Over the past quarter century, my colleagues and I have spent our work and volunteer lives turning innovative ideas into life-changing solutions for those afflicted with rare diseases. With the help of scientists, clinicians, donors, investors, other colleagues, caregivers and those affected, we have used drug repurposing to make a profound and positive impact in many rare disease communities. Our most recent endeavor, Fortuity Pharma, reflects our unwavering commitment to creating effective, affordable therapies for underserved populations by finding new uses for existing drugs and nutraceuticals.
Discovering the Power of Drug Repurposing
My journey into the rare disease space began with the fortuitous realization that many existing drugs hold untapped potential for new applications. As the founder and former CEO of Cures Within Reach, I was presented with a number of drug repurposing grants that had the potential to actually improve patient lives in a very short time for a small amount of money. These grants were being judged against grants that were proposing new and exciting ways to improve patient lives, but the impact was much more speculative, very far off, and very costly. We decided to focus on drug repurposing to leverage the speed, safety, cost and near-term impact. Cures Within Reach helped pioneer the development of repurposed therapies by funding proof of concept clinical trials in patients to bridge the gap between promising scientific possibility and patient improvement. While I was involved at Cures Within Reach, we delivered over a dozen redeveloped drug and device therapies to patients through proof-of-concept clinical trials. And the work of Cures Within Reach continues to this day.
I have continued my work in the rare disease space at the biotech Healx, which uses AI and machine learning to create therapies for rare diseases. Our first AI predicted drug is approved to begin clinical trials early in 2025.
Founding Fortuity Pharma to Accelerate Treatments
My impetus for starting the public benefit corporation Fortuity Pharma stems from my deep belief in the potential of already-approved compounds to address rare diseases. The idea isn’t just to bring new hope to patients but to do so efficiently, ethically, and cost-effectively. Fortuity Pharma is inspired by patient stories like Lucia’s—a young girl with WOREE Syndrome whose life was briefly transformed by the now discontinued drug amlexanox. Stories like hers fuel Fortuity Pharma’s determination to provide access to treatments that might otherwise be lost to history.
Fortuity Pharma’s approach—leveraging existing scientific knowledge and repurposing compounds—perfectly aligns with our team’s expertise in translational medicine and drug redevelopment. This strategy allows us to accelerate timelines, reduce development costs, and focus on diseases that traditional pharmaceutical models often overlook.
Personal Connections Drive Our Passion
Beyond our technical expertise, our commitment to rare disease patients is deeply personal. I have a nephew with Fragile X syndrome who is in his early 40s. Although there have been many attempts to create a therapy for Fragile X syndrome, none exists. For my nephew and his family, there are drugs that exist that could be repurposed, but there is no economic incentive to drive industry involvement. Philanthropy is interested, but often does not have the expertise of the funding capacity. Fortuity Pharma lives in between industry and philanthropy, having the expertise of industry and the passion, perspective and commitment of philanthropy.
Whether through our leadership roles at Fortuity Pharma, Healx, the Kabuki Syndrome Foundation, or other organizations, we remain tireless advocates for collaboration and patient-centric innovation. Our work not only brings hope to patients and families but also inspires a new generation of scientists, entrepreneurs, and advocates to reimagine what’s possible in rare disease research.
