A Transformative Therapy for Parkinson's Disease
I did something I did not expect to do or plan to do. I discovered a new way to treat Parkinson's Disease (PD). PD is a horrible disease; after it gradually takes away your ability to move and care for yourself, it starts to affect your ability to communicate, understand or think normally.
I began to study PD to help my dear friend and mentor as he tried to navigate the disease and the health care system. And as I continued to dig deeper into the scientific literature, I started to see the disease differently.
The standard teaching (since the 1970s) is that patients with PD suffer because a deficiency of dopamine, and therefore, that dopamine should be supplemented or stimulated. However, studies show that the individual neurons vital for our ability to move any muscle are producing near-normal amounts of dopamine even in the end stages of PD. At the same time, these neurons cannot protect themselves from dopamine-related toxicity as can normal neurons, so the small number of living neurons to control movement become more dysfunctional and die because of the dopamine they are producing.
With diseases having similar cell physiology and pathology, we've learned that the best long-term outcome is realized when the average levels of the neurotransmitter (in this case dopamine) are reduced while still allowing for temporary increases as activity requires.
I figured out how to control dopamine levels according to this model, which will protect the critical and delicate neurons deep in the brain responsible for the disease, and allow them to function better so people can function better.
Once the target for treatment was recognized, I sought drugs that would block the enzyme I wanted to block, and found one that was approved by the FDA in 1979 for treatment of a complication from a rare cancer. It's well tolerated and well understood, so I filed a Method of Use patent and created the development plan for repurposing this old drug for the new use, treatment of PD.
This is not the typical approach that investors like, or at least not one they typically fund. It offers the possibility of "only" hundreds of millions in revenue instead of billions, so the challenge is finding open minded investors who see the potential impact, understand that the risks are far lower than would be the case of a new chemical, and are ready to make the commitment.
To make it easier for investors, I engaged with the FDA informally to identify a rapid path to market, so that instead of 10 years it should take less than 5 to reach the market, and with higher likelihood of success too.
It's not easy to get experts to admit they are wrong about how to do their jobs. PD experts have been teaching and treating on the premise that more dopamine is optimal, when data support my view that less dopamine is optimal.
My first challenge was finding PD experts who are open to such a contrarian idea, and after a bunch of the big names expressed disinterest (before engaging in a scientific discussion), I found several bright, accomplished, respected PD experts who are excited to pursue this path in clinical trials.
Next, I needed to convince professional investors to fund the program - acknowledging that their standard model does not include investing in repurposed drugs. I did not expect this to progress smoothly as I started pitching (January), and from the outset, expected that I'd fail a lot but only needed one success. This was a key perspective: that my batting average did not matter, and instead, I needed to keep swinging until I finally got one hit. That was all it would take to be successful overcoming this major barrier.