Why am I doing this?

I've shared the personal story that started me on this path to conquer Parkinson's. And looking at the big picture, the possibility (or in my mind, high probability) that our work will yield a transformative therapy for millions is also motivation enough for this work. I don't need to ask why I am going to conquer Parkinson's.

But I do need to keep focus on why I write this newsletter. Yesterday a close friend shared that he was not sure of its purpose. I need to address that.

My goal from the start was to ask questions that could help us learn, enable us to see new opportunities and ultimately lead us to conquer Parkinson's. And recognizing that I had no reason to believe I could be part of conquering Parkinson's back when I started on this path, I wanted to embody the "why not" mentality. After all, so many transformations in medicine started with an idea that seemed crazy and/or was dismissed by the leading experts. And some breakthroughs come from people and places that would never have been predicted to deliver they way they did.

Here are a few examples of contrarian and dismissed ideas that ultimately led to a major medical breakthrough:

1. Stomach ulcers and gastritis (inflamed stomach lining) can be caused by a bacterial infection, so treatment should be antibiotics and not antacids. Barry Marshall infected himself to develop gastritis and then used antibiotics as treatment - not antacids.
2. mRNA vaccines, also known broadly as nucleic acid vaccines were thought to be impossible but eventually their success as COVID-19 vaccines created huge number of potential therapies.
3. Thalidomide caused huge and horrible congenital defects when used for morning sickness in pregnancy, so it was withdrawn from the market. Decades later, scientists discovered that its effect on the immune system could make it the first effective treatment for multiple myeloma. Eventually they were able to conduct the studies and conquer the disease.
4. A Swedish cardiologist (Finn Waagstein) thought the standard treatment for heart failure was backwards, and that instead of stimulating the heart muscle, beta-blockers should be used. He shared that perspective in the eary 1970s. I was a research fellow in 1990 on the team starting the first Phase 2 trial of a beta-blocker for heart failure (when they were contraindicated). Dr. Waagstein was proved to be correct; beta-blockers improve quality of life and lifespan for people with heart failure.

Breakthroughs start with people asking questions. And the questions could be directly tied to the eventual solution or represent the beginning of a circuitous route. They can lead quickly to the eventual solution or take decades. The common theme to success is the willingness to be open-minded, curious and dogged in pursuit of the possibilities.

This newsletter is a way to share how a curious, motivated, data-driven person learns about Parkinson's and applies himself to proving this path will conquer the disease. Occasionally, I'll find a question compelling to discuss even if off-topic. And sometimes the topics may seem mundane (such as my current evaluation of the platform we will use to collect data in the trials). Topics will be selected based on feedback and suggestions you provide.

Ultimately, I want to bring you along on this journey of discovery and (hopefully) success.