Patients with an incurable form of lung cancer could lead normal lives by taking a daily pill that “stops cancer in its tracks”, a breakthrough study has revealed.
Scientists have hailed the drug, called lorlatinib, for its “off the charts” results after a trial found it improved survival rates by the longest period ever recorded.
In the trial, lorlatinib stopped the cancer from spreading for at least five years in 60 per cent of patients, including just over half of the participants whose cancer had already spread to the brain before the trial commenced.
It has been so effective that at the time of publishing the results, the researchers have not been able to give an average number of months that patients were able to live without their cancer worsening because the majority are still living progression free.
Experts said it was “the longest progression-free survival ever reported” for a lung cancer treatment.
The drug significantly outperformed the 8 per cent who were progression free at five years in a control group taking an alternative treatment called crizotinib, with the cancer spreading after 9.1 months on average.
Dr David Spigel, chief scientific officer at Sarah Cannon Research Institute and American Society of Clinical Oncology (ASCO) member, said the industry had “not seen anything close to this”.
“The results with lorlatinib are the best we’ve ever seen,” he added.
“We just haven’t seen results like that in oncology that often, much less in non-small cell lung cancer. These are among the best results we’ve seen in advanced disease in any setting…a really major step forward in lung cancer care.”
‘Could prevent spread to brain’
The treatment is for a deadly sub-type of lung cancer that is more common in younger people and non-smokers, who have a specific mutation of the anaplastic lymphoma kinase (ALK) gene.
It is often diagnosed at a later stage when it is harder to treat and accounts for around five per cent of non-small cell lung cancers (NSCLC) – the most common form – affecting about 350 patients each year.
Around 49,000 people are diagnosed with lung cancer in the UK each year.
The drug works by binding to a protein on the tumour cells and stopping it from growing and spreading.
Professor Charles Swanton, chief clinician at Cancer Research UK, said the study “could present us with an effective way of stopping cancer in its tracks and preventing it from spreading to the brain”.
“Research like this is vital to find new ways to treat lung cancer and help more people survive for longer.”
It is available on the NHS on a limited basis for patients who have not had success with other treatments, but is only given to around 80 patients a year. The pill costs around £167 per day based on its list price.
It was rejected for wider use last year by spending watchdog, the National Institute for Health and Care Excellence (NICE), owing to a lack of evidence but could be reconsidered soon in light of the new data.
‘Unprecedented outcomes for patients’
Benjamin Solomon, lead author and head of lung medical oncology at research institute Peter MacCallum Cancer Centre, Melbourne, Australia, said: “The progression-free survival benefit observed with lorlatinib in this five-year analysis corresponds to the longest progression-free survival ever reported in ALK+ non small cell lung cancer, and indeed, to our knowledge, of any targeted therapy in lung cancer to date, and speaks to the progress made in this area.”
He said the findings, which are being presented at the ASCO conference today, “indicate that this provides an unprecedented improvement in outcomes for patients”.
The research team split 296 patients with the ALK mutation into groups and gave half of them lorlatinib and half crizotinib.
Lorlatinib was also more effective at halting the disease in the quarter of participants whose cancer had spread to the brain before the trial began, with 53 per cent living for five years without the disease worsening, compared to an inestimable amount in the control group.
All patients had brain scans on entry into study and every eight weeks. Lorlatinib prevented progression of existing cancer that had spread to the brain and prevented or delayed progression of new metastases.
Dr Julie Gralow, the chief medical officer at ASCO said: “You don’t need a magnifying glass to see the difference between these two drugs. It’s just a profound difference… 60 per cent five-year progression-free survival in non-small cell lung cancer is just unheard of.”
Debra Montague, chair of ALK Positive Lung Cancer UK, said: “Lung cancer often spreads to the brain and Lorlatinib is very successful at stopping this. The drug is not yet in use for first line treatment in England but hopefully, after these results, it will get approval.”