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Cancer therapy in a little girl first cured with cell therapy
British doctors have achieved a medical sensation. Using genetically engineered immune cells, they were able to cure a particularly aggressive form of leukemia in a one-year-old girl. As the Great Ormond Street Hospital in London (GOSH) reports, the child was the first patient in the world to use this innovative procedure.
Blood cancer is discovered three months after birth
Doctors at London's Great Ormond Street Hospital (GOSH) have been able to save the lives of people suffering from cancer through an innovative process. The clinic reports that this is the first case in the world in which genetically engineered immune cells - known as "UCART 19 cells" - were used to fight leukemia. "We were overjoyed that it worked. Her leukemia was so aggressive that such a reaction is almost a miracle, "the girl's attending physician, Paul Veys, is quoted in the message.
Child could be freed from severe cancer. Image: Sebastian Kaulitzki - fotolia Acute Lymphocytic Leukemia curable in most cases
The little Layla Richards was born June 10, 2014 healthy and happy. But at the age of three months, the doctors discovered a particularly aggressive form of blood cancer, which is referred to in the jargon as infantile acute lymphoblastic leukemia (ALL). The disease primarily affects the blood and bone marrow but may also affect the lymph nodes, liver and other organs. Whereas only a few years ago, ALL led to the death of the vast majority of patients, today it is curable, especially in children, in about 80 percent of cases with intensive chemotherapy.
But with Layla Richards, the therapy did not start. Instead, the cancer returned despite chemo and bone marrow transplantation, making the hope for a cure smaller and smaller. The physicians at the GOSH told parents that there were no other treatment options for Layla that could cure them, so they suggested palliative care.
T-helper cells of the healthy donor are modified
"We did not want to accept palliative care, so we asked doctors to try everything for our daughter, even if it had not been tried before," Lisa Richards said, according to the GOSH statement. It worked, because the physicians suggested to the parents an innovative treatment method, which was developed at this time at the GOSH. In this, the white blood cells ("helper T cells") of a healthy donor are modified so that they are prepared for the leukemia and can fight the cancer.
Intervention takes only a few minutes
"The doctors told us that even if we tried the treatment, there was no guarantee of success. But we prayed that it would work, "said the girl's father, Ashleigh Richards. "It was frightening to think that the treatment had been used on a human before," adds the father. But despite possible risks, there was no doubt. "She was sick and in great pain, so we had to do something."
Finally, the child received a small injection of the genetically engineered cells, taking only a few minutes to complete. A few weeks later came the good news that the treatment had been successful. Layla's father surprised his wife on the phone when she was picking up the eldest daughter from school. "At first I thought it was bad news, but then he said 'it works!' and I just cried with happiness, "says the child's mother.
Child can be discharged just one month after the transplant
About two months later and free of cancer, Layla returned to the GOSH for a second bone marrow transplant to replace the bone marrow that had been attacked by the treatment. From then on, her blood cell counts increased, and one month after the transplant, she was strong enough to be discharged.
Despite the unbelievable success, Waseem Qasim, the cell and gene therapy specialist at the hospital, warned against too much optimism: "So far we have only used this treatment on a single, very strong little girl and therefore we need to be careful this could be a suitable treatment option for all children. However, this is a milestone in the use of new genetic engineering and the effect on this child is breathtaking. "Accordingly, a repeat of the success" could be a big step forward in treating leukemia and other cancers, "Professor Waseem Qasim continued. (No)