Following the first patient to receive the novel therapy and experience remission, the groundbreaking world-first treatment for an aggressive form of leukemia was hailed by UK medics. 

T-cell acute lymphoblastic leukemia, also known as Alyssa's disease, was discovered in a 13-year-old girl in 2021. However, her blood cancer did not respond to conventional treatment, including chemotherapy and a bone marrow transplant.

At London's Great Ormond Street Hospital for Children (GOSH), she participated in a clinical trial for a novel therapy that used genetically modified immune cells from a healthy volunteer.

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In a 28-day period, her cancer was in remission, allowing her to receive a second bone marrow transplant to restore her immune system. She is "doing well" at home in Leicester, central England, six months after the incident and is getting follow-up care.

"Without this experimental treatment, Alyssa's only option was palliative care," the hospital said in a statement on Sunday.

Her recovery was "quite remarkable", according to Robert Chiesa, a GOSH consultant, though the outcomes still needed to be watched and verified over the coming few months.

Cutting edge 

Acute lymphoblastic leukemia (ALL), which affects the B and T cells of the immune system that defend and fight viruses, is the most common type of cancer in children.

The first patient to receive base-edited T cells, which entail chemically altering single nucleotide bases, which are letters in the DNA code and carry instructions for a specific protein, was Alyssa, according to GOSH.

Researchers from GOSH and University College London helped develop the use of genome-edited T cells to treat B-cell leukemia in 2015. The team encountered a challenge when attempting to treat some other forms of leukemia, however, because T cells developed to recognize and eliminate cancerous cells accidentally killed one another while being produced.

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To be able to target cancerous cells without harming healthy cells, the base-edited cells needed numerous additional DNA alterations. "This is a great demonstration of how, with expert teams and infrastructure, we can link cutting-edge technologies in the lab with real results in the hospital for patients," said GOSH consultant immunologist and professor Waseem Qasim.

"It's our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children." 

According to Alyssa's statement, she was motivated to participate in the trial not just for herself but also for other kids. Her mother, Kiona, added, "Hopefully this can prove the research works and they can offer it to more children."

During the past week, the researchers presented their findings at the American Society of Hematology's annual meeting.

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