A stem cell therapy could help treat a serious disease brain

According to scientists at the University Paris-Descartes, in France, it is the disease known as adrenoleukodystrophy, an inherited disorder caused by the absence of a protein called ALD involved in the degradation of fatty acids. Those with the disease, described in the U.S. movie "Lorenzo's Oil" lose the myelin shield that protects neurons. The disease affects children between 6 and 8 years and death occurs when they reach adolescence.

In a pilot study in two patients followed for two years, scientists slowed the disease by a lentiviral vector with which a therapeutic gene introduced into the bloodstream.
According to scientists, although further studies are needed with larger numbers of patients, the results indicate that therapy with lentiviral vectors, which are canceled versions of human immunodeficiency virus (HIV) could be used in the treatment of a wide range of disorders .

"This is the first time we've been using an HIV-derived lentivirus gene therapy in humans and is also the first time is a brain disease with gene therapy," said Patrick Aubourg, a professor of pediatrics at the University of Paris Descartes. In therapy involving stem cells also have the capacity to become cells that produce the myelin that protects neurons. "The lentiviral vector allows for expression of therapeutic gene is inserted into the chromosomes. Therefore, cells derived from stem cells continue to express the therapeutic gene," said the doctor. For the study, the scientists extracted stem cells from the patient's blood, which were genetically modified and reinserted into your body.

Two years later, one could see the presence of ALD protein in the bloodstream of both patients, who experienced neurological improvement and less progression of the disease, the scientists said. However, the researchers cautioned that the therapy can have side effects that could be serious because it alters the cell biology and patients may end up with leukemia. "The HIV-derived lentiviral vector carries basically the same risk, though its design makes patients less sensitive to this side effect," said Aubourg in the study.