British doctors are praising a pioneering treatment for aggressive leukemia after a teenage girl became the first patient to receive a new treatment and go into remission.
A 13-year-old girl named Alissa was diagnosed with T‑cell acute lymphoblastic leukemia in 2021.
But her blood cancer didn’t respond to conventional treatments like chemotherapy and a bone marrow transplant.
She was enrolled in a clinical trial at London’s Great Ormond Street Children’s Hospital (GOSH) for a new treatment using genetically engineered immune cells from healthy volunteers.
After 28 days, the cancer was in remission and I was able to receive a second bone marrow transplant to restore my immunity.
Six months later, he is “well” under observation at his home in Leicester, central England.
Without this experimental treatment, Alyssa’s only option was palliative care,” the hospital said in a statement on Sunday.
GOSH consultant Robert Chiesa said Alyssa’s progress was “very remarkable”, but results will be monitored and confirmed in the coming months.
Acute lymphoblastic leukemia (ALL), the most common type of cancer in children, affects cells of the immune system that fight and protect against viruses called B and T cells.
Alissa is the first patient to receive base-editing T cells, which chemically converts individual nucleotide bases into letters of the DNA code that contain instructions for specific proteins, GOSH said. increase.
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 leukemia, they had to overcome the challenge that T cells designed to recognize and attack cancer cells kill each other during the manufacturing process. It is.
The base-edited cells had to make many more changes to their DNA before they could attack cancer cells without harming each other.
Waseem Qasim, Professor and Consultant Immunologist at GOSH, said: “It’s a great demonstration of how with a team of experts and infrastructure, you can combine cutting-edge technology in the lab with real patient outcomes in the hospital. ’ said.
“This is the most sophisticated cell engineering to date, paving the way for other new treatments and ultimately a better future for sick children.”
In her statement, Alyssa said she felt she had to participate in the trial not only for herself, but also for other children.
Her mother, Kiona, said: “I hope this research will prove effective so that more children can be reached.
The researchers presented the results this weekend at the American Society of Hematology annual meeting.
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