New UK clinical trial for relapsed neuroblastoma planned

LONDON — November 7, 2011 — A new clinical trial testing ¹⁷⁷Lutetium-DOTATATE (LuDO) for relapsed neuroblastoma is due to start in the UK next year. The UK is the first country to plan a clinical trial for this new molecular radiotherapy treatment for treating relapsed 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. Dr Jenny Gains presented the promising results of the of this pilot study at the Neuroblastoma Children’s Cancer Alliance parents’ meeting July 23, 2011 in London.

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 targeted 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.”

Notes:

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).

The pilot was funded by the JACK appeal of the Neuroblastoma Alliance. The JACK appeal was set up by Yvonne and Richard Brown to raise money for their son Jack’s neuroblastoma treatment. Jack sadly died in May 2009.

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.

The Neuroblastoma Children’s Cancer Alliance is a registered charity (no. 1135601), based at 3-4 Sentinel Square, Brent Street, London, NW4 2EL.