Medical experts presented new targeted therapy options and clinical trial opportunities for adults with neurofibromatosis during a 2026 [1] symposium.

These developments provide critical management strategies for patients facing complex genetic conditions. By focusing on adult care, the symposium addresses a gap in long-term treatment for those living with neurofibromatosis type 1 (NF1) and schwannomatosis related to NF2.

The event was hosted by the Cleveland Clinic Neurofibromatosis Program. The University of Alabama at Birmingham (UAB) and Children’s of Alabama served as co-hosts for the gathering.

Neuro-oncologist Dr. Alyssa Lucas shared insights on the future directions of care. The program aimed to educate patients and their family members on the latest medical advancements and how to access emerging trials.

Targeted therapies aim to treat the underlying causes of tumor growth associated with these conditions. This approach differs from traditional symptom management by attempting to modify the disease progression in adult patients.

The collaboration between the Ohio-based Cleveland Clinic and the Alabama-based institutions underscores a multidisciplinary effort to standardize care. The symposium provided a platform for families to learn about the specific criteria required for clinical trial enrollment.

Participants discussed the transition from pediatric to adult care, which often presents challenges for NF patients. The symposium emphasized that adult-specific strategies are necessary as the disease manifests differently in mature patients than in children.

“Medical experts presented new targeted therapy options and clinical trial opportunities.”

The shift toward targeted therapies and adult-specific clinical trials indicates a move away from purely palliative care for neurofibromatosis. By coordinating efforts between major research hubs like the Cleveland Clinic and UAB, the medical community is attempting to create a more cohesive pipeline for adult patients who have historically fallen through the gaps after leaving pediatric care.