Earlier this year, the NYU Langone Medical Center launched a new clinical trial led by pediatric neuro-oncologist Dr. Sharon Gardner. The trial is exploring the effects of a new drug on children with a class of tumors that contain a devastating mutation, known as H3 K27M. This mutation is associated with one of the most deadly forms of pediatric brain tumors, known as DIPG (the survival rate is less than 1%).
Clinical trials, utilizing this new drug on adults with the mutation, have been underway for some time, and have shown promising results. A handful of children have already been treated with the drug under the “compassionate use” protocol, and some of them have also responded well. While no one can yet speak to the drug’s efficacy, Dr. Gardner is enthusiastic about the possibilities: “There is no cure for this kind of tumor. None. We’re hopeful that this new therapy can improve the dire prognosis for these children.”