Bruce Korf, M.D., Ph.D., associate dean of Genomics.
A $25 million award from the Department of Defense will extend funding for the Neurofibromatosis Clinical Trials Consortium for another 10 years. The NFCTC was initially funded in 2006, with renewal funding received in 2011, 2016 and late 2022. The new 10-year, $25 million award comes from the DoD through the Neurofibromatosis Research Program, one of the Congressionally Directed Medical Research Programs.
Bruce Korf, M.D., Ph.D., associate dean of Genomics and chief genomics officer at the University of Alabama at Birmingham Marnix E. Heersink School of Medicine, serves as the principal investigator of the NFCTC and leads the NFCTC Operations Center at UAB. The NFCTC Data Center is based at the Children’s Hospital of Philadelphia, in Pennsylvania.
“The funding from the DoD allows unique opportunities to partner with well-established neurofibromatosis centers both nationally and internationally, pooling expertise and resources toward the common goal of testing new treatment opportunities for neurofibromatosis,” Korf said.
The consortium has grown from nine to a total of 24 clinical sites, including two sites in Australia. The NFCTC has conducted 18 clinical trials involving more than 500 study participants and produced 18 peer-reviewed publications. There are five additional trials in development to fulfill the obligation of up to 12-15 trials over the next 10 years, and several more publications are planned.
The neurofibromatoses are a set of three different conditions involving the development of tumors that may affect the brain, spinal cord, and the nerves that send signals between the brain and spinal cord and all other parts of the body. They are caused by changes in genes that play key roles in suppressing growth in nervous system cells. These genetic changes keep the genes from making normal proteins that control the ability of the cells to function properly. Without the normal function of these proteins, cell growth increases, leading to the formation of tumors, as well as multiple other medical problems.
Neurofibromatosis 1 is the most common of the three conditions. Although many people with NF1 inherit the genetic change that causes the condition, approximately 50 percent of cases arise from a spontaneous genetic mutation in the NF1 gene. Aside from tumors of the nervous system, affected individuals may develop abnormal bone growth, cardiovascular problems and malignant tumors, among many other manifestations. Persons with NF1 also often demonstrate behavioral problems, attention deficit disorder and learning disabilities. Neurofibromatosis type 2 causes tumors of both hearing nerves, as well as other cranial and peripheral nerves, and also tumors of the membranes that cover the brain and spinal cord. Schwannomatosis, the third form of neurofibromatosis, leads to painful tumors along peripheral nerves.
Neurofibromatosis occurs in both sexes and in all races and ethnic groups. There is no cure for neurofibromatosis, but treatments can address symptoms.
According to Korf, the NFCTC has allowed its investigators to develop innovative protocols through collaborative discussions with various disease and discipline committees, allowing for quicker turnaround of scientific reviews and regulatory approvals.
Additionally, the NFCTC has partnered with pharmaceutical companies to acquire therapeutic agents and with other organizations to assist with funding to conduct additional trials.
This work was supported by the Office of the Assistant Secretary of Defense for Health Affairs and the Defense Health Agency J9, Research and Development Directorate, of the U.S. Army Medical Research Acquisition Activity at the U.S. Army Medical Research and Development Command, in the amount of $25 million through the DOD FY2022 Research other Transaction Agreement – Neurofibromatosis Clinical Trials Consortium Award under Award No. W81XWH223001. Opinions, interpretations, conclusions and recommendations are those of the author and are not necessarily endorsed by the Department of Defense.