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Long-term clinical outcome of fetal cell transplantation for Parkinson disease : two case reports

Kefalopoulou, Zinovia; Politis, Marios; Piccini, Paola; Mencacci, Niccolo; Bhatia, Kailash; Jahanshahi, Marjan; Widner, Håkan LU ; Rehncrona, Stig LU ; Brundin, Patrik LU and Björklund, Anders LU , et al. (2014) In JAMA Neurology 71(1). p.7-83
Abstract

IMPORTANCE: Recent advances in stem cell technologies have rekindled an interest in the use of cell replacement strategies for patients with Parkinson disease. This study reports the very long-term clinical outcomes of fetal cell transplantation in 2 patients with Parkinson disease. Such long-term follow-up data can usefully inform on the potential efficacy of this approach, as well as the design of trials for its further evaluation.

OBSERVATIONS: Two patients received intrastriatal grafts of human fetal ventral mesencephalic tissue, rich in dopaminergic neuroblasts, as restorative treatment for their Parkinson disease. To evaluate the very long-term efficacy of the grafts, clinical assessments were performed 18 and 15 years... (More)

IMPORTANCE: Recent advances in stem cell technologies have rekindled an interest in the use of cell replacement strategies for patients with Parkinson disease. This study reports the very long-term clinical outcomes of fetal cell transplantation in 2 patients with Parkinson disease. Such long-term follow-up data can usefully inform on the potential efficacy of this approach, as well as the design of trials for its further evaluation.

OBSERVATIONS: Two patients received intrastriatal grafts of human fetal ventral mesencephalic tissue, rich in dopaminergic neuroblasts, as restorative treatment for their Parkinson disease. To evaluate the very long-term efficacy of the grafts, clinical assessments were performed 18 and 15 years posttransplantation. Motor improvements gained gradually over the first postoperative years were sustained up to 18 years posttransplantation, while both patients have discontinued, and remained free of any, pharmacological dopaminergic therapy.

CONCLUSIONS AND RELEVANCE: The results from these 2 cases indicate that dopaminergic cell transplantation can offer very long-term symptomatic relief in patients with Parkinson disease and provide proof-of-concept support for future clinical trials using fetal or stem cell therapies.

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@article{4b85e87e-a236-4161-9c34-bca5880e90d6,
  abstract     = {<p>IMPORTANCE: Recent advances in stem cell technologies have rekindled an interest in the use of cell replacement strategies for patients with Parkinson disease. This study reports the very long-term clinical outcomes of fetal cell transplantation in 2 patients with Parkinson disease. Such long-term follow-up data can usefully inform on the potential efficacy of this approach, as well as the design of trials for its further evaluation.</p><p>OBSERVATIONS: Two patients received intrastriatal grafts of human fetal ventral mesencephalic tissue, rich in dopaminergic neuroblasts, as restorative treatment for their Parkinson disease. To evaluate the very long-term efficacy of the grafts, clinical assessments were performed 18 and 15 years posttransplantation. Motor improvements gained gradually over the first postoperative years were sustained up to 18 years posttransplantation, while both patients have discontinued, and remained free of any, pharmacological dopaminergic therapy.</p><p>CONCLUSIONS AND RELEVANCE: The results from these 2 cases indicate that dopaminergic cell transplantation can offer very long-term symptomatic relief in patients with Parkinson disease and provide proof-of-concept support for future clinical trials using fetal or stem cell therapies.</p>},
  author       = {Kefalopoulou, Zinovia and Politis, Marios and Piccini, Paola and Mencacci, Niccolo and Bhatia, Kailash and Jahanshahi, Marjan and Widner, Håkan and Rehncrona, Stig and Brundin, Patrik and Björklund, Anders and Lindvall, Olle and Limousin, Patricia and Quinn, Niall and Foltynie, Thomas},
  issn         = {2168-6157},
  keyword      = {Corpus Striatum,Dopaminergic Neurons,Fetal Tissue Transplantation,Follow-Up Studies,Humans,Levodopa,Male,Mesencephalon,Middle Aged,Parkinson Disease,Positron-Emission Tomography,Time Factors,Treatment Outcome,Case Reports,Journal Article,Research Support, Non-U.S. Gov't},
  language     = {eng},
  number       = {1},
  pages        = {7--83},
  publisher    = {American Medical Association},
  series       = {JAMA Neurology},
  title        = {Long-term clinical outcome of fetal cell transplantation for Parkinson disease : two case reports},
  url          = {http://dx.doi.org/10.1001/jamaneurol.2013.4749},
  volume       = {71},
  year         = {2014},
}