Understanding the genetics behind brain tumors.

Alexander H. Stegh, PhD
Northwestern University

In his laboratory at the Northwestern University Robert H. Lurie Cancer Center (Chicago, IL), personal experience drives Alexander H. Stegh, PhD, to find new hope for people fighting glioblastoma (GBM). 

When Dr. Stegh’s grandmother developed metastatic brain tumors after being diagnosed with end-stage breast cancer, he witnessed the vicious toll the disease had on her and everyone around her. Her battle inspired him to confront this insidious disease, and his education and training led him to do so by pioneering a better understanding of the genetics behind brain tumors. 

Dr. Stegh appealed to the Alliance for Cancer Gene Therapy for support. He had a hunch about the IDH1 gene enzyme and its role in fostering the growth of brain tumors. The Alliance for Cancer Gene Therapy’s investment in Dr. Stegh’s innovative ideas allowed him to take a closer look and to prove that when compared to normal brain tissue, GBM tumors have significantly higher levels of the IDH1 gene enzyme — a pro-tumor gene enzyme that Dr. Stegh learned could reprogram the metabolism of cancer cells, protect them, and enable them to thrive. 

This important Alliance-funded breakthrough was reported in the May 2017 issue of Cell Reports and has contributed greatly to today’s understanding of brain tumor progression. Dr. Stegh is “extremely grateful for the Alliance’s support.” The momentum it created helped catapult Dr. Stegh’s research forward, generating promising data that justified major subsequent funding from the U.S. National Institutes of Health. 

Dr. Stegh’s research has continued to evolve and in 2018, he and collaborators at the Northwestern University Center for Cancer Nanotechnology Excellence and the Northwestern Brain Tumor Specialized Programs of Research Excellence (SPORE) launched an early-stage clinical trial that combines insight from his genetic research with a novel nanotechnology drug delivery platform. 

If the trial proceeds as they hope, a revolutionary new therapeutic will emerge to effectively reach and efficiently destroy GBM tumors in humans by changing the genetic makeup of the tumor cells, dampening their ability to divide, and making them susceptible to standard-of-care therapies. 

“When my grandmother developed metastatic brain tumors after being diagnosed with end-stage breast cancer, I experienced the vicious toll the disease had on her and everyone around her. Her battle inspired me to confront this insidious disease, and my education and training led me to do so by pioneering a better understanding of the genetics behind brain tumors.”


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