In more promising news from the American Heart Association s Scientific Sessions in California, researchers have reported a key advance in using stem cells to repair damaged hearts.
Although the study involved just 30 patients, it demonstrated that allogeneic stem cells those taken from a different person are as safe and effective as autologous (the patient s own) stem cells in restoring heart tissue and allowing the heart to pump more effectively. Although this small study must clearly be replicated in a much larger trial before this approach is used more widely, the results show promise.
In other stem cell news, the first gene therapy to be approved in a regulated market was announced by the European Commission, which granted a stamp of approval to a treatment for the rare inherited disorder lipoprotein lipase deficiency (LPL). This genetic disorder leaves people unable to store fat in adipose and muscle cells. The new drug has been named Glybera by its maker, UniQure.
The therapy, which manufacturers say will be available next year, uses a virus to insert a working copy of the gene into muscle cells. This is a true breakthrough, says ACSH s Dr. Gilbert Ross. Hopefully we will see the actions of the European Commission echoed here in the States.
ACSH s Dr. Josh Bloom is cautiously optimistic: Gene therapy has been a very hot topic for more than 20 years with little to show for it. Although this study is focused on a rare defect, any technological advances that facilitate the introduction of the right gene into the right cell could be extremely valuable when applied to common diseases.