The Children’s Tumor Foundation is a national nonprofit organization dedicated to finding effective treatments for neurofibromatosis (NF), the term for three genetic disorders: NF1, NF2 and schwannomatosis. NF causes tumors to grow on nerves throughout the body and can lead to deafness, blindness, learning disabilities, and some forms of cancer. It affects one in every 3,000 people, more than cystic fibrosis, Duchenne muscular dystrophy, and Huntington’s disease combined. To learn more, please visit www.ctf.org\
From 2010 to 2016, the Children’s Tumor Foundation’s primary objective has been to fill the clinical drug development pipeline. We strategically integrated our investments into this clinical pipeline, resulting in substantial accomplishments by the end of those five years:
-Over 70% of all NF data in the world has been funded or co-funded by CTF.
-CTF investments in the last five years ($26.6M) have already attracted $38.4M in follow-up funding from other sources.
-CTF’s NF preclinical initiatives have generated 115 preclinical studies which have led to 16 clinical trials.
-One of those trials (MEK inhibitor) resulted in over 50% of participants in a clinical trial seeing a reduction of at least 20% in their inoperable plexiform neurofibromas.
-CTF created the NF Registry, which, with over 8,000 patients thus far, has been utilized 22 times to recruit patients to clinical trials.
-The CTF Biobank collected more than 200 tissue samples and a body donation program is in place.
-CTF built an innovative research model called Synodos, which attracted a “dream team” of scientists who pledged to work together collaboratively.
-CTF launched an NF data hub, providing an open access data platform to all.
-CTF collaborated with the National Institutes of Health (NIH), Congressionally Directed Medical Research Program (CDMRP), and Neurofibromatosis Therapeutic Acceleration Program (NTAP) to build a common grant repository for an easy visualization of the NF funding landscape.
The accomplishments of the past enable us to turn our eyes toward lofty goals. Our strategy for the next five years is to double the speed of clinical therapies to the clinic. We are advancing our Synodos consortia, increasing our NF data hub, growing the CTF Biobank, and expanding the NF Registry. With your help, our hope for the future includes partnerships with the pharmaceutical industry and the creation of an NF Biomarker Center to discover measureable biological indicators for NF.
