The grant application is in...

The Michael J. Fox Foundation sponsors several programs for novel products including the therapeutics pipeline program to support early clinical development of novel products.

After some discussion, it appeared that they would consider supporting our program at Right Brain Bio, though I write that with the understanding that they knew only the high level view when they welcomed us to apply for funding.

This program has two parts, and after the first we were invited to submit a full application, which is not a small undertaking. Last Wednesday we completed our submission (it was due Thursday) and now we wait for their response, which should come by the end of June.

As part of the application, we submitted a lay abstract. This is that summary of our proposal.

Project Title:   Tyrosine Hydroxylase inhibition to Reduce Dopamine in Parkinson’s Disease – a Phase 2A Clinical Trial

Study Rationale: Standard treatment for Parkinson’s is to increase the amount or activity of dopamine, but that does not prevent the disease from worsening. This hints that the disease is not as simple as a dopamine shortage. We recently discovered that the brain cells that control movement have too much dopamine inside them, which could harm these cells and limit people from moving. Lab studies show that lowering the amount of dopamine can restore the balance of these cells towards normal. The challenge now is to learn whether lowering dopamine in people with Parkinson’s might help them.

Hypothesis: Our hypothesis is that brain cells that control movement are dysfunctional and die because they have too much dopamine inside, and by reducing dopamine production, the cells can return towards normal, and the disease can be reversed for people with Parkinson’s.

Study Design: People with Parkinson’s will randomly receive either the active medicine or a placebo for 20 weeks. The drug will be started very carefully to avoid worsening of Parkinson’s, which is a risk with this approach.

Impact on Diagnosis/Treatment of Parkinson’s Disease: If the results in people are similar to that seen in lab studies, then this drug and this approach of reducing dopamine would represent an entirely new way to treat the disease and would present hope that the drug is a breakthrough.

Next Steps for Development: If this first clinical trial shows that the experimental drug can be taken by people with Parkinson’s without complications, then further studies will be conducted to learn if the drug can reverse the disease, and if so, if there are certain types of people more likely to benefit than others.

The goal is to reduce the amount of dopamine the dopaminergic neurons make, while still allowing these brain cells to make surges of dopamine as required for normal function. And we do not believe reducing dopaminergic drug therapy will have the same effect as reducing dopamine synthesis. It is the excess dopamine produced by the cells that we see as the problem, not the amount of dopamine (by way of levodopa) that is prescribed by your doctor.