A leading New Zealand researcher hopes to quickly begin human trials of gene therapy to control obesity after proving the technique in animal experiments. Professor Matthew During, of Auckland University and Ohio State University in the U.S., leads a research team that announced the ground-breaking animal results this week in the online edition of Nature Medicine. “We believe we could be in the clinic with this approach within 12 months treating morbid obesity,” During says. The treatment is similar to During’s previously developed gene therapy for Parkinson’s disease, which is now in phase 2 human clinical trials.

The obesity experiments involved injecting up to three kinds of genetic material into the brains of mice and measuring the effects on their body weight and markers of diabetes. The genetic material is injected into the hypothalamus after boring two holes through the skull. It is carried in a harmless virus whose genetic material has been replaced by the therapy. The main active component is part of the human gene that causes the production of a substance called brain-derived neurotrophic factor (BDNF), a protein involved in weight regulation. It is coupled with genetic material that triggers auto-regulation of the BDNF, preventing weight loss from continuing beyond therapeutic levels. A third genetic component was developed for a subsequent neurosurgery injection, if needed, to knock out the introduced BDNF gene as a rescue device that halts the weight-loss process. In one of the trials, the body weight of obese mice reduced by 20% in three weeks and stabilized for the remainder of the 11-week experiment. According to During, many genes have been implicated in obesity, but little had been achieved in converting that knowledge into effective treatments — until now. “The efficacy and the degree of weight loss is very dramatic. We haven’t seen that in any study previously,” he comments. “We have very high expectations that this would also work in humans.”

Go to: The New Zealand Herald

Posted March 11th, 2009 under Tech Transfer

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