Inside Pediatrics Spring 2025

On the pediatric side, non-medical specialists such as social workers, child life specialists and school liaisons provide the holistic level of support children and their families require. “There’s so much that needs to be to be managed outside of our clinic with these children,” Metrock said. “So the social worker and school liaison really help bridge the gaps between school and life.” The clinic also works closely with the Hope and Cope team to help address neurocognitive and mental health issues. “We’re very committed to providing care for these patients, not just for their tumors, but for how the disease affects their life outside of our clinic,” she said. “But I always felt we could grow. So I’m very excited that Dr. Brown is here and that we have a new push for what we can do for these families.” That includes building on the existing multidisciplinary foundation and working on streamlining care for families so they don’t have visit the hospital—often hours away from their homes— for multiple appointments.

To learn more about Metrock’s leadership at Children’s and UAB, check out this episode of our INSIDE PEDIATRICS podcast.

Neurofibromatosis Clinical Trials Consortium (NFCTC), which coordinates research across 24 sites internationally. Girish Dhall, M.D., who directs the Division of Pediatric Hematology, Oncology and the Blood and Marrow Transplantation Program at Children’s, 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 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 robust genetic labs for the disease, which has identified more than 3,000 NF type 1 mutations. The research team also played a crucial role in developing 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 said. “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 and are very aware of the responsibility we have to move forward for these patients,” Metrock said. ●

“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 clinics. UAB is the headquarters for the

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