Clinical trials are a critical part of research. When a scientist is developing a new therapy or treatment for a disease, clinical trials are how they make sure it’s safe and effective before it can become available to anyone who needs it. But for someone with amyotrophic lateral sclerosis, or ALS, a disease that has no known cure, clinical trials are about so much more than moving science forward — they’re their only shot at curing, and sometimes even slowing down, their disease.
Navigating clinical trials with ALS can seem daunting, especially when you’re not familiar with scientific research. So we sat down with Phil Green, an ALS Champion and advocate since his diagnosis in 2018 and a member of Augie’s Quest Leadership Council, to clear up some of the mystery surrounding clinical trials from a patient perspective. Drawing from his own clinical trial experience and ALS advocacy work, he gave these pointers for anyone wondering if a clinical trial is right for them.
- There’s a limited window where you might qualify.
To qualify for any clinical trial, you must meet certain requirements. For example, to enroll in many ALS trials, you cannot be more than two years beyond your first symptoms. And since it can take a long time from when symptoms first start to finally get a diagnosis — sometimes a year or more — you’ll want to act fast.
The researchers also need to be sure that your ALS is progressing at a certain pace before you start the trial. Unlike other diseases, there are no known biomarkers in the blood that researchers can look for to see if the therapy is working, and there’s no growth or tumor they can measure, as in cancer. With today’s knowledge of ALS, the only way to determine if a therapy is working is by measuring your disease progression, so if you aren’t progressing enough to begin with, researchers won’t be able to tell if the therapy is helping.
- You’ll have to take some initiative.
You should always talk to your doctor about clinical trial opportunities and any questions you might have. But your doctor might not be aware of every ALS trial that’s happening around the country, so it’s a good idea to do your own research on what trials are open and where. You can search for ALS on clinicaltrials.gov, the NIH’s clinical trials database, or you can use the ALS Signal Dashboard created by our friends at I Am ALS to search for clinical trials by state, trial phase, genetic target and more. Then, it’s up to you to consult with your doctor and reach out to the trial site to find out if you qualify.
- It’s a big commitment.
Clinical trials ask a lot of their participants. Though some trials may enable remote participation, the majority require you to travel to the clinic for every visit, and if you’re unable to drive, you’ll need to arrange your own transportation. It can be a big time and financial commitment, especially if the clinic is out of town.
“From a personal commitment level, the investment is pretty high,” Phil said. You’re also committing your body to a therapy that may or may not work, and if you’re participating in a double-blind controlled study, like Phil did, there’s a fair chance — in his case, a 50/50 shot — that you’re in the placebo group and not getting any real treatment at all.
But if it does work, the payoff can be huge. Since the study hasn’t been published yet, Phil doesn’t know for sure if he got the investigative treatment or a placebo, but he’s almost certain he got the treatment because his disease stopped progressing for a year. “If I was in the placebo group, how can I explain how my disease progression stopped for the majority of 2019?” he said.
- No matter the outcome, you’re helping people with ALS everywhere.Phil Green and Lynne Nieto addressing the crowd at the 2021 Cure ALS Golf Classic to support Augie’s Quest
Even if the treatment doesn’t work, or if you get a placebo, you’re still making an invaluable contribution to research for ALS cures — one that will help everyone with ALS, now and in the future.
“Without the ALS community’s participation in these trials, a therapy that might help us will never reach the market,” Phil said. “So we need to be the research partners for these companies so they can bring their potentially impactful therapy to everyone else who currently doesn’t have access to these investigational therapies.”
- You won’t be left in the dark.
So what happens once you enroll? Every trial is different, but each trial site has a clinical trial coordinator who will map out the study for you and make sure you understand what’s happening every step of the way.
In Phil’s case, he received a schedule at the start and was deliberately walked through what would happen and when. “I felt very informed up front, which I thought was very helpful,” he said. He was also encouraged to reach out to the trial coordinator by text or email at any time, so he always had a direct communication line for any questions or concerns he might have.
- You need to find the right fit for you.
Phil’s top advice for someone looking to enroll in a clinical trial?
“Find a trial that will meet your individual needs.”
Consider how long the trial will run, how many trips you’ll have to make to the trial site or if you’ll be able to check in at your home clinic, what interventions it will entail, what side effects you might experience, and whether you might be compensated for some of your out-of-pocket expenses (like flights and hotel stays if you’re traveling out of state). These are all questions you’ll want to ask when you first reach out to the trial coordinator.
- You still have other options to participate in research.
Even if you don’t qualify for a clinical trial, or if you decide clinical trials aren’t for you, you might be able to enroll in an observational study, another important part of clinical research.
“There are so many observational trials you can participate in that not only help advance the research and understanding of the disease, but also can give you insights into your own progression and help you stay on top of your own personal journey,” Phil said. While you won’t gain access to a potentially helpful new therapy, you’ll still be contributing to ALS research by providing important data to scientists studying the disease.
Augie’s Quest is proud to support our research partners at the ALS Therapy Development Institute (ALS TDI) to fund critical science and drug development through work happening at the Augie’s Quest Translational Research Center. In 2021, through a partnership with the Eddie and Jo Allison Smith Family Foundation, Augie’s Quest also funded all new enrollees of the Precision Medicine Program (PMP).
The PMP is the most comprehensive and longest running translational research study in ALS. People from around the world share their data on movement, lifestyle, medical history, genetics, biomarkers, voice recordings and patient cell biology. By collecting the unbiased data, researchers are able to better understand ALS. This incredible program comes at no cost to its participants and is funded thanks to generous Augie’s Quest donor support.
- The decision is yours.
Clinical trials are a crucial part of scientific research, and especially for diseases like ALS, they’re a path to discovering truly helpful therapies — and hopefully, one day, cures.
“We’ll never have FDA-approved therapies for ALS that are somewhat helpful if people don’t participate in clinical trials,” Phil says. Your clinical trial participation benefits the greater ALS community, but it also stands to benefit you, he explains.
“Clinical trials are an opportunity to try an investigational therapy that might actually help slow down your disease progression,” Phil says. “We already know what the alternative is, so right now our only chance at hope is through these trials.”
Ultimately, the decision to participate in clinical research is one that only you can make for yourself. But if you’re having trouble weighing the pros and cons, Phil suggests talking it through with other members of the ALS community who’ve been where you are today. “I think leveraging people who walked in your shoes or sat in your wheelchair is critical,” he says. “I like to think that we are the experts on living with this disease, and I’m not a doctor so I’m not going to tell you what to do, but I can tell you what I’ve done and hopefully that can help you make a decision.”
Augie’s Quest is committed to funding cutting-edge research to find effective treatments, and ultimately cures, for ALS. Your support means hope for people living with ALS. Donate today.