An experimental gene therapy that modifies the genetic code of key blood cells to help them fight cancer showed promise in a preliminary study, with more than one-third of lymphoma patients showing no signs of the disease six months after one treatment.
National Cancer Institute (NCI) originally developed the gene approach and licensed it to Kite Pharma, which conducted the study and released the preliminary results. If FDA approves the therapy, it will be the first gene therapy available in the United States.
Kite Pharma plans to present the full results at the American Association for Cancer Research presentation in April. The study has not been published or reviewed by other experts, the Associated Press reports.
How it works
For the study, researchers administered a so-called CAR-T cell therapy. The treatment involves filtering patients' blood to remove T-cells, a key part of the immune system. The cells were then modified to contain a gene that fights cancer—turning the cells into "cancer killers," according to AP—and reintroduced into patients' bloodstreams. The treatment permanently modifies the cells, which multiply within the patient, the AP reports.
The study included 101 patients who had one of three types of a blood cancer called non-Hodgkin lymphoma, which had not responded to treatment. According to AP, the median survival time for such patients is about six months.
After six months, 36 percent of patients were in complete remission, 41 percent of patients' cancer had shrunk by at least half, and 82 percent of patients had their cancer shrink at least by half at some point during the study's six-month time period. Moreover, the number of patients in complete remission at six months barely changed from the number in remission at three months, indicating that "this one-time treatment might give lasting benefits for those who do respond well," according to AP.
After nine months, more than half of patients in the study were still alive.
While researchers say the results are promising, there were also some serious complications. Thirteen percent of patients developed a serious issue wherein their immune response dangerously overreacts to the treatment. However, the rate declined over the study period as doctors became more adept at identifying and treating the issue sooner.
About one-third of patients developed anemia or blood-count-related problems during the study. According to AP, 28 percent of patients had neurological problems—such as sleepiness or difficultly sleeping—although they typically only lasted a few days. Three of 101 patients in the study died for reasons unrelated to their cancer, with two of those deaths thought to be related to the treatment.
Roy Herbst, cancer medicines chief at the Yale Cancer Center, called the results "extraordinary" and "extremely encouraging." He said patients will need to be monitored longer than six months to determine if the treatment becomes less effective over time—but added that the treatment was "certainly is something I would want to have available."
NCI's Steven Rosenberg, who was not involved in the study, said the treatment was safe—especially in comparison to progressive lymphoma. "It's a safe treatment, certainly a lot safer than having progressive lymphoma," he said (AP/Los Angeles Times, 2/28; Farley Steele, HealthDay, 2/28).
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