How I Used ALS TDI's Precision Medicine Program to Measure my Disease Progression During an ALS Clinical Trial
As an athlete for most of my life, I have been in tune with my body and able to notice differences due to a change in my training or injuries resulting from a game. When I was diagnosed with ALS in August of 2018, I knew how important it would be to keep in tune with the changes in my body as my disease progressed.
Shortly after my diagnosis, I was fortunate enough to get connected with Augie and Lynne Nieto, founders of Augie’s Quest to Cure ALS. They introduced me to Dr. Steve Perrin from the ALS Therapy Development Institute (ALS TDI) – the world’s largest nonprofit biotech company and the most comprehensive research laboratory dedicated solely to finding treatments for ALS. I learned about the great things that ALS TDI was doing and I became very interested in their Precision Medicine Program (PMP). I learned that through the PMP, ALS TDI partners with people with ALS to gather data on voice, movement, lifestyle, personal medical and family medical histories, genetics, biomarkers, and patient cell biology.
I also learned that as a participant I would able to view my own data via a secure online portal. I decided to enroll because the PMP seemed like a better way for me to consistently and accurately monitor my disease progression. My clinic visits were once every 3 months but every month the PMP was collecting data to monitor my disease.
During this same time, I was exploring clinical trial options and recognized that the PMP could provide me with insights as to whether or not a therapy was having a positive effect on my disease progression. As it all worked out, I was selected to participate in the BrainStorm Phase 3 NurOwn Trial. I was also able to travel to Boston and officially enroll in the PMP at the same time as my 3-month observation period started for the Brainstorm trial.
The BrainStorm trial is a double-blind randomized 50/50 placebo trial, meaning that there was a 50% chance that I would receive actual treatment and a 50% chance that I would be in the placebo group. Being double-blind means that neither the company nor the trial site would know which group I was randomly assigned to. For these reasons, it was even more critical for me to look to the PMP data to help me deduce if I was getting treatment, and if so, was it having a positive effect on my progression. Understanding this, I made certain to do my monthly PMP exercises diligently and purposefully.
We won't know for certain whether I actually received treatment or placebo until the trial is completely finished at the end of 2020, but my PMP data during my time in the trial shows that my progression was effectively paused for the 9 months or so after my first stem cell transplant. From the perspective of someone living with ALS, that has very little options for effective treatments, there is value in having access to data that shows if a therapy, supplement, or other intervention is helping slow (or even accelerate) your disease progression. This made me feel as if I had some control over a disease that offers very little control and I am very thankful for that.
The work that ALS TDI is doing – both the Precision Medicine Program and their research to find effective treatments for ALS, is extremely important to people living with ALS and their families – and they need our support to keep it going. So, as a former federal employee and a person with ALS, I’m asking you to consider supporting them as part of the Combined Federal Campaign, using the code #98547.