As a Patient, My Contributions to Research Are Important

As a Patient, My Contributions to Research Are Important

I recently visited my neurologist and FA clinical team at UCLA. I am participating in a clinical trial, and UCLA is my nearest study site. Oftentimes, clinical trials are “blind” and placebo-controlled, meaning multiple patient groups take an investigative therapy or a placebo, and no one knows who receives which. (One of my doctors refers to the placebo as the “fake drug.”)

This particular trial is in its extension phase, and those participating are all taking the investigational therapy, omaveloxolone.Omav,” as we refer to it, currently is beyond the “secret” stages of collecting data, so I’m comfortable bringing this up. I often avoid the topic of clinical participation so as not to negatively affect the process. In a sense, I find it taboo even to mention it.

The details, however, are not the point of this post. Instead, I want to focus on the value of participation overall.

For a drug or a therapy to gain any traction and have a chance of finding “success” in treating or curing a condition, patients must be involved. I believe the role of a patient is like a scope on a rifle. A scope allows the user to take aim at the target. The better the scope, the better the chances of hitting the target. Sophisticated scopes, especially for long-range situations, help identify wind speed, temperature, humidity, and probably the weather report for the next three months. The reliability of the scope combined with a sturdy rifle and the skill of the rifle’s user all help to ensure the target is hit.

Doctors are incredibly skilled and talented in their field of study. If we think of medical science as being the high-powered rifle wielded by the ingenuity of skilled doctors, and the patient as the sophisticated scope, together we have a powerful system for hitting the target. If the doctor, the science, or even the patient is removed from the equation, we end up with a piece of metal that is not optimized for high-level performance.

The target may as well be tied to a chicken wearing a blindfold — constantly moving with no rhyme or reason. A gun can’t take aim or fire a round on its own. Without a scope, a trained sniper will have difficulty hitting a long-range target. Of course, without the rifle, someone’s just taking in the views with a monocular.

All three work in unison to hit the target.

Medical science, skilled medical professionals, and willing patients must work in unison to increase the chances of finding a cure, or “hitting the target.”

I don’t have the skills of a doctor, nor do I have endless resources to fund a research grant or finance a scientific approach to a cure or treatment. However, my DNA and the scientific data my body can produce is invaluable and can’t (yet) be reliably manufactured to a beneficial degree.

I believe it’s important that everyone consider clinical trial participation to some degree. The data a patient group can provide the medical world will only help add sophistication to the scope being used.

I know that eligibility criteria may exclude some patients. I understand that some folks are unable to engage with blood draws or muscle biopsies. I respect that many patients prefer to be reserved in their approach to living with their conditions. However, I believe even a simple conversation can help doctors and scientists better understand what patients are up against and may help direct their attention to symptoms or root causes that seem to be most concerning.

My involvement in clinical trials is fueled by an insatiable desire to be a part of the process of finding a cure. My data may not provide any breakthrough insights, but my contribution certainly won’t slow down anyone or anything. However, if my data, and that of many other patients, aren’t available to the professionals, those scientists are aiming blindly.

Thankfully for my condition, a database and a system for observing the natural history of Friedreich’s ataxia was established about two decades ago. This natural history allows doctors to establish baselines for many aspects of the condition and offers starting points for the work of researchers. I’d consider this natural history to be the starting point, or the basic level, at which to be involved. Perhaps blood draws or investigational treatments are unappealing or uncomfortable for you. Your opinions, personal experiences, and specific symptoms still matter, and all data are important data.

Your words and your life experiences have power, as long as you allow others to hear your words and observe your experiences. Consider what level of participation you are comfortable with and take responsibility for getting involved. Excuses will always hold you back. Identify something that works for you and make a plan you can follow.

You are the fancy and sophisticated scope if you choose to be.  

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Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Sean Baumstark lives with Friedreichs ataxia and embodies the mantra “get stuff done.” He believes excuses hold us back from being our best. He is the founder of de:terminence, a non profit helping disabled individuals experience the beauty and power of physical achievement. He also co-hosts a weekly podcast, Two Disabled Dudes, encouraging listeners to “live beyond circumstances.” He lives and works in Sacramento, CA.
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Sean Baumstark lives with Friedreichs ataxia and embodies the mantra “get stuff done.” He believes excuses hold us back from being our best. He is the founder of de:terminence, a non profit helping disabled individuals experience the beauty and power of physical achievement. He also co-hosts a weekly podcast, Two Disabled Dudes, encouraging listeners to “live beyond circumstances.” He lives and works in Sacramento, CA.

4 comments

  1. Ginny Browne says:

    I was part of a trial for Altzheimer’s several years ago. The drug worked for me, but not for a high enough percentage of the participants, so the trial was halted. The director of the trial gave me all the left over medication and I took it until it was gone. I can tell I’m slipping back into the disease, but it is moving slowly . . . I’m glad I had several years without worrying about it.

    • Sean Baumstark says:

      Thank you for sharing, Ginny! I think you touch on one of the hardest things we might face after a diagnosis – the lack of “significant statistical data.” I can only imagine the difficulty of facing such an outcome after experiencing benefits. I’m glad you had several years without worrying about it and I am hopeful that the fight against Altzheimer’s is only getting stronger and closer to a treatment.

  2. Carla Hagler says:

    BRAVO….well said!!!!!! I totally agree. We FAers [those who can] have a responsibility to others to participate in research in whatever way we can. I have proudly given my time, experiences, knowledge, tissue, & blood to researchers in USA, CA & Austrailia.

    • Sean Baumstark says:

      Thank you, Carla! Not only for reading and commenting, but for participating in the process of finding a treatment and a cure. Keep it up, we aren’t through yet!

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