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59th Annual Meeting of the German Society of Neurosurgery (DGNC)
3rd Joint Meeting with the Italian Neurosurgical Society (SINch)

German Society of Neurosurgery (DGNC)

1 - 4 June 2008, Würzburg

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Human fetal striatal transplantation in Huntington's disease. The first Italian clinical trial

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  • corresponding author A. Di Rita - Department of Neurosurgery, University of Florence, Italy
  • M. Paganini - Department of Neurosurgery, University of Florence, Italy
  • G. B. Vannelli - Department of Neurosurgery, University of Florence, Italy
  • L. Lombardini - Department of Neurosurgery, University of Florence, Italy
  • M. Campolo - Department of Neurosurgery, University of Florence, Italy
  • P. Gallina - Department of Neurosurgery, University of Florence, Italy
  • N. Di Lorenzo - Department of Neurosurgery, University of Florence, Italy

Deutsche Gesellschaft für Neurochirurgie. Società Italiana di Neurochirurgia. 59. Jahrestagung der Deutschen Gesellschaft für Neurochirurgie e.V. (DGNC), 3. Joint Meeting mit der Italienischen Gesellschaft für Neurochirurgie (SINch). Würzburg, 01.-04.06.2008. Düsseldorf: German Medical Science GMS Publishing House; 2008. DocDI.05.07

The electronic version of this article is the complete one and can be found online at: http://www.egms.de/en/meetings/dgnc2008/08dgnc181.shtml

Published: May 30, 2008

© 2008 Di Rita et al.
This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by-nc-nd/3.0/deed.en). You are free: to Share – to copy, distribute and transmit the work, provided the original author and source are credited.

Outline

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Objective: We report on preliminary results of the first Italian clinical trial in Human Fetal Striatal Transplantation (HFST) in Huntington’s Disease (HD).

Methods: After approval of the National Ethics Committee, eight clinically and genetically defined HD patients underwent HFST at our institution. Subject selection, enrollment and evaluation followed a modified version of the Core Assessment Program for Intracerebral Transplantation (CAPIT).

Seven patients received bilateral grafts in two sessions 1 month apart, while one patient has not received the contralateral graft yet. A cell suspension obtained from the whole ganglionic eminence of a 9-12 weeks post-conception fetus was implanted in the head of the caudate nucleus and in the pre- and post-commissural putamen by means of an image-guided robotic-assisted stereotactic procedure. For each procedure five to nine stereotactic tracks were performed. Striatal atrophy prevented grafting of the posterior putamen in four out of fifteen procedures. Immunosuppression was started one day before the first grafting with cyclosporin A, prednisolone and azathioprine.

Results: The procedure resulted feasible and well tolerated. In all patients, post-operative MR imaging did not detect any complication related to surgery and showed the needle tracks and appropriate location of grafted material within the targets.

Follow-up ranged from 2 to 18 months. Patient 2 experienced, one month after each procedure, the occurrence of a subdural haematoma necessitating surgical evacuation. Seven patients show a slight improvement in their neurological status and in daily living activities, while one patient is clinically stable.

In patient 1, MR and 18-FDG PET studies show the neo-generation of metabolically active tissue with striatum-like imaging features. In other patients imaging findings are consistent with grafted tissue survival and growth.

Conclusions: HFST in HD is a feasible and safe procedure. Neuroimaging data suggest grafted tissue survival and growth. Based on early follow-up a clinical improvement, or at least a prevention of disease progression, can be achieved. Longer follow-up and a larger number of patients are needed to assess the efficacy of HFST in HD.