Therapy Analysis - Gene therapy - Still the Next Big Thing?
Current gene therapies
Despite a slow start, gene therapy still has the potential to effectively treat devastating inherited diseases for which there is little hope of finding a conventional cure, and it certainly seems that eventually gene therapy will become a fundamental part of modern medicine, although the timescale for this is less than certain.
With the impending approval of three gene therapies, it is hoped that 2009 could prove a significant year for gene therapy. Furthermore, Vical, Oxford BioMedica and MolMed all expect approvals for their lead gene therapy drugs, currently in Phase III trials, over the next three years. Recent results from preclinical studies have also suggested the successful use of gene therapy for the treatment of both obesity and diabetes, and researchers will be looking to move to human trials in these indications in the near future.
The end of 2008 also saw the startling case of an AIDS patient in Germany, who underwent a stem cell transplant with CCR5-deficient cells that are resistant to HIV. After more than 600 days, doctors are still unable to detect the virus despite terminating all conventional AIDS medication. These results suggest a new, genetic method of controlling the AIDS virus and effectively provide proof of concept for the use of gene therapy in combating a disease that has thus far proved most resistant to conventional pharmacological intervention. This example serves to highlight the astonishing potential future of human healthcare - to cure a viral infection not by attacking the virus itself but by altering our own genetic makeup. In this lies the true power of gene therapy, not just to control and contain disease, as with conventional pharmaceuticals, but actually to rewrite our own human nature at its most fundamental level.
Parita Patel
Pharmaprojects Analyst
