2025 Annual Report

Neurofibromatosis Clinical Trials Consortium

72+

Began in 2006

Grown from 9 to 24 Sites 19

Investigators

17 500

$ 5 , 000 , 000 In funding acquired

Clinical trials launched involving more than

Papers published and 5 more in progress

patients

launched 17 clinical trials involving more than 500 patients, with eight trials currently in development; published more than 19 peer-reviewed papers with five in progress; and landed more than $5 million in funding from pharmaceutical companies, foundations and government sources. In addition, UAB boasts one of the world’s most ro bust neurofibromatosis genetic labs, which has identified more than 3,000 NF type 1 mutations. The research team also played a crucial role in developing selumetinib, the first FDA-approved drug for NF, which blocks the action of an abnormal protein that signals tumors to grow. This can stop or slow tumor growth. While selumetinib has been a major step forward, more fast-acting targeted therapies are needed, Brown said. “These patients can develop new and enlarging tumors in a relatively short period of time,” she added. “There is very much a need and value in finding medications that can stabilize or shrink those tumors over the long term.” In the meantime, she and Metrock focus on proactive management. “We’re very proud of what we have here,” she said, “and are very aware of the responsibility we have to move forward for these patients.” ●

plinary foundation and working on streamlining care for families so they don’t have visit the hospital—which might be hours away from their homes—for multiple appointments. “They have other children, they have jobs, they have everything outside in life. And so, us asking them to ‘come back, come back,’ can be quite overwhelming,” Metrock said. “So, how can we streamline their care so that they’re getting the best care they can in a way that allows them to keep living their life away from clinic in the hospital?” That involves bringing more clinicians interested in the condition into the clinic as well as expanding an already robust clinical research program. Indeed, research is embedded in the mission of the clinic. UAB is the headquarters for the Neurofibromatosis Clinical Trials Consortium (NFCTC), which coordinates research across 24 sites internationally. Girish Dhall, M.D., director and professor in the Divi sion of Pediatric Hematology, Oncology and the Blood and Marrow Transplantation Program at Children’s and UAB, leads the consortium. Since its inception in 2006, it has grown from nine to 24 sites with more than 72 investigators, according to Karen Cole-Plourde, the NFCTC operations center program director. It has also

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