Alport syndrome is genetically inherited condition that can lead to serious kidney damage. It is a anomaly found in both men and women, but in females the disorder is usually quite mild, with little or no symptoms ever manifesting.

It is a different story for men afflicted with the condition though. In males the symptoms tend to be worse, progress quickly and in many cases lead to chronic kidney failure, even in adolescence. Many patients also lose their hearing permanently as a result of the progression of their disease.

Now, researchers at the Harvard School of Medicine believe that the work that they have been doing with stem cells may offer new hope to the victims of this fairly rare but potentially fatal condition.

Led by Dr Valerie LeBleu, a team from the prestigious Boston, MA facility tested a number of different cell based therapies in mice who had a genetic defect similar to that found in humans suffering from Alport’s Syndrome.

Through their work, the researchers were able to demonstrate that stem cell treatments could indeed repair the damaged DNA of the afflicted mice.  According to another author on the study, Raghu Kalluri MD, PhD, the team found that adult stem cells taken from bone marrow were just as effective as embryonic stem cells. Since the use of embryonic stem cells is still very restricted in the United States, this discovery should help translate the team’s findings into a real workable treatment in a reasonable amount of time.

By transplanting bone marrow from healthy specimens into the bodies of the Alport stricken mice the researchers found that they could achieve very significant improvement in kidney function even in those mice that had end stage renal failure. Perhaps more importantly still it seemed that bone marrow transplantation could be successful without the need for potentially harmful radiation treatments.

The team also found that a simple blood transfusion could also lead to improvements in kidney function in the affected mice. According to Kalluri both blood transfusion and bone marrow transplantation has been approved for clinical use in Alport’s patients in the past, which may mean that the benefits to be derived from the new work may be able to be implanted immediately, although all the study’s authors admit that until human trials can be begun the real benefit from the treatment cannot be fully accessed.

The study will be published in an upcoming issue of the professional publication Journal of the American Society of Nephrology.