Last week, the Wall Street Journal reported on the FDA approval of the drug Benlysta as a treatment for lupus. Lupus and NF are biologically very different but the approval of Benlysta can nevertheless prove instructive as we work to find treatments and a cure for NF.
At first look the numbers are daunting. GlaxoSmithKline, or more specifically its predecessor SmithKline Beecham, invested more than $125 million in research and development to bring Benlysta to the market. GSK’s investment came on the heels of hundreds of millions, if not billions, spent by Human Genome Sciences, who partnered with GSK in 2006 to help advance the project.
To put those numbers in perspective, the Children’s Tumor Foundation is the leading non-government funding source of NF research, last year we invested $4M in research into all of the manifestations of NF1, NF2, and schwannomatosis. But while on the surface that number seems small it represents an investment of a far larger scope.
Through our Research Advisory Board, our research programs seed-fund early stage ideas that go on to receive attention, and in many cases follow-on funding from biotech and pharmaceutical companies. Further, our NF Preclinical Consortium and Clinical Trial & Research Awards test drugs that we acquire from biotech and pharma companies to prove safety and/or efficacy (including an on-going clinical trial on vestibular schwannomas with lapatanib, a drug provided by GlaxoSmithKline).
In this way we engage companies, show them the potential for treatments in NF, and rapidly provide them with data to consider the opportunities of advancing the research and development of drugs. Last year alone we partnered with Novartis, Genentech, Pfizer, Avila, AstraZeneca, and Merck, among other companies. This year those partnerships will continue and expand as we search to find treatments for all of the manifestations of NF.
The Foundation is never going to be able to invest hundreds of millions of dollars to bring a drug from discovery to FDA approval. That’s not our role and that’s not something non-profits are designed to do. What we can do is leverage the commitment and resources of our supporters to provide information to companies that make each dollar we invest become many more in follow-on funding. With your help we’re succeeding in this realm like never before.
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