When I first jumped on board with angiosarcoma awareness, my mission was to get some research done by directly funding labs. I could never have anticipated what would unfold from these efforts. In addition to our success in raising funds, I have discovered that opening up a dialogue with scientists and clinicians interested in this orphan disease can lead to inter-institutional collaborations. As a result of the network that we have weaved, major grants are being co-written by talented researchers who are dedicated to the mission of eradicating angiosarocma! Although we've raised quite a bit of money, science is expensive, and multi-million dollar grants will fuel this area of research far greater than anything we could possibly do alone. By no means am I trivializing the efforts of all who have donated, to the contrary, that "seed money" has and continues to ignite the field!
I feel strongly that ALL orphan diseases could benefit greatly by modeling this system. I'm calling it the one year plan, from disease non grata to R01 (that's a nice fat grant for all my non-geeky friends). Here's a breakdown of the top 5 lessons that I've learned, maybe they could be grafted onto other diseases that suffer from obscurity. You just need a dedicated biomedical researcher with no need for sleep to get the ball rolling:)
1) Clinicians and basic researchers who are thriving while studying diseases that get little or no government funding are reallllly good. While other researchers are securing nice fat grants, they are publishing on the backs of non-profits (like ours!) which don't offer large multi-year pay offs. Basically, they are able to stay afloat in a publish or perish world holding onto a little yellow ducky.
2) The dedicated few who are actually interested in your rare disease, welcome collaborations with open arms. They are passionate about their work and if you provide them an avenue through which they can answer their questions faster, they're eager to participate.
3) Basic research advances from the publications of others in the field. If you think about a paper, it's like the tip of a pyramid, you couldn't possibly describe all the results, all the reagents, all the resources used to generate the "highlights" that make it to publication. If you engage with researchers who have published something interesting, you WILL find out more, and that more can lead to an entire project with like minded researchers. Scientists often have no idea what's going on in related labs outside their publications, so just getting them to talk to each other can lead to the birth of a new project...one that is already in the current funding mechanism of the labs!
4) If you open up your search parameters from those directly studying your disease, to those studying related diseases that may have some overlap, you could potentially tack a project onto one that's addressing a larger indication. This may draw researchers from other areas into your field if you can make a case for the disease being a good model system, or having some, ANY relevance to their research.
5) More projects lead to more data which leads to more publications which draws in more researchers. Once there is enough compelling data to suggest that the mechanism of an orphan disease has been discovered/and or a cure is in the works, the government will take a closer look at grants that are submitted and the field will continue to grow.
5b) This should be 6, but I said upstairs that I'd only write 5. Bridging the gap between clinician experts and basic scientists is 100% necessary for there to ever be a clinical manifestation of the basic findings. Just getting clinicians up to date with current basic research can result in the administration of brand new compounds that might not be FDA approved on label, but are a better option than hospice! There could be a million different cures sitting on someone's bench in a lab, but if the clinicians don't know about them, they might as well be tick tacks.
Wouldn't it be fabulous if we could convince scientists to do a little pro-bono work? Can you imagine if scientists picked a fledgling charity and for one year, got as much done as possible?