A rare nerve disease was successfully treated using stem cell gene therapy in two patients in Hannover, Germany.
Doctors at the Hannover Medical School in Hannover conducted the experimental treatment in two boys who were diagnosed with Wiskott-Aldrich syndrome (WAS) soon after their birth. WAS is a rare X-linked recessive disease with symptoms including eczema, thrombocytopenia (low platelet count), immune deficiency, and bloody diarrhea.
The two boys were treated with their genetically altered hematopoietic stem cells so that the cells expressed the WAS protein. After six months of therapy which included gene transfer, the researchers found that the proportion of leukocytes that were positive for the WAS protein increased sharply while the proportion of monocytes expressing the protein has ranged between 7 percent and 28 percent.
In their report, which was published in the New England Journal of Medicine, the researchers said that such a treatment can be viewed as "feasible" in treating people suffering from WAS, adding that they have not encountered any treatment-limiting adverse events in the follow-up.