Celebrating 35 Years

NF Midwest Research Program

Lobbying for federal funds and NF Midwest grants to a clinic in our region conducting clinical trials, helped in the development of a drug now showing promise in the treatment of plexiform tumors.

NF Midwest Work Aids Development of Orphan Drug for NF

In February AstraZeneca and Merck announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1). Research and clinical trials on selumetinib and other MEK inhibitors have been possible in great part due to the lobbying work of NF Midwest and other NF organizations. Every year we push for federal funding of NF research through the Congressionally Directed Medical Research Program (CDMRP) and language encouraging neurofibromatosis and schwannomatosis research through the National Institutes of Health.

In 2017, our lobbying efforts resulted in $15 million for NF research, plus the NIH spent around $31 million on NF research. Since 1996, over $300 million has been provided to NF research through the CDMRP alone!  The value of our lobbying efforts is incredible and multiplies tenfold! We are seeing the fruit of our efforts in the development of Selumetinib and other drugs.

Also, many of the clinical trials for MEK inhibitors have been done through the NF Consortium which is funded through the CDMRP. Several patients have participated in MEK inhibitor trials through the Consortium at the University of Chicago. Funding for a research coordinator for those trials and other research is provided by NF Midwest through direct grants for many years. So far, over 70% of participants in this MEK clinical trial are showing reductions in tumor size of 20-50% of plexiform tumors.

Read more about the designation of selumetinib as an orphan drug here.

Comments (4)

  • I wanna know how to try this out I have neurofibromatosis type 1 and i tried the Mekinist and It just made me really sick

  • My 40 year old son has NF1 and the tumors are growing and mulipling including one by his eye.

    Where can he have this drug administered to him?

    • Susan, Sorry this post didn’t come through to us previously. This drug is not available yet. Orphan status gives pharmecutical companies incentives, etc to do testing and clinical trials. Also, it is for plexiform tumors…which the one by his eye could be. You could see an experienced surgeon about removing them.

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