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. Its purpose is to increase awareness about Glioblastoma (GBM), the most aggressive and deadly brain cancer.
Nearly half of all primary brain tumors are GBM. 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 GBM therapies are so limited that the National Comprehensive Cancer Network (NCCN)  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. 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 analysis for better clinical development.
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, including our work with:
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 in their Phase 2 study to help justify and design a pivotal, Phase 3 study leveraging an external control arm (ECA).
The result of this partnership was a ground-breaking agreement from the FDA to use a hybrid-ECA in a pivotal 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  and offers significant hope to patients.
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 leveraged a Medidata AI SCA to contextualize overall survival in two uncontrolled GBM cohorts increasing scientific-certainty and aiding internal decision making.
The Medidata Difference
Medidata AI’s SCA are external control arms 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 an SCA 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 National Comprehensive Cancer Network (NCCN) advises patients to join trials with investigational therapies, external control arms can maximize the number of patients that will be able to receive the experimental drug, rather than serving as a standard of care control.