A second meeting called for sponsors of gene therapy clinical trials to follow-up with patients for as long as 20 years. Doctors at the National Institutes of Health in Bethesda, Maryland gave the girl an IV containing her own white blood cells that had been genetically altered in the hopes of fixing an inherited birth defect. In the decade since that revolutionary first genetic step, over 400 clinical trials to test gene therapy have been launched, mostly targeted at cancer treatments. Unfortunately however, amid great promise, there has been little actual clinical success to date. Human gene therapy involves the replacement of a person's faulty genetic material with normal genetic material to treat or cure a disease.
Human genes are pieces of genetic material recorded on a DNA molecule.
Genes are also believed to play a role in some cancers, either by stimulating the growth of cancer cells or by failing to stop them before they grow out of control. Advances in human gene therapy will eventually allow doctors to treat a disease by turning off a faulty gene, stopping the growth of a cancerous tumor, or enabling the body to produce a missing protein or enzyme. The FDA has not yet approved any human gene therapy product for sale. However, the amount of gene-related research and development continues to grow at an extraordinary rate. Panelists noted that the risks associated with altering genetic material are still unknown and, in some instances, side effects could take decades to develop.