Gene therapy for curing a number of disorders has become a need today. Doctors have got a huge success from a new cell and gene therapy for a blood cancer, myeloma that is increasing day by day. “Other therapies like chemotherapy help 10 to 30 percent of patients; immune system drugs at the most 35 to 40 percent while some gene-targeting drugs 70-80 percent, but the number never increases to 100”, said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society. “There are impressive results but time will reveal everything if they last”, he further added.
Multiple myelomas directly attack plasma cells responsible for making antibodies to fight infection. 30,000 multiple myeloma cases are seen each year in the US and around 115,000 worldwide. According to the National Cancer Institute, cancer rises by 2 to 3 percent each year and has proved as second fastest growing cancer for men and third for women.
Nine new drugs were found since 2000 to cure this cancer but it proved useless. Only half of US patients stay alive for 5 years after diagnosis of this disease. Dr. Frank Fan, the chief scientific officer of Nanjing Legend Biotech said, “With cell therapy, I can’t say we may get a cure but at least we bring the hope of that possibility.” He developed a new therapy named CAR-T therapy that involves removal of immune system soldiers called T-cells by filtering patient’s blood. These T cells are bio-engineered and altered in the lab to contain a gene that targets cancer cells. The altered cells are then injected back into the patient’s body.
This treatment is called as “living drug”. It is a one time treatment that means the cells are permanently altered once that multiply inside the body forming an army to fight cancer. Initially, most of the people had the side effects after taking this treatment like fever, low blood pressure and trouble in the breathing process. According to doctors, among the treated patients only 2 of them were severe and rest were treatable and temporary.
Another study laid down by researchers at Bluebird Bio and Celgene, US tested four different dose levels of cells in a total of 21 patients. Among them, 18 are long enough for treatment to judge effectiveness and all 15 who got an adequate number of cells had a response. Four of them have reached full remission till now and some of them are more than a year past treatment. They showed a remarkable result.