USF neuroscientist translates cell therapy for stroke from lab to clinical trial
Preclinical work in the laboratory of USF Health neuroscientist Dr. Cesar Borlongan laid the groundwork for clinical testing of SanBio Inc.’s regenerative cell therapy for ischemic stroke, the leading cause of adult disability.
The translational research of Dr. Borlongan and his team was critical to a groundbreaking new clinical trial evaluating the safety and effectiveness of adult bone-marrow derived stem cells in treating stroke survivors. Renewed media interest in stem cells for brain repair was generated with the announcement April 30 that the University of Pittsburgh joined Stanford University as one of two trial sites enrolling a limited number of stable patients with disability from stroke.
Long before the first of dose containing millions of SanBio’s SB623 cells was injected into the brain of a stroke patient at Stanford, Dr. Borlongan’s team, based at the USF Center of Excellence for Aging and Brain Repair, demonstrated in the laboratory that these cells protected neurons and improved neurological behavior in a rat model of stroke. SB623 cells are unique in that the bone-marrow derived cells are genetically modified to behave more “neuron like,” said Dr. Borlongan, professor and vice chair for research in the USF Department of Neurosurgery and Brain Repair. “It’s a stem-cell based gene therapy.”
USF’s preclinical studies have shown that the stem cells aren’t replacing neurons that have been lost, because they disappear relatively soon after transplantation. Rather, Dr. Borlongan said, they appear to secrete substances, such as growth and anti-inflammatory factors, that may recruit the body’s endogenous stem cells to repair brain damage from stroke. And, he added, there are indications that the SB623 cells encourage brain repair by initiating a “bio-bridge” that rebuilds neural connections between the core tissue damaged by stroke and surrounding spared tissue.
Three of six current NIH-registered clinical trials in the U.S. testing stem cell therapies for ischemic stroke were translated from laboratory to clinic with the help of research led by Dr. Borlongan. In addition to their work with bone marrow stem cells, USF researchers are investigating stem cells derived from umbilical cord blood, placenta and skin fibroblasts.
“We’re not putting all our eggs into one basket,” Dr. Borlongan said. “We’re working with a range of different cells to try to find the best cell type that will be safe and effective for stroke patients.”