British doctors have backed the effectiveness of an innovative treatment against an aggressive form of leukemia, the most common cancer in children, after it went into remission after the first patient benefited from it.
Alyssa, a 13-year-old girl, was diagnosed with T-cell acute lymphoblastic leukemia in 2021. However, his leukemia did not respond to conventional treatments, including chemotherapy and a bone marrow transplant.
So he 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, he was in remission, allowing him to receive a second bone marrow transplant to restore his immune system, researchers explained this weekend at the annual meeting of the American Society of Hematology.
Six months later, he is “well” and back home in Leicester, central England, where he is receiving medical attention.
“Without this experimental treatment, Alyssa’s only option is palliative care,” the hospital said in a statement on Sunday.
Acute lymphoblastic leukemia affects cells of the immune system, B and T lymphocytes, which fight and protect against viruses.
Alyssa is the first patient to receive the base transplanted T cells, the hospital said. Processing involves chemical modification of the letters of the DNA code.
Researchers from the hospital and University College London helped use engineered T cells to treat B-cell leukemia in 2015.
But these T cells, designed to attack cancer cells, kill each other during the manufacturing process, prompting scientists to think of other solutions.
“With expert teams and infrastructure, this is a great demonstration of how cutting-edge technologies in the lab can be linked to real results in the hospital for patients,” said Wasim Qasim, consultant immunologist and professor at GOSH.
It “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 tested it for herself and other sick children.
“I hope this will prove that the research works and that it can be given to more children with the disease,” said his mother Kiona.