Researchers Using HIV Virus to Heal People

French medical researchers have made a significant breakthrough in the fight against the rare genetic disease adrenoleukodystrophy (ALD), which became well-known following the release of the 1992 movie “Lorenzo’s Oil”.  What’s ironic is that the HIV virus is an important part of the new procedure.  By mixing gene therapy and bone marrow transplants in two boys, lead researcher Dr. Patrick Aubourg of the University Paris – Descartes disabled HIV virus cells so they would not cause AIDS, and then used them as couriers to insert the healthy new gene.

In the treatment, stem cells  are harvested from the patient’s soft bone tissue.  Then a correcting gene is introduced using inactivated AIDS virus cells.  These are used because they are the only ones which can penetrate into the heart of the stem cells.  Re-injected into the patients, the altered cells navigate to the brain where they permanently correct the gene deficiency which causes ALD.

Although a small first step, Dr. Kenneth Cornetta, president of the American Society of Gene and Cell Therapy, said the procedure has “exciting implications” for other blood and immune disorders.  “This study shows the power of combining gene therapy and cell therapy,” according to Dr. Cornetta, who is conducting similar research at Indiana University.

ALD is a rare genetic disease that destroys the coating nerve fibers in boys’ brains.  Without that coating – known as myelin – the neurological system breaks down and causes blindness, deafness, dementia, a loss of muscle control and, ultimately, death.

Two years after receiving the therapy, both boys are showing no sign of deteriorating brain damage.  They function well with 15 percent of their blood cells producing the healthy protein, according to Dr. Aubourg, who plans to use the procedure with additional patients.  The Stop ALD Foundation, an American advocacy group, is raising money to fund a similar study in the United States.

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