Ami Vijay Desai, MD
Three patients with targeted gene fusions responded to the multikinase inhibitor entrectinib (RXDX-101), according to results from an ongoing phase I/Ib study of children and young adults with advanced, previously treated CNS tumors.
, Desai discussed the ideal patient population for entrectinib and what the future could hold for the agent.
OncLive: Can you provide some insight into the rationale and design of this trial?
We know there are some myosarcomas and various other types of tumors that do have our gene fusions of interest, and a lot of this initial study was developed due to some preclinical data in the neuroblastoma population also looking at TRKB overexpression and work conducted in the laboratory of Dr Garrett M. Brodeur at the Children's Hospital of Philadelphia.
, was our recommended phase II dose in the pediatric population.
What are the next steps?
Now that we have the recommended phase II dose, our dose-expansion cohorts are open and the cohorts that we're looking at include patients who have either primary CNS tumors or other extracranial solid tumors that have our gene fusions of interest. Those are the populations we will be focusing on moving forward.
What is the ideal patient population for entrectinib?
gene rearrangements are found in similar populations, including some rare sarcomas such as infantile fibrosarcoma [and] inflammatory myofibroblastic tumors. We also know there are some glioblastoma tumors and primary brain tumors that actually have TRK fusions that have been identified as well. Those are potential populations that may benefit from this drug.
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