Nav: Home

Promising gene replacement therapy moves forward at Ohio State

August 21, 2019

COLUMBUS, Ohio - Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic effects in mice.

These research findings are published online in the journal Science Translational Medicine.

Prior to joining Ohio State as a professor of neurosurgery, Bankiewicz conducted this translational gene therapy research at the University of California at San Francisco, in conjunction with researchers in New York, Massachusetts and Spain.

Niemann-Pick disease primarily affects children, has no known cure and is often fatal. It's caused by mutations in a gene that causes 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, said Bankiewicz, who also is a member of Ohio State's Neurological Institute.

"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," Bankiewicz said. "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, Bankiewicz and his team, including co-author Lluis Samaranch, 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.

"Dr. Bankiewicz is a world leader in neuro-restorative medicine - particularly gene therapy. With his recruitment here - along with the expertise of his five-member research team he's bringing with him - The Ohio State University College of Medicine is making a strong commitment to developing a gene therapy program to develop treatments for this and other diseases, such as Parkinson's and Alzheimer's," said Dr. K. Craig Kent, dean of Ohio State College of Medicine.

Throughout his career, Bankiewicz has maintained a strong focus on the development of translational approaches to drug, gene and cell replacement therapies.

His research has focused on new treatments of serious diseases, including brain cancer, Parkinson's disease, Huntington's disease, Alzheimer's diseases, pediatric neurotransmitter deficiency and lysosomal storage disorders. At Ohio State, Bankiewicz will work closely with the department of neurological surgery to conduct other gene replacement therapy clinical trials in humans.
-end-
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.

Ohio State University Wexner Medical Center

Related Science Articles:

75 science societies urge the education department to base Title IX sexual harassment regulations on evidence and science
The American Educational Research Association (AERA) and the American Association for the Advancement of Science (AAAS) today led 75 scientific societies in submitting comments on the US Department of Education's proposed changes to Title IX regulations.
Science/Science Careers' survey ranks top biotech, biopharma, and pharma employers
The Science and Science Careers' 2018 annual Top Employers Survey polled employees in the biotechnology, biopharmaceutical, pharmaceutical, and related industries to determine the 20 best employers in these industries as well as their driving characteristics.
Science in the palm of your hand: How citizen science transforms passive learners
Citizen science projects can engage even children who previously were not interested in science.
Applied science may yield more translational research publications than basic science
While translational research can happen at any stage of the research process, a recent investigation of behavioral and social science research awards granted by the NIH between 2008 and 2014 revealed that applied science yielded a higher volume of translational research publications than basic science, according to a study published May 9, 2018 in the open-access journal PLOS ONE by Xueying Han from the Science and Technology Policy Institute, USA, and colleagues.
Prominent academics, including Salk's Thomas Albright, call for more science in forensic science
Six scientists who recently served on the National Commission on Forensic Science are calling on the scientific community at large to advocate for increased research and financial support of forensic science as well as the introduction of empirical testing requirements to ensure the validity of outcomes.
World Science Forum 2017 Jordan issues Science for Peace Declaration
On behalf of the coordinating organizations responsible for delivering the World Science Forum Jordan, the concluding Science for Peace Declaration issued at the Dead Sea represents a global call for action to science and society to build a future that promises greater equality, security and opportunity for all, and in which science plays an increasingly prominent role as an enabler of fair and sustainable development.
PETA science group promotes animal-free science at society of toxicology conference
The PETA International Science Consortium Ltd. is presenting two posters on animal-free methods for testing inhalation toxicity at the 56th annual Society of Toxicology (SOT) meeting March 12 to 16, 2017, in Baltimore, Maryland.
Citizen Science in the Digital Age: Rhetoric, Science and Public Engagement
James Wynn's timely investigation highlights scientific studies grounded in publicly gathered data and probes the rhetoric these studies employ.
Science/Science Careers' survey ranks top biotech, pharma, and biopharma employers
The Science and Science Careers' 2016 annual Top Employers Survey polled employees in the biotechnology, biopharmaceutical, pharmaceutical, and related industries to determine the 20 best employers in these industries as well as their driving characteristics.
Three natural science professors win TJ Park Science Fellowship
Professor Jung-Min Kee (Department of Chemistry, UNIST), Professor Kyudong Choi (Department of Mathematical Sciences, UNIST), and Professor Kwanpyo Kim (Department of Physics, UNIST) are the recipients of the Cheong-Am (TJ Park) Science Fellowship of the year 2016.
More Science News and Science Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Listen Again: Reinvention
Change is hard, but it's also an opportunity to discover and reimagine what you thought you knew. From our economy, to music, to even ourselves–this hour TED speakers explore the power of reinvention. Guests include OK Go lead singer Damian Kulash Jr., former college gymnastics coach Valorie Kondos Field, Stockton Mayor Michael Tubbs, and entrepreneur Nick Hanauer.
Now Playing: Science for the People

#562 Superbug to Bedside
By now we're all good and scared about antibiotic resistance, one of the many things coming to get us all. But there's good news, sort of. News antibiotics are coming out! How do they get tested? What does that kind of a trial look like and how does it happen? Host Bethany Brookeshire talks with Matt McCarthy, author of "Superbugs: The Race to Stop an Epidemic", about the ins and outs of testing a new antibiotic in the hospital.
Now Playing: Radiolab

Dispatch 6: Strange Times
Covid has disrupted the most basic routines of our days and nights. But in the middle of a conversation about how to fight the virus, we find a place impervious to the stalled plans and frenetic demands of the outside world. It's a very different kind of front line, where urgent work means moving slow, and time is marked out in tiny pre-planned steps. Then, on a walk through the woods, we consider how the tempo of our lives affects our minds and discover how the beats of biology shape our bodies. This episode was produced with help from Molly Webster and Tracie Hunte. Support Radiolab today at Radiolab.org/donate.