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FARAFARA Cure FA

Patient Perspective: Kyle's Reflection on the Importance of Being an Active Participant in Research

PatientPerspectiveKB1After the stressful early morning drive on the expressway through Philadelphia traffic, I arrive at the Children’s Hospital of Philadelphia (CHOP) to find all the accessible parking spots taken. I park at the far end of a row so I can have enough room to pull my wheelchair out.

I have been at the hospital a lot lately.

Blood tests, heart exams, neurological exams, and exercise tests. Not because I have been sick but because I was a participant in one of the clinical trials for a potential treatment for Friedreich’s ataxia (FA).

I live about 35 miles away from CHOP (the FA Center of Excellence), so anytime there is an opportunity to participate in a study, I am game. 

Turning the Crank Toward a Treatment and a Cure

The primary outcome measure of this study is exercise on a stationary recumbent bike so I head straight to the exercise lab.

It’s like being in a Gatorade commercial, hooked up with wires all over my chest, nose plugged and a snorkel in my mouth quantifying everything that enters and exits.

Next time you are exercising to your absolute limit try doing it with your nose plugged and a snorkel on your mouth. When spit accumulates, there is nowhere for it to go so the snorkel at the hospital is equipped with a spit trap.

The resistance on the bike starts at zero and then every minute it is cranked up a bit until you can’t pedal anymore. My test takes about 10 minutes. It feels defeating to be slowly stopped by this machine.

Like dealing with the effects of a degenerative disease, it’s almost like I have to make a choice whether to push myself or just stop pedaling.

The resistance eventually forces me to stop pedaling.

Clinical Trials: The Hope of a Rare Disease

FA is a genetic, debilitating disease that occurs in approximately 1 in 50,000 people. Statistically that means that there are 5,000 - 6,000 people living with FA in the US.

Many clinical trials in general fail because of lack of participation. So with numbers like in FA, each person has a big responsibility to do their part.

Since this is a rare disease with relatively few patients, the FA community is spread out across the world. The average travel distance for someone participating in a clinical trial is 600 miles.

With this thought, my troubles become insignificant and I carry on with purpose.

Volunteering has its Perks

One of the benefits of being in a clinical trial is you get to talk with the top doctors in the field and ask questions to make sure you are getting the best care possible.

As we talk, Dr. Dave Lynch uses his stethoscope to listen to my chest and then takes out his little hammer to check my reflexes. He holds out his index finger and asks me to touch my nose and then his finger as fast as possible.

As usual this does not go very well.

I end up overshooting the target or missing completely.

Frustrating, but I know all these tests are necessary parts of the process.

PatientPerspectiveKB2When Dr. Lynch’s duties are complete, my study coordinator, Lauren pulls out the dreaded pegboard. In this test of dexterity there are 9 holes in a plastic board and 9 small pegs to go into them. The test starts with the pegs in a little dish and the subject (me) must put all 9 pegs in, and then take them out.

You accomplish this task while the clock runs and judges you on your hand eye coordination.

Who Sticks Pegs in a Pegboard?

This test annoys me. Who puts pegs in little holes on a regular basis?

However, it measures a familiar frustration—the same frustration I experience when zipping up my jacket, or unlocking my front door, or typing on my keyboard. If it takes less time, I experience less frustration.

No Cure Without the Effort

Participating in clinical trials makes me feel extremely hopeful. Any study promises a chance of therapeutic benefit, but to me, none of these drugs work until proven, so therapeutic benefit is not my expectation.

But it can't be proven until it is tested in patients. We hold the key.

Are you interested in participating in a research study or clinical trial for Friedreich's ataxia? Please find all current opportunities HERE

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About the Author

The FARA Ambassador Program

The FARA Ambassador Program

The FARA Ambassadors are a united team of patient volunteers living with FA who are committed to supporting FARA in the search for a treatment and cure.
 
Together we seek to know more about FA, and FARA so we can be prepared to represent the community when the opportunity arises; speaking at events, to volunteers, potential donors, scientific groups, pharma partners, media interviews and other awareness and fundraising opportunities. We believe support is key to continued success toward our ultimate goal of treatment and a cure. Participants in the FARA Ambassador Program are passionate about building and upholding relationships within the FA community.
 
The FARA Ambassadors are positive, supportive, peer representatives for the FA community, actively raising awareness and funds for FARA. To learn more about the FARA Ambassador Program or to have a FARA Ambassador speak at your event, please contact: info@cureFA.org.

 

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