Identifying new drugs for the treatment of retinoblastoma

Retinoblastoma is the most common childhood cancer of the eye. Although treatable, it can lead to blindness and with new cancers elsewhere in the body. Basic treatments involve eye removal, cryotherapy (use of extreme cold to freeze and remove abnormal tissue), laser therapy, chemotherapy or radiotherapy. These result in various negative side effects such as hair loss, weight loss, kidney damage and hearing impairment. This all shows that there is a clear need for new treatments with fewer side effects.

If the disease has spread from the eye to other parts of the body, such as the brain, even intensive treatment will often fail to cure a patient. To improve retinoblastoma survival rates globally, it is essential that new treatment options are developed that are accessible worldwide, that are cheap and easy to store and transport.

Professor Majlinda Lako and her team at Newcastle University have been studying the molecules inside retinoblastoma cells to find out what genes are active. They found what triggers lead to healthy cells becoming cancerous and identified which drugs could target these triggers. The research team have also created a laboratory model of retinoblastoma, which acts just like real retinoblastoma cancer at the start of tumour formation.

Now, the researchers are ready to test the new drugs they found. They will use the retinoblastoma model to test how well the drugs can kill cancer cells. Whichever drugs are the most successful will then be tested to see whether they can reach the retina of the eye through the bloodstream, and how long they work for once there. Professor Majlinda Lako has gathered an expert team with expertise in stem cell biology and retinal disease, treatment of retinoblastoma, drug discovery and pharmacokinetics, which she hopes will put this project in a unique position to revolutionise retinoblastoma treatment.