PITTSBURGH, April 11 -- Douglas Kondziolka, M.D., professor of neurological surgery and radiation oncology at the University of Pittsburgh department of neurological surgery, reported today that some patients who underwent neuronal transplantation following stroke showed improvement in motor function.
His study, presented at the annual meeting of the American Association of Neurological Surgeons in San Francisco, reported on the six-month neurologic outcome and 12-to-18 month safety data of patients in the first research study of a treatment aimed at reversing neurological deficits from stroke.
Dr. Kondziolka's study involved 12 patients in a phase I trial that evaluated the safety and feasibility of implanting human neuronal cells for treatment of chronic stroke in the basal ganglia region of the brain.
The nine men and three women in the study varied in age from 44 to 75 years. In eight patients, the stroke involved only the basal ganglia region of the brain and in four patients, both the basal ganglia and regional cortex were involved. All patients had stable neurologic deficits at least two months prior to implantation. The first four patients were treated with two million neurons implanted at three sites along a single needle pass within the basal ganglia. The remaining eight patients were randomized to receive either two million neurons along one needle pass or a total of six million neurons implanted along three trajectories.
Assessment of safety and feasibility was performed using the NIH Stroke Scale (NIHSS), European Stroke Scale (ESS), Short Form 36 (SF36) and Barthel Index (BI).
Following implantation, subjective improvement, which included increased strength, sensation and coordination, was reported in eight patients.
Objectively, six patients had an increase in ESS score ranging from three to 10 points, with four of these patients improving by eight or more points. Three patients had no change from their baseline scores and three patients deteriorated slightly. Six patients improved from one to eight points on the motor component of ESS with four patients improving six points or more.
"The changes in performance on the motor function of each of these scales accounted for the majority of improvement noted," reported Dr. Kondziolka. "Functional improvement appeared to be independent of the time interval since occurrence of the strokes. Increases in cerebral glucose metabolism as measured by PET scanning appeared to correlate with functional improvement."
The implanted human neuronal cells are provided by Layton BioScience, Inc., located in Atherton, Calif. LBS-Neurons originate from a human teratocarcinoma, a tumor of the reproductive organs that is composed of embryonic-like cells, removed from a 22-year-old cancer patient in the early 1980s. Layton BioScience, Inc., has licensed a patented process that uses several chemicals to transform this 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 and the surgical team makes its final decision for location of cell implantation.
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.
After the 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 a small opening in the skull, and patients generally leave the hospital the next day. Dr. Kondziolka will soon begin a second, much larger multi-center trial to assess the effectiveness of the treatment.