While each rare cancer individually affects a very small number of patients, rare cancers collectively account for almost 1 in 4 of cancer cases according to EU data.

And yet despite that, research into rare cancers remains very much a minority pursuit, and we face a series of challenges in improving treatment for these patients. In my field of sarcoma research, we have used the same first-line chemotherapy for patients where the disease has spread for the past 40 years!

There are more than 70 subtypes of sarcoma—many of which are not well understood at all. Even with advances in technology and leaps forward in cancer treatment generally, developing treatments for such a diverse group of cancers remains challenging.

Despite these challenges, there are already signs of more targeted future therapies for rare cancers.

Advances in treatment

At the Institute of Cancer Research, London, we have led a number of studies into sarcomas, including research into a novel combination treatment for an otherwise incurable cancer in children.

More recently, ICR researchers have discovered a novel mechanism of targeting a rare, but aggressive, sarcoma affecting predominantly young people. This research will hopefully be translated into the clinic—improving outcomes for these patients.

As part of the International Sarcoma Kindred Study (ISKS), a project led by researchers in Australia, the ICR have also helped understand genetic mutations that increase the risk of developing sarcomas.

Studies like this collect genetic data from a diverse range of patients, which is helping us begin to understand the possible link between inherited mutations and sarcoma risk—helping us to treat these cancers in a more targeted way.

Selecting patients by their tumor genetics

A more routine and integrated approach to genomics in research and health care will be key to improving outcomes for patients with rare cancers.

By placing patients in different groups, or sub-types, based on the mutations in their cancers, we can select the targeted treatments that are most likely to work for each person.

Increasingly, we are moving toward treating patients according to the particular set of genetic mutations in their cancer rather than simply on the location of their tumor. But, moving fully to such a precision medicine approach will require significant changes in how we diagnose patients and monitor them during treatment.