News & Stories

Patient Perspective on ALS Research: Stacy Farber

Exactly 3 years ago I heard 3 letters that forever altered the trajectory of my life: ALS. It was something I studied in medical school, but I never imagined it would apply to me. I learned about researching drug trials, rare diseases and so much more that I never thought would enter my world.

Boy, was I wrong.

After taking a moment to absorb the shock of my diagnosis, I dove into learning everything about ALS – accessibility, treatments, ventilators, FRS (Functional Rating Scale), clinic visits, social security benefits, insurance coverage, voice banking, support groups, ongoing trials and research.

The list was overwhelming, but breaking down each category helped me navigate forward. Since there is no cure and very few available approved drugs to treat ALS, I chose to research and become my own ambassador for learning about ongoing clinical drug trials.

Deciding to enter a clinical drug trial is a personal choice with new terminology to understand:

Placebo: A substance that mimics the appearance and mode of delivery of the therapy being tested but has no therapeutic effect and is used as a control in a trial.
Blinding-test subjects: Being unaware that they are being given placebo or actual therapy.
Open Label Extension or OLE: A period of time when the blinded portion of a trial is concluded, and the patient receives the actual therapeutic modality.
Expanded Access Program or EAP: Programs that enable patients to receive particular study therapies when they are no longer eligible for any drug trials. This may or may not involve data collection.

Different types of trial studies exist, from therapy intervention to observational. Resources like clinicians, and trial search engines created by organizations such as I AM ALS and ALS TDI help find available trials. Choosing a trial involves factors like location, number of in-person visits, placebo to drug ratio, costs and/or reimbursement, inclusion/exclusion criteria, therapy delivery, trial duration, and potential for OLE.

All of the above is only scratching the surface. I knew at the time of my diagnosis I would want to participate in clinical trials. Engaging in clinical trials is not just for personal benefit but also for future ALS patients. Most trials accept patients within two years of first symptom onset. My first symptom was in July of 2020 and luckily my diagnosis was not that long after, in February of 2021. Paramount is that time is of the essence. At the time of my diagnosis, there were only two approved therapies that applied to me, Riluzole and Radicava. Once I was enrolled in those, I opted to apply for the Healey Platform Trial. The 3:1 drug to placebo ratio and the multi-platform drug study were very appealing to me. In addition, the active part of the study was for 6 months with an available OLE to follow.

I was accepted into the Healey Platform D, or Pridopidine study. The in-person visits were not taxing and the study involved me taking a capsule twice a day. I still do not know if I received the drug or a placebo for the first six months. I am nearing the end of my OLE and have done very well with this trial. Unfortunately, the phase of trial I was on did not meet its primary end points, however potentially showed some promise in speech and breathing. My understanding is that a further trial is being planned to investigate this. While I am now wheelchair bound with limited use of arms, hands, breathing is compromised, but speech and swallowing are still strong for which I am extremely grateful. I can only at this point wonder if this is because of Pridopidine. Next up looking for availability of Pridopidine in EAP, as I am no longer eligible for clinical drug trials at this point in my disease.

Participation in clinical trials limits further entries into any additional clinical trials. However, there is no limit to the number of observational studies you can partake in. I have found this very rewarding and have been a part of speech studies, brain computer interface studies, and studies that evaluate tools that measure muscle strength. It is a passion of mine to pay it forward for the help I have received from the ALS community.

Following the theme of paying it forward, I was enrolled in a two-day course with the Critical Research Learning Institute to become a NEALS Research Ambassador. This has allowed me to sit as a patient advisor for various pharmaceutical companies, attend virtually the International Alliance Meeting for the past two years and spread the knowledge gained to other persons and caregivers living with ALS. I am active in many ALS organizations and gladly engage in advocacy and fundraising. I’m thankful to ALS United Mid-Atlantic for providing the opportunity to pursue these endeavors.

For further questions about ALS research and if you could be a candidate for a study, please contact a member of the ALS United Mid-Atlantic care team.

Share This Page: