Hundreds of people with a rare and aggressive blood cancer will
benefit from a potentially life-saving treatment from today
(Tuesday 25 November), following NICE's recommendation of a CAR-T
(chimeric antigen receptor T-cell) treatment developed by a
British spinout company.
NICE has recommended obecabtagene autoleucel (obe-cel or
Aucatzyl) for routine NHS use in England, offering new hope to
over 150 people over the next the three years with relapsed or
refractory B-cell acute lymphoblastic leukaemia (ALL), who have
limited treatment options.
Obe-cel, developed by Autolus, a University College London
spinout company, is a next generation CAR-T treatment,
personalised to the needs of each patient. It has the potential
to increase people's likelihood of going into remission and has
lower toxicity, meaning more people could benefit from it
compared to other treatments. It works by reprogramming a
patient's own immune cells to recognise and destroy cancer cells.
Evidence from a clinical trial has shown high rates of remission
in patients whose cancer has either returned (relapsed) after
treatment or failed to respond to initial therapy (refractory). A
study of nearly 100 people found that 77% of those who had at
least one obe-cel infusion went into remission.
Evidence suggests obe-cel improves how long people live compared
to other immunotherapies used at this stage of treatment. It also
suggests that obe-cel works as well as tisagenlecleucel, the CAR-T
therapy available for people aged 25 and under. Obe-cel is
recommended as a treatment only for those aged 26 and over.
NICE's independent committee applied greater weighting to the
benefits that obe-cel would offer over current treatments due to
the severity of the condition.
Helen Knight, director of medicines evaluation, at NICE,
said: “I am delighted that we have been able to
recommend this new treatment, which offers real hope to people
living with this rare and aggressive blood cancer.
“This drug has the potential to offer a more effective and less
toxic alternative to standard treatments, with fewer side
effects. This could potentially be a life-saving drug, which will
make a huge difference to people's lives, including spending less
time in hospital.”
This recommendation supports the government's Plan for Change
commitment to build an NHS fit for the future by boosting access
to innovative treatments that improve patient outcomes and
showcases the UK's world-leading life sciences
sector.
Clinical trials for the treatment were conducted at a number of
sites across the UK and part-funded by the National Institute for
Health and Care Research (NIHR), with support from UCLH
Biomedical Research Centre (BRC).
B-cell acute lymphoblastic leukaemia is a rare blood cancer,
affecting fewer than 5 in 10,000 people in the UK.
Dr Maria Koufali, Life Sciences Industry Director at NIHR,
said: "The UK's integrated research system is built to
help life science companies move faster from promising science to
robust evidence. By connecting Autolus with NHS sites, specialist
facilities, and experienced investigators, NIHR support is
helping bring innovative cell therapies closer to patients.”
Dr Claire Roddie, UCLH consultant haematologist and
associate professor at the UCL Cancer Institute said: “I
am delighted to hear of NICE's decision. Many more patients now
stand to benefit from this CAR-T cell therapy on the NHS and we
are still working to widen its application. "Working on proving
the safety and efficacy of this drug has brought together
clinical and research teams from UCL and UCLH, with support from
government and arms-length bodies like the NIHR and the BRC as
well as the pharmaceutical industry. The many people involved in
this work can feel immensely proud of this achievement which will
help save the lives of many more patients.”
Read the final draft guidance for obecabtagene autoleucel for
treating relapsed or refractory B-cell acute lymphoblastic
leukaemia.
ENDS
Notes to editors
- An embargoed copy of NICE's final draft guidance on
obecabtagene autoleucel for treating relapsed or refractory
B-cell acute lymphoblastic leukaemia can be downloaded here:
ID6347 Obecabtagene
Final Draft Guidance.pdf.
- The usual treatments recommended by NICE for this
form of cancer are blinatumomab, inotuzumab ozogamicin or
ponatinib, targeted immunotherapies. These can be followed by a
stem-cell transplant for some people.
-
Obecabtagene autoleucel was granted
a conditional marketing
authorisation by Medicines and Healthcare products
Regulatory Agency (MHRA) in April this year.
- Since 2015, the National Institute for Health and Care
Research (NIHR) has supported Autolus - and the development of
this treatment - with funding, specialist facilities, expert
investigators and trial delivery, enabling progress from early
studies through to trials and regulatory approvals. Support and
funding predominantly came from NIHR UCLH Biomedical Research
Centre (BRC), an NIHR i4i Challenge Award and the NIHR Research
Delivery Network (RDN).
