New kind of ‘de­signer’ im­mune cells clear baby’s leukemia

Kuwait Times - - HEALTH & SCIENCE -

NEW YORK: A baby whom doc­tors thought al­most cer­tain to die has been cleared of a pre­vi­ously in­cur­able leukemia in the first hu­man use of an “off-the-shelf” cell ther­apy from Cel­lec­tis that cre­ates de­signer im­mune cells. One-year-old Layla had run out of all other treat­ment op­tions when doc­tors at Bri­tain’s Great Or­mond Street Hospi­tal (GOSH) gave her the highly ex­per­i­men­tal, ge­net­i­cally edited cells in a tiny 1-mil­li­liter in­tra­venous in­fu­sion. Two months later, she was can­cer-free and she is now home from hospi­tal, the doc­tors said at a brief­ing about her case in Lon­don on Wed­nes­day.

“Her leukemia was so ag­gres­sive that such a re­sponse is al­most a mir­a­cle,” said Paul Veys, a pro­fes­sor and di­rec­tor of bone mar­row trans­plant at GOSH who led the team treat­ing Layla. “As this was the first time that the treat­ment had been used, we didn’t know if or when it would work, so we were over the moon when it did.” The ge­need­ited cell treat­ment was pre­pared by sci­en­tists at GOSH and Univer­sity Col­lege Lon­don (UCL) to­gether with the French biotech firm Cel­lec­tis, which is now fund­ing full clin­i­cal tri­als of the ther­apy due to start next year. It is de­signed to work by adding new genes to healthy do­nated im­mune cells known as T-cells, which arm them against leukemia. Us­ing a gene-edit­ing tech­nol­ogy called TALEN, which acts as “molec­u­lar scis­sors,” spe­cific genes are then cut to make the T-cells be­have in two spe­cific ways: Firstly, they are ren­dered in­vis­i­ble to a pow­er­ful leukemia drug that would usu­ally kill them and se­condly they are re­pro­grammed to only tar­get and fight against leukemia cells.

Other drug­mak­ers in­clud­ing No­var­tis, Juno Ther­a­peu­tics and Kite Pharma have tested ge­net­i­cally mod­i­fied T-cells ex­tracted from an in­di­vid­ual pa­tient. How­ever, this is the first time cells from a healthy donor have been used in a process could lead to a ready off-the-shelf sup­ply for use in mul­ti­ple pa­tients. Some sci­en­tists have ques­tioned Cel­lec­tis’ ap­proach be­cause of po­ten­tial prob­lems with pa­tients re­ject­ing for­eign cells.

But the French biotech, work­ing with the US gi­ant Pfizer, as well as No­var­tis be­lieves its method is faster and cheaper than cre­at­ing sin­gle pa­tient-spe­cific gene ther­a­pies. Re­sults from Layla’s case were due to be pre­sented at the Amer­i­can So­ci­ety of Hema­tol­ogy’s an­nual meet­ing in Or­lando on Wed­nes­day. “This is a land­mark in the use of new gene en­gi­neer­ing tech­nol­ogy and the ef­fects for this child have been stag­ger­ing,” said Waseem Qasim, a pro­fes­sor of Cell and Gene Ther­apy at UCL and im­mu­nol­o­gist at GOSH who worked on her med­i­cal team.

If the suc­cess in this case is sus­tained and repli­cated in other pa­tients, he said, the ther­apy “could rep­re­sent a huge step for­ward in treat­ing leukemia and other can­cers.” Matt Kaiser, head of re­search at the leukemia and lym­phoma char­ity Blood­wise, said that while the con­cept of edit­ing im­mune cells to rec­og­nize and hunt out leukemia cells is “very ex­cit­ing,” pa­tients and their fam­i­lies should note that the tech­nique is still in the very early stages of de­vel­op­ment. “We need to es­tab­lish whether it can of­fer a long-term cure, whether there are any side ef­fects and which pa­tients are most likely to ben­e­fit from it,” he said. — AP

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