Nov
5 A baby whom doctors thought almost certain to die has been cleared of a
previously incurable leukaemia 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-millilitre 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
leukaemia 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
gene-edited 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 leukaemia.
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 leukaemia
drug that would usually kill them and secondly they are reprogrammed to only
target and fight against leukaemia 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 U.S. 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
leukaemia and other cancers".
Matt
Kaiser, head of research at the leukaemia and lymphoma charity Bloodwise, said
that while the concept of editing immune cells to recognise and hunt out
leukaemia 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. (Additional reporting by Ben Hirschler Editing by Mark Heinrich)
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