What is targeted therapy?

Posted in: Blog | February 12, 2015

What is targeted therapy and how can it benefit children suffering from cancer?


A recent episode of the BBC TV program Panorama has focused on the benefits of so called “targeted therapy” or  “precision medicines” for the treatment of diseases such as cancer. So what is targeted therapy and how can it benefit children suffering from the disease?


Traditional treatments for cancer, such as chemotherapy drugs, work non-specifically against any dividing cells whether they are normal or cancerous. This results in the typical side effects associated with patients being treated with chemotherapy. These side effects are particularly important in the treatment of children’s cancer, with late effects manifesting themselves months or many years after treatment has successfully concluded. They can affect future development of the child and their quality of life into adulthood and beyond. The main side effects seen in children, brought on by non-targeted therapies such as chemotherapy are problems with puberty and fertility, growth and development, heart and lungs, kidney problems, intellectual capacity/education and secondary cancers. Targeted cancer therapy is an approach that deliberately sets out to minimise these side effects by only killing the cancer cells within the patient and not their normal cells.


Research suggest that one in 1000 adults in wealthy nations are survivors of childhood cancer, and around 50% of these survivors experience the adverse effects of their treatment throughout the rest of their life. New therapeutic “smart drugs” that only target cancer cells would certainly minimise these short and long term side effects.


Another reason for pursuing targeted therapies, apart from minimising the side effects to the patients, is that they will provide the means to make so called “personalised cancer medicine” a reality. Personalised medicine uses information about the patient’s genes and proteins to prevent, diagnose and treat the cancer.  The technology to do this is now commonly available and has produced a wealth of knowledge, such as, identification of new cancer genes and identification of changes in children’s DNA linked to their cancer.  So personalised medicine is a reality, the problem is that there are not enough licensed drugs available that can be used to take advantage of all the personalised patient data.  This is why it is so important to translate this knowledge on an individual’s cancer into new-targeted therapeutic smart drugs and this is the proposed focus of the “Kidscan centre of excellence in targeted therapy”.


A further issue for children suffering from cancer is that almost all of the new targeted cancer drugs are aimed at adult cancers, this needs to be addressed if children are to benefit from advances in personalised therapy and targeted therapeutics. Kidscan is fundraising for the establishment of a targeted cancer therapy centre, which would enable us to contribute to the growing number of smart drugs available for the treatment of cancer. More importantly it would allow us to focus on developing smart drugs against targets specifically seen in children’s cancer.


Giving children the best access to the full range of currently available treatment options and the delivery of new smart drugs specifically aimed at targets in children’s cancer must be a priority for the UK and the wider world.


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