PITTSBURGH, February 4, 1999 — Lawrence Wechsler, M.D., director of the University of Pittsburgh Medical Center (UPMC) Stroke Institute , reported today that some patients in the first research study to reverse neurological deficits from stroke reported improvement in their symptoms after receiving brain implants of human neuronal cells.
Dr. Wechsler’s presentation was made at the 24th annual International Conference on Stroke and Cerebral Circulation of the American Heart Association, in Nashville.
The on-going Phase I study is an open label, uncontrolled evaluation of the safety and feasibility of human neuronal cells for the treatment of chronic stroke. Efficacy is also being assessed.
"Several of the patients have reported subjective improvement in motor or speech skills," said Dr. Wechsler, professor of neurology and neurologic surgery, University of Pittsburgh School of Medicine. But he cautioned, "It is not yet clear whether the functional gains some patients report are due to the implanted neurons or to other factors." Additionally, objective measures of improvement are still being collected.
Researchers at UPMC performed the first procedure in a single-site, FDA cleared approved phase I trial on June 23, 1998. The primary goals of a phase I trial are to evaluate the safety and tolerability of the therapy in patients.
"All nine surgeries performed to date have proceeded safely," said Douglas Kondziolka, M.D., professor of neurologic surgery and radiation oncology, University of Pittsburgh School of Medicine, and the study’s principal investigator. "Other than occasional fatigue or nausea, the patients are feeling well. Serious side effects attributed to the therapy have not been seen. In early follow-up, this procedure appears to be a safe and feasible approach."
Current plans are to complete the initial study, obtain longer follow-up evaluations and formalize plans for a second, much larger trial, Dr. Kondziolka added. The phase II trial would be a multi-center study to assess the effectiveness of the treatment.
The implanted human neuronal cells are provided by Layton BioScience, Inc., located in Atherton, Calif. LBS-Neurons originate from a human teratocarcinoma found in a 22-year-old cancer patient. Teratocarcinomas are tumors of the reproductive organs that are composed of embryonic-like cells. Layton BioScience, Inc., has licensed a patented process that uses several chemicals to transform this rapidly dividing cell line into fully differentiated, non-dividing human neuronal cells (LBS-Neurons) that can be used in clinical applications. In extensive preclinical testing, implants of LBS-Neurons reversed cognitive and motor deficits in animals in which stroke had been induced.
The procedure begins with the placement of a stereotactic frame on the head of the patient. The frame is a standard tool in neurosurgery to provide a fixed way to find specific locations within the brain. The patient then receives a CT or MRI scan of the brain. The surgical team then makes its final decision for location of the human neuronal cells.
Concurrently, the University of Pittsburgh Immunologic Monitoring and Diagnostic Laboratory team thaws the human neuronal cells that were frozen by and transported from Layton BioScience, Inc. Researchers then check the LBS-Neurons to ensure there are the requisite number of viable cells to implant.
After the human neuronal cells are transferred to a long-needled syringe, the surgeon uses CT to guide their injection at multiple sites. The surgeon injects these cells through an opening of the skull smaller than a pea. After injection, the opening is closed with one stitch.
Stroke is the third leading cause of death and the most common cause of adult disability in the United States, according to the National Institutes of Health. Each year, 700,000 Americans suffer a stroke. About 30 percent die and 20–30 percent become severely and permanently disabled. The total cost for caring for all aspects of stroke is $41.9 billion annually in the United States.
Currently, rehabilitation through physical and occupational therapy is the only treatment available for people with established stroke. No direct treatment is recognized as safe and effective for reversing the neurologic damage months after the event.
For additional information on the Department of Neurological surgery at the University of Pittsburgh, please access the web site at http://www.neurosurgery.pitt.edu. For information regarding Layton BioScience, Inc., please access http://www.laytonbio.com.