Ohio State College of Medicine is committed to genetic therapy: focus on disease treatment and prevention

World-renowned neuroscientist Krystof Bankiewicz, MD, PhD, known for his work in translational neurological gene therapy research, joined The Ohio State University College of Medicine to further his cutting-edge research. 

“I came to Ohio State to continue my research and to develop an internationally recognized gene therapy center here,” Dr. Bankiewicz says. “I have been involved in genetic research since the early 1990s, and I am committed to seeing breakthroughs in genetic therapy and editing.”

Gene therapy, or genetic editing, is precision medicine for the brain’s circuitry and helps restore connections that are missing. It allows genetic material to be added, removed or altered at particular locations in the genome. 

Through previous work done at the University of California, San Francisco, Dr. Bankiewicz brings with him several clinical trials focused on preventing the progression of Parkinson’s disease and separately restoring effectiveness of medication used in management of Parkinson’s patients, as well as gene therapy clinical trials for Alzheimer’s disease and rare disorders such as neurotransmitter deficiency. These clinical trials will be supported at Ohio State.

“Gene- and cell-based therapies represent a solution for both rare and common forms of complex disease,” explains Peter Mohler, vice dean of Research at The Ohio State University College of Medicine. “With Dr. Bankiewicz’s arrival here, along with our brilliant other researchers in neuroscience at Ohio State, we have the unique ability to fight diseases that were once thought to be incurable.”

NIH Grant for Gene Editing Technology

 Dr. Bankiewicz’s team of researchers at the Ohio State College of Medicine and researchers at the University of California, Berkley, were recently awarded a four-year, $2.1 million grant from the National Institutes of Health to develop gene editing technology to treat neurological conditions.

This technology will enable doctors to edit defective genes using CRISPR-based therapy on incurable brain diseases including Huntington’s, Parkinson’s and Alzheimer’s diseases. The CRISPR system allows genetic material to be added, removed or altered at specific locations in the genome. 

Through this new technology, treatment of brain diseases once thought to be fatal may be reversed, or progression slowed, leading patients to a better quality of life.

In addition, Dr. Bankiewicz’s team will be developing a next-generation, high precision, neural interface system to be used for therapeutic restoration of sensory deficits — visual, auditory and somatosensory. This is through a funded grant that’s a continuation of a multicenter project in the field of optogenetics.

Past Success in Treating Brain Diseases

Dr. Bankiewicz has conducted research on treatments for rare genetic diseases. His work and clinical trials for AADC have shown significant success. AADC is a disease that severely impacts a child’s motor skills to the point that the child cannot move his or her limbs. His research proved that mid-brain infusion can be safely delivered to children with AADC, that MR-guided delivery is essential for safety and accuracy, and the results support the increase in brain AADC activity and improvement.

The 15 individuals in the study received the treatment one time, and they continue to improve. Dr. Bankiewicz still tracks these patients and will continue the AADC study at Ohio State.

Recently, Dr. Bankiewicz and researchers in California, New York, Massachusetts and Spain had a breakthrough for gene therapy for Niemann-Pick type A. Niemann-Pick is an incurable, inherited disease and is often fatal. 

Researchers evaluated the safety and effectiveness of gene therapy in trials, and Dr. Bankiewicz’s goal is to translate this data into human clinical trials in the next few years at Ohio State. He and his team will move forward with additional research in preparation for clinical trials in humans.

Future of Treating Neurological Diseases

Dr. Bankiewicz and his team will continue to search for treatments for brain cancer, Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, pediatric neurotransmitter deficiency and lysosomal storage disorders.

The new technology Dr. Bankiewicz is researching and CRISPR-based gene editing related to the brain have the potential to revolutionize the treatment of neurological diseases.