My stalled relay race to get access to ALS drugs in development

One night in November 2018, I fell while crossing a street in Subic Bay, a U.S. naval logistics station in The Philippines. My face whacked the asphalt. Lying in the path of oncoming traffic, I couldn’t move a muscle. After 45 seconds of terror, I was able to stand up and haul myself to the other side.

Six months later, after dealing with symptoms like my left leg buckling unexpectedly and being extra tired hauling my tools around on the job, a doctor told me I had amyotrophic lateral sclerosis — a 100% fatal disease. “What’s going to happen?” I asked. “You’re going to be paralyzed,” he said.

At the time I was a 59-year-old father and husband. My work entailed climbing down stairs and ladders to work on the bottom of ships, repairing propellers and hydraulics, and climb up them to fix controls on the bridge.

As my condition worsened, my doctor told me, “It’s not safe for you to climb ladders. You need a new profession right away!”

I swallowed a 50% pay cut to work part-time in the office. Six months later, I couldn’t drive safely to the office, and was terminated from my job.

Within a year of my diagnosis, the only way I could get around was with a powered wheelchair. Then I needed breathing machines, and found myself fighting a continuous battle to find and retain in-home care providers.

Available drugs didn’t do much to help me. And since I was well into my diagnosis after one clinical trial, I wasn’t eligible for further clinical trials. What could I do?

The Food and Drug Administration has a program to allow expanded access to experimental therapies. It lets people like me with serious or immediately life-threatening diseases get access to investigational products outside of clinical trials when no alternatives are available.

Learning about that program was good news — at first — since at the time there were three promising drugs for ALS the FDA had approved for expanded access. Yet it’s been like an uneven relay race. The first three legs went incredibly well, but the final anchor leg is going in slow motion.

Leg 1: At the start, expanded access programs (EAPs) — which had proved useful in other disease areas to get investigational therapies to people who can’t get into clinical trials — were practically nonexistent for people living with ALS. The major excuse was that small companies working in ALS couldn’t afford to provide people with their therapies. ALS advocates proposed the idea for public funding to prime the expanded access pump for small companies. But the seeds were planted for legislation that would provide public funding and other resources for the National Institutes of Health to work with expanded access programs.

Leg 2: In incredibly fast bipartisan fashion, legislators listened to advocates, and developed and passed the Accelerating Access to Critical Therapies for ALS Act. It was signed into law by President Biden at an impressive ceremony at the White House. After that, the big excuse for not making expanded access available to people ineligible for Phase 3 trials was gone. Once funding was appropriated, the baton passed to NIH to issue grants, paired with studies intended to add scientific insights into these investigational therapies.

Leg 3: The NIH moved quickly, providing grant opportunities for investigators to propose expanded access programs and companion studies. One grant was given in 2022, the first year, then the NIH issued three substantial grants for EAPs in October 2023 totaling $32.5 million. NIH did its job. People living with ALS looked forward to a swift, efficient anchor leg.

Leg 4: As the baton was passed to those who were to finish the EAP race — people like me — the ALS community sensed it was close to getting access to investigational therapies. Yet eight months after the October 2023 announcement, very few people have received any of these therapies. The principal investigators of the trials understand the urgency of the ALS clock, but clinic administrators along with legal and institutional review boards don’t. The track is now filled with bureaucratic hurdles such as protocols, contracts, compliance, ethics, policy, payment, training and more.

In the meantime, people are dying every day from ALS, stopped in their tracks without access to investigational therapies. Since October 2023, approximately 3,000 people in the U.S. have died from ALS. During the same time, a similar number of people have heard the dreaded words, “You have ALS.”

People living with ALS — and those with other fatal diseases — deserve a seamless system that helps them get access to treatments that might extend their lives. Procedural hurdles that block access to experimental therapies that might help those who no longer qualify for clinical trials must be flattened. My life, and the lives of many others, depend on it.

Lynn Brielmaier is an electronics engineer who is involuntarily unemployed due to extreme disability and a member of the Institute of Electrical and Electronics Engineers’ neuroethics subcommittee.