Krystof Bankiewicz, MD, PhD, Brings Promising Gene Therapy Research to Ohio State

Internationally renowned for his work in translational neurological gene therapy research, neuroscientist Krystof Bankiewicz, MD, PhD, recently joined The Ohio State University Wexner Medical Center to further his work in gene therapy and pursue other new treatments for brain cancer, Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, pediatric neurotransmitter deficiency and lysosomal storage disorders.

Throughout his career, Dr. Bankiewicz has maintained a strong focus on the development of translational approaches to drug, gene and cell replacement therapies. Before joining Ohio State as a professor of neurosurgery and member of Ohio State’s Neurological Institute, Dr. Bankiewicz conducted his translational research and clinical trials at the University of California at San Francisco (UCSF).

Groundbreaking Gene Replacement Therapy for Niemann-Pick Type A Disease

Among Dr. Bankiewicz’s most recent breakthroughs is a gene replacement therapy for Niemann-Pick type A disease, which he and his lab at UCSF found to be safe for use in nonhuman primates. It also has been demonstrated to have therapeutic effects in mice. The Bankiewicz lab worked in conjunction with researchers in New York, Massachusetts and Spain.

Their research findings were published online in August 2019 in the journal Science Translational Medicine.

Niemann-Pick disease primarily affects children, has no known cure and is often fatal. It is caused by mutations in a gene that lead to a deficiency of a specific enzyme, acid sphingomyelinase (ASM). With this disease, the body's ability to metabolize fat within cells is affected, causing these cells to malfunction and, eventually, die. This inherited disease can affect the brain, nerves, liver, spleen, bone marrow and lungs.

Researchers evaluated the safety and effectiveness of adeno-associated viral vector serotype 9 (AAV9)-based gene therapy. Using a newly developed cerebrospinal fluid delivery method into a specific area of the brain allowed widespread gene expression in the brain and spinal cord of nonhuman primates without signs of toxicity.

In addition, the treatment provided a “bystander effect” by preventing motor and memory impairment and increased survival in a mouse model of the disease, says Dr. Bankiewicz.

“This is the ‘proof of concept’ of gene therapy in animal models for this disease. It’s a big jump in the development process as we work toward one day being able to safely give this therapy to children who otherwise would die by age 3,” Dr. Bankiewicz says. “Our plan is to translate this exciting data into human clinical trials at The Ohio State University Wexner Medical Center within the next couple of years.”

Based on this research, Dr. Bankiewicz and his team, including co-author Lluis Samaranch, PhD, an assistant professor of neurological surgery at Ohio State, will move forward with additional animal studies in preparation to eventually begin clinical trials in humans.

Other Endeavors

“I want to establish dominance in gene therapy here at Ohio State,” Dr. Bankiewicz says. He is working closely with the department of neurological surgery to conduct other gene replacement therapy clinical trials in humans.

“I’m already bringing two Parkinson’s trials here,” he notes. “One of them is to help prevent the progression of Parkinson’s through stimulation of the remaining innervation in the brains of patients affected by Parkinson’s disease. It’s imminent that we will be treating those patients.”

Dr. Bankiewicz continues, “Since my group participated in co-development of gene therapy clinical trials for Huntington’s and Alzheimer’s, we are happy to support these clinical trials at Ohio State.”

Dr. Bankiewicz has co- founded MedGenesis Therapeutix Inc., Voyager Therapeutics Inc. and Brain Neurotherapy Bio Inc. and also invented several devices currently used clinically to administer gene therapy to the brain.

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