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

  • Venture capitalist Jonathan Silverstein was diagnosed with Parkinson's disease, with the rare GBA mutation, at age 49.
  • VCs can often be reluctant to swing for the fences when approaching rare diseases due to a lack of understanding of the disease and worry about the risk for return on investment.
  • He says we need to approach rare diseases with a collaborative "hive mind" to find a cure and is calling on the medical and financial communities to come together.

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.

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. We must consider a "hive mind" approach that enables deeper collaboration between scientists, academics, pharmaceutical innovators, operations experts, venture capitalists and others to identify the most cutting-edge research, and to manage the rapid go/no-go decisions. In other words, we need to tackle the puzzle from a variety of angles.

In the US, a disease is "rare" if it affects less than 200,000 people. Yet there are approximately 6,000 diseases classified as "rare" – translating to millions of Americans waiting, and hoping, for help. The facts are daunting – there is limited knowledge around many of these diseases, few treatments and even fewer cures. Unfortunately, research dollars are limited, federal funding is shrinking, and for many people suffering from these diseases, there is little time to waste.

The rare GBA mutation in Parkinson's (affecting approximately 50,000 people in the US) often causes earlier onset of symptoms which are common to all Parkinson's patients. There is no specific treatment for GBA-PD, and there have been no truly innovative therapies approved by the FDA for any type of Parkinson's over the past three decades. I know firsthand that the impatience felt by those dealing with PD and their loved ones, and the pace of research and development has not kept up with their urgent need.

We believe that by creating a collaborative approach, backed by a philanthropic foundation, focused singularly on one specific rare disease, we will generate novel breakthroughs. We can provide greater attention and speed, accelerating the process and moving science to medicine, from the lab to the patient, more rapidly. This proposed re-orientation of the venture philanthropy model means that nothing is off the table in terms of funding research or pursuing promising leads. The level of talent and experience would focus concerted knowledge and resources on one disease, until a cure is found.

It's time to rethink how we organize ourselves when taking on rare diseases which stymie the medical establishment and receive too little government funding. Uncoordinated, fragmented activity is the enemy of insight and innovation. Only through ambitious collaboration, only by integrating thought and action, can we possibly achieve the critical mass needed to take our efforts to another, infinitely more effective, level.

I hope others in the biotechnology, research and venture capital communities will join me in bringing this model's promise to life. I understand now, even more than I did over the first 20 years of my career, that we need renewed energy in fighting these rare diseases and finding the treatments that will save lives. We have no time to waste. We must strive to be innovators and leaders in this race against the clock, ultimately providing the most important element in the equation: hope.

Commentary by Jonathan Silverstein, co-head of global private equity at OrbiMed, a health-care fund with approximately $14 billion under management. He is also the co-founder of The Silverstein Foundation for Parkinson's with GBA.

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