Highlights of the ATS congress 2017- By Marlies Wijsenbeek and Francesco Bonella

Marlies Wijsenbeek and Francesco Bonella, members of the EU-IPFF Scientific Advisory Board participated in the American Thoracic Society Conference (ATS) which took place in Washington from 19-24 May 2017. With around 17,000 attendees, the ATS conference is one of the largest conferences offering ground-breaking research in pulmonary, critical care, and sleep medicine. This year’s congress provided an excellent opportunity to discuss different aspects and up to date research in IPF treatment and care:

Factors influencing  IPF onset anddisease course

A study onIPF patients monitored at home revealed that exposure to air pollution could increase the risk of acute exacerbations and mortality (Johannson K.A.). Another study assessing the effect of occupational exposure to metal, wood and agricultural dust on IPF found that the occupational exposure was associated with IPF disease burden. Prevention measures in the workplace could potentially reduce thedisease burden by as much as 13% (Cummings K.J et al).

In addition, Toby Maher from Royal Brompton Hospital reported that the bacterial composition in IPF patients lungs affect the hosts’ immune response and can significantly influence progression-free survival. Also fungicould be involved in the worsening of IPF; however, these new findings require further investigation.

Use of supplemental oxygen during routine daily activities

Although oxygen is often provided for patients with fibrotic interstitial lung disease (fILDs), little research on its benefit exists. A group of UK researchers investigated the impact of supplemental oxygen on routine daily activities and quality of life for people with fILDs. Using patient reported outcome measures, the study showed a significant improvement in breathlessness and activity with the use of ambulatory oxygen. However, no improvement in the psychological domain was found.

Views on IPF management

 A study on patients’ and physicians’ views on IPF management (Mahler) found that only 56% of interviewed patients feel that they received enough information at diagnosis, while less than half discussed disease prognosis and were told about treatment options. Most patients felt that the ability of anti-fibrotic drugs to slow disease progression was more important than side effects. Among the patients who had received antifibrotic therapy, 86% felt confident in managing those side effects.

Meanwhile, physicians with more of a watch-and-wait attitude (initiation of anti-fibrotic treatment more than 4 months after diagnosis) were less likely to discuss prognosis and were more concerned about side effects. On the other side, proactive physicians (initiation of anti-fibrotic treatment less than 4 months after diagnosis) were more concerned about disease progression and more likely to discuss prognosis.

Role of the multidisciplinary team in diagnosis and treatment

When it came to diagnostic tools for IPF and ILDs, the focus was on the role of the multidisciplinary discussion (MDD) to reach a definitive diagnosis and to decide the appropriate treatment for patients. A study conducted by Walsh et al came to the conclusion that ILD experts diagnosed IPF correctly more often than non-experts, thereby confirming the diagnostic value of the MDD as a valid tool, especially within reference centres. Another study found that the involvement of a rheumatologist in the MDD led to a diagnosis of connective-tissue disease-related ILD in about 20% of IPF patients, thereby further increasing diagnostic accuracy (Israeli-Shani L. et al).

 Current and future treatment options for IPF

Data from long-term observational studies as well as real-life experiences with the two approved drugs pirfenidone and nintedanib confirmed the results on safety and efficacy from the clinical trials. Interestingly, treatment with pirfenidone or nintedanib has not found to be associated with cardiac, bleeding or airway complications in IPF patients who received lung transplantation (Dorey-Stein et al). Moreover, the results of a phase IV single-arm, open-label study on the combined use of pirfenidone and nintedanib in IPF did not suggest a different safety profile to that expected for either treatment alone (Flaherty et al). It should be stressed that for the moment combination treatment can only be used in the context of controlled trials.

Several phase 2 trials with promising preliminary results are ongoing. Prometic‘s PBI-4050 is an orally active lead drug candidate which demonstrated a strong safety profile and promising efficacy, either alone or in combination with nintedanib or pirfenidone.

TD139, an inhibitor of galectin-3 that affects receptor signalling including the pro-fibrotic molecule TGF-β – has been tested against a placebo in 24 IPF patients for two weeks. The compound appeared to be safe and well tolerated and the results regarding efficacy in terms of suppressed Gal-3 expression on BAL macrophages were promising.

In conclusion, the ATS conference 2017 provided new insights in the pathogenesis, management and treatment of IPF and ILD in general, confirming that research and clinical practice are close each other in this rare disease group.

Breathing hope for IPF treatment in Europe

Political attention to idiopathic pulmonary fibrosis could make a difference for the hundreds of thousands of Europeans living with the disease, writes Elena Gentile.

Diseases are often referred to as 'great equalisers'. No one chooses to fall ill nor what symptoms they suffer. Yet not all diseases are equal in terms of morbidity and mortality. Idiopathic Pulmonary Fibrosis is one of those diseases that is not only rare, chronic and ultimately fatal but is also one that also lacks both a treatment and a cure. 

This is why I, as a Member of the European Parliament and also as a trained doctor, have chosen to take political action to raise awareness of this fatal disease, with its devastating impact on patients  and their families. 

Imagine feeling out of breath after climbing just a few stairs, or suffering a violent coughing attack for which no explanation can be found. You consult a number of doctors and eventually discover the cause of your symptoms; a condition you have never heard of before. 

This scenario and diagnosis has become a reality for around 111,000 Europeans that live with Idiopathic Pulmonary Fibrosis (IPF). A further 35,000 people are diagnosed with IPF every year. To date, no cure has been developed.

Where patients experience the feeling that there is no way out, political attention at European level can make a difference in the daily battle of IPF patients and their loved ones. This is why, together with colleagues from across Europe and across the political spectrum, we have put forward a written declaration on IPF to raise awareness about this disease. 

Through this written declaration, we hope to encourage the European Commission and member states to work together to support timely access to treatment. This way, we seek to address delays in pricing and reimbursement by national bodies and to address inequalities in access to lung transplantation. 

My colleagues that, with me, support the written declaration also believe that we can advance treatment through research and innovation in the pharmaceutical field. The Commission can play a key role in funding IPF research activities and in supporting opportunities for patient participation. In this context, we are pleased to see that last month the call for European Reference Networks (ERN) was published by the Commission. 

An ERN on rare lung diseases, including IPF, would be a crucial milestone towards improved cross-border collaboration of healthcare professionals and the establishment of a standardised approach for IPF care.

As my relationship with the IPF patient community and the newly established European IPF & Related Disorders Federation has grown over the past two years, so has my motivation to ensure that IPF is placed on the EU's agenda. 

I strongly encourage all Members of the European Parliament to support us in passing the written declaration on IPF onto the Commission and member states and showing European unity in standing up for its most vulnerable citizens.

EU must do more to combat disease described as 'more lethal than most cancers'

Idiopathic pulmonary fibrosis sufferers across Europe are struggling to receive consistent and equal access to care, argues Andrey Kovatchev.

It starts out as shortness of breath and dry cough. But it’s more rapidly lethal than most cancers. Idiopathic pulmonary fibrosis (IPF).

It is one of the most frightening diseases you’ve never heard of. There is no known cause for this disease, which makes it increasingly difficult for the patient to breathe as it thickens the lungs. Only one or two out of five IPF patients survive five years.

An estimated 80,000 to 110,000 people in Europe live with IPF, and up to 35,000 new cases are identified each year. These numbers, however, do not reflect the full magnitude of the disease.

IPF is often difficult to diagnose with initial symptoms resembling those of other, more common, diseases. What’s worse is that IPF treatment options are limited and there are huge discrepancies in access to care between and within EU member states.

Access to the best available healthcare is a right for all European citizens. As an elected citizens' representative to the European parliament, it is my duty to voice the recommendations of the patient organisations that have come together to create the first European charter for patients suffering from IPF, calling for consistent and equal access to care and treatment.

This includes early and accurate diagnosis, better access to palliative care and end-of-life care as well as a holistic approach to standardise the management of the disease.

The EU has done a lot to ensure a high level of human health protection, yet there are still simple measures that should be implemented at both European and national level to improve the situation of thousands of patients living with this disease.

First, the EU can play a decisive role in the improvement of standards for IPF care across Europe by ring-fencing funding for research within the Horizon 2020 framework.

Second, the EU can create a Solidarity Fund to allow access to orphan drugs that, though having received European medicines agency (EMA) approval, are not yet sold in some member states due to delays in approval by national regulators.

Along with more than a dozen other members of the European parliament, I will formally endorse the first European IPF patient charter.

We will also send a joint letter to the delegates of the expert meeting on chronic respiratory diseases to underline the importance of establishing multidisciplinary teams and regional networks to provide optimal care for IPF patients.

I firmly believe that the EU institutions must work together with member states to drive changes in national healthcare systems. National governments have a responsibility to influence clinical practice in partnership with local health authorities, medical societies and patient organisations.

If adopted, which we hope it will, the charter’s recommendations will improve quality of life for all IPF patients across Europe, supporting the development of better long-term treatments and hopefully contributing towards finding a cure.

Idiopathic Pulmonary Fibrosis, here’s the European Charter

Idiopathic Pulmonary Fibrosis is a rare pathology and is not well known

And just to step out of the shadows, the European IPF Charter was presented yesterday at the EU Parliament. The initiative has been promoted by 11 patient organizations and will be discussed on occasion of the IPF World Week. Their aim is to raise awareness of this fatal condition and promote better access to care for patients in Europe. Firstly, by collecting 35,000 signatures in representation of the number of new cases diagnosed every year in Europe. Idiopathic Pulmonary Fibrosis, that hits mainly people aged over 65, is a progressive and irreversible chronic lung disease. In those affected, the normally thin and pliable tissue of the lungs becomes increasingly thick and prevents oxygen from moving into the bloodstream, thereby leading to a continuous decline in lung function. In Europe, there are currently between 80,000 and 110,000 people living with IPF and an approximate 35,000 people are newly diagnosed every year.

First European Patient Charter on IPF Presented in European Parliament by 11 Patient Organizations


For the first time ever, a new European Patient Charter for patients who suffer from idiopathic pulmonary fibrosis (IPF) was developed by a group of advocates and presented to the European Parliament on September 30, during IPF World Week 2014. Biotechnology company InterMune, which is dedicated to research, development, and commercialization of innovative therapies in pulmonology and orphan fibrotic diseases, lobbied hardest for the release of the charter.

The first of its kind European Patient Charter was developed in a collaboration with 11 patient organizations and healthcare professionals from nine different EU countries as a way of advocating for improved quality of life for patients living with IPF. The purpose of the charter is to promote the standardization of care provision among patients, as well as equality of access to diagnosis and treatment procedures among IPF patients all over Europe.

“The development of the Charter represents the greatest joint effort that was ever realized by the IPF community in Europe. For the first time patient organizations from across Europe spoke with a unified voice to shed light on the needs of IPF patients,” stated Rosalba Mele, the president of the Italian association AMA Fuori dal Buio, one of 11 organizations that is promoting the initiative.

“This would not have happened without the support of InterMune, which has supported us and provided the opportunity for patient groups across Europe to come together, share their challenges and develop this Charter which we hope will support the need for change in the management of IPF and give a better future to patients living with IPF.”

Representing top priorities for IPF patients, the charter is a result of a series of meetings between advocacy groups and healthcare professionals, who are seeking to make their case to policy makers with one voice. It is composed of recommendations not only about diagnosis and therapy accessibility, but also about managing the disease in a holistic manner, and making updates to palliative care. Presenting at the European Parliament is a way of garnering attention about the issues related to the disease in order to raise awareness among European policy makers.

“We are proud to play our part in supporting the Charter initiative, which will be essential for improving care and access to healthcare services across Europe. The development of a Charter provides clinicians, patient advocacy groups and patients the opportunity of working together to identify the key concerns that can and must be addressed by policy makers,” said the executive vice president and managing director of InterMune in Europe, Giacomo di Nepi.

The charter is also expected to engage key stakeholders in each country of the EU, in order to conduct changes in their countries and improve patients’ lives. The initiators of the charter are now looking to collect 35,000 signatures through an online public petition as a symbolic act, since 35,000 is the number of people that are newly diagnosed every year in Europe with the irreversible, progressive and deadly disease that causes scarring of the lungs, irreversible destruction of the lungs, and hindering breathing. The disease has a survival rate of only 20-40% after 5 years.