Rare Diseases

We need to approach rare diseases with a hive mind to find a cure

I was diagnosed with Parkinson’s disease with the rare GBA mutation at age 49. For most people with Parkinson’s, symptoms emerge in their 70’s or 80’s. My family has no history of the disease. Like many of the 60,000 Americans who are diagnosed every year with this disease, I was overwhelmed by this news, and debated keeping it private or sharing it publicly.

For 20 years, I have worked in health-care venture capital, collaborating with scientists and doctors to find solutions to difficult medical problems, particularly in rare diseases. I’ve been fortunate enough to invest in life-saving and quality-of-life-enhancing therapies, including three different investments in companies that, coincidentally, will soon produce drugs to treat the symptoms of Parkinson’s. While important steps forward, none of those drugs will provide a cure for this disease.

Tune in to CNBC’s “Squawk Box” at 6:30am ET. Jonathan Silverstein will be a guest.

Venture capitalists are often reluctant to swing for the fences for cures because we lack a complete understanding of the disease, leading to a low probability of a return on investment and high expense to reaching clinically meaningful results.

However, as a patient, all I want is to find a cure.

After my diagnosis, my professional life suddenly took a very personal turn. I went public with my story in Forbes on April 27, and was quickly inundated by emails and calls from all over the globe. I realized my unique position to make a real impact on Parkinson’s disease. I can raise awareness and the funds necessary to accelerate research into therapies that provide not only incremental improvement in the symptoms, but hopefully, a cure. I can generate philanthropic support, not only from concerned individuals and foundations, but major pharmaceutical companies, as well. But beyond contacts and an ability to mobilize minds and money, something else is needed.

Achieving progress for GBA-PD, and many other rare diseases like it, might require a different mindset within a philanthropic medical discovery endeavor….