Precision Medicine – childhood cancer treatment

Precision medicine is an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle.

This includes a personalised approach to cancer treatment which is tailored to the patient’s specific type of cancer. Scientists use state of the art genetic sequencing to identify key genetic and proteomic changes in a patient’s tumour.

These individual variations in genes allow doctors to allocate young patients to very focused tumour categories and prescribe treatments to their cancer’s individual makeup. As well as being more effective, highly targeted treatments are less likely to have toxic side effects.

Precision Medicine has two main goals:

• To improve the cure rate
• To reduce toxicity and so reduce the long-term effects of treatment

Children with Cancer UK are helping to fund the development of a national infrastructure for rapid sequencing of tumour DNA from young cancer patients. This will include:

• A structure for the genetic analysis of patient tissue which will allow for highly accurate diagnosis and treatment recommendations
• The development of a tissue bank, which will build a large source of patient samples – this is extremely valuable in childhood cancers because of their rarity
• Establishing a hub where cancer cells are extracted from samples and analysed using a next-generation sequencing tool which provides a highly-defined genetic picture of individual tumours
• A new bioinformatics team, which will interpret the genetic data to help decide on the best treatment and to aid discovery of new genetic signals and mutations that could be targeted
• A national database is being set up which will allow the bioinformatics team to track the many different tumour types and how they respond to treatment, which will be a powerful aid to future treatments and prognosis
• The development of a panel of oncology experts (known as the national molecular tumour board) who will discuss the findings of the genetic analysis and bioinformatics teams, before making informed recommendations for patient treatment. This will include assigning patients to clinical trials for new targeted drugs and therapies.

Our Precision Medicine project will actively offer Precision Medicine to young patients, who will benefit from rigorous diagnosis and highly targeted treatments.

The impact of the project will go much further than this:

• We’ll improve our genetic understanding of childhood cancers, identifying new targets for treatment where available
• The project will lead to more clinical trials for promising new treatments
• The initiative will increase the amount of tissue that is banked, allowing research to access precious tumour samples.

Watch our Little Translators Grace and Louis explain Professor Chesler’s Precision Medicine research: