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New Hope for Pulmonary Fibrosis from the 2014 ATS Meeting

MD-FM Thursday May 22nd, 2014 

 

SARAH:

MD FM: the latest medical news from around the world. Here’s Peter Goodwin.

 

SEGMENT 1: IDIOPATHIC PULMONARY FIBROSIS: Two new molecules shown to improve lung function

 

PETER:

Hello! And we begin at the annual meeting of the American Thoracic Society, held this week in San Diego, where delegates heard about the first hope of effective treatment for patients with idiopathic pulmonary fibrosis. Here’s Sarah Maxwell with more…

 

SARAH:

Yes, previously, these patients’ only treatment option had been lung transplantation, but two randomized studies, published in the New England Journal of Medicine, now show efficacy of not only one but two molecules for reducing disease progression. One is nintedanib, which inhibits a variety of tyrosine kinases; while the other molecule, pirfenidone, approved in Europe but not in the US because of previous mixed results, has an unclear mechanism of action. Both drugs significantly reduced the decline in forced vital capacity (FVC) at one year –a measure of patients’ lung capacity. Dr. Gary Hunninghake wrote an editorial on the findings:

 

HUNNINGHAKE:

The reasons why prior drug therapies may not have worked very well was partly the target, and these drugs address a different way of going after this disease so… This is a very big breakthrough for these patients. These medicines both met fairly similar outcomes, reducing the decline in lung volume is really an important outcome in pulmonary fibrosis and I would say that, arguably, both had similarly positive findings.

 

SARAH:

Gary Hunninghake, from Brigham and Women’s Hospital in Boston. And he said both molecules also showed a trend towards reducing mortality. He also wrote about a third study, using acetyl-cysteine, that did not improve lung function.

 

PETER:

Well, finding two molecules is a good start! But could patients at all disease stages benefit from these drugs?

 

SARAH:

Well… These results were only among patients with mild to moderate disease, and study’s lead author explained why patients with severe disease weren’t included here

 

RICHELDI:

The patients with an FVC below 50 percent were not included and the reason for that is that these patients are very sick and they have difficulties in providing FVC measurements, and they can easily drop out for several reasons... But that’s another unmet medical need because there is basically no trial testing any drug in that patient population so I’m hopeful that, maybe, it’s possible that these drugs will be tested in a properly designed trial in this segment of the population.

 

SARAH:

Dr. Luca Richeldi, Professor of Respiratory Medicine at the University of Southampton in the UK. And he told us that identifying patients as early as possible is important.

 

RICHELDI:

We have seen that with these drugs, we only decrease disease progression --we are not reverting the disease. So the earlier we find these patients, the earlier we treat the patients, the best treatment effect we have. And there is a very typical sound which is called velcro --reminding of the velcro --and we know that it’s very typical of this disease, and we are looking forward to studies showing that the detection of these early signs, in particular through electronic stethoscopes, which is non-invasive, cheap --it’s really feasible at the point of care, like in a GP’s office --might help to identify patients with early disease.

 

SARAH:

Professor Luca Richeldi, from the UK.

 

SEGMENT 2: Statins not effective in preventing COPD exacerbations 

 

PETER:

Simvastatin is not effective at reducing the number or severity of flare-ups of chronic obstructive pulmonary disease. These are the findings of the first randomized trial to examine the efficacy of statins in preventing COPD exacerbations. The results — which contradict data from several previous retrospective studies — were also presented this week at the American Thoracic Society's annual meeting in San Diego and they appear in the New England Journal of Medicine.

 

SEGMENT 3: The majority of patients with adenocarcinoma of the lung are suitable for more effective, less toxic, targeted therapies

  

PETER:

The majority of patients with adenocarcinoma of the lung carry genetic mutations which make them suitable candidates for targeted therapies. That’s according to a study reported in JAMA

 

SARAH:

…And these targeted therapies, which are more effective and less toxic than current treatments, appeared to improve survival. 1000 patients with lung adenocarcinoma were tested for 10 genetic mutations, “oncogenic drivers,” which can be targeted with drugs that are either already approved or under development. 64 percent of patients tested positive for at least one driver and Dr. Boris Pasche from the USA wrote an editorial on the study:

 

PASCHE:

It’s probably the best data we’ve ever had… Today, you know, when we speak about personalized therapy in oncology, the word is that approximately 30 percent of tumors may have a driver. So 64 percent is very high. If we now were to re-start a similar study we would probably have 15 drivers and you may even further increase the coverage from 64 to maybe 70+

 

SARAH:

That was Boris Pasche, from Winston Salem, North Carolina. Patients who got a treatment targeting their mutation, clearly had the longest survival: Three and a half years, compared to those who either had a mutation and didn’t get therapy in this trial, or tested negative for any of the 10 drivers.

 

PASCHE:

3.5 years of life expectancy: it’s about 3 or 4 times longer than the average metastatic non-small-cell lung cancer.

 

SARAH:

And Dr. Pasche concluded that a new treatment era is on the horizon:

 

PASCHE:

We are really entering a new phase in oncology where treatment of tumors based on genetic abnormalities is not only theoretically possible but is a reality. A lot of drugs are currently in development. We are speaking of close to 120 drugs now in development for lung cancer. Most of them are targeted for certain genomic or genetic abnormalities of these tumo rs.

 

 

SEGMENT 4: Patients with RA hoping to become pregnant need a rheumatologist to improve their chances!

 

PETER:

Women with rheumatoid arthritis who want to have a baby, could increase their chances of getting pregnant if they first got optimal treatment from a rheumatologist…

 

SARAH:

Yes, that’s the implication of data from a study, published in Annals of the Rheumatic Diseases. It showed that women with RA have more difficulty getting pregnant and there are clinical factors associated with this delay in pregnancy:

 

DOLHAIN:

Disease activity as well as the use of NSAIDS and as well as the use of prednisone more than 7.5 mg on a daily basis –all were independently associated with a prolonged time to pregnancy.

 

SARAH:

That was senior author Dr Radboud Dolhain, from the Erasmus Medical Centre in Rotterdam. He insisted that women with RA hoping to get pregnant should be treated by a rheumatologist, rather than a general practitioner, to decrease disease activity as much as possible:

 

DOLHAIN:

We haven’t shown that if you decrease disease activity that they get pregnant more easily but I think it makes sense to lower disease activity as actively as possible without using NSAIDS and without using prednisone more than 7.5 mg on a daily basis…

 

SARAH:

And Dr. Dolhain hypothesized on what possible mechanism affecting fertility might be at play here:

 

DOLHAIN:

I think that the disease activity, because of high inflammation, high levels of circulating cytokines, might make women less likely to become pregnant…. For example, we know that women with active Crohn’s are less likely to become pregnant, although in Crohn’s it might be more than only active inflammation but, in my opinion, it’s quite likely that active inflammation, for whatever cause, makes women less likely to become pregnant.

 

SARAH:

That was Dr. Radboud Dolhain, from The Netherlands.

 

IN BRIEF 1: Physical therapy no better than sham treatment for improving symptoms of hip osteoarthritis

 

PETER:

Finally, in brief:  For patients with painful hip osteoarthritis, physical therapy didn’t appear to improve pain and physical function any better than having a sham treatment. And the authors of a randomized study on this, published in JAMA, are now questioning the value of physical therapy for these patients. It’s currently recommended by clinical guidelines, but these data showed that physical therapy was associated with mild but relatively frequent adverse effects.

 

IN BRIEF 2: For pregnant women who smoke: daily supplements of vitamin C improved new-borns’ lung functions

 

PETER:

For women who continued smoking during pregnancy, taking 500mg of vitamin-C a day improved their babies’ lung function test results at birth. These are the findings of a randomized, placebo-controlled study, published in JAMA, that found that infants’ wheezing was also reduced for up to a year. But the authors encourage, of course, that giving up smoking during pregnancy is obviously the best solution for both mother and child.

 

PETER:

That's all from MDFM for now. Sarah Maxwell and I will be back with more next week, so until then, from me Peter Goodwin, goodbye!

 

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