Nov. 29, 2000 -- In a small study, three of five patients with the devastating progressive disorder Huntington's disease saw their disease greatly improve. How? By receiving transplants of brain cells from fetuses.
The treated patients gained better control of their movements and improvements in their ability to carry out daily tasks when compared with untreated patients. The results of the study are scheduled to be published in the Dec. 9 issue of the British medical journal The Lancet.
Huntington's disease is a progressive genetic disorder that usually begins in middle age. It's characterized by rapid and jerky involuntary movements, accompanied by a mental decline leading to dementia, and death follows about 15 years after the symptoms first appear. Perhaps the most famous victim of Huntington's disease was the legendary folk singer Woody Guthrie, creator of the song "This Land is Your Land," who died from the disease in 1967.
The disease is caused by shrinkage of a portion of the brain and by the loss of brain cells that produce chemicals necessary for the control of movement. Several drugs to treat the disorder are in human studies, but there is currently no proven treatment that can significantly slow or reverse the disease. Currently the only ways to deal with it are through genetic screening to identify people who carry the gene, genetic counseling, and/or abortion if a fetus is found to be affected.
But Marc Pechanski, MD, and colleagues from France's National Institute of Health and Medical Research report in the studythat the improved patients had previously experienced marked deterioration but had significant progress after receiving transplants of fetal brain cells in two surgeries performed one year apart.
All three patients were able to resume bicycling. In addition, according to the authors, "Patient 1 plays indoor games and takes children to school; patient 2 mows his lawn and takes care of children's homework; patient 3 swims and plays the guitar," the authors report.
"Patient 2 drives and, although he reduced his workload to part-time after the first [operation], was still working up to three years later. Patient 3 has started to do odd jobs in his house. Relatives indicated that patients were intellectually much more 'present,' [were] physically much fitter, and got weary less quickly."
In contrast, two of the five patients who received the transplants -- and an additional 22 patients with the disease who did not have the operation -- had declines in most tests and worsening of bodily control.
"These new data are important because they provide the first evidence that [transplants] of tissue taken from the human fetal [brain] can survive and induce measurable functional improvement in patients with Huntington's disease," write Olle Lindvall and Anders Björklund, who are both professors at the Wallenberg Neuroscience Center at Lund University in Sweden, in an accompanying editorial.
But the editorialists caution that the study is quite small, that durability and long-term effectiveness of the transplants is still unknown, and that researchers still don't know how much tissue will need to be transplanted.
One problem with this kind of approach to Huntington's is that transplanting nerve cells into just one area of the brain addresses only part of the problem, because the disease can cause damage across wide areas of the brain, Kenneth H. Fischbeck, MD, chief of the neurogenetics branch at the National Institute of Neurological Disorders and Stroke, tells WebMD.
"On the other hand, whatever other kind of treatment you might expect to come up with over the long run would not be expected to bring back nerve cells that have died, so it is something that is worth pursuing, and it's good to hear that they're making some headway," Fischbeck says.
The researchers have organized a larger study to determine whether the encouraging early results will translate into a useful therapy for patients with Huntington's disease.