HD research news - medical research into treatment & prevention
Preliminary
success of fetal Brain-Cell Transplantation
in Huntington's disease
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.
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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".
The decision to publish the
paper early on The Lancet's website ahead of the scheduled print publication
date of December 9 was made after some of the research findings were
made public by French Government officials on Monday 27 November. The
full research study and accompanying Commentary are freely available
to doctors, patients, journalists and the general public on the journal's
website (www.thelancet.com).
The data will be presented
at a press conference organised by INSERM at 1030 am (French time),
Thursday November 30; venue: 101 rue de Tolbiac, Paris 75013, France.
- Contact: Dr Marc Peschanski,
Inserm U, 421/IM3, Faculte de Medecine, 94010, Creteil, France; T) +33
1 49 81 36 82; F) +33 1 49 81 37 09; E) peschanski@im3,inserm.fr
- Professor Olle Lindvall,
Wallenberg Neuroscience Center, Lund University, SE-223 62 Lund, Sweden;
T) +46 46 222 0543/0542; M) +46 705 171466; F) +46 46 222 0560; E) olle.lindvall@neurol.lu.se
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