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Relapsed Neuroblastoma: UK starting a phase II clinical trail with new radiotherapy treatment
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Submitted By: Information, News and Press Releases
Source: Neuroblastoma Alliance UK (November 7, 2011)
UK clinical trial offers new hope for sufferers of childhood cancer neuroblastoma
A clinical trial is due to start in the UK next year that will offer hope to children who suffer from relapsed neuroblastoma. The UK is the first country to trial the new molecular radiotherapy treatment for treating high-risk neuroblastoma, although it is already used to treat rare adult neuro-endocrine cancers.
Neuroblastoma is the most common 'solid' cancer diagnosed in babies and children. It accounts for around 15 percent of cancer deaths in children and around 100 children are diagnosed every year in the UK. Of these 100, approximately half will have high risk disease and the long term survival rate for these patients is less than 40 percent.
A pilot study that was given funding by the Neuroblastoma Children’s Cancer Alliance, a charity that helps children and families affected by neuroblastoma, has investigated a new radiotherapy treatment that targets a particular receptor on neuroblastoma cells.
Following the success of the pilot study, a three-year Phase II clinical trial is expected to start in the UK in early 2012. During the trial, the new radiotherapy treatment will be offered to all UK patients who meet the eligibility criteria - primarily patients who have relapsed neuroblastoma. Although it is a UK-wide trial, the treatment itself will take place at the Royal Marsden and University College Hospital (UCH) in London.
The pilot was carried out by a team of researchers, including Dr Mark Gaze, Consultant Clinical Oncologist at UCH and Great Ormond Street and Dr Jenny Gains, Clinical Research Fellow at UCH . Six children were offered the new radiotherapy treatment, of which five were found to have stable or improved disease following treatment.
Currently the main radiotherapy treatment offered to neuroblastoma patients is a compound called 131I-mIBG. However, this treatment has various limitations as some neuroblastoma cells do not have the receptors which this compound attaches to, so will not be killed by this treatment. Also 131I-mIBG can have serious side effects for neuroblastoma patients as it is toxic to bone marrow, which is already depleted in neuroblastoma patients.
The new treatment uses a compound called 177Lutetium -DOTATATE, or LuDO for short, which attaches to a different receptor that many neuroblastoma cells have on their surface. It is hoped that LuDO will be as effective in killing neuroblastoma cells as 131I-mIBG, but have fewer and less severe side effects. It is possible that it will be offered in combination with other radiotherapy drugs in the future.
"This pilot study, offers new hope for the families of children affected by the disease," said Dr Gaze. "If the trial proves that the new drug is safe and effective, it may become part of the standard treatment for neuroblastoma patients receiving radiotherapy treatment. It is likely to be offered with other drugs in the future - combining drugs is likely to be more effective as more cells will be killed."
For more information, contact Alison Moy, Chief Executive of the Neuroblastoma Alliance UK on 020 8203 0100, 07580 964 709 or alison@nballiance.org.uk, or Ingrid Marson on 01707 328 511 or ingrid@acornpr.org.uk
The clinical trial has been approved by National Cancer Research Institute, Clinical and Translational Radiotherapy Research Working Group (CTRad) and Clinical Trials Advisory Committee (CTAC), although it is still awaiting approval from the Ethics Committee, Medicines and Healthcare products Regulatory Agency and Administration of Radioactive Substances Advisory Committee (ARSAC).
More information on the pilot study are available in: J. E. Gains, J. B. Bomanji, N. L. Fersht, T. Sullivan, D. D'Souza, K. P. Sullivan, M. Aldridge, W. Waddington, M. N. Gaze. 177Lu-DOTATATE Molecular Radiotherapy for Childhood Neuroblastoma. Journal of Nuclear Medicine, 2011; DOI: 10.2967/jnumed.110.085100
The Neuroblastoma Alliance UK works to help children and families affected by neuroblastoma through providing financial assistance for treatment and to fund leading clinical research in recognised cancer centres.
Neuroblastoma is a type of childhood cancer. In the UK around a hundred children are diagnosed every year. The condition arises when a tumour develops from particular nerve cells which run in two chains from the skull down the back of a child’s chest and abdomen on each side.
Children with advanced neuroblastoma can receive chemotherapy, surgery, radiation therapy, stem cell transplant, and immunotherapy. Specialists in America and Europe are working together, sharing information and trialling different treatments in different countries. New treatments that utilise special antibodies are being trialled in Europe—including the UK—and the USA. The criteria for participation in these trials vary to some extent, which is why some children have to travel to America or Germany. The Neuroblastoma Alliance UK funds their treatment abroad which is why we need your support.
The Neuroblastoma Children's Cancer Alliance is a registered charity (no. 1135601), based at 3-4 Sentinel Square, Brent Street, London, NW4 2EL.
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