Treating brain tumors – how can we approach this differently?

Khalid Shah, MS, PhD
Massachusetts General Hospital

“Malignant glioblastomas have virtually defied all therapeutic modalities to date,” says Khalid Shah, MS, PhD, director of the Center for Stem Cell Therapeutics and Imaging and vice chair of the Department of Neurosurgery Research at Brigham and Women’s Hospital (Boston, MA); and a principal faculty member at the Harvard Stem Cell Institute (Boston, MA). 

“We know they (brain tumors) can’t be treated with normal, systemically delivered drugs, because most traditional cancer fighting drugs cannot reach the brain. To have an impact, we need to think outside of the box.” 

It was Dr. Shah’s creative thinking that attracted the interest of the Alliance for Cancer Gene Therapy early in his career. “Alliance for Cancer Gene Therapy was one of the first major funding agencies to believe in my vision. Its funding helped me get started, build a foundation for my future research and eventually attract more funding from other sources.” 

“I always wanted to find a cure for tumors in the brain,” says Dr. Shah who once lost a good friend and last year ironically lost his father to glioblastoma. “The goal of our research is always to figure out how to realistically translate our work into the clinic, and ‘how can we approach this differently?’ is the question we always ask.” 

Dr. Shah’s initial research focused on homing and engineering stem cells with tumor-specific agents. As his studies evolved, the scope of work expanded to address causes of therapy resistance; the need for better, more appropriate tumor models that could authentically replicate what happens in clinical settings; and reverse-engineering approaches to gene editing using a patient’s own cells. 

The progress Dr. Shah achieved with Alliance for Cancer Gene Therapy funding was presented at several major scientific conferences and in groundbreaking papers published in Nature Neuroscience, Journal of the National Cancer Institute , and in a review paper entitled “Stem Cell-Based Therapies for Cancer Treatment: Separating Hope from Hype” in Nature Reviews Cancer. Dr. Shah’s engineered stem cell work also has been showcased around the globe in feature stories on the BBC and in Smithsonian to name a few. 

Fast forward to today and we find Dr. Shah working closely with the U.S. Food and Drug Administration and two start-up companies, preparing to launch promising new therapies into the clinic to treat brain cancer patients. 

In the meantime, the fight continues at Harvard Medical School where the Shah Laboratory is now equipped with powerful tools and technologies for investigating questions about cancer, including hundreds of tumor cell lines and different types of mouse models, DNA expression constructs, stem cells, oncolytic viruses and CAR-T cells. 

Recently, he and his team have been focused on developing cell-based immune therapies and their achievements have been published in high-impact scientific journals, including Proceedings of the National Academy of Science of the United States of America (PNAS) and Science Translational Medicine with feature stories in the New York Post, Boston’s NPR News Radio, Scientific American and many others. 

This Alliance for Cancer Gene Therapy Research Fellow is funded by Swim Across America. 

“Without question, our achievements of the past decade have their genesis in the support and encouragement I received from great mentors, great institutions, and Alliance for Cancer Gene Therapy.”

Related Research Study

Malignant glioblastomas have virtually defied all therapeutic modalities to date. Successful therapies will need novel strategies targeting the underlying pathogenesis which may ultimately offer an innovative approach for glioblastoma patients.   This proposal aims at using neural…

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