Our next steps. Your next step?

Many of you send emails asking questions about the clinical trial, ranging from scientific to geographic, and for some the incredible offer to participate in the initial experiments.

As one who started working in research in high school and moved into clinical research in college before launching my career as an academic cardiologist, I am humbled by the willingness of people to enter trials. Of course I understand that for many the motivation is to pursue an experimental option when nothing else is possible. But for so many - including some people who have participated in more than one trial - the motivation includes the desire to advance knowledge and help others, even if not themselves.

The plan is to share all the information about the clinical trial here - as we hit meaningful milestones. In that context, here are links that will help you understand how clinical trials work and what you should understand before you participate.

The NIH: why participate? It's worth following the link with information about the basics of clinical trials.

I like the way the National Cancer Institute discusses clinical trials. I believe it is because trials in cancer used to include the use of drugs with a lot of toxic effects, they tend to communicate more clearly the risks of participating in clinical trials, as you will see by following this link.

So this educational approach could be your next step.

More Next Steps

We've written about manufacturing and chemistry. We've alluded in various interviews posted on the internet about our engagement with the FDA. Our next steps focus on FDA-regulatory matters.

To conduct a clinical trial in the USA, an application needs to be filed with the FDA called an Investigational New Drug application (IND). People usually talk about getting the FDA to approve an IND but technically there is not an approval but rather an allowance or authorization. And along with a successful IND filing comes legal permission for the drug to be imported into the US and be transported across state lines. That's important for us because we are manufacturing drug outside of the USA.

Once an IND is filed, the FDA has 30 days to respond. Technically, no response in that time period means the trial can begin. We do not anticipate that to happen for our program because when a commercial entity is developing a drug the FDA understands it needs to guide the program in its initial stages so that subsequent requirements can be met. Simply, if the first trial is sloppy scientifically or operationally then the FDA may not find its results sufficiently reliable to allow the next study. So there is always feedback at this stage.

Traditionally, the back and forth between FDA and Sponsor for a program like ours would take up to 3-4 months to complete, which would then allow the trial to begin. I'm hoping to avoid any political interpretations to this next matter, as it is a scientific, clinical and logistic matter that could affect your chances of seeing this or any breakthrough approach move foward in development. With the reductions in force and other attrition at the FDA over recent months, that goal of successfully completing the IND review is unlikely to be 3-4 months, and more likely to be at least double that amount of time.

That means another next step for you could include contacting your Representative and Senator and demand that the FDA be allowed to revert to its prior staffing and resources. So many voices are clamoring for changes or no changes. If you want options for your Parkinson's, it is time to join that effort with others with the same needs and lobby for preservation/restoration of the FDA.

Our Next Step: Focus on the IND

For us, the next few months will be mostly focused on preparing the IND. If you look at the required content of the IND and read some of the 15-page list of items the FDA requires, it would be easy to recognize that this is a huge undertaking. Because it is.

But as I shared when discussing manufacturing and chemistry, I know that there is no way to successfully write a first-rate IND by looking at the entirety of its requirements. Instead, I will look at each subsection and the subsections of it. I'll make the IND a large number of very small projects - each on a scale I can complete. And when there are areas outside my expertise, particularly the manufacturing parts, I'll leave those to other experts who are helping me with the operations already. And when I need, I'll reach out to my network to get assistance from those with more expertise than mine.

It's not one project. It's several hundred, each of which become manageable. And as I start to string them together, the progress will energize me and enable completion and filing of a first-rate IND.

And that IND will lead to the specifics of what the trial will look like - the type of people to study and the nature of the measurements to be made. Once the IND is authorized/allowed, that's when I can share much more information, which will also be posted in clinicaltrials.gov.

Then the clinical work will begin.