Impact report for the Mangurian Foundation: The Pelotonia Research Center Opens Its Doors 

Support from the Mangurian Foundation was instrumental in constructing the Pelotonia Research Center, which opened in June. This five-story, 305,000-square-foot laboratory building is composed of research “neighborhoods” for interdisciplinary teams of experts to advance discoveries in several areas of health care, including gene therapy and cancer.

Because of the Mangurian Foundation’s significant investments in two neighborhoods within this anchor facility for Carmenton, Columbus’ innovation district, researchers and leaders are envisioning a bright future for gene- and cell-based therapies as well as cancer care. 

Leading the Way in Gene Therapy 

When Shillann Rodriguez-Pena and her husband Mike welcomed their second child, Rian, they noticed something was wrong just days after she was born. Following testing for over a thousand conditions, physicians diagnosed her with an ultra-rare genetic disorder known as AADC deficiency that dramatically changed the entire family’s life.

Rian’s condition left her disabled and sick. She couldn’t hold herself up, let alone walk or talk. Everyday situations would cause sensory overload that made her physically ill, and she was eating through a feeding tube. Rian was taking so much medicine each day that her teeth needed reconstruction.

“This disease didn’t leave her a little bit sick or a little bit disabled,” her mom says. “Every aspect of Rian’s existence — and every aspect of our life as a family — was affected.”

Rian’s family sought answers during hundreds of visits with physicians, and then they finally found someone who could help: Krystof Bankiewicz, MD, PhD, who serves as director of Ohio State’s Brain Health and Performance Center and holds the Gilbert and Kathryn Mitchell Chair. Dr. Bankiewicz is a world-renowned neurosurgeon and a pioneer in gene therapy.

“I remember looking at his hands and thinking that those hands could change everything for Rian,” Shillann says. “And they did.”

Since her gene therapy treatment in 2019, Rian has made a “miraculous” transformation, according to Shillann.

“That was the day my daughter was reborn,” she says. “Rian went from bedridden to holding her head up two months after gene therapy, and then batting at toys, and then sitting. She came out a different person.”

Because of the Mangurian Foundation’s commitment to name the Pelotonia Research Center’s Dorothy J. Mangurian Foundation Neighborhood, members of Dr. Bankiewicz’s lab will have dedicated space to keep pursuing positive outcomes for families like Rian’s. The team’s research will continue to focus on developing treatments for genetic disorders such as Huntington’s and Parkinson’s diseases, with federal funding from the National Institutes of Health (NIH).

Ohio State’s interdisciplinary Gene Therapy Institute, where Dr. Bankiewicz is chief scientific officer, recently was a recipient of an NIH grant totaling $25 million to further study the genome-editing technology known as CRISPR. This work has the potential to address multiple diseases, and it will be elevated through the Pelotonia Research Center.

For Rian, who is now 8 years old, it’s been four years since her treatment. She can now walk, and earlier this year, she even rode a roller coaster. She takes six doses of medication per day, down from the 21 she was taking before gene therapy.

Her mom credits this progress to the care she received — and it would not be possible without the research that the Mangurian Foundation is helping to accelerate.

“I don’t say gene therapy is our happy ending. It’s our hopeful beginning,” Shillann says. “I know one day, Rian will be up at a podium with me, and she will be helping to tell the world about the miracles that happened in her life.” 

Finding Cancer Drug Delivery Solutions 

In brain cancer research, a staggering 90% of new drugs never make it to market. Imagine all the work that goes into developing medicine, only for it to go unused in treating cancer patients.

“The problem is delivery. Manufacturers don’t want to deal with the challenge,” says Jessica Winter, PhD, a bioengineer with Ohio State’s College of Engineering. “If we can show that we’ve already solved the delivery challenge here, that will speed up our ability to have those medicines reach patients.”

As an investigator in the OSUCCC – James’ multidisciplinary Center for Cancer Engineering, drug delivery is a primary focus of Dr. Winter’s research. Thanks to the Mangurian Foundation’s generosity in naming the Pelotonia Research Center’s Harry T. Mangurian Neighborhood – which houses the Center for Cancer Engineering – Dr. Winter will be able to further bolster her work in difference-making ways.

Dr. Winter often partners with researchers at the Ohio State Wexner Medical Center who contact her about drugs they’re developing. Every time a new drug enters the field, a novel way to administer it must follow. Since each one is different, it can interact with other materials in ways that diminish or even cancel its intended effects.

For some drugs, it can take 10-15 years to develop an effective carrier, and design and manufacturing are costly. That’s why Dr. Winter is passionate about finding solutions that improve the process of getting these treatments to patients.

“I’m trying to figure out how to bridge the gap between research and commercial manufacturing,” she says. “When a pharmaceutical company shows interest in Ohio State’s research and wants to buy it, I want them to be able to run with it instead of immediately encountering the next roadblock.”

Dr. Winter’s work is both practical and personal. As a cancer survivor, she recognizes how much research never leaves the lab. But with the technologies, resources and synergies that the Pelotonia Research Center provides, Dr. Winter says working in the Harry T. Mangurian Neighborhood will help lead to practical applications in frontline cancer drug therapies.

“Surviving cancer really changed my perspective. I’m always asking myself whether this research is going to help people,” she says. “I’ve come to realize that it doesn’t matter what you make in the lab if it never reaches a patient who needs it.”