Gene-editing technology for drug-resistant cancer could be a ‘scientific arrangement’ for treating other diseases


For the first time, a new gene-editing technology called base edit was used to modify immune cells and successfully treated a teenager with treatment-resistant leukemia. A month later, Alyssa, 13, was in remission and she continued to do well for the next few months.

The treatment is a modification of mosaic antigen receptor, or CAR, T-cell therapy. But instead uses Gene editing technique CRISPR To modify the patient’s immune cells, clinicians used a more precise base-editing technique to alter the donor immune cells.

Those edited cells are given to patients to “rapidly find and destroy T cells in the body, including leukemia T cells,” according to one report. New information posted from University College London.

Dr. Otis Brawley, a professor of oncology at Johns Hopkins University, told CNN that the potential of base editing could be extended to other diseases.

Brawley, the former chief scientific and medical officer of the American Cancer Society, described the development as a “scientific arrangement”.

“That technology will help us in so many other diseases. For example, we used CRISPR to cure sickle cell anemia. We could very well use this gene-editing tool to make curing sickle cell anemia easier, better and less expensive,” said Brawley, who was not involved in the new study.

For now, he’s cautiously optimistic and wants to see more of these therapies before declaring success.

Basic editing changed Alyssa’s life. Teenagers UK to be diagnosed with T-cell acute lymphoblastic leukemia, or T-ALL, in 2021. Acute lymphoblastic leukemia is one of the most common childhood cancers. em and T-ALL account for 10% to 15% of ALL cases.

After chemotherapy and a bone marrow transplant, Alyssa’s cancer persisted, making palliative care her only remaining treatment option.

In May, Alyssa was admitted to Great Ormond Street Children’s Hospital in London to become the first person in the world to receive a transfusion of basically edited immune cells.

About a month after the treatment, Alyssa was in remission and received a second bone marrow transplant to restore her immune system. Six months after that transplant, Alyssa continued to be healthy.

Alyssa says she has no questions about whether to try the experimental therapy.

“Once I do that, people will know what they need to do, one way or another, so doing this helps everyone – of course I will,” she said in a hospital press release.

The findings of Alyssa’s case were presented at the annual meeting of the American Society of Hematology, and the team at Great Ormond Street hopes to enroll at least 10 more patients to trial the therapy.

Baseline editing is an even more precise gene editing technique than CRISPR and has less risk of unwanted effects on the chromosomes and therefore less risk of side effects.

Through basic editing, researchers at Great Ormond Street and University College London were able to take healthy donor T cells and remove two gene markers that prevent the modified cells from being destroyed by the immune system. the patient’s immune system or chemotherapy drugs they may be taking.

“This is our most sophisticated cellular technique to date,” said Waseem Qasim, professor of cell and gene therapy at University College London, who leads Alyssa’s donor cell production programme. and pave the way for other new treatments and ultimately a better future for sick children.” , in a statement.

Dr Robert Chiesa, consultant in bone marrow transplantation and CAR T-cell therapy at Great Ormond Street, said: “This is quite remarkable, although this is still a preliminary result, which should be followed up. monitor and confirm in the next few months. , in the statement.


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