High risk Neuroblastoma is one of the hardest cancers to treat in
children. An 18 country Europe wide
study has demonstrated the benefit of the new European high dose chemotherapy
The trial was funded by Cancer Research UK, the Children’s Cancer Research Institute in Vienna, and also by the European
Commission Framework Five. The research was led by Great
Hospital, The Royal Marsden and The
Institute of Cancer Research in the UK, and international
As reported to this month’s American Society of Clinical Oncology conference
(ASCO), the European protocol offered 16 per cent better disease free survival
at three years compared with a treatment based on the current American
protocol. It has also proved to have fewer toxic side effects. From
the end of last year, when these figures were validated, the randomised
controlled trial was halted on ethical grounds so all European patients could
receive the better treatment.
Figures reported were 49 per cent v 33
per cent Event Free
Survival and 61 per cent v 48 per cent overall survival at three years.
Peppy Brock, UK
lead for the trial, consultant paediatric oncologist at Great Ormond
and President of SIOPEN (European Neuroblastoma Group of the International Society
for Paediatric Oncology) said:
“It has taken since 2002 to prove the advantages of the European protocol.
It could never have been achieved without pan European co operation, since no
one country has large enough numbers to run an effective trial in a realistic
time frame. The high dose regime was pioneered in France, the study
chair is Austrian, and the UK
enters the largest number of patients onto this trial.
“This trial predates the introduction of immunotherapy which this group of
children are also now benefitting from. We are now beginning to see real
improvements in survival, even in this very high risk group of children, right
across the UK and Europe. Funding is now needed to ensure that we can
develop this work and continue to make progress against this particularly
difficult cancer in children.”
Senior trial investigator Professor Andrew Pearson, from The Institute
of Cancer Research and The Royal Marsden, added:
"Neuroblastoma is one of the
most common types of childhood cancer in the UK and high risk Neuroblastoma is
one the major causes of death in children from malignancy.
"In this study, we found that the combination of chemotherapy drugs given
for consolidation therapy used in Europe led to children living longer on
average than a regimen based on the US standard.
“This very conclusive result will
alter the way that doctors treat their patients world wide, and will mean fewer
children die from this disease each year.
“These chemotherapy drugs - busulphan
and melphalan - were actually discovered and developed at The Institute of
Cancer Research in the 1950s, so it is especially pleasing that drugs
discovered here in the UK so many decades ago may now make a difference to the
lives of children all over the world."
Julie Hearn, head of
clinical trials at Cancer Research UK, said:
“The results of this
study offer new hope for children diagnosed with high-risk Neuroblastoma. It
shows how drugs Cancer Research UK were at the forefront of developing 30 or 40
years ago have stood the test of time and are still proving useful today. But
while we are making progress in treating childhood cancers, today almost 80 per
cent survive, there is an urgent need to develop new treatments for hard to
born in March 2008, a normal, healthy baby.
After two months at home, her mum Karen noticed she was ‘chesty’ and
having trouble breathing. Doctors diagnosed bronchitis.
noticed Kacie rolled her eyes frequently, and in December 2008 Kacie was
diagnosed with infantile spasms (when a small part of the brain doesn’t
developing well, and having around 18 months of good health, in September 2010
Karen noticed a rapid decline in Kacie. She wouldn’t eat or drink, didn’t want to walk
and screamed in pain whenever she was lifted up.
tests and scans at her local hospital, which revealed a tumour the size of a
melon in Kacie’s abdomen and pelvis, she was finally diagnosed with
Neuroblastoma on 20 December 2010 at Great
never even considered Kacie might have cancer, and I had never heard of
Neuroblastoma. Once we had the diagnosis everything started to happen so
Neuroblastoma had spread to Kacie’s bones through her bone marrow, reaching her
thighs, fingers and skull.
December 23, Kacie started her first round of chemotherapy. My two other children, 11 year old Jake and
four year old Alfie, came and spent Christmas Day in hospital so we could be
together as a family.
Kacie had eight rounds of chemotherapy, spending three days out of ten in
hospital over a 70 day period. That
finished in February 2011, and we had some time at home before Kacie came back
to hospital for a stem cell harvest.
followed by surgery to attempt to remove the tumour from her abdomen and
pelvis. However, the Neuroblastoma had
wrapped itself around some of Kacie’s major blood vessels and the surgeon
decided going ahead would be too risky.
started on this high dose form of chemotherapy. She is currently doing really
well. All the Neuroblastoma from her bone marrow has been killed off, the aim
is to make sure this also happens to the tumour in her pelvis and abdomen; and
that this shrinks and becomes benign. Kacie will also need radiotherapy once
she finishes these rounds of high dose chemotherapy, and will then be put on to
an antibody trial.
finish treatment in March 2012. We know we are in the best hands at Great Ormond
and that the doctors and nurses are doing everything they can for Kacie.“
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