Game-Changing Gene Therapy Saves Life Of 13-Year-Old From An Untreatable Cancer

Scientists in the UK have made use of a game-changing gene therapy to treat a young patient with relapsed T-cell leukaemia. The technique, which was administered for the first time in the world, could help offer a way to take down other kinds of childhood cancers, reports the Guardian.

Alysa via The Guardian

To the unaware, T-cell leukaemia is a kind of cancer that affects a category of white blood cells dubbed T-cells. The disease affects their development, making them grow too quickly and affecting the blood cell growth in the body.

Normally, in order to treat this, two treatments are prescribed -- bone marrow transplants and chemotherapy.

The novel treatment was administered to Alyssa, a 13-year-old from Leicester. She had earlier undergone chemotherapy and a bone marrow transplant in a bid to cure her cancer but it didn¡¯t prove to be as successful. Sadly, no further treatments remained, leaving the future truly uncertain for her.

However, the new treatment devised by doctors involved administering an infusion of donated T-cells, which were altered and designed to kill other T-cells including her leukaemia cells. This was done using base editing that allowed scientists to make a single change in the billions of letters of DNA that form an individual¡¯s genetic code.

While there are other techniques that allow changing of genetic code, they also result in a ton of side effects. Base editing on the other hand allowed researchers to make a series of separate changes to the donated T-cells. They had to make sure they attacked harmful T-cells and leukaemia cells and not each other. They also made the cells to allow them to work after chemotherapy while also preventing it from affecting normal cells.

Unsplash

The new treatment with a second bone-marrow transplant to restore her immune system, Alyssa has been leukaemia-free for over six months.

Researchers at London¡¯s Great Ormond Street Hospital who treated Alyssa are now looking to bring on board 10 more T-cell Leukaemia patients who don¡¯t have any other treatments remaining, for trials.

Immunologist Professor Waseem Qasim, one of the project¡¯s leaders said in a statement, ¡°This is our most sophisticated cell engineering so far, and it paves the way for other new treatments and ultimately better futures for sick children.¡±

¡°This is an ¡®off the shelf¡¯ universal cell therapy and ¨C if replicated ¨C will mark a huge step forward in these types of treatments,¡± added Dr Louise Jones of the Medical Research Council, which funded the project.

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