British doctors have defended the effectiveness of an innovative treatment against an aggressive form of leukemia, the most common cancer in children, after the first patient to benefit from it went into remission.
Alyssa, a 13-year-old girl, was diagnosed with T-cell acute lymphoblastic leukemia in 2021. Her blood cancer, however, did not respond to conventional treatments, including chemotherapy and a bone marrow transplant.
So she took part in a clinical trial at Great Ormond Street Hospital for Children in London for a new treatment that uses genetically modified immune cells from a healthy volunteer.
Within 28 days, she was in remission, allowing her to receive a second bone marrow transplant to restore her immune system, researchers explained this weekend at the annual meeting of the American Society of Hematology.
Six months later, she is “doing well” and is back home in Leicester, central England, where she is receiving medical attention.
“Without this experimental treatment, Alyssa’s only option was palliative care,” the hospital said in a statement Sunday.
Acute lymphoblastic leukemia affects cells of the immune system, B and T lymphocytes, which fight and protect against viruses.
Alyssa is the first known patient to receive base-modified T cells, the hospital said. The processing involves the chemical conversion of the letters of the DNA code.
Researchers at the hospital and those at University College London helped develop the use of engineered T cells to treat B-cell leukemia in 2015.
But these T cells designed to attack cancer cells ended up killing each other during the manufacturing process, prompting scientists to think of other solutions.
“This is a great demonstration of how, with expert teams and infrastructure, we can combine cutting-edge technologies in the laboratory with real results in the hospital for patients,” said Waseem Qasim, consultant immunologist and professor at GOSH.
This “paves the way for other new treatments and ultimately a better future for sick children,” he added. Leukemia is the most common type of cancer in children.
Alyssa said in a statement that she did the trial for herself, but also for other sick children.
“I hope this will prove that the research works and that it can be offered to more children” who suffer from the disease, added his mother, Kiona.