A fetal striatal transplantation performed in a patient with moderately severe Huntington's disease (HD) has apparently been successful, with long-term evidence of engraftment and clinical improvement.
As reported in the August issue of the Journal of Neurology, Neurosurgery and Psychiatry, Dr. Paola Piccini and colleagues at Imperial College and Hammersmith Hospital in London, UK, performed bilateral fetal striatal allograft transplantations in a 52-year-old man with a six-year history of HD and a 53 year-old woman with a four-year history of HD.
Six patients with HD of comparable severity and duration who did not receive allograft transplantation served as controls.
The male patient demonstrated increased striatal D2 receptor binding, evident at five years with C-raclopride positron emission tomography. He also demonstrated prolonged clinical improvement over five years, "suggesting long-term survival and efficacy of the graft."
The female patient did not improve clinically or radiologically.
The male patient "is now 12 years into his disease and continues to be independent in all aspects of activities of daily living," Dr. Piccini and associates note.
"It is possible that the resolution of his depression, as reflected by the improvement in his Beck Depression scores, may account for some of his clinical improvement," the researchers comment. "However, neither this nor placebo effects could explain the prolonged improvement in PET and the subsections of his UHDRS (Unified Huntington's Disease Rating Scale) motor scores, including chorea, over five years."
"Furthermore," they explain, "the profile of cognitive improvement points not to a global change, as would be expected in someone with resolving depression, but rather shows changes more in fronto-striatal tasks."
A larger randomized trial is currently underway, the team reports, and this "may provide a more definitive answer on the efficacy of this procedure."
J Neurol Neurosurg Psychiatry 2008;79:948-951
