Neuroblastoma Drug Trial

The Ronan Thompson Foundation
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Lorlatinib, an investigational drug candidate currently in late stage clinical development for the treatment of lung cancer may also prove effective for the treatment of the pediatric cancer neuroblastoma. The phase I trial co-led by Dr. Mossé at CHOP and Dr. Marachelian at CHLA will be conducted in 14 hospitals in the US/Canada and at 2 European sites in London and Paris. The study focuses on a subset of neuroblastoma patients with mutations in the anaplastic lymphoma kinase (ALK) gene. The primary objective of this study is to identify a Phase II dose and assess the anti-tumor activity as a result of administering this investigational drug in children with ALK-driven neuroblastoma. The drug was specifically designed to inhibit tumor mutations that drive resistance. One important feature of this drug is that it penetrates the blood brain barrier and may be effective in patients with relapsed neuroblastoma in the central nervous system.


of $50,000 goal