If this is not your name, click here.
| | If this is not your name, click here. | | |
| | Contact Us | Order Now | Journals | Bookstore | Register a colleague | | |
| |  AAN MEETING: NeuroCell Products For Parkinson’s Show Clinical Improvement CAMBRIDGE, MA and CHARLESTOWN, MA -- April 27, 1998 -- Phase I clinical trial results show that Genzyme Tissue Repair’s and Diacrin Inc.’s NeuroCell(TM)-PD and NeuroCell(TM)-HD porcine neural cell products for implantation into people with advanced Parkinson's and Huntington's diseases were well-tolerated in transplanted patients. The results of the ongoing studies will be presented tomorrow at the American Association of Neurology meeting in Minneapolis, MN. The phase I trials are open in design. Every patient in the trial will be followed and reported on regularly over a period of three years. In addition to tolerating the treatment well, some Parkinson's disease patients continued to show evidence of clinical improvement 12 months following surgery even though the cells were implanted on only one side of the brain. In the study's 10 evaluable patients who were systematically tested at a time when their L-dopa medication was wearing off, data indicated an average improvement of 16.9 points on a standardised scale that measures a patient's ability to perform a variety of movements. This result, representing an improvement of approximately 19 percent, was statistically significant, compared to an evaluation that was taken prior to surgery. "We are continuing to see very encouraging sustained improvement in some patients at the 12-month time point," said Samuel Ellias, M.D., Ph.D., assistant professor of neurology at Boston Medical Center, who is presenting the NeuroCell-PD results. "One of my patients who had significant difficulty walking, bathing and performing other routine tasks of daily living had a 43-percent improvement in his motor skills at three months after surgery and is continuing to improve. Seeing this patient go from a very disabled state to being able to perform routine tasks is truly remarkable." The Huntington's patients also tolerated the treatment and those who were evaluated six months after surgery showed no decline in their functional status. Patients with the disease are expected to decline by an average of 0.6 point to 1.0 point per year on a standardised test designed to assess their functional status. Parkinson's and Huntington's diseases both result from a loss of nerve tissue in the brain. In Parkinson's disease, this nerve damage occurs at areas in the brain that control movement and can result in a variety of debilitating motor symptoms, including tremors, falls, rigidity, slowed movement, and difficulty with speech. In Huntington's disease, patients can develop severe abnormal movements, called chorea, as well as psychological and cognitive symptoms, including dementia and depression. Huntington's disease progresses more quickly than Parkinson's disease. Huntington's patients usually die from the disease approximately 20 years after symptoms appear. NeuroCell-PD and NeuroCell-HD are being developed to reverse or delay the progression of some of the disabilities caused by Parkinson's and Huntington's diseases. NeuroCell-PD and NeuroCell-HD are fetal pig neural cells which, when implanted, are intended to function as a replacement for the brain cells lost in the disease process. Medical experts believe that approximately 500,000 Americans have Parkinson's disease and another 50,000 cases are diagnosed each year. More than 100,000 of these patients have reached the later stages of disease in which current therapies provide little benefit. Medical experts estimate that 25,000 Americans have Huntington's disease and that another 1,500 are diagnosed each year. For the past 30 years, the drug L-dopa has been used to treat Parkinson's patients. By increasing brain levels of dopamine, the brain chemical deficient in Parkinson's disease patients, treatment with L-dopa can result in nearly complete control of symptoms in early-stage patients, but it does not stop progression of the underlying disease. L-dopa begins to lose its effectiveness in patients after six to 12 years of treatment because of the continuing loss of nerve cells in the brain. The drug has also been known to produce serious side effects in some patients. NeuroCell-PD is designed to help Parkinson's patients for whom L-dopa is no longer working as effectively. In the study, 12 patients received an implant of porcine cells into three sites in the brain, located just below the cortex, that are involved in smoothing and co-ordinating movement. Half of the patients received the drug cyclosporine to suppress the immune system to avoid rejection of the implant. The other six patients received a new technology in which the donor cells are treated with an antibody prior to implantation so that the patient's immune system does not recognise them as foreign and thus does not attack them. The patients were evaluated both before and after surgery using a standardised scale for measuring a patient's ability to perform several different types of movement, timed movement tests and neuropsychological tests. Patients were measured when their L-dopa medication was working and when its therapeutic effects had worn off. There were no serious adverse events definitely attributable to the porcine cells. Two of the patients were not included in the analysis. One patient had Parkinson's disease symptoms that were too severe to be evaluated before surgery. According to physician reports, that patient has improved significantly compared to his condition before surgery. The other patient died suddenly of a pulmonary embolism -- which was not related to the implant -- seven months after surgery. Histological studies of that patient showed that the porcine cells survived and showed signs of reconnecting nerve tissue damaged by the disease. The histological results were published in the March 1997 issue of Nature Medicine. Improvement in scores for the 10 evaluable patients was evident by three months after surgery and this improvement has persisted to the 12-month evaluation. Overall, scores for eight of the 10 patients improved at 12 months compared to their condition before surgery, while scores for two of the patients were unchanged or had worsened slightly. The improvement in some patients was substantial at 12 months compared to their condition before surgery. Two of the patients in the study improved by 34 and 46 points respectively on the standardised tests. The NeuroCell-HD study was conducted in much the same fashion as the NeuroCell-PD study, except that cells were implanted in six sites in patients' brains instead of three. The 12 patients in the study were evaluated both before and after surgery using a standardised scale to measure total functional capacity. The scale ranges from zero, representing patients with the most disability, to 13, representing patients with the least amount of disability. Although an evaluation six months after treatment with NeuroCell-HD showed no evidence of improvement in Huntington's patients' hyperkinetic movements, small changes in functional scores were seen. Four patients' scores improved slightly, one patient's score did not change and seven patients' scores worsened slightly. None of these changes were significant, however. "Even though there was no decline in patients' functional status, it is too early to make a judgement about the benefits of the porcine cells in this group of patients," said Huntington's study presenter Marie Saint-Hilaire, M.D., FRCPC, assistant professor of neurology, Boston Medical Center. The companies noted that further follow-up of the Huntington's patients is needed to properly assess possible efficacy. The procedure was well tolerated and no safety issues related to the cells were reported. These patients will continue to be evaluated at three-month intervals over a period of three years. More information on: Neurocell; Genzyme Tissue Repair and Diacrin Inc..
|
