2022 FBI Research Scholars - Rebecca M Brown, MD, PhD and Nicholas David Gulati, MD, PhD скачать в хорошем качестве

2022 FBI Research Scholars - Rebecca M Brown, MD, PhD and Nicholas David Gulati, MD, PhD

2022 Friedman Brain Institute Research Scholars Ram Sundaram Research Scholar Award “Diphencyprone Immunotherapy in Cutaneous Neurofibromas" Associated with NF1 Neurofibromatosis type 1 (NF1) is the most common genetic tumor predisposition syndrome, affecting up to 1 in 2500 individuals, or ~3400 children and adults in New York City. Mutation in the NF1 gene leads to the growth of benign tumors known as neurofibromas along large nerves and in the skin. Cutaneous neurofibromas begin to appear in late childhood and they never spontaneously resolve, resulting in a tumor burden that can sometimes reach the tens of thousands. They are disfiguring and can be painful or itchy, leading to loss of confidence, anxiety, and depression. Currently, the only treatment is surgery or laser therapy, however, only a limited number of tumors can be simultaneously removed, recovery can be painful, and there is significant risk of regrowth. There is a strong need for noninvasive topical treatments for cutaneous neurofibromas. Diphencyprone (DPCP) is a topical immunotherapy that works by activating immune cells within the region of application. DPCP has previously been used to eliminate warts, restore hair growth in idiopathic baldness (alopecia areata), and treat malignant melanoma. We are planning a phase 1 clinical trial for DPCP in adult patients with NF1 and 4-20 symptomatic/bothersome cutaneous neurofibromas with a goal accrual of 40 patients. Outcomes include safety, tumor regression, immune activation measures, and quality of life. Learn More about the Friedman Brain Institute: https://icahn.mssm.edu/research/friedman