Run in partnership with the Gorlin Syndrome Alliance.

Overview

Gorlin syndrome, or nevoid basal cell carcinoma syndrome, is a rare genetic disorder typically linked to PTCH1, SUFU, or other Hedgehog pathway variants. It causes a lifelong risk of basal cell carcinomas (BCCs), jaw cysts, and developmental issues. While BCCs usually affect adults, pediatric patients face unique challenges with early-onset and recurrent tumors. Managing BCC risk in children requires lifelong surveillance, frequent procedures, and multidisciplinary coordination. This creates a significant burden, including physical scarring, treatment fatigue, and psychosocial anxiety. Gaps remain in understanding disease severity, optimizing prevention, and refining pediatric treatments. To address these needs, the Gorlin Syndrome Alliance (GSA) and the Pediatric Dermatology Research Alliance (PeDRA) established the GSA-PeDRA Gorlin Syndrome Challenge Grant. This partnership funds high-impact research to improve the diagnosis, treatment, and quality of life for affected children and their families.

Focus

Proposed research should bridge critical knowledge gaps or address significant clinical requirements pertaining to pediatric basal cell carcinoma within the context of Gorlin syndrome. Submissions may encompass basic, translational, clinical, epidemiologic, or patient-oriented investigations exploring the underlying biology, preventative measures, diagnostic techniques, surveillance protocols, therapeutic interventions, or the psychosocial impact of basal cell carcinomas on pediatric and adolescent populations. Potential areas of inquiry include, but are not limited to the following: mechanisms driving early-onset or extensive tumor burdens; genetic, molecular, or environmental modifiers affecting disease phenotype; prognostic factors for tumor development and recurrence; innovative photoprotection and prevention strategies; refining pediatric-specific surveillance and biopsy standards; establishing minimally invasive or more tolerable treatment modalities; techniques to mitigate scarring and procedural fatigue; clinical decision-making frameworks for youth; and the creation of patient-centered outcome measures specific to the pediatric Gorlin syndrome experience. The GSA and PeDRA strongly encourage clinical research, especially projects designed to enhance care pathways, identify unmet management needs, assess multidisciplinary care models, or provide foundational data for prospective clinical trials. Where applicable, investigators are invited to utilize available GSA resources, such as the GSA Natural History Study, to bolster their research efforts.

Important Details

• Up to $25,000 to support one year of research
• One award anticipated for this funding cycle
• Application deadline: Thursday, December 3, 2026