‘Chink’ Found in Armour of Aggressive Childhood Cancer


 Grace Kelly was diagnosed with malignant rhabdoid tumours two years ago and ‘went from being a happy, healthy schoolgirl to passing away within three weeks’, her mother said. Photograph: Grace Kelly Ladybird Trust

Grace Kelly – Photograph: Grace Kelly Ladybird Trust

Scientists believe they may have found a way to treat malignant rhabdoid tumours, a rare but devastating cancer that can kill young children within mere months of their diagnosis.

There is currently no treatment for these malignant rhabdoid tumours, which usually develop in the kidneys and show no symptoms in the early stages. By the time these tumours are diagnosed, the cancer has often spread to other organs and is fatal.

The mutated gene that causes the tumours is well-known, but scientists have not been yet to find a way to target it. Most of those affected are infants and toddlers.

This cancer is very rare (one case in 2 million children), but the work the ICR is doing will pay dividends in other cancers too. About 20% of all cancers have genes from this class.

Scientists at the Institute for Cancer Research (ICR) believe they have a made a breakthrough. They have now identified two other genes that appear to be implicated in driving the growth of the tumour, which produce proteins that can be inhibited by drugs that are already in use in other cancers or are in the development pipeline.

Prof Paul Huang, team leader of the protein networks laboratory at the ICR, says they have found “a chink in the armour” of these very aggressive tumours, using drugs that attack proteins known as kinase inhibitors.

There are a lot of kinase drugs out there, so we don’t have to re-invent the wheel,” he said. “In many cases in drug discovery you find a new target and spend 10 to 20 years developing drugs against it.”

In this case, they hope that will not be necessary. “We have been able to re-purpose existing drugs, some of which are already approved.”

So far, the drugs have been tested against cancer cells in the laboratory. The scientists, who have published their results in the journal Cell Reportsfound that inhibiting proteins produced by one of the genes had little effect, but attacking both of them killed the cancer cells.

The drugs pazopanib, dasatinib and sunitinib were all successful at inhibiting the protein from the PDGFRA gene – but if high levels of protein from the other gene, FGFR1, remained, the cancer returned and would not respond to treatment even at higher doses. This may explain why, when doctors have tried chemotherapy with their young patients, even where it has worked at first, resistance has quickly set in.

An existing drug called ponatinib, currently in use as treatment for leukaemia, can target both proteins at once. There are questions however, regarding the safety of the drug in children, so there will be a need to screen other drugs to find one with fewer side effects that works.

Prof Paul Workman, chief executive of the ICR in London, said: “We need to see much better, more targeted treatments for children with cancer,  that further drive up survival rates and spare children the serious long-term side-effects that conventional chemotherapy can cause.

This study provides an exciting pointer for how we might treat more effectively an aggressive childhood cancer for which there is currently no cure. It’s crucial, when as here there is a strong scientific rationale for a new treatment approach in children, that we can assess them as rapidly as possible in clinical trials. At the moment, it’s much too easy for pharmaceutical companies to avoid evaluating their drugs in children, even when there is good evidence that they could benefit.”

Jennifer Kelly’s four year-old daughter Grace was diagnosed with malignant rhabdoid tumours two years ago and ‘went from being a happy, healthy schoolgirl to passing away within three weeks’, her mother said. Her mother set up the Grace Kelly Ladybird Trust to fundraise research into childhood cancers.

Grace went from being a happy, healthy schoolgirl to passing away within three weeks,” said her mother. “She started school in September and didn’t make it to half-term. She was an athlete – she was the monkey hanging upside down from the monkey bars. Even when she was admitted to hospital initially she seemed quite well. It progressed very rapidly.

It is basically a diagnosis of death, whether they try to hold it at bay with chemotherapy for a while or not. They simply can’t treat them. There is a lack of funding for childhood cancers as a whole. People don’t realise childhood cancer is the biggest medical cause of death in children in the UK. People think it is rare and it actually isn’t. It is that that shocked us more than anything else.”



About LFCT

This is a blog about CHILDHOOD CANCER and CHILDHOOD CANCER AWARENESS Little Fighters Cancer Trust is a non-profit organisation that offers support and aid to Children with Cancer and their families. When a child is diagnosed with cancer it affects the whole family. One of the parents, usually the mother, must give up their job to care for the child and this creates financial problems for the family. In South Africa especially the majority of these families are not well-to-do; many of them are rural. A diagnosis of cancer can wipe out any family’s finances, let alone a poor family. The costs of special medications, special diets, hospital stays, transport to and from the hospital or clinic and accommodation and food costs for the mother who spends most of the time at her child’s bedside are astronomical. These are the people and problems that fall through the cracks, and these are the people that Little Fighters Cancer Trust has pledged to help in any way possible. LFCT takes a holistic approach to assisting the Children with Cancer and their Families, with the main aim to be the preservation of individual dignity and pride. Little Fighters Cancer Trust also focuses on promotion and advocacy of National Childhood Cancer Awareness in an effort to increase awareness of Early Warning Signs of Childhood Cancer. This would result in earlier diagnosis, giving the Child with Cancer more of a chance at Treatment and Survival. See "About" for more Background info

Posted on 2 November, 2016, in Blog, Research and tagged , , , , , , , , . Bookmark the permalink. Leave a comment.

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