DAGMAR’S STORY

By Dagmar Munn

“Have you ever participated in clinical trials?”

That’s a common question among ALS patients. What’s my standard answer? “It’s complicated. I’m participating in several studies, but even though I’d like to, I haven’t been in any clinical trials. I’m excluded because I’ve had ALS for too many years.”

‘What? Tell me more’

Shortly after my ALS diagnosis in 2010, I searched the internet to learn about all the research being done to find a cure for the disease. When I discovered ALS research was still studying whiteflies, cells in dishes, and mice on treadmills, it was a huge disappointment, to say the least.

Then, a few years later, clinical trials began with actual patients, but most required participants to live within 50 miles of the study site. My nearest study site was Houston, Texas, which is a long, long way from where I live in Arizona.

As time went on, clinical trials expanded. Advances in data collection and technology allowed for easier access and reporting. But my feelings of being excluded continued every time I read the fine print of almost every trial’s criteria, which limited participation to patients whose ALS onset began less than three years ago. At 11 years post-onset, I was waaay over the limit.

The question that kept bouncing around my brain was the following: Even though I’m still functioning, mobile, and have no severe, debilitating symptoms, my longevity of 11 years isn’t worth investigating?

A Catch-22 situation
Recently, a discussion went viral in the ALS community about a new drug shown to be favorable in the treatment of ALS. The hubbub was that doctors and medical insurers were hesitant to prescribe it to patients with disease longevity like mine. The reason? It had only been studied on patients within one to three years of their ALS disease onset. It seems that those of us with four years or more are stuck on the outside, looking in.

The situation is getting noticed
In 2018, a conference titled “Evaluating Inclusion and Exclusion Criteria in Clinical Trials” produced an in-depth workbook that identified barriers and recommendations for positive change. A few recommended strategies included improving trial transparency, increasing patient involvement in a trial’s design, and reexamining exclusion and inclusion practices.

A year later, a 2019 study focusing on the ALS community concluded that, “The majority of patients with amyotrophic lateral sclerosis (ALS) are excluded from participating in clinical trials, ” according to a report by ALS News Today‘s Alice Melão. Not only were patients excluded who had the disease longer, but the study also found that “enrolled patients were mainly younger, male, and with a slower disease progression rate. They also had a better risk profile, with a longer survival prognosis than the general ALS population.”

Who knows how long it will take for clinical trial participant criteria to evolve. Let’s hope it will be soon.

Other options are available
For the past three years, I’ve been participating in the ALS Therapy Development Institute‘s Precision Medicine Program (PMP). Every two weeks, I complete an online medical and lifestyle history survey, plus an online ALS Functional Rating Scale-Revised. To assess my voice, the PMP lab computer calls my phone, and I read five different phrases aloud, then hang up. It’s easy to do, everything is done online, and I am helping the institute in their quest to discover new targeted treatments for ALS and make clinical trials faster and more efficient.

I’m also enrolled in two voice studies. The first is sponsored by the nonprofit group EverythingALS. This study aims to create a high-quality database of speech data for early detection and improved diagnosis of ALS. The second study is Project Euphonia, sponsored by Google, which has the goal of helping teach voice recognition software how to better understand atypical speech.

If you feel excluded by clinical trial criteria, don’t give up. There are many other opportunities available. Participating in ALS research not only feels good, but also is a good thing to do. Let’s help the researchers learn more about ALS so that we can live well while living with ALS.