Adolescent cancer deemed incurable goes away after innovative gene therapy News

British Alyssa, 13, was diagnosed with T-cell acute lymphoblastic leukemia in May 2021. In the battle against the disease, the teen underwent all available conventional treatments, but did not reach remission.

That changed after she was the first person in the world to undergo a gene therapy using CD7 CAR-T cells, which researchers classify as the most advanced cell engineering model today.

Alyssa was about to undergo palliative care (prevention and alleviation of suffering) because chemotherapy and a bone marrow transplant had failed. At that moment, the British woman became the first patient to be enrolled in an unprecedented study of a treatment for this disease.

The young woman thought, according to the newspaper daily MailIt was: “Once I do this, people will know what they need, one way or another, doing this will help people, of course I will.”

T-cell acute lymphoblastic leukemia is a condition in which T cells, which are supposed to move around the body and “destroy” defective cells, fail to perform this function, get out of control and end up becoming a danger to the patient.

With this situation in mind, the gene therapy, conducted at Great Ormond Street Hospital for Children (GOSH), reconstructed DNA from T cells from a healthy volunteer donor and transferred them, in stages, into Alyssa’s body.

The new cells were modified to allow them to find and kill only cancerous T cells.

In just 28 days, the teen battled the disease, according to the researchers. The second step was a bone marrow transplant to rebuild Alyssa’s immune system.

“Since Alyssa developed leukemia in May last year, she has not made a full recovery from the disease. It was only after she received CD7 CAR-T cell therapy and a second bone marrow transplant at GOSH that she was free of leukemia.” Robert Chiesa, consultant bone marrow transplant and CAR T cell therapy at the hospital.

“We were so touched by how brave she was, and nothing would make me happier than seeing her out of the hospital and back to a more normal life,” he adds.

Currently, the British woman is at home and recovering from the procedures with her family, but with observation and monitoring by the hospital. Keona, Alyssa’s mother, told Gosh that the family is “on cloud nine” with the outcome and that it’s “amazing to be home.”

“I hope this proves to be a successful research and they can introduce it to more kids, it has to be all for something. We are now trying to live between commitments. Alyssa wants to go back to school and that could become a reality soon. She is already teasing her brother because he was He should come back for the fall.”

Scientists are still hopeful that the treatment can be applied to other children and other diseases, if the treatment continues to be successful in the coming months.

“This is a fascinating demonstration of how, through expert teams and infrastructure, we can connect cutting-edge technologies in the lab with real outcomes in the hospital for patients. It is our most advanced cell engineering yet and paves the way for other new therapies and treatments and ultimately, a better future for pediatric patients,” he said. Immunologist Waseem Qasim, GOSH consultant.