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Archive - Jun 18, 2012

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Implanted Device Has Therapeutic Potential in Huntington’s Disease

Studies suggest that neurotrophic factors, which play a role in the development and survival of neurons, have significant therapeutic and restorative potential for neurologic diseases such as Huntington's disease. However, clinical applications are limited because these proteins cannot easily cross the blood-brain barrier, have a short half-life, and cause serious side effects. Now, a group of scientists has successfully treated neurological symptoms in laboratory rats by implanting a device to deliver a genetically engineered neurotrophic factor directly to the brain. The scientists report on their results in the May 31, 2012 issue of Restorative Neurology and Neuroscience. Researchers used Encapsulated Cell (EC) biodelivery, a platform which can be applied using conventional minimally invasive neurosurgical procedures to target deep brain structures with therapeutic proteins. "Our study adds to the continually increasing body of preclinical and clinical data positioning EC biodelivery as a promising therapeutic delivery method for larger biomolecules. It combines the therapeutic advantages of gene therapy with the well-established safety of a retrievable implant," says lead investigator Dr. Jens Tornøe, NsGene A/S, Ballerup, Denmark. Investigators made a catheter-like device consisting of a hollow fiber membrane encapsulating a polymeric "scaffold," which provides a surface area to which neurotrophic factor-producing cells can attach. When implanted in the brain, the membrane allows the neurotrophic factor to flow out of the device, as well as allowing nutrients in. Dr. Tornøe and his colleagues used the neurotrophic factor Meteorin, which plays a role in the development of striatal projection neurons, whose degeneration is a hallmark of Huntington's disease.

Anti-Cocaine Vaccine Effective in Mouse Study

A single-dose vaccine capable of providing immunity against the effects of cocaine offers a novel and groundbreaking strategy for treating cocaine addiction [Referential web site: http://drugabuse.com/library/how-to-help-a-cocaine-addict/ and is described in an article published in the May 2012 issue of Human Gene Therapy. "This is a very novel approach for addressing the huge medical problem of cocaine addiction," says James M. Wilson, M.D., Ph.D., Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia. In the article, a team of researchers from Weill Cornell Medical College, The Scripps Research Institute, and Cornell University used a virus-based delivery vehicle in mice to transfer a gene that produces a protein capable of binding to cocaine present in the blood, preventing the cocaine from crossing into the brain. The protein is a monoclonal antibody that sequesters cocaine, making the vaccinated mice resistant to the drug's effects. Whereas unvaccinated mice exhibited hyperactivity when exposed to intravenous cocaine, the immunized mice showed no effects, according to the authors. [Press release] [Human Gene Therapy article]

Newly Discovered Fruit Fly Gene Could Provide Clues to New Cancer Drugs

Loyola University Chicago Stritch School of Medicine researchers are taking advantage of a quirk in the evolution of fruit fly genes to help develop new weapons against cancer. A newly discovered fruit fly gene is a simplified counterpart of two complex human genes that play important roles in the development of cancer and some birth defects. As this fruit fly gene evolved, it split in two. This split has made it easier to study, and the resulting insights could prove useful in developing new cancer drugs. "Evolution has given us a gift," said Andrew K. Dingwall, Ph.D., senior author of a paper that describes how his team identified and analyzed the split gene. Their findings are published in the June 1, 2012 issue of Development. Based on the importance of the findings, the paper was recently selected as an "Editor's Choice" in Science Signaling, published by the American Association for the Advancement of Science (AAAS). When normal cells develop, they differentiate into particular types, such as bone cells or muscle cells, and reproduce in an orderly manner. The process is governed by genes and hormones that work in concert. Two of these genes are known as MLL2 and MLL3. Cancer cells, by contrast, undergo uncontrolled division and reproduction. Since 2010, a growing number of cancers have been linked to mutations in the MLL2 and MLL3 genes. These cancers include non-Hodgkin’s lymphoma, colorectal cancer, kidney cancer, bladder cancer and a brain tumor called medulloblastoma. There also is evidence that MLL2 and MLL3 mutations are involved in breast and prostate cancers. The MLL2 and MLL3 genes are similar to one another. Each has more than 15,000 building blocks called base pairs -- more than 10 times the number found in a typical gene. Because these genes are so large and complex, they are difficult to study.