An Innovative Trial Is Changing the Way We Find New Treatments for Glioblastoma

An Innovative Trial Is Changing the Way We Find New Treatments for Glioblastoma

A phase 2 clinical trial led by Dana-Farber Cancer Institute and several major brain tumor centers is revolutionizing the search for new treatments for glioblastoma, the most common primary brain tumor. The trial, called INSIGhT (Individualized Screening Trial of Innovative Glioblastoma Therapy), is the first of its kind in neuro-oncology and aims to rapidly identify therapies that benefit patients.

Traditionally, investigational therapies for glioblastoma are tested individually against standard therapy or in single-arm trials. INSIGhT, however, takes a different approach by using a shared control arm to simultaneously test multiple investigational therapies. This design is more efficient and cost-effective than conducting separate randomized phase 2 trials. The trial has so far tested a control arm of standard therapy against three therapeutics: abemaciclib, neratinib, and CC-115.

The trial employs a strategy called Bayesian Adaptive Randomization, where complex statistical analysis is continuously applied to learn from each patient’s response. The algorithm adapts the trial based on patient outcomes, ensuring that future patients have an increased chance of receiving the most beneficial treatment. This approach reduces the number of patients exposed to ineffective therapies and expedites the identification of promising treatments.

The initial results of the trial, published in the Journal of Clinical Oncology, showed that abemaciclib and neratinib extended progression-free survival compared to standard therapy and CC-115. However, none of the therapies improved overall survival.

INSIGhT is an evolving trial that continues to add new treatment arms. Currently, new patients are being assigned to receive a novel brain penetrant chemotherapy, an immunotherapy regimen, or standard therapy. The trial also requires tumor genomic sequencing for all patients to better understand the influence of genetic biomarkers on treatment response.

This innovative trial not only provides a more efficient and streamlined approach to finding new treatments for glioblastoma but also facilitates a better understanding of why patients respond or do not respond to different therapies. It holds the potential to significantly impact the search for effective therapies and improve outcomes for patients with this challenging disease.


– Dana-Farber Cancer Institute

– Journal of Clinical Oncology,

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