Cell transplantation in Parkinson´s disease

Findings by SBU Alert

Version: 2

Technology and target group

In Parkinsons disease, the brain cells that produce dopamine die. Approximately 15 000 patients in Sweden have Parkinsons disease. In its early stages, the disease can often be treated successfully by drugs. However, in most patients the disease progresses after a few years to a stage with complications where drugs no longer have a sufficient effect. In patients with Parkinsons disease, cell transplantation is intended to replace dead brain cells. A solution containing small fragments of brain tissue is injected into the patients brain. The results to date suggest that cell transplantation mainly benefits patients who still have a positive effect from medication but who have started to have complications. The size of this patient group in Sweden is estimated to be between 100 and 200 per year.

Patient benefit

A valuable level of symptom amelioration was achieved in three open studies of 15 patients who received cell transplantation. This outcome remained at 2-year followup. There are two randomized placebo-controlled trials including 74 patients with advanced disease. Patients were followed for one or two years post implantation. Small, but statistically confirmed, improvements were noted in the first trial in patients under 60 years of age and in the second study in patients with less advanced Parkinson´s disease. Survival of the transplanted dopamine neurons was, however, substantially below what had been reported in earlier studies. Stereotactic surgery, the method used in transplantation, carries a risk for cerebral hemorrhaging. Furthermore, there is a risk for immunological rejection. The results to date suggest, however, that these risks are small.

Scientific evidence

Cell transplantation in treating Parkinsons disease is currently an experimental method under development. There is poor* documentation on the effects of treatment. There is no* documentation on the cost-effectiveness of the method.

Until further evidence becomes available the method should be used only within the framework of scientific studies.

*This assessment by SBU Alert uses a 4-point scale to grade the quality and evidence of the scientific documentation. The grades indicate: (1) good, (2) moderate, (3) poor, or (4) no scientific evidence on the subject.

This summary is based on a report prepared at SBU in collaboration with Olle Lindvall, Professor, Department of Neurology, Lund University Hospital. It has been reviewed by Sten-Magnus Aquilonius, Professor, Department of Neurology, University Hospital, Uppsala.

SBU Alert is a service provided by SBU in collaboration with the Medical Products Agency, the National Board of Health and Welfare, and the Federation of Swedish County Councils.

References

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3. Hagell P, Crabb L, Pogarell O, Schrag A, Widner H, Brooks DJ, et al. Health-related quality of life following bilateral intrastriatal transplantation in Parkinsons disease. Mov Disord 2000;15:224-9.
4. Kordower JH, Freeman TB, Snow BJ, Vingerhoets FJ, Mufson EJ, Sanberg PR, et al. Neuropathological evidence of graft survival and striatal reinnervation after the transplantation of fetal mesencephalic tissue in a patient with Parkinsons disease. N Engl J Med 1995;332:1118-24.
5. Lindvall O, Brundin P, Widner H, Rehncrona S, Gustavii B, Frackowiak R, et al. Grafts of fetal dopamine neurons survive and improve motor function in Parkinsons disease. Science. 1990;247:574-7.
6. Lindvall O, Hagell P. Clinical observations after neural transplantation in Parkinsons disease. Prog Brain Res. 2000;127:299-320. Review.
7. Piccini P, Brooks DJ, Björklund A, Gunn RN, Grasby PM, Rimoldi O, et al. Dopamine release from nigral transplants visualized in vivo in a Parkinsons patient. Nat Neurosci. 1999;2:1137-40.
8. Piccini P, Lindvall O, Björklund A, Brundin P, Hagell P, Ceravolo R, et al. Delayed recovery of movement-related cortical function in Parkinsons disease after striatal dopaminergic grafts. Ann Neurol. 2000;48:689-95.
   
   New references in update November 7, 2003
9. Hagell P, Piccini P, Bjorklund A, Brundin P, Rehncrona S, Widner H et al. Dyskinesias following neural transplantation in Parkinson's disease. Nat Neurosci 2002;5(7):627-8.
10. Ma Y, Feigin A, Dhawan V, Fukuda M, Shi Q, Greene P et al. Dyskinesia after fetal cell transplantation for parkinsonism: a PET study. Ann Neurol 2002;52(5):628-34.
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12. Olanow CW, Goetz CG, Kordower JH, Stoessl AJ, Sossi V, Brin MF et al. A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann Neurol 2003;54(3):403-14.