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European Biopharmaceutical Review

Q&A: Idiopathic Pulmonary Fibrosis

EBR:What were some of main themes at the American Thoracic Society’s recent event, ATS 2016?

Dr Marilyn Glassberg: ATS covers a lot of ground, with asthma and chronic obstructive pulmonary disease (COPD) probably being the biggest attractions. My speciality is interstitial lung disease, and idiopathic pulmonary fibrosis (IPF) in particular, where we got to explore the growing evidence base for the recently approved drugs, as well as get a first look at some potential future therapies.

Why do you think conferences such as these are so important for the industry?

When it comes to critically evaluating the best and the worst of new research, there is no substitute for live meetings. I co-chaired a fascinating session on stem cell therapy, including some really promising research from around the world. Dampening some of the enthusiasm, however, was alarming information from Dr Ikonomou from Boston University on the expanding threat from expensive and unsafe stem cell-related biotourism. Worried patients are enrolling in very poorly regulated ‘clinical trials’ – they are listed on but they do not deserve to be! – at private medical centres that are essentially selling untested therapies to misinformed and desperate patients under the guise of medical research.

You mention that you are particularly involved in IPF research. What do the ATS 2016 data releases add to developments in this area, and what do you predict will be their impact?

ATS 2016 went some way to addressing the paucity of data characterising disease progression and drug effectiveness. For instance, we showed that patients whose forced vital capacity (FVC) had declined over six months of therapy – the traditional definition of ‘treatment failure’ – would, in fact, continue to benefit from therapy with pirfenidone. This is tremendously helpful in treating physicians and their patients who are trying to figure out what to do next.

How do you feel approaches to treating IPF are likely to change over the coming decade?

As the therapeutic arena is relatively young, up to now IPF patients have been treated as a single homogenous group, but the reality is that they are very varied – and that this is not just one disease. At ATS, there was discussion of a wealth of different biomarkers, including some excellent microRNA work uncovering their genetic regulators in a session co-chaired by Dr Kaminski from Yale School of Medicine and Dr Geraci at Indiana University. I fully expect that biomarkers will become increasingly important to IPF research, diagnostics and therapeutics (3).

What are the main obstacles when treating IPF? How might new therapies manage to tackle the shortcomings of current approaches?

IPF is progressive and the two current therapies can only slow the decline. Equally, the two drugs have adverse events: pirfenidone can be associated with nausea, rash and dyspepsia (4), while the majority of nintedanib patients experience diarrhoea (5). So, similar to the situation in cancer medicine, physicians and patients need to be really well informed about what the drugs can achieve and their potential side effects before they start therapy. I hope that in the future stem cell therapies could actually help to reverse the disease. I have completed the first FDA-approved clinical trial of bone marrow-derived mesenchymal stem cells (MSCs) as a potential therapy for patients with IPF.

Why are advances in IPF treatment crucial for the development of pharma as a whole?

Because this idiopathic disease is so serious and so poorly understood, there is now a lot of great work going on to get to grips with IPF biomarkers and their regulators. To an extent, this is reflective, of and a great example of, the broader push towards personalised treatments in many fields of medicine. Speaking more personally, tell us a little about your background.

How did you end up working in this industry?

I have been fortunate enough to have some great teachers and mentors throughout my career who helped me recognise and translate new ideas into actual research and that, in turn, guided my career.

What is the best part about your role?

I get a real kick out of being a mentor myself to researchers and clinicians at all stages of their careers. It’s crucial to not try to create clones of myself – you have to figure out what each person loves to do. I travel to teach as much as my research career allows!

And what is the worst or most frustrating part?

The poor prognosis my patients have and the time it takes to turn research into medicine. In 2014, it was estimated that IPF kills up to 65,000 Europeans and 17,000 people in the US every year (6). And it was only in 2014 that the FDA approved pirfenidone and nintedanib (approved by the EMA in 2011 and 2015, respectively). We need more data and more therapeutic options. We need a cure.

What is the most important lesson you have ever learnt?

[Laughs] There are way too many! I guess that “perseverance promotes success”. Much of the time you just have to keep jumping hurdles!

1. Ikonomou L et al, A note of caution in stem cell therapies: Separating the wheat from the chaff, Presented at American Thoracic Society 2016 International Conference, San Francisco, CA, 13-18 May 2016. Adapted from Connolly R et al, Travel Med Infect Dis 12: pp695-701, 2014 and Deans RJ et al, Cytotherapy 18: pp138-142, 2016
2. Nathan SD et al, A4995 – Effect of continued treatment with pirfenidone following a ≥10% relative decline in percent predicted forced vital capacity in patients with Idiopathic Pulmonary Fibrosis (IPF), Poster presented at American Thoracic Society 2016 International Conference, San Francisco, CA,13-18 May 2016
3. Kaminski N and Geraci M (Chairs), C21 – Applying 'omics technologies to reveal gene regulators and biomarkers in lung development and disease, Session at American Thoracic Society 2016 International Conference, San Francisco, CA, 13-18 May 2016. Visit: planner?shared=B4
4. Esbriet Summary of Product Characteristics. Visit: www.ema. library/EPAR_-_product_information/ human/002154/WC500103049.pdf 5. Ofev Summary of Product Characteristics. Visit: www.ema. library/EPAR_-_product_information/ human/003821/WC500182474.pdf
6. Hutchinson JP et al, Ann Am Thorac Soc 11: pp1,176-1,185, 2014

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Dr Marilyn K Csete Glassberg is a pulmonologist specialising in interstitial and rare lung diseases. She has conducted NIH-funded, industry-sponsored, and investigatorinitiated clinical trials in patients with IPF, pulmonary hypertension and lymphangioleiomyomatosis. She has completed the first clinical trial on the safety of intravenous infusion of bone marrow-derived MSCs in patients with IPF endorsed by the FDA and reported safety. Her laboratory studies MSCs, steroid hormones in lung disease and the aging lung. As the Director of the Interstitial Lung Disease Program at the University of Miami Miller School of Medicine, Marilyn is well-versed in basic and clinical research, as well as database development.
Dr Marilyn Glassberg
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