Guest Author: Sam Kirton, NAF Board of Directors President
Fighting Ataxia is a cause that is close to Sam Kirton’s heart. He lost several family members to the disease. He volunteers his time to help make sure that NAF continues to move boldly forward, championing efforts to accelerate development of treatments and the search for a cure. Although Sam does not have Ataxia himself, he is no stranger to having a rare disease. Sam has Idiopathic Pulmonary Fibrosis (IPF), which is a rare disease that causes irreversible and progressive lung damage.
Sam’s year-long participation in a clinical trial for a new drug for IPF caused him to reflect on NAF’s work to mobilize the Ataxia community toward participating in research opportunites. Read his thoughts below.
Sam's Experience Participating in a Clinical Trial
Recently I received a call from one of my Doctors and a Clinical Research Coordinator concerning a Clinical Trial I am supporting. I have been enrolled in this Investigational New Drug (IND) trial for over a year. While this IND is not Ataxia related, I believe my experience is relevant.
Every day for over a year I have taken a dose of the IND, made a diary entry confirming the dose, and when necessary made a diary entry for observations or if I had a change in any of my other medications. The trial was terminated for “futility,” meaning that during the course of the trial the research team was not able to see any difference between the patient populations (IND vs placebo).
This brings me to my point for sharing this experience. It was not a year plus wasted, but a year plus invested in exploring the efficacy of the IND. Every single investment in research whether it is by NAF, pharmaceutical companies, or government, pulls a single thread which gives us more insight, deeper understanding, and potentially one day moves us closer to treatments and a cure for the different Ataxias.
I remain confident that the key to discovering treatments lies in the work of organizations like NAF supporting research and it is why we have to continue to help members understand why their participation in research opportunities is key to helping unravel the mystery.
A Few of Sam's Thoughts on Clinical Trials
- Clinical trials are in an investment in not only your future but the future of the next generation.
- Clinical trials are rigorously monitored for the safety of the participants.
- Clinical trials provide “us” an opportunity to leverage science to continue to investigate and explore a variety of Ataxia related diagnoses.
- Healthy volunteers are also needed for clinical trials.
- Joining the patient registry provides investigators a resource pool to identify and locate the right candidates for a given research effort or clinical trial.
- Tell your care team that you want to support clinical trials and research efforts that they become aware of.