May 19, 2014

New hope for sufferers of mystery lung disease

Breakthrough medication proven to successfully slow down the progression of a mystery lung disease affecting around 5,000 Australians could be available as early as next year, according to Australian experts involved in landmark research released today.

Idiopathic Pulmonary Fibrosis (IPF) is a rapidly progressive and fatal lung disease that usually occurs in adults over the age of 50.1,2

The condition results in irreversible scarring of tissue in the lungs, which makes it increasingly difficult for oxygen to transfer into the bloodstream.3

The cause of the disease is still unknown4 and people with IPF often remain undiagnosed as symptoms are similar to severe asthma, chronic obstructive pulmonary disease, congestive heart failure and other treatable forms of lung fibrosis. Approximately half of all patients die within two years of diagnosis5, and until now there has been no treatment approved for this ‘mystery lung disease’.

Lung Foundation Australia has welcomed the findings of two major studies released at the American Thoracic Society 2014 Congress in San Diego, which show that IPF can be successfully treated with new medicines that slow disease progression by ‘blocking the signalling pathways’ that cause the scarring of lung tissue.6

The INPULSIS trial, which involved 1,066 patients worldwide, including 33 Australians, found that patients who received nintedanib for one year experienced a 50 per cent reduction in lung function decline and an increase in their overall health compared to those treated with traditional therapies.7

Similarly, the ASCEND study demonstrated that pirfenidone significantly reduced the proportion of IPF patients with disease progression or death over a 52 week period compared to placebo.

More than 90 per cent of patients who participated in the INPULSIS and ASCEND studies have opted to continue with the new therapies as part of on-going trials.

Dr Tamera Corte, Respiratory Physician at the Royal Prince Alfred Hospital, who was one of the Australian researchers involved in the INPULSIS study said, “IPF is an unpredictable, debilitating and ultimately fatal disease but the results of this study mark a major turning point for Australians living with the illness”.

“Without effective treatment, people with IPF find that the scarring of lung tissue impedes the delivery of sufficient oxygen to the body. This places additional strain on the heart,” said Dr Tamera Corte.

Over the last few years there has been an improvement in both diagnosis and treatment, with doctors currently using a range of treatments for patients with IPF, including supplemental oxygen therapy, often supplied through a small portable tank, an exercise program known as pulmonary rehabilitation, and in some circumstances, lung transplant surgery.5

William Darbishire, Chief Executive Officer, Lung Foundation Australia said, “The findings from these studies pave the way for improved treatment options. Lung Foundation Australia looks forward to working with industry and government to make these treatments available in Australia as soon as possible”.

Lung Foundation Australia is calling for increased funding to support the prevention and early detection of IPF, as well as encouraging Australians with the disease to sign-up to the Lung Foundation’s national Registry of IPF patients.

“The Australian Registry of Patients diagnosed with IPF aims to enrol all Australians with the condition so that the data collected can help researchers learn more about this insidious disease,” said Mr Darbishire.
“With greater understanding, earlier diagnosis and new treatment options we can take the mystery out of this serious lung condition,” he added.

Click here to learn more about Lung Foundation’s national Registry of IPF patients.


The two studies were published in the New England Journal of Medicine.

Efficacy and Safety of Nintedanib in Idiopathic Pulmonary Fibrosis

A Phase 3 Trial of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis




1. Pulmonary Fibrosis Foundation. Prevalence. Available at: Accessed March 2014.
2. American Thoracic Society. Idiopathic Pulmonary Fibrosis: Diagnosis and Treatment. Am J Respir Crit Care Med. 2000; 161:646–664.
3. Collard H, et al. Acute Exacerbations of Idiopathic Pulmonary Fibrosis. Am J Respir Crit Care Med. 2007; 176(7):636–643.
4. NHLBI, NIH. What Is Idiopathic Pulmonary Fibrosis? Accessed at: Accessed March 2014
5. Raghu G, et al. An official ATS/ERS/JRS/ALAT statement: idiopathic pulmonary fibrosis: evidence-based guidelines for diagnosis and management. Am J Respir Crit Care Med. 2011;183(6):788–824.
6.Richeldi L, et al. Efficacy of a tyrosine kinase inhibitor in idiopathic pulmonary fibrosis. N Engl J Med. 2011;365(12):1079–87.
7. Richeldi L, et al. Efficacy and Safety of Nintedanib in Idiopathic Pulmonary Fibrosis N Engl J Med. 2014. In press.