A seven-year-old girl has been 'cured' of leukaemia after
a new therapy in which her T-cells — the immune system's killer cells — were
bio-engineered to become 'guided missiles' to seek out and destroy the
leukaemia, a new study shows. She had relapsed following conventional treatment
for acute lymphoblastic leukaemia (ALL), which can sometimes avoid detection by
the T-cells.
Ken Campbell, Clinical Information Officer at Leukaemia
& Lymphoma Research, said the results of the study were encouraging for
both children and adults diagnosed with leukaemia. 'Treatments which modify the body’s own immune
system to fight leukaemia have shown much promise in recent years,' he said. 'What is significant about [the] therapy is
that the severe side-effects associated with this form of treatment seem to be
greatly reduced when combined with other drugs. 'This is a small study of just
12 patients. Larger clinical trials are needed to determine how effective this
treatment could be and as a result it should be some time before it is
available in the UK.'
Previous studies have developed modified T-cells as a
treatment for ALL, which is a B-cell leukaemia — a type of lymphoid leukaemia
which affects the B-cells of the immune system.
The new study looked at CTL019 therapy, which programmes
the T-cells to recognise and attack specific invading disease cells — in this
case, cancerous B-cells. This can help attack cancer cells which fly under the
radar of immune surveillance and evade detection by T-cells, according to
researchers from the Children's Hospital of Philadelphia and University of
Pennsylvania.
Results of the clinical trial, which will be presented at
the American Society of Hematology annual meeting in Atlanta, revealed nine of
the 12 patients with advanced leukaemia — including two kids — responded to
treatment.
One of the nine responding patients is the seven-year-old
girl with acute lymphoblastic leukemia (ALL) — the most common childhood
leukaemia and most common childhood cancer.
Although physicians can cure roughly 85 per cent of ALL
cases, the remaining 15 per cent of such cases stubbornly resist treatment.
The researchers found the activity that destroyed
leukaemia cells also stimulated a highly activated immune system, which made
the child very ill.
To counteract the toxic side effects, they used two
immunomodulating drugs that blunted the over-active immune response and rapidly
relieved the child's treatment-related symptoms.
The immunomodulating drugs did not interfere with the
CTL019 therapy's anti-leukaemia benefits, which have persisted six months after
the infusion of cell therapy.
This persistence is essential, because the engineered
T-cells remain in the patient's body to protect against a recurrence of the
cancer.
These results were so effective that the approach is now
being successfully incorporated into CTL019 treatments for adults as well.
Paediatric oncologist Dr Stephan Grupp of The Children's
Hospital of Philadelphia said: 'These engineered T-cells have proven to be
active in B-cell leukaemia in adults. 'We
are excited to see that the CTL019 approach may be effective in untreatable
cases of paediatric ALL as well. 'Our
hope is that these results will lead to widely available treatments for
high-risk B-cell leukaemia and lymphoma, and perhaps other cancers in the
future.'
Dr Susan Rheingold said: 'This type of pioneering
research addresses the importance of timing when considering experimental
therapies for relapsed patients. 'To
ensure newly relapsed patients with refractory leukaemia meet criteria for
options like CTL019, we must begin exploring these innovative approaches
earlier than ever before. 'Having the
conversation with families earlier provides them more treatment options to
offer the best possible outcome.'
SOURCE: DAILYMAIL UK
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