TRANSPLANTS of genetically engineered skin cells can slow the progress of Alzheimer’s disease, the first human trial of its kind has revealed.

The new therapy, which involves injecting the modified tissue deep into patients’ brains, increased neural activity and substantially reduced the rate of mental decline, scientists report today.

Although the results come from a preliminary safety trial of only eight patients and need to be repeated on a much larger scale, they are seen as hugely promising by researchers.

There is no effective treatment for Alzheimer’s and the research offers the first proof of the principle that cell transplants have the potential to help people with the degenerative brain condition. “If validated in further clinical trials, this would represent a substantially more effective therapy than current treatments for Alzheimer’s disease,” Mark Tuszynski, a Professor of Neurosciences at the University of California in San Diego, who led the study, said.

“This would also represent the first therapy for a human neurological disease that acts by preventing cell death.”

He pointed out, however, that the study was designed to test only the safety and not the efficacy of the technique — thus the experiment had no control group receiving placebo treatment against which its value could be judged. A placebo-controlled double-blind trial is under way at Rush University Medical Centre in Chicago and its results will be a more accurate measure of therapeutic potential.

In the research, which began in 2001, Professor Tuszynski’s team took skin cells from eight patients in whom the early stages of Alzheimer’s had been diagnosed. These cells were then genetically modified to make a protein called nerve growth factor (NGF), which prevents nerve death and stimulates activity.

Extensive previous research has shown that transplants of cells that express NGF restored atrophied brain cells in aged monkeys and that the technique is also effective on mice.

The eight patients who took part in the trial were awake but under light sedation during the operation. Two of the patients moved as the cells were being injected but the six patients who completed the surgery without any ill-effects all appeared to benefit from their treatment. Injections in the full clinical trial will be performed under general anaesthesia.

The results are published today in the journal Nature Neuroscience.

“When administered into the central nervous system . . . [NGF] treatment is well tolerated and had the potential to improve symptoms and modify neurological disease progression,” Professor Tuszynski said.

BRAIN DISEASE

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