A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease

Ann Neurol. 2003 Sep;54(3):403-14. doi: 10.1002/ana.10720.


Thirty-four patients with advanced Parkinson's disease participated in a prospective 24-month double-blind, placebo-controlled trial of fetal nigral transplantation. Patients were randomized to receive bilateral transplantation with one or four donors per side or a placebo procedure. The primary end point was change between baseline and final visits in motor component of the Unified Parkinson's Disease Rating Scale in the practically defined off state. There was no significant overall treatment effect (p = 0.244). Patients in the placebo and one-donor groups deteriorated by 9.4 +/- 4.25 and 3.5 +/- 4.23 points, respectively, whereas those in the four-donor group improved by 0.72 +/- 4.05 points. Pairwise comparisons were not significant, although the four-donor versus placebo groups yielded a p value of 0.096. Stratification based on disease severity showed a treatment effect in milder patients (p = 0.006). Striatal fluorodopa uptake was significantly increased after transplantation in both groups and robust survival of dopamine neurons was observed at postmortem examination. Fifty-six percent of transplanted patients developed dyskinesia that persisted after overnight withdrawal of dopaminergic medication ("off"-medication dyskinesia). Fetal nigral transplantation currently cannot be recommended as a therapy for PD based on these results.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Aged
  • Brain Tissue Transplantation / adverse effects
  • Cell Survival
  • Dihydroxyphenylalanine / analogs & derivatives*
  • Dihydroxyphenylalanine / metabolism
  • Dopamine / metabolism
  • Double-Blind Method
  • Dyskinesias / etiology
  • Female
  • Fetal Tissue Transplantation* / adverse effects
  • Functional Laterality
  • Humans
  • Male
  • Middle Aged
  • Neurons / pathology
  • Parkinson Disease / complications
  • Parkinson Disease / diagnostic imaging
  • Parkinson Disease / pathology
  • Parkinson Disease / surgery*
  • Substantia Nigra / metabolism
  • Substantia Nigra / pathology
  • Substantia Nigra / transplantation*
  • Tomography, Emission-Computed
  • Treatment Outcome


  • fluorodopa F 18
  • Dihydroxyphenylalanine
  • Dopamine