benefiting in the EAP, and also begin enrolling more people, so that more people benefit and more data is collected—a win-win. But getting more people enrolled is another up-hill battle. “Now, we’re working with the National Institutes of Health to submit a grant application that would make this EAP available to more patients, so that we can get more statistically significant results,” he said. 

Meanwhile, Anne continues to experience positive impacts from the drugs on the program. “When she first got the disease, Anne was not a slow progressor. She had initial limb and bulbar symptoms and it was difficult,” he said. “Her breathing is good now and getting better. Anne is still able to move around, she’s ambulatory, but she needs a chair lift to get off the floor because her arms are useless.”

John believes that EAPs are a lifeline for people living with ALS. “You have just two years after symptoms or diagnosis to get on a clinical trial. If you age out, and there aren’t these EAPs, you just have to wait to die.”

John believes more EAPs are needed to increase access and create “more seats on the bus,” or pathways for effective treatments. Currently, since EAPs are few and far between: many people across huge swaths of the country have to travel for 500 miles or more to get to a hospital for treatment. “That’s a really high burden for people living with ALS and their families,” John says. 

So, while Anne is an outlier, she shouldn’t have to be. If EAPs can continue to be developed and funded, as they are through the ACT for ALS, they can create life-extending pathways for patients living with ALS, while giving researchers the chance to gather significant patient data that can serve future generations through scientific study. 

As the ALS community prepares for the reauthorization of ACT for ALS, John says he will continue his volunteer activism work, in honor of Anne, with more access for everyone living with ALS as his end goal. “I’m going to keep doing this advocacy work… it’s the only option,” he says.