An Innovative Clinical Trial for Glioblastoma Identifies Potential Therapies

An Innovative Clinical Trial for Glioblastoma Identifies Potential Therapies

An adaptive phase 2 clinical trial called INSIGhT, led by Dana-Farber Cancer Institute in collaboration with 10 other brain tumor centers, has reported initial results in the Journal of Clinical Oncology. The trial aims to find new potential treatments for glioblastoma, the most common primary brain tumor. While none of the three therapeutics tested so far improved overall patient survival, this innovative trial design has the potential to efficiently identify therapies that could benefit patients.

Traditionally, investigational therapies for glioblastoma are tested against standard therapy in head-to-head trials or in single-arm trials with no control group. In contrast, INSIGhT uses a shared control group to simultaneously test multiple investigational therapies. This approach is more economical and faster than conducting separate randomized phase 2 trials.

In the first analysis of results, the trial enrolled 237 patients with newly diagnosed MGMT unmethylated glioblastoma. Patients were randomly assigned to receive one of four treatments, each with a 25% chance of being assigned.

The trial adapts based on new information. Complex statistical methods are applied to learn from each patient’s response to their assigned treatment. This adaptive randomization reduces exposure to ineffective therapies and increases the likelihood of assigning future patients to the most promising treatment options.

The trial also requires tumor genomic sequencing for all patients, providing researchers with insight into how genetic biomarkers influence treatment responses. In the initial analysis, patients receiving abemaciclib and neratinib experienced longer progression-free survival compared to standard therapy or CC-115 but did not show improved overall survival.

The trial intends to add new treatment arms and is currently testing a novel brain penetrant chemotherapy, an immunotherapy regimen, and standard therapy. This dynamic and evolving trial aims to discover therapies that could potentially benefit patients with glioblastoma.

The study provides a streamlined and rigorous approach to testing new therapies, increasing the reliability of the trial’s findings. The trial’s infrastructure also facilitates understanding of why patients respond or do not respond to specific therapies.

This approach of an adaptive platform trial with shared control arms has the potential to revolutionize neuro-oncology research and accelerate the identification of effective therapies for glioblastoma.

– Dana-Farber Cancer Institute, Journal of Clinical Oncology

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