Round 2!

Last October (2024) I attended the Michael J. Fox Foundation annual therapeutics conference in New York City. The showcase of scientists and science was impressive.

As I approached the lecture hall, I saw a member of the scientific team at the Foundation that I had not previously met. I introduced myself and was surpised ty the response that recent presentation was quite compelling. I did not know how to respond. I couldn't think of a presentation where any MJFF people were evident. Others started to engage - trying to get the attention of a Foundation leader. I felt like a failure. Here was an important voice within the Foundation showing interest, and I completely missed the opportunity. We've all heard the importance of having the elevator pitch ready. And I thought mine was. But I was so surprised that I froze and failed.

I was able to reconnect later in the day and we discussed ways to engage the Foundation which included submitting a grant for funding. Of course this sounds obvious, but up to that point, the Foundation was not warm to a submission. The problem was more than my advocacy for such an unusual approach (dopamine reduction instead of the standard of care which was dopamine replacement). They knew I needed $5M and they preferred not to go over $2M. For that amount I could manufacture drug and get ready to start a trial but not generate any data which is what they wanted.

Round 1.

With this shift in interest in our approach, I got busy organizing a grant proposal. In January the submission process began; in June the rejection was delivered.

Thanks to you expressing interest in our approach to the Foundation and wanting to understand why our application was rejected, Foundation leadership met with us and explained how we could make a resubmission stronger. And that was fantastic to hear - their talk about how a resubmission should look. We asked explicitly and they encouraged us to resubmit.

This week we submitted the pre-proposal for our resubmission. And we're excited to submit the full grant application early in December. We've learned a long over the year and in particular, from the engagement with the Foundation's scientific leadership.

They pointed to areas where we need to be better communicators. And we will be.

They raised concern about our engagement with the FDA, which is a big concern to us as well. And so instead of working with the understaffed FDA which cannot possibly respond to the massive amount of submissions as well with 15-20% less people, we shifted the clinical trial to Australia, which provides more predictability and the same quality of care and expertise.

The grant reviewers raised concerns that we did not have enough Parkinson's expertise (I think that was based on the application not making the roles of experts sufficiently clear so the reviewers raised that concern because I am a cardiologist and non a neurologist). Our alignment with experts in Australia addresses this concern. And that alignment is providing amazing input as we move towards requesting approval to start the study in Australia.

Round 2.

So here we are. Starting Round 2. We're ready to go the distance - 10, 12 or even 15 rounds - because we can feel how close we are to launching the trial. And based on the preponderance of data, we're extremely optimistic that we'll see initial signals that we are on the right path as soon as the end of 2026 or early 2027. Of course, that depends on getting support.

While conventional thinking is not with us, the science on our side. That's what keeps us going.

What's next?

In addition to celebrating the holidays with family and friends, we will focus 110% over the next weeks to our grant resubmission to the Michael J. Fox Foundation. We are working to address the concerns shared in response to our prior submission by the Foundation leadership and their expert reviewers in such a clear and compelling manner that they commit to fund this program.

So this will be our last posting until the new year.

We wish you happy holidays and a happy, healthy and fulfilling new year.