Cooking up a cure for extremely rare types of children’s cancer

We have all been there, we go to visit the doctors, they listen to your symptoms, conduct an examination and make a diagnosis but how do they make that decision?

The doctor will have seen similar symptoms time and time again and will return to their own pattern of drug choice/decision making to prescribe for the patient. Many common illnesses have prescribing guidelines based on good clinical evidence, and its usually easy to match this to the symptoms of the patient as they present themselves. Some patient situations occur very frequently and although there may be no specific guidelines, there are treatments learned by doctors during their career that they know will give good results. If a specific patient does not correspond to the first two scenarios then prescribing becomes an art based on the clinicians knowledge of existing and new treatments .

In essence the clinicians are creating their own recipe book based on the evidence, clinical guidelines, information on new available treatments and their own experiences to cook up a cure for the patient.

But what happens when clinicians are presented with a less common disease such as a childhood cancer? This is a difficult situation for GP’s who might see only a handful of childhood cancer patient during their career, however if they do suspect cancer then they don’t have to concern themselves about what treatment to prescribe they simply refer them on. Thankfully clinicians exist who specialise in childhood cancer that have the background knowledge and experience to make the best treatment choice for the commonest cancers.

Childhood cancers are classed as rare or “orphan” diseases, affecting just 1 in 500 children before the age of 14. The knowledge built up over many years in the medical profession means that there is a comprehensive recipe book of treatments for the more common types of children’s cancer and some availability to become involved in new clinical trials. However, within each type of childhood cancer, extremely rare cancers can be found that even clinicians specialising in children may see very infrequently.

Because of the lack of developments in childhood cancer treatments, there are often only a few limited treatment options available to clinicians, when they encounter a very rare version of a cancer. The course of treatment can be hard to define, as the recipe book of treatments (clinical guidelines) contains little information for these patients.

With more typical children’s cancers, clinicians have a choice, they can use the treatments currently available from the recipe book for that type of cancer or they enrol the patient onto a clinical trial aimed at developing ground breaking drugs, or treatments. Unfortunately, for patients with the extremely rare cancers, neither of these options is usually available, leaving the child and their family with few choices of treatment.

A good example of this is Atypical Teratoid Rhabdoid Tumour (ATRT), a very rare childhood cancer with only 3 from 1,000,000 children diagnosed each year. This disease is so rare that not enough evidence exists for the development of accurate clinical guidelines. So the choices in the clinicians recipe book is very limited and this is reflected in the poor survival rate for ATRT patients, 2-year survival rates are less than 20%.

The clinical evidence gathered over the last 60-years has been used effectively to produce recipe books for a ranges of childhood cancers and has helped to increase overall survival rates to greater than 90%. However, much more needs to be done for those patients with very rare childhood cancer. ATRT is very rare with no treatments delivering long-term survival patients and no guidelines or recipe books to help clinicians make decisions exist. Clearly more research and clinical trials are needed for all childhood cancer but even more so for the extremely rare types where cooking up a cure is all but impossible.