Researchers at UCLH have begun to test whether a drug can stop the growth of tumours in chordoma, a rare bone cancer which affects the spine.
Chordomas, which affect roughly 1 in 1 million people each year and for which no drugs are currently available, usually begin at the base of the skull or the tailbone. They may be slow-growing but can lead to serious complications including severe pain, disability and paralysis. Tumours often reoccur, and can spread to other parts of the body, and patients often die prematurely.
Treatment involves surgery to remove the tumour followed by radiotherapy with proton beam therapy increasingly being used, but surgery can be difficult due to the tumour’s proximity to critical structures such as the brainstem and spinal cord. Chemotherapy is not effective in this disease.
Now, researchers at UCLH led by Dr Sandra Strauss will test whether the drug afatinib can shrink or halt the growth of tumours in patients whose disease has returned or spread to other parts of the body. This study is being conducted at the NIHR UCLH Clinical Research Facility which is fully dedicated to expedite translational research and the early evaluation of novel treatments in patients.
Afatinib works by inhibiting the EGFR protein which is believed to in involved in driving the growth of chordoma tumours. Research led by UCL’s Professor Adrienne Flanagan found that compounds which inhibit EGFR are effective against chordoma cell growth in the laboratory; this discovery being instrumental in providing the evidence to set up the clinical trial. The study is the result of an academic partnership developed between Leiden University, UCL, and The Istituto Tumori, Milan and is funded by the Chordoma foundation and Boehringer Ingenheim.
Study participants will take the drug orally on a daily basis in four-week cycles. Researchers will use scans to assess changes in tumour size during and after the treatment.
Dr Strauss said: “Treatment options are limited for the chordoma patients we see at the moment, and there is a huge unmet need for new drugs. Chordoma is a rare disease but can be devastating and significantly affect a person’s quality of life. We hope that a new treatment will emerge as a result of our research.”
Three European centres will participate in the study (UK, Netherlands, and Italy). UCLH will be the only site in the UK.
‘I have been eagerly awaiting this trial’ – a patient’s perspective
Peter Kenrick, from London, was diagnosed with a sacral chordoma – which starts from the base of the spine – in January 2014 after suffering from lower back pain for 9 months. He then had carbon ion radiation treatment in Italy in 2014, but his chordoma has been recurrent.
Mr Kenrick said: “I learnt through the Chordoma Foundation that the trial was in the pipeline 2 years ago. Since then I have been eagerly waiting for it to open, because with recurrent chordoma there are very few treatment options. I had also learnt that afatanib looked promising in laboratory tests, so it looked like the best option for me.”
He added: “The trial has been a very positive experience so far. I feel extremely well supported at the Clinical Research Facility, and can always speak to someone or drop in if any help or advice is needed. The staff at the facility are concerned and interested, and the general atmosphere is calm and optimistic, which is extremely helpful to patients with serious disease. It is also good to feel part of a bigger picture that will be helpful to other patients.”
