Experimental treatment helps 13-year-old girl achieve leukemia remission: ScienceAlert
British doctors have hailed a pioneering treatment for an aggressive form of leukaemia, after a teenager became the first patient to receive a new therapy and went into remission.
The 13-year-old girl, identified only as Alyssawas diagnosed with T-cell acute lymphoblastic leukemia in 2021.
But his blood cancer has not responded to conventional treatment, including chemotherapy and a bone marrow transplant.
She was enrolled in a clinical test of a new treatment at Great Ormond Street Hospital for Children in London (GOD) using genetically modified immune cells from a healthy volunteer.
Within 28 days, his cancer was in remission, which allowed him to receive a second bone marrow transplant to restore his immune system.
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Six months later, she is “doing well” at her home in Leicester, central England, and is receiving follow-up care.
“Without this experimental treatment, Alyssa’s only option was palliative care,” the hospital said in a statement Sunday.
Robert Chiesa, GOSH consultant, said its turnaround had been “quite remarkable”, although the results were yet to be monitored and confirmed in the coming months.
On the cutting edge
Acute lymphoblastic leukemia (ALL) is the most common type of cancer in children and affects cells of the immune system, called B cells and T cells, which fight and protect against virus.
GOSH said Alyssa was the first known patient to receive base-edited T cellswhich involves chemically converting single nucleotide bases – letters of the DNA code – that carry instructions for a specific protein.
Researchers from GOSH and University College London helped develop the use of genome-edited T cells to treat B-cell leukemia in 2015.
But to treat other types of leukaemia, the team had to overcome the challenge that T cells designed to recognize and attack cancer cells ended up killing each other during the manufacturing process.
Several additional DNA changes were required for the base-modified cells to allow them to target cancer cells without damaging each other.
“This is a great demonstration of how, with expert teams and infrastructure, we can combine cutting-edge technologies in the lab with real results in the hospital for patients,” said GOSH Consultant Immunologist and Professor Waseem Qasim.
“This is our most sophisticated cellular engineering to date and paves the way for other new treatments and ultimately a brighter future for sick children.”
Alyssa said in the statement that she was prompted to participate in the trial not just for herself but for other children.
His mother, Kiona, added“I hope this proves that the research works and that they can offer it to more children.”
The researchers were presenting their findings this weekend at the annual meeting of the American Society of Hematology.
