Stem Cell Therapy Helps Huntington's

Stem Cell Therapy Helps Huntington's

Researchers Say Stem Cell Transplant May Offer Several Years of Improvement

 

Feb. 27, 2006 - Stem cell transplants may provide benefits in treating Huntington's disease, according to a new study.

Researchers found the benefits of experimental stem cell therapy in reducing symptoms -- such as muscle stiffness and memory loss -- peaked after two years and then faded four to six years after the procedure in people with Huntington's disease.

While not a cure for the rare and difficult-to-treat condition, the study suggests that transplanting healthy stem cells to replace those brain cells damaged by Huntington's disease may offer patients long-term improvements and stability. The progressive neurological disorder causes rapid, jerky movements, loss of memory, and behavior problems.

In the study, published in The Lancet Neurology, researchers followed five patients with Huntington's disease for up to six years following experimental treatment with stem cell transplants.

Previously, a small pilot study showed the treatment led to improvements in movement and brain function for two years after surgery, but the long-term benefits of stem cell therapy in treating Huntington's disease was unclear.

The results showed that the improvements reached a plateau two years after surgery and then faded away at a variable rate for four to six years after surgery. Two patients who did not benefit from stem cell transplantation two years after the procedure continued to decline in the same way as untreated patients.

Researchers say further refinements in stem cell transplantation technique as well as selecting patients appropriate for the procedure may improve the potential benefits of stem cell therapy in treating Huntington's disease.

 

SOURCES: WebMD

AC Bachoud-Lévi et al. Effect of fetal neural transplants in patients with Huntington's disease 6 years after surgery: a long-term follow-up study Lancet Neurology Feb. 27, 2006 early online edition

 

Background

Although we have shown in three out of five patients with Huntington's disease that motor and cognitive improvements 2 years after intracerebral fetal neural grafts are correlated with recovery of brain metabolic activity in grafted striatal areas and connected regions of the cerebral cortex, neural grafts are not known to have protective effects on the host brain per se. We undertook long-term follow-up of previously reported patients with the disease to ascertain the nature and extent of any secondary decline after grafting.

Methods

Five patients with Huntington's disease from our pilot study were assessed annually with the unified Huntington's disease rating scale, neuropsychological tests, and MRI, for up to 6 years after neural grafting. Resting cerebral activity was recorded at 2 and 6 years.

Findings

Clinical improvement plateaued after 2 years and then faded off variably 4–6 years after surgery. Dystonia deteriorated consistently, whereas chorea did not. Cognitive performance remained stable on non-timed tests, whereas progression of motor disability was shown by deterioration on timed tests. Hypometabolism also affected the brain heterogeneously, sparing the benefits in the frontal cortex and at the precise location of the grafts, but showing a progressive deterioration in other areas. Two patients who had no benefit from grafting at 2 years continued to decline in the same way as non-grafted patients.

Interpretation

Neuronal transplantation in Huntington's disease provides a period of several years of improvement and stability, but not a permanent cure for the disease. Improvement of the surgical procedure and in patient selection could improve the therapeutic value, but neuroprotective treatment seems to be unavoidable in the disease.