Our grant submission to the Michael J. Fox Foundation

Before working at the FDA and DARPA, I was part of the team in the 1990s that launched the first Phase 2 trial of a beta-blocker for heart failure when beta-blockers were considered contraindicated. Beta-blockers are now standard-of-care. In 2003, I was the one voice raising concerns about the safety of a newly approved drug for heart failure, nesiritide, which was poised to generate over a billion dollars in revenue annually (J&J paid $2.4 billion for the US rights). Shortly after publishing my analysis the use of the drug all but stopped, although it took a decade before the drug was removed from the market.

Upending the status quo requires sharp insight, bold imagination and unwavering resilience. But even with clear and compelling data for a therapy that addresses an unmet need, overcoming long-standing perceptions and practices is not a simple process. (Who else is a fan of Thomas Kuhn’s The Structure of Scientific Revolutions?)

I was reminded of this challenge when the Michael J. Fox Foundation (MJFF) turned down our grant proposal to support a clinical trial of dopamine-reducing therapy with RB-190. I am sharing this news with you, a community I’m proud to be part of, who ask me about this grant and others.

First, I deeply respect MJFF’s decision-making process and their funding priorities, awarding grants based on a proposal’s scientific merits and its fit within the Foundation’s funding portfolio. And I recognize that running a major organization such as the MJFF requires detailed forecasting and financial planning that make it challenging to incorporate a novel approach such as ours to conquer Parkinson’s.

MJFF draws from the world’s leading Parkinson’s experts to review grant applications. And these experts did not provide any evidence that our scientific insights were flawed or lacked merit. They did not say we misunderstood the disease, that we overlooked important studies or that we misrepresented the data — all of which validates the scientific basis for developing RB-190 for Parkinson’s.

Over the arc of my career, I’ve learned to trust my intuition and ability to see and understand trends in data that others overlook because they diverge from the status quo. We’ll lean on the lessons learned by so many who proposed radically different perspectives. In science and medicine conventional wisdom must be questioned. Data must be looked at from different perspectives — especially when focused on a condition like Parkinson’s where the need is so great and we are not getting the results that patients need.

So, to my community, take heart — we have much to celebrate over this past year. We’re taking in investors from around the world. We secured a manufacturer and are already making the drug for our Phase 2A trial. We are laying the groundwork for this historic clinical trial. The rejection of the grant proposal is not an ending, but rather, another step forward in the process of conquering PD.