title: Unilateral transplantation of human primary fetal tissue in four patients with Huntington's disease: NEST-UK safety report ISRCTN no 36485475.
abstract: OBJECTIVE
  Huntington's disease (HD) is an inherited autosomal dominant condition in which there is a CAG repeat expansion in the huntingtin gene of 36 or more. Patients display progressive motor, cognitive, and behavioural deterioration associated with progressive cell loss and atrophy in the striatum. Currently there are no disease modifying treatments and current symptomatic treatments are only partially effective in the early to moderate stages. Neural transplantation is effective in animal models of HD and offers a potential strategy for brain repair in patients. The authors report a safety study of unilateral transplantation of human fetal striatal tissue into the striatum of four patients with HD.
  
  
  METHODS
  Stereotaxic placements of cell suspensions of human fetal ganglionic eminence were made unilaterally into the striatum of four patients with early to moderate HD. All patients received immunotherapy with cyclosporin A, azathioprine, and prednisolone for at least six months postoperatively. Patients were assessed for safety of the procedure using magnetic resonance imaging (MRI), regular recording of serum biochemistry and haematology to monitor immunotherapy, and clinical assessment according to the Core Assessment Protocol For Intrastriatal Transplantation in HD (CAPIT-HD).
  
  
  RESULTS
  During the six month post-transplantation period, the only adverse events related to the procedure were associated with the immunotherapy. MRI demonstrated tissue at the site of implantation, but there was no sign of tissue overgrowth. Furthermore, there was no evidence that the procedure accelerated the course of the disease.
  
  
  CONCLUSIONS
  Unilateral transplantation of human fetal striatal tissue in patients with HD is safe and feasible. Assessment of efficacy will require longer follow up in a larger number of patients.
authors.affiliation: School of Biosciences, Cardiff University, Museum Avenue Box 911, Cardiff CF10 3US, UK. RosserAE@cf.ac.uk
authors.forename: A E
authors.initials: AE
authors.lastname: Rosser
authors.forename: R A
authors.initials: RA
authors.lastname: Barker
authors.forename: T
authors.initials: T
authors.lastname: Harrower
authors.forename: C
authors.initials: C
authors.lastname: Watts
authors.forename: M
authors.initials: M
authors.lastname: Farrington
authors.forename: A K
authors.initials: AK
authors.lastname: Ho
authors.forename: R M
authors.initials: RM
authors.lastname: Burnstein
authors.forename: D K
authors.initials: DK
authors.lastname: Menon
authors.forename: J H
authors.initials: JH
authors.lastname: Gillard
authors.forename: J
authors.initials: J
authors.lastname: Pickard
authors.forename: S B
authors.initials: SB
authors.lastname: Dunnett
mesh_terms: D000328:Adult; D016380:Brain Tissue Transplantation / Q000502:physiology; D026661:Clinical Trials Data Monitoring Committees; D003342:Corpus Striatum / Q000503:physiopathology / Q000601:surgery / Q000637:transplantation*; D004292:Dominance, Cerebral / Q000502:physiology*; D005240:Feasibility Studies; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D006816:Huntington Disease / Q000503:physiopathology / Q000601:surgery*; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use; D008279:Magnetic Resonance Imaging; D008297:Male; D009048:Motor Skills / Q000502:physiology; D009483:Neuropsychological Tests; D011183:Postoperative Complications / Q000175:diagnosis / Q000209:etiology*; D013238:Stereotaxic Techniques; D014180:Transplantation / Q000502:physiology; D006113:United Kingdom
chemical_list: D007166:Immunosuppressive Agents