Pirfenidone to Treat Hypertrophic Cardiomyopathy

[Wayne]

Does anyone have any information regarding this trial proceedure at N.I.H. The drug is also called Deskar. :?:

[Sarah]

Dear Wayne,

THe NIH recently disbanded its HCM clinic. Can you be more specific--is this an older study you just read about or is it something new? etc

S

[Sarah]

Hi,

I got curious, so I poked around. I found the NIH clinical trial, but it closed on April 12th this year. It is not clear if or when any results will be published from what I could find.

However, from another web site, it seems that "Pirfenidone is an orally active small molecule drug that appears to inhibit collagen synthesis, down regulate production of multiple cytokines and block fibroblast proliferation and stimulation in response to cytokines. Pirfenidone, which has demonstrated activity in multiple fibrotic indications, is currently in Phase II clinical development for fibrotic diseases of the lung, kidney and liver. Investigational, not an FDA approved indication."

Basically, this means that the drug reduces collagen buildup and is being studied. Preliminary results in mice show that it can reduce liver cirrhosis (sp?).

How is this relevant to HCM? In SOME --big emphasis on SOME-- genetic mutations of HCM cause collagen to deposit in between the muscle cells, which is why we get hypertrophy and those disorganized cells. So if you could reduce the fibrosis (what those deposits are called) then you would have a non-surgical way of reducing the hypertrophy --in theory.

It is a long, long way from being put to use or even proven to work.

But here's hoping!!

S