The immune systems of two men who were born with an "incurable" genetic disorder that made them almost defenceless against bacterial and fungal infections has been repaired with Gene therapy. Sixteen months after an operation to repair a defective gene, the once severely ill patients show signs of complete recovery, a German-based team of scientists said Sunday.
Scientists believe that this latest success will lead to further operations for treating other such chronic and incurable conditions due to inheritance of defective genes. These cutting-edge techniques have mainly been used in the past to help sick children. Dorothee von Laer, gene therapy coordinator at the Georg Speyer laboratory in Frankfurt, said two males, aged 25 and 26, had been treated for a hereditary autoimmune disease over the past two years at Frankfurt University Hospital. She said the new technique might prove suitable for other diseases where people are born with a defective gene.
Gene therapy is a procedure involving the alteration of a person's genome to repair or replace a mutated gene. After years research, the technique has finally seems to have proven its potential with a handful of successful operations. The treatment of two men with chronic granulomatous disorder (CGD) is the latest success. The men suffered repeated life-threatening infections from bacteria and fungi from an early age had to rely on powerful antibiotics and antifungal medicines. After their operations 16 months ago, both patients have gained weight and have been free of the severe infections that have plagued them since childhood.
Manual Grez of the Institute for Biomedical Research in Frankfurt, one of the leaders of the study published in the journal Nature Medicine, said the results suggest that it may be possible to use gene therapy to treat other similar inherited diseases. Stating that the work was a landmark study he said that though the results are very positive there still has to be a lot of work to make this technology available to all patients.
Chronic granulomatous disorder (CGD) is a rare immunodeficiency disease caused by a defective gene on the maternally inherited X chromosome. In this condition their bodies cannot kill invading bacteria and fungi and they often have internal infections. Currently the disease is treated using bone-marrow transplants. The new technique involved the scientists to take blood stem cells from the men and introduced a healthy gene to the cells. The cells were then re-injected into the men. The key to the technique was a new, more efficient method of inserting the genes in many cells at the same time.
Concern remains that such therapy could have a negative side effect like causing cancer of the blood. Research is continuing on how to make such techniques safer. Professor Adrian Thrasher of the Great Ormond Street Hospital in London says that further improvements and refinements in the technology are now being done and studied so as to maximise the effectiveness and safety of the system.