Gene therapy is representing a significant shift in medicine and disease treatment. In 2017, gene therapy was approved by the Food and Drug Administration as an additional treatment plan for individuals in the U.S. Gene therapy allows doctors to go in and alter an individual’s DNA by removing bad genes and replacing them with healthy genes and can work towards curing certain diseases. Researchers are still working to help patients with rare, life-threatening conditions, but the following advancements in gene therapy treatments are helping shape the future of medicine.
Caused by a single genetic mutation in the body, sickle-cell is an inherited blood disorder that affects people around the world. Scientists have found that removing bone marrow and then modifying them in a lab by adding large amounts of healthy genes to red blood cells to keep them from becoming “sickled.” Once the treated cells are infused back into the body, they can begin to make healthy blood cells. Early treatments have proven successful, but sickle-cell gene therapy is still experimental.
The FDA has approved two treatments that could potentially change the way cancer is treated in patients. Known as CAR-T therapies, the treatment is created by extracting T cells from the patient and genetically altering them. The extracted cells are then infused back into the body and are genetically altered to go after and destroy any cancer carrying cells. While this has only been tested as a last resort when traditional treatments such as chemotherapy fail, patients have recovered and stayed in remission for months.
Caused by a hereditary lack of a coagulation factor, individuals who suffer from hemophilia can significantly benefit from gene therapy treatments. Some clinical trials have shown that patients who received gene therapy had increased blood-clotting proteins that they lack because of hemophilia. While treatment takes longer than a year, patients who have gone through the clinical trial have shown that they have fewer bleeding issues and have had to receive fewer blood infusions to help with clotting.
While these treatments are still very new to the market, gene therapy will continue to evolve and one day replace most disease treatments.