An image from an MRI during a gene therapy perfusion procedure. Credit: Courtesy of Dr. Russell Lonser
For patients with rare and devastating neurological disorders, treatment options can seem few and far between. However, gene therapy research, led by Dr. Russell Lonser and Dr. Brad Elder, is hoping to offer better care.
Lonser is the director of the Gene Therapy Institute at Ohio State, and Elder is the director of Neurosurgical Oncology. Their work focuses on treating disorders like Alzheimer’s and Parkinson’s Disease, both of which affect over 8 million Americans combined, by injecting healthy growth genes to help recover dying brain cells.?
“What we do is [that] we can infuse viral vectors in the brain. These vectors aren’t pathogenic, and we put a gene of interest that we want to do into those vectors, because it delivers those genes into the cells,” Lonser said. “Cells that take those genes produce a protein. We can treat an entire circuit from a very small area.”
One of the main ways this infusion occurs is through a method called convection-enhanced delivery, which involves inserting catheters in the brain down to the submillimeter, Elder said.
“That can be done with a technique called convection-enhanced delivery, where we put catheters in the brain, we infuse under a little bit of pressure, and that helps the larger molecules like viral particles or big chemotherapy agents to distribute more broadly in the brain,” Elder said.?
An advantage to infusing growth genes into the brain instead of stem cells or a blood transfusion is the strength of the genes.?
Currently, growth genes are infused by blood which can be diluted and cause a negative immune reaction and doesn’t have a specific target, Lonser said.
Another difference between neurological gene therapy is the high level of imagery possible from brain scans, many of which offer insights into the procedures in the research phase, Elder said.??
“Somebody can watch it all happen live with an MRI that we have in these operating rooms, so we have lots of trials for tumors and for neurodegenerative disorders,” Elder said.?
Once used on a patient, the MRIs can help surgeons evaluate the decisions made in the operating room.?
“If we have a patient while they’re asleep in the intraoperative MRI, and we can actually track that distribution of where we’re treating, we get real-time feedback,” Lonser said. “It allows us as surgeons to modify and do things on the fly.”
Because of the ever-changing landscape of both the medical field and the public’s general health, doctors and researchers are constantly looking for new ways to improve treatments.?
“Unfortunately, brain tumors are very difficult to treat,” Elder said. “We’re always looking to push that boundary a little bit in terms of overall survival by doing clinical trials.”
The biggest aspect of the research isn’t the quantifiable results in patients; it’s how the work can be widely applied to more than just isolated clinical trials, Lonser said.?
“What you really need is a research outlet where you’re looking to not just change or impact a singular patient, you’re looking at what can I do to improve the field that we’re in,” Lonser said. “How can I improve that field in a much more lasting way, and that’s where the research comes in. That’s what really keeps you going to the end.”
