NFRP 2018 Vignette

Title: Gaining Momentum Through Innovation and Recruitment

Vincent Riccardi, MD; The Neurofibromatosis Institute

Neurofibromatosis type one is the single most common human inherited genetic disorder—about 1 in 3,000 people—and it gets its name from the mass lesions that are called neurofibromas.

It also involves some skeletal problems, some blood vessel problems, and pretty close to 60 percent of individuals have some degree of learning or performance disorder. Some it’s marginal, and I know a number of people with NF1 who have medical degrees or PhDs, so it doesn’t preclude that, but it has a burden in terms of brain activity.

The single best program in terms of consistency of the research that’s done, the consistency of attracting good proposals, has been the DoD Neurofibromatosis Research Program (NFRP). Without any question, it is the best—because the longevity and the openness and the constant recruitment at all levels. I just don’t think we’d be anywhere near making the progress that we’ve made without the NFRP.

If you look at the overall history, the program has gotten better over the years for a number of reasons. One is there’s a much larger number of people who are willing to be able to identify their career as being part of neurofibromatosis. And two, there’s a lot more people coming in who are interested in just cancer in more general terms, or skin problems in more general terms, or the macrocephaly that’s part of it. I think people are being gathered into the cadre of participants from a larger number of fields. And I think that has made a big difference. And I think, if there’s any one accomplishment of the DoD compared to other granting programs, it’s the steady progression of attracting and utilizing people who otherwise wouldn’t be involved in NF. It really has been a power in that way.

It’s the expansive element. It’s expanding into involving more people, more institutions, more aspects of human physiology, more aspects of human anatomy. And since neurofibromatosis is a very complex disorder, with so many facets to it, and more than just neurofibromas, it’s this that’s pointing in the right direction. We’re still gaining momentum, and I think that’s what the NFRP has done—has given a unique momentum in the research process.

I’ve done programmatic review, probably more than anybody else, and thinking about grants that you find exciting, turning you on, yeah. I think that happened more for me this time than any time previously. For 64 applications, my sense was we’re not going to be able to fund all the good ones; it just—there’s too many good ones. So the issue of high-risk, high-gain is ultimately a matter of money. Do you have enough money to do the high-risk, high-gain? It’s, yeah, it’s high-risk, but golly, it’s going to have some high-gain if it works. And I think the NFRP has allowed for that to be the case more often—that there’s enough funds and process of comparisons that it’s possible to take some legitimate risks that usually you don’t get to do.

So that’s part of the success of this program. I think it’s been a format, a setting for people to think out loud in a potentially rewarding way, and the reward goes, not only to the people who get funded, but for those who participate in the reviewing and making the award. There’s developed sort of a fraternity, a much more cohesive community, as a function of the NFRP.