Posted for Erika Check

Is gene therapy inching its way towards a comeback? Today, doctors report that they have used the technique to cure 8 children with one form of the rare illness called severe combined immunodeficiency, or SCID. And the achievement is so far free from the complications that accompany gene therapy to treat a form of SCID caused by a different genetic glitch.

The result is welcome positive news for gene therapy, which has been struggling to rehabilitate its image after some stunningly bad news rocked the field 6 years ago. At that time, doctors treating SCID patients were cautiously optimistic that they were writing gene therapy’s first success story by infused corrective genes into patients with so-called “X-linked SCID.” But the success stories were marred in 2002 when one of the X-linked SCID patients developed cancer. The trials were later allowed to restart, but five of 20 patients treated with gene therapy have since developed cancer, and one has died.

Today, doctors led by Maria-Grazia Roncarolo of the San Raffaele Telethon Institute for Gene Therapy in Milan publish the results of gene therapy trials in children with a different form of SCID that is caused by a deficiency of an enzyme called adenosine deaminase. Children with this version of SCID can be treated with bone marrow transplants and enzyme replacement. But in 8 of 10 children who lacked a matching donor, doctors report, treatment with a corrected adenosine deaminase gene allowed them to survive without enzyme injections.

“The prospects for continuing advancement of gene therapy to wider applications remain strong,” write two scientists who were not involved in the study in a commentary accompanying the report.

Coverage

Gene therapy cures form of ‘bubble boy disease’ – AP