HD research news - medical research into treatment & prevention

Preliminary success of fetal Brain-Cell Transplantation in Huntington’s disease

September 2001

A preliminary study published on The Lancet’s website - www.thelancet.com - outlines a surgical technique involving transplantation of fetal brain cells which could be of future benefit to people with Huntington’s disease.

Huntington’s disease is a genetic neurodegenerative disease that mainly affects the area of the brain called the striatum. It has severe motor and cognitive consequences and, up to now, no treatment. Motor and cognitive functions can be restored in experimental animal models by means of transplantation of fetal brain cells. Marc Peschanski and colleagues from INSERM, Paris, France, explored whether grafts of human fetal striatal tissue could survive and have detectable effects in five patients with mild to moderate Huntington’s disease.

After 2 years of preoperative assessment, patients were grafted with human fetal nerve cells into the right then, after a year, the left striatum. Final results were assessed 1 year later. Assessment of the effects was done by a range of neurological and psychiatric tests. These results were compared with a control group of 22 untreated patients at similar stages of the disease who were followed up in parallel. Repeated magnetic resonance imaging (MRI) and positron emission tomography (PET) scanning was also done to assess metabolic activity.

The final PET-scan assessment showed increased metabolic activity in various parts of the striatum in three of five patients, contrasting with the progressive decline recorded in the two other patients in the series, as seen in patients with untreated Huntington’s disease. As a result, motor and cognitive functions were improved or maintained within the normal range, and functional benefits were seen in daily-life activities in these three patients, but not in the other two.

In an accompanying Commentary, Olle Lindvall and Anders Bjorklund from Lund University, Sweden, comments: "Although the findings reported by Peschanski and colleagues are promising, the clinical usefulness of cell-replacement therapy for Huntington’s disease remains unclear". They conclude: "The encouraging results reported by Peschanski and co-workers indicate that this strategy may work in Huntington’s disease. Future studies will have to clarify whether cell therapy can be developed into a clinically useful treatment for patients with this disease’.