Andrew Carnegie, the 19th century business magnate and philanthropist, once said, “It is more difficult to give money away intelligently than to earn it in the first place”. I wholeheartedly agree and here’s why.
Making wise and effective funding decisions requires a rigorous process and unwavering attention on multiple fronts: 1) a thorough understanding of the Rett research landscape at large; 2) synthesizing that knowledge into a strategic research plan; 3) ongoing vigilant scrutiny of novel technologies and scientific advancements that can be leveraged to discover and develop novel therapeutics; 4) recruiting pioneer scientists to our cause; 5) objective and rigorous peer review of every proposal to evaluate scientific integrity and relevance; 6) nurturing collaborative teams focused on advancing science toward a cure.
None of this is easy. But with years of experience and the right team in place, RSRT does it well. Rett families, donors and supporters should feel confident that our team has a thorough understanding of the Rett research landscape. And by now you know that we have a strategic research plan in hand, Roadmap to a Cure. How do we execute on this plan?
Most disease focused non-profits follow the NIH format when it comes to soliciting proposals. They distribute a Request for Proposal (RFP), review the proposals that are submitted, prioritize and fund the ones they can afford. Indeed, this is the method that I employed during my tenure at the Rett Syndrome Research Foundation (which later merged with IRSA to become IRSF, dba Rettsyndrome.org).
RFPs are an effective way to entice researchers to work on Rett. But they are passive by nature, meaning there’s no control over who is submitting or when, or what they are proposing, and often foster competition amongst top scientists. The funding agency waits and hopes to receive cutting edge proposals and researchers compete with their colleagues for a limited pool of funds.
We’ve opted for a more proactive and unique approach. With the benefit of strong relationships with our advisors, researchers (RSRT-funded and not), clinicians and industry, our team identifies projects that we feel will move us, not incrementally, but significantly towards a cure. Once a project is identified we make an internal list of researchers with track records of success and who are innovative, out of the box thinkers, passionate and collaborative. Then we set about recruiting them. Sometimes a proposal is forthcoming after a single phone call and sometimes it takes several years of courtship. Often the researcher is welcomed into an existing RSRT-led collaboration where open sharing facilitates progress.
With this approach we’ve funded projects that fill glaring knowledge gaps (eg. the MECP2 Consortium tasked with identifying the function of MECP2); and projects that open an entirely new area of Rett research that didn't exist previously (the Reactivating MECP2 Consortium or the new methods of MECP2 RNA modifications) or projects that accelerate the development of an important approach to a potential cure (the Gene Therapy Consortium). We’re always looking for new ways to move the field forward.
We recognize that sometimes great ideas come out of left field so we also accept and consider unsolicited proposals from any researcher at any time.
Our goal is to fund scientists who enjoy collaborating with other top scientists to leverage complementary knowledge, expertise and insights to expedite the generation of high quality, reproducible data that can be licensed to an industry partner with the funding and expertise to move into human clinical trials. We achieved this goal with AveXis in gene therapy, and hope to repeat with our other approaches as well.
One of our core values at RSRT is transparency. By that I mean accountability for every dollar donated and its impact on the research. It also means giving Rett families and our supporters a clear and open view of how RSRT works and makes decisions. I hope this article gives you a good sense of how the projects we support come to be. Stay tuned for next week’s blog that addresses the important topic of peer review. I always welcome thoughts or questions, so please feel free to reach out any time.