LONDON, Nov 5 (Reuters) – 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)
