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Great Ormond Street trial clears previously incurable disease in 12 of 14 patients, with five remaining in remission.
A new treatment for an incurable form of leukemia has been hailed by experts after it left patients without a trace of the disease.
The patients were treated with a faster acting version of CAR-T therapy, in which the body’s immune system is used against the malignant cells.
The trial at Great Ormond Street Hospital in London was carried out with the aim of reducing the side-effects of treatment for the patients, most of whom are children.
The treatment for acute lymphoblastic leukemia (ALL) sees the patient’s own immune cells genetically modified and then used to target cancer.
During the trial, 14 patients with a previously incurable strain of ALL were given the therapy and 12 saw the disease cleared after three months. Five of those patients remain leukemia-free.
Professor Persis Amrolia, the study’s chief investigator, said: “CAR-T therapy is a fantastic example of using the power of the immune system to specifically target cancer cells.
“While it doesn’t work for everyone, it can offer hope for those children who have run out of all other options.
“We’re just at the beginning of this new treatment and over the next few years, I hope we can refine it further to make it safer and more effective.
“The side-effects of CAR-T therapies can be severe, so we hope that this new technology can reduce the risk for patients.”
Austin Sweeney, 10, was diagnosed with ALL when he was two and was subsequently invited to take part in the trial after he had exhausted all other treatment options.
His father, Scott Sweeney, said Austin was “so fortunate” to have been able to participate.
“He had the cells at Great Ormond Street Hospital in October 2016 and we found out on his birthday at the end of that month that the cells were doing exactly what we needed them to do,” Mr. Sweeney said.
“Two-and-a-half years later, Austin is doing so well. He is more physical than he has ever been. It is lovely to see him full of energy.”
ALL affects around 400 children in the UK each year, according to the hospital, and while most patients can be cured with standard treatments including chemotherapy and transplant, some relapse.
Survival for children suffering from ALL increased from under 10 percent in the 1960s to 90 percent in 2015, though the survival rate is lower for babies.
The hospital said the research, which was published in the Nature Medicine journal on Monday, offers great hope to those with relapsed ALL, which is the most common cause of childhood cancer death in the UK.
Source: Independent
