Key findings made by researchers funded by The Little Princess Trust

A team of researchers from Newcastle University has been able to identify two drugs that could form the basis of more effective and less damaging treatments for retinoblastoma eye cancer.

Funded by The Little Princess Trust, Professor Majlinda Lako’s team tested over 30 drugs in the lab. They worked on specially created ‘mini eyes’ that could mimic how retinoblastoma grows and behaves in patients’ eyes.

Prof Lako said: “Testing whether a drug kills cancer cells is only half the battle, because the eye is protected by the blood-retinal barrier. This barrier is designed to keep harmful substances out of the delicate retinal tissue – but it also blocks many life-saving medicines.

“If a drug cannot cross this barrier, it will never reach the tumour without risky, invasive injections directly into the eye.”

Current treatments can be very tough for children, sometimes affecting their vision or requiring major surgery – in addition to the usual complications of chemotherapy.

In Prof Lako’s recent paper, published in Molecular Therapy: Oncology, her team identified two medicines that can cross the blood-retinal barrier and effectively fight retinoblastoma cells. Because the drugs are able to kill the cells that cause retinoblastoma, they could form part of new and effective targeted treatments.

Prof Lako added: “The research also focuses on how drugs cross the eye's natural barriers, paving the way for life-saving treatments that can be taken intravenously or as a simple pill – potentially saving the sight and lives of children worldwide without the need for invasive surgery or harsh side effects.”

Retinoblastoma can be tricky to treat because there are a number of different genetic mutations in the cancer cells. This changes how the cancer behaves, so can mean that different children need different treatments.

In order to fully develop the two candidate treatments, the researchers need to ensure that they work against all of the different genetic variations of retinoblastoma.

While this will take time, the team have already jump-started the development process by using existing medicines that are known to be safe for humans.

If they can prove that the treatments are more effective than other options, these medicines could be available for children with retinoblastoma more quickly.

Young patients deserve treatments that give them the best chance of curing their cancer.

Phil Brace, CEO of the LPT, said: “It is exciting to hear that Prof Lako has found safe, promising medicines that could help children with retinoblastoma.

"These young patients deserve treatments that give them the best chance of curing their cancer, without taking their sight away.

"We’re proud that our funding has played a role in advancing such important research.”