Advancing Hope for Patients on Glioblastoma Awareness Day

Glioblastoma Awareness Day takes place on the third Wednesday of every July. This initiative was started in 2018 by a group of bi-partisan senators, following the death of Senator John McCain from glioblastoma multiforme (GBM). Its purpose is to increase awareness about GBM, the most aggressive and deadly brain cancer.

Nearly half of all primary brain tumors are GBM. [1] These patients experience higher rates of mortality and lower survival timelines compared to patients with other cancers—demonstrating the dire need for increased funding and research. Available glioblastoma therapies are so limited that the National Comprehensive Cancer Network (NCCN) [2] recommends patients who receive a GBM diagnosis join an investigational glioblastoma clinical trial for a potentially more successful treatment than the four FDA-approved drugs.

Supporting Research and Development in Glioblastoma

Medidata is proud to partner with and support many of the 500+ active glioblastoma clinical trials. [3] In today's increasingly complex world of precision medicine, Medidata’s cloud-based platform connects stakeholders to cohesively deliver seamless, end-to-end clinical operations, data integration, quality control, and analyses for better clinical development to improve patient care.

Specifically, the Medidata AI Synthetic Control Arm® (SCA®) team has worked with sponsors on innovative approaches to study design that accelerate GBM research and expedite bringing potentially life-saving treatments to patients through the creation of external control arms (ECAs). This collaborative work includes the following:

Medicenna Therapeutics 

Medicenna Therapeutics is a clinical-stage biotechnology company that uses immunotherapy to combat the world’s most challenging diseases and inspire hope in patients with unmet needs, including those living with GBM and recurrent GBM (rGBM). For their MDNA55 program, Medicenna partnered with the Medidata SCA team to contextualize the treatment effect of their drug in a single arm Phase 2 study to help justify and design a pivotal, Phase 3 study leveraging an ECA.

The result of this partnership was a ground-breaking agreement from the FDA to use a hybrid-ECA in their  pivotal Phase 3 study. As a result, Medicenna will be able to enroll fewer patients in their standard of care control arm—which will ease recruitment challenges, help speed up overall trial timelines, and most importantly increase the proportion of patients receiving a potentially promising drug. Early clinical trials of MDNA55 have demonstrated a survival rate 2x greater in patients with the most aggressive forms of rGBM [4],something that may offer significant hope to patients [5].

Plus Therapeutics

Plus Therapeutics is a clinical-stage pharmaceutical company focused on developing safer, more effective radiotherapeutics specifically targeted at cancers that have been historically difficult to treat, like GBM. Plus Therapeutics leveraged a Medidata AI SCA (i.e., ECA) to contextualize overall survival in two uncontrolled single arm GBM cohorts which increased the scientific-certainty of results and aided in internal decision making [6].

The Medidata Difference

Medidata AI’s SCAs are ECAs built using historical clinical trial data (HCTD) to assist in the development of new therapies by providing important comparative insights. HCTD, as opposed to real-world data (RWD), is made up of patient-level data — complete with the traditional clinical trial endpoints and covariate information as they were captured as a part of their original protocols.

In the case of life-threatening diseases with inadequate standards of care, like GBM, utilizing such HCTD ECAs can be vital to the success of a clinical trial by bolstering evidence for single-arm studies or easing recruitment challenges for later-stage randomized trials where patient drop-out can be of concern. In cases like this, where the NCCN advises patients to join trials with investigational therapies, ECAs can maximize the number of patients that will be able to receive the experimental drug, rather than serving as a standard of care control.

If you’d like to learn how an external control arm could help your trial in GBM or rGBM, please request to get in-touch or learn more here.