Michael’s Story
By Michael Morale
Marcella: Michael, can you talk about your experience with SMA clinical trials?
Michael: Back in 1998, after officially receiving my diagnosis of spinal muscular atrophy (SMA) type 3, my neurologist asked me if I would be willing to participate in a doubleblind study that they were going to be doing for patients with SMA. This clinical trial only involved 15 patients in the United States, and I believe I was the only participant in Texas. The other 14 participants were scattered throughout the U.S.
Marcella: What did participation entail?
Michael: I was required to go through strength tests for six months, and during my first visit, I was given a prescription that needed to be filled. Since this was a double-blind study, neither myself nor my doctor knew if I was on the actual drug or if I was going to be receiving a placebo. When I went to fill the prescription, I received eight large prescription bottles of capsules. I was instructed to take one capsule each day for the first week, and two capsules each day for the second week, and was required to increase the dosage until I was taking six capsules each day. During the strength tests, they would lay me down in bed and attach wires that were hooked up to monitoring stations, and they would test how much resistance I could generate with my arms and legs. The clinicians that were performing these tests on me were not allowed to give me any information with regards to the results of the strength tests, but a total of six tests were performed over the six months.
Marcella: Were you able to stay in the trial until completion, and if yes, what happened after?
Michael: Yes, I was. Once the clinical trial ended, I was required to reduce the dosage that I was taking by one pill each week until I transitioned completely off of the medication. After the clinical trial, I was notified by my doctor that I was on the actual medication and not the placebo. The results of the clinical trial were not positive with regards to increasing strength in any of the patients. As a matter of fact, during this sixmonth clinical trial, I experienced a decrease in strength, which was considered normal due to the natural history of the disease itself.
Marcella: Michael, knowing what you know now, after many years as a patient advocate, what’s the most positive thing about that experience?
Michael: One of the most positive aspects of my participation is that I was asked to do a clinical trial by my doctor as opposed to having to look for a clinical trial; this is one of the most daunting tasks for patients even today.
Marcella: And was there anything that could have made your patient experience easier?
Michael: Yes. A better knowledge about SMA patients would have helped. If my father had not been with me, they would have not been able to lift me as the personnel in charge of the clinical visits were not strong enough and not qualified enough to pick me up. They should have been more prepared to assist the patients with SMA unable to lift themselves from the wheelchair.
Marcella: You are highly involved with the SMA community. Are people still experiencing these challenges or what challenges do people face today as it relates to clinical trial participation?
Michael: Pharma companies are certainly investing time and efforts in understanding the patient journey. Many people struggle with falling just outside the range (aka, for inclusion and exclusion criteria like age and physical criteria); a lot of info is delayed; and patients need quicker response. One of the biggest challenges patients complain about is finding the clinical trial, lack of awareness and not being able to easily navigate tools and updated tools.
Marcella: “Thank you, Michael. Do you have any suggestions for patients with SMA who are interested in learning more about clinical trials?”
Michael: Patients have to be their best advocate; they have to be self-starters and determined, because unfortunately nobody knocks on your door to bring you solutions. Again, I was lucky that my doctor spoke to me about clinical trials, but many patients are not so lucky.
Marcella: And do you have any suggestions for pharma companies who are investing so much in these trials?
Michael: Finding a regulated way to involve doctors and specialists who are not directly part of those clinical trials but who are the ones who see the patients who would benefit from participation. Also, include patient navigators who could work with patients on all aspects of participation. Lastly, invest in awareness: Patients use clinicaltrials.gov, but it’s not easy to navigate and not always updated.
