The Children's Tumor Foundation is a 501 foundation dedicated to improving the health and well-being of individuals and families affected by neurofibromatosis. Their four-part mission includes propelling drug research and development through a series of strategic investments, strengthening patient support, increasing public awareness of NF and establishing best practices in clinical care for affected individuals. The Foundation is incorporated in all 50 states with active chapters and affiliates in 37 states. CTF is the largest private funder of NF research.
History
Established in 1978 as the National Neurofibromatosis Foundation by Lynne Ann Courtemanche, RN, neurologist Allen E. Rubenstein, MD and Joel S. Hirschtritt, Esq., the organization changed its name to Children's Tumor Foundation in 2005. In the early years, the organization's focus was on providing patient support and organizing the NF community. From the late 1980s through the mid-1990s, their aim incorporated discovering the genes that cause NF. CTF began concentrating on translational research in 2005; in 2008 CTF also began to fund clinical trials. Under the leadership of President and Chief Scientific Officer Annette Bakker, the organization has shifted from a more traditional funding model to a funder-partner model in order to accelerate the drug discovery process.
Notable achievements
The Foundation awarded its first grants in 1988, launching the first neurofibromatosis research program in the world. In 1985, they organized the NF Conference, the first major gathering of NF scientists and clinicians. In 1990 and 1993, respectively, labs funded by grants from the Foundation identified the genes for NF1 and NF2. In 1997, CTF launched an international summer camp for youth affected by NF. In 2006, the Foundation began funding a drug discovery initiative and piloted a program for a network of NF clinics. They launched a patient registry in 2012 and a specimen biobank in 2013. In 2014, the Foundation established Synodos for NF2, a first-of-its kind collaboration of NF scientists working across institutions to find a cure for NF2. A similar project, Synodos for NF1, is planned to begin in 2015.
Funding model
In recent years, CTF has shifted its funding model from that of a more traditional non-profit organization to one more aligned with the venture capital approach advocated by the Milken Institute'sFasterCures model. CTF now situates itself as a catalyst of NF research and has created active partnerships with patients, scientists, research institutions and both the biotechnology and pharmaceutical industries.
Major initiatives and key investments
The Young Investigator's Award : Begun in 1985, this two-year award is granted to post-doctoral and early-career scientists. It is intended to establish awardees as independent investigators. Many former awardees have become leaders in the field of NF research and within the clinical community.
Drug Discovery Initiative : Begun in 2006, this award facilitates preclinical screenings to develop data of both in vitro and in vivo screening of NF therapeutics.
Children's Tumor Foundation NF Clinic Network: CTF established the NF Clinic Network in 2007. This network currently includes 47 affiliate clinics throughout the United States, all of which adhere to current consensus clinical care guidelines for NF. Over 10,000 patients with NF are treated at these clinics annually.
NF Conference: In 1985, CTF convened the first-ever scientific meeting focusing on neurofibromatosis with the goal of encouraging research and fostering collaboration in the scientific community. Held annually, it is now considered the preeminent meeting of NF scientists and clinicians worldwide.
NF Forum: In response to continued layperson interest in the proceedings of the NF Conference, CTF initiated the NF Forum. Held annually, the Forum is attended by those affected by NF and their families as well as scientists and advocates in the NF field. It is held contingent to the NF conference on a biannual basis.
NF Preclinical Drug Screening Consortium : The NFPC was a $4M, 4-year multi-site preclinical drug screeningconsortium designed to accelerate identification of candidate drug therapies and speed their progress to the clinic.
Neurofibromatosis Therapeutic Consortium : Launched in 2013, this collaboration with the NF Therapeutics Acceleration Program at Johns Hopkins School of Medicine is a three-year program that continues preclinical testing using animal models established during the NF Preclinical Drug Screening Consortium.
Patient Registry: The registry's intended primary outcome is “to speed the development of promising new treatments.” It serves as a referral source for clinical trials and provides registered patients with up-to-date, personalized information about their particular manifestation of NF. It currently has over 5000 registrants.
Synodos for NF2: Launched in 2014, Synodos for NF2 is consortium of scientists from a variety of institutions who have agreed to collaborate, sharing data in real time and working together to find treatments for NF2.
Synodos for NF1: Announced in 2014 and launching in early 2015, Synodos for NF1 is a consortium of scientists from a variety of institutions who have agreed to collaborate, sharing data in real time and working together to find treatments for NF1.
Patient support
The Foundation publishes educational brochures for patients, their caregivers and other interested parties on a variety of subjects. Many of these brochures are available in both English and Spanish. Participation in the NF Registry offers additional support to patients and their families. In addition to providing up-to-date information about applicable clinical trials, the registry allows patients and their families the opportunity to receive information targeted to their specific NF-related symptoms. In addition to these efforts, The Foundation also sponsors an annual summer camp for youth living with NF.
Advocacy
CTF advocates on a national level for funding of neurofibromatosis research. The Foundation is considered instrumental in securing both initial and ongoing funding through the Department of Defense Congressionally Directed Medical Research Program Neurofibromatosis Research Program. As a part of their efforts, CTF organizes volunteers to petition their representatives in Congress and the Senate online, by letter and in person, to urge continued and increased funding through both the CDMPR-NFRP and the National Institutes of Health.
Fundraising programs
The Foundation sponsors a number of programs designed to raise money and bolster NF awareness, as well as provide a sense of community for those who live with NF. These programs include Racing4Research, NF Endurance, NF Walk and Cupid's Undie Run.
