I am finally rested up from my trip and have so much to share about our experience at the American Thoracic Society. A big shout out and thank you to everyone that helped out including: Candice and Crystal Sipe, Donna and Ashley Appell, Demetria Saffore, Kristen Brantner and Carrie Frew.
I’ll start with what everyone really wants to know – what happened with the drug trials being discussed for pulmonary fibrosis??? Grin!
The meeting was very exciting. I went to a class on diagnosing pulmonary fibrosis and the class just sort of fell apart into one big party. One of the docs told me, “I’ve been treating patients with pulmonary fibrosis for 30 years and for the first time I will have a real treatment to offer them.”
The results of the Pirfenidone trial (the second one ordered by the FDA) were encouraging. Everyone seems to think it will be approved with no trouble this time and that it will be on the market in a year. I’m hoping for sooner, but to be realistic, a year is probably about right if no other snags occur.
The company that makes the drug has announced an Early Access program for the drug, but it is only for patients with idiopathic pulmonary fibrosis, not HPS. At this time it does not include HPS. We are working on some strategic advocacy right now to try to make that happen, but I want to be very realistic. It could take some time if it is successful at all.
The good news is I do not think we will have any trouble at all finding doctors in the US to prescribe Pirfenidone off label to HPSers once it is on the market. The issue is going to be whether insurance is going to cover it.
The feedback we got on that issue was about 50/50.
Some thought it might be a problem, while others thought it would be no big deal. I think we’re going to have to wait on that issue until it is on the market, and act like we think of course we’re going to get it. Do nothing to red flag to anyone that it even might be an issue, and see what happens. One doc said to me that he didn’t think it would be an issue because there are so few HPSers that it wouldn’t be worth it to the insurers to fight about it. It’s expensive, but they’ll be paying for it for thousands of people with IPF, thus HPSers will be a drop in the bucket.
In the meantime, we need to build the case for HPS. We need those that were in the drug trials to go back to NIH for follow up. I can’t stress how important this is now.
There was a second drug trial for another drug called Nintedanib (BIBF1120) that reported phase III results that showed benefit. We do not have any history with HPS and this drug. I am not totally sure about the biochemistry, or what part of the disease pathway it hits etc. to know if it would be helpful to us. We’re asking our researchers now. Everyone seemed to think this drug would also be on the market in about a year, assuming that it also doesn’t run into any bumps like Pirfenidone did.
Lastly, they reported on a trial with NAC and this trial showed no benefit.
Let me be very clear. Neither one of these drugs is a cure. They might just slow things down a bit, and they might not work for everyone. It’s a first step in a journey that is ongoing.
We are working on the issue of access for HPSers. Right now I don’t want to say more than that because I don’t want to do anything to endanger a delicate process. Please know, however, that we are actively working on this. When and if the time comes, we will need everyone on deck ready, but I hope the process turns out to go smoother than that and major lobbying isn’t needed. Still, be ready if we send out the call!
Lastly, about that journey.
Dr. Zhou (he was one of the researchers we funded) presented some research at the meeting that had everyone buzzing, even the non HPS researchers. It’s all a little above my head, but he thinks he’s found a possible therapeutic target that would be HPS specific and directed therapy, rather than these drugs coming out now that are sort of more general in nature. Dr. Zhou even won an award for his research at the meeting! So, there are better things in the pipeline.
I know we all want them now. I get it as I need a therapy yesterday – but this was perhaps one of the most promising and exciting meetings we’ve had yet!
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