“Hey, Curmudge, what’s with the title? I was anticipating more discussion on
neuroplasticity.”
“That will be delayed a bit, Julie. I encountered an editorial in the Wall
Street Journal (3/03/14) that dumps on the Food and Drug Administration (FDA)
for their handling of a drug that is effective against idiopathic pulmonary
fibrosis (IPF).”
“Oo, that’s the disease that killed Mrs. Curmudgeon in
January 2011.”
“Right. That
plus a generous dollop of pneumonia as the coup
de grâce. Actually, we’re not
completely certain which was the ultimate culprit, but I’m always on the alert
for articles on pulmonary fibrosis.”
“So what did the Journal say, Old Guy? Whatever it was, it seems to have
stoked your boiler.”
“A small biotech firm called InterMune released the results
from a third randomized controlled (Phase III) trial of pirfenidone. The FDA ruled that the results of the
first two trials were insufficient for approval and demanded the third.
Trial details, showing a delay in the decline of patients’
lung function and a prolonging of life, are shown in the InterMune website. Furthermore, the Journal said that
pirfenidone had been available in
Japan since 2008, in the EU since 2011, and Canada since 2012.”
“You recall, Professor, that in our earlier postings we
discussed clinical
trials and searching the medical
literature. Then more recently we mentioned the challenges of diagnosing
and treating
your wife’s disease. One
pathologist (local) said it was nonspecific interstitial pneumonia (treatable)
and the other (at Mayo Clinic) said it was idiopathic pulmonary fibrosis
(fatal).”
“For whatever disease it was, the treatment prescribed was
that which was recommended by the American Thoracic Society in 2000 for
idiopathic pulmonary fibrosis.
That was a combination of a corticosteroid (prednisone)
and an immunosuppressant agent (Imuran) which, by the way, was never evaluated
in a randomized, placebo-controlled trial.”
“I would bet, Curmudge, that these meds mucked up your
wife’s resistance to her subsequent case of pneumonia.”
“Very likely.
So I began scouring the literature in search of a better med for IPF, and I learned of
trials of many classes of potentially valuable compounds. (1) When I asked
about this work in the fall of 2010, the response from the local medical
community was that nothing looked very promising.”
“But
that response was out of date, Old Guy.
A
report from a meeting on May 17, 2009—over a year before your question—was
titled, Pirfenidone shows promise for
idiopathic pulmonary fibrosis.
And there was a Cochrane Review (2), updated July 7, 2010, that
concluded, ‘pirfenidone appears to improve progression-free survival and, to a
lesser extent, pulmonary function in patients with idiopathic pulmonary
fibrosis.’ “
“You
know, Julie, even if everyone had known about pirfenidone, there wasn’t much
that could have been done without FDA approval. And according to the Journal, that hasn’t been achieved even
yet. The only possibility would
have been to enroll Mrs. Curmudgeon in the next trial.”
“So
what’s the bottom line, Old Widower?
We can’t bring her back to life.”
“I
guess I should apologize to her ghost.
On behalf of myself for not raising more hell with the medical
community, on behalf of the medical community for being behind the curve in
reading the literature, and on behalf of the FDA for being self-serving,
foot-dragging bureaucrats. Sorry about that, Mrs. Curmudgeon.”
Kaizen
Curmudgeon
1.
http://emedicine.medscape.com/article/301226-medication The report at this site accessed in
2010 is no longer available.
2.
Spagnolo, P., et al. Non-steroid agents for idiopathic pulmonary fibrosis. Cochrane
Database of Systematic Reviews 2010, Issue 9, Art. No. : CD003134. First published online: July 21,
2003. Last assessed as up-to-date:
July 27, 2010.
P.S.—In the Letters to the Editor section of today’s Wall
Street Journal (3/12/14, p. A18) L. Salzman states, “My dad took pirfenidone
during the last two years of his life.
He believed, as I do, that it dramatically slowed his pulmonary fibrosis… He couldn’t travel regularly to Japan,
where the drug is approved, due to his health, so he bought it online—a
crime.” “…on one occasion his
medicine was seized by U.S. Customs, leaving him without the drug for nearly a
month during which time his condition took a rapid turn for the worse.”
Link to posting from blog archives: A day in a hospital in Prague 4/23/09
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