Second baby gets Cellectis "designer" cells to clear leukaemia

LONDON (Reuters) - A second baby with aggressive leukaemia has been treated in London with “designer immune cells” developed by Cellectis and, six months after treatment, remains in remission, the French biotech firm said.

Cellectis shares jumped 14 percent following Friday’s news.

The first human use of the company’s cell therapy made headlines worldwide in November when one-year-old Layla was cleared of previously incurable leukaemia at Britain’s Great Ormond Street Hospital (GOSH).

Details of the latest compassionate care case, also treated at GOSH, were presented at the American Society of Gene & Cell Therapy annual meeting in Washington.

The so-called UCART19 injection works by adding new genes to healthy donated immune cells known as T-cells, which arm them against leukaemia.

More time is needed to see whether the therapy has cured the disease, or simply slowed its progression. But the fact that Layla is still doing well 11 months after her injection and the second case has so far been successful is encouraging.

“This is not yet statistical proof but we do now have two cases,” said Cellectis Chief Executive Andre Choulika. “It shows that the therapy has some potential.”

The second baby was diagnosed with leukaemia at four weeks old and she was treated with UCART19 in December aged 16 months, after failing to respond to other treatments.

Other drugmakers including Novartis, Juno Therapeutics and Kite Pharma have tested genetically modified T-cells extracted from an individual patient.

The Cellectis approach, however, uses cells from a healthy donor in a process that could lead to a ready “off-the-shelf” supply for use in multiple patients.

The biotech company, working with U.S. giant Pfizer and French drugmaker Servier, believes its method is faster and cheaper than creating single patient-specific gene therapies.

Clinical trials of UCART19 are due to start this year.

Cellectis has been viewed in the past as a potential takeover target for Pfizer or another large pharmaceutical company, but Choulika said it was not his aim to sell up.

“My game plan is not to be acquired. The company is very well financed and we can go with a very aggressive business plan up to the end of 2018,” he told Reuters.

Editing by Susan Thomas and Alexander Smith