Working Together to Make an Impact on Patient Care - Friedreich’s Ataxia

At Cydan, we are dedicated to accelerating impactful therapies for people with rare genetic diseases, but first and foremost we’re dedicated to helping improve the lives of patients with rare diseases across many facets of their life. Members of the Cydan team commit time, expertise, and resources to many different groups working to improve the lives of patients with rare diseases.

Our co-founder and President, R&D, James McArthur, PhD, sat down to share more about his relationship with the Friedrich’s Ataxia Research Alliance (FARA), including his appointment to both their scientific advisory board and board of directors.

Why did you choose to join the board of FARA?

I would like to say that I chose FARA, but in reality, they chose me. It started after an early interaction with their team. We needed their help identifying investigators who could be a source of new potential projects in Friedreich’s ataxia (FA) for Cydan. With FARA’s help, I was able to connect with many of these investigators. FARA found my feedback on the investigators’ work helpful and asked me to join their scientific advisory board and, later, their board of directors.

What impact are you able to make for patients by sitting on the board?

I believe my experience and background in developing new therapeutics across a range of therapeutic platforms offers helpful insights to the patient community through FARA. In particular, I’m in the position to help FARA identify projects that are worthy of funding and more likely to be successful — critical to organizations like FARA that need to make every dollar count. In addition, providing a biotechnology perspective to efforts that more broadly support drug development is helpful in advancing projects that are most likely to lead to the development of new therapies.  Lastly, Cydan’s desire to help the rare disease community has enabled me to spend valuable time with FARA and provide financial support.

What responsibility do biotech/pharma execs have in giving back to rare disease organizations?

The phrase “rare disease community” gets tossed around a lot. But I truly believe it is a community — an interdependent ecosystem of patients, parents, care providers, companies and government agencies, all of whom wish to improve the lives of people living with rare diseases. This effort requires input from people with diverse backgrounds, and we all have a responsibility to contribute that knowledge to advance this goal.

Is there an emotional connection for you when you work with FARA or other rare disease organizations?

Yes. There is an emotional connection that is both inspiring and sometimes difficult. I attend many rare disease patient events where I have the opportunity to connect with patients and parents, and better understand their lives and challenges. Interacting with this community energizes me and helps me maintain my focus on finding therapies to help them.

One of my first interactions with a Friedreich’s ataxia patient occurred several years ago when Ron Bartek, a founder of FARA, asked me to walk with a family whose daughter had FA to view posters at a Muscular Dystrophy Association meeting. Their daughter was in a wheelchair and had already experienced a tremendous loss of motor control. The mother asked me to interpret the content of a poster on a new potential treatment for the disease that was still in pre-clinical mouse studies. The poster highlighted the hopeful aspirations of the investigators that this could be a new therapy for FA, even though it was years away from human clinical testing and regulatory approval. These latter points were lost on this family who thought this potential treatment would be able to change their daughter’s life now.

I spent the next hour with them going through many posters in that scientific meeting, explaining the underlying biology, the challenges, and the potential of these new ideas. My experience that day highlighted for me the responsibility that we have to connect the patient community to the science that is behind drug development because, for them, these therapies are closely tied to hope.

What’s the barrier to treatment/research for FA?

Cydan’s work is done on the shoulders of people who’ve gone before us, who have tried and failed. Unfortunately, this is the nature of most drug development stories; many projects that start with huge promise into the clinic, ultimately fail to accomplish what was hoped for. Part of this process is the need to learn more in patients versus in mice or cells.

FARA has done an extraordinary job of encouraging many companies to enter into human clinical studies with new therapies for FA. Some of these have failed but, as highlighted above, that is the beginning of success. I believe FARA’s next big hurdle will be its ability to keep patients, companies and investors excited about the development of new therapies, despite these failures. They’ll also need to increase awareness in the medical community around the disease to help clinicians more rapidly and accurately diagnose patients with the disease, so they can get appropriate treatment. This is much tougher than it might appear, but I believe FARA is taking steps to do this as well and I am very hopeful for the future. I believe FARA is a model that many patient organizations could emulate.