Skip to main content

Another step on the road to a treatment

Many of you that follow these things closely will have already seen this news yesterday - but since it means a little something different for us, I wanted to share.

Intermune (the company that makes the drug pirfenidone, brand name Esbriet) announced the results of their ASCEND trial yesterday and they were good! This means that the company will be moving forward to try to get FDA approval for the drug to treat IPF (idiopathic pulmonary fibrosis).

So, for those who haven't followed this saga or for the new folks, here's a little background on all this and the tie-in for HPS.

Back in the late 90s the NIH conducted a small phase II trial of this drug to treat the PF of HPS. The results were promising. It is NOT a cure, but did slow down the progression of the lung disease.

We were ready to do our phase III trial, but the drug was sold to another company and our trials were put on hold for several years (very frustrating). Finally, in 2006 we were able to start our phase III trial. Sadly, in 2009 the trial was halted half way through because the review board at NIH felt that the study, as constructed, would never be able to prove whether the drug worked. (It was not halted because the drug didn't work, only because our study would not be able to prove it.)

This is part of the challenge of rare diseases. When you have trials with such small numbers, one little thing can statistically really throw things off.

The much larger phase III trials for IPF continued around the world. In the US there were two trials. One showed benefit and the other was inconclusive. Even though the FDA's own review board recommended approval, the FDA required the drug company to conduct yet another phase III trial. Meanwhile, around the world other countries have been approving the drug and it is in use overseas.

While this was going on, there have been some slow reforms happening at the FDA. You all might remember signing a petition about a year ago asking the FDA to add pulmonary fibrosis to a list of 16 diseases that would be getting special attention because of their severe impact on patients and lack of other treatment options. Originally, PF wasn't even on the list of diseases to be evaluated for this program.

The PF community, (other PF organizations and the Network) got so many signatures on that petition that PF was not only considered, but made the final list. This was important because now the FDA will be under even more pressure to get treatments for PF to market.

So, then yesterday the results of the extra phase III study were announced. They were very exciting and positive.

Intermune says it plans to resubmit their application for approval by the third quarter of this year and they are estimating that it will take the FDA six months to review it (this is on a fast track as it can otherwise take a year or two at least.) The company is hopeful that they will bring the drug to the US market by the second quarter of 2015. Óf course, we've hoped for things before and been slowed up by snags in the system, but right now, that's the timeline.

So, here is where things may be tricky for us. It will approved for use in IPF, not HPS PF. This means it will be an off label use, thus insurance companies may be able to get out of paying for it for us. It is expected to cost around $50,000 a year, so coverage is a big deal! So, behind the scenes the Network is trying to get the stage set to advocate for us to also get access to this drug. We have several options and are already discussing them with our docs and outside resources to plot the best course forward.

I am very excited about this news because, while I think we have a battle ahead, we could not have even fought that battle if the results of yesterday's announcement had been negative. Now, we can get our ducks in a row and go into action! We will need EVERYONE's help with this. So, please pay attention to calls for advocacy coming up.

There is other good news here for HPSers around the world as well.

1. None of the data suggested any additional safety concerns with the drug. Also, Intermune reported that follow up studies on patients getting the drug overseas haven't yielded any additional concerns. There is a small percentage of people who develop some GI distress and/or a rash from the drug, and in some cases, had to stop taking it. In the now thousands and thousands of patients studied, there were two reports of liver toxicity, but it resolved as soon as the patients stopped taking the drug. Everyone is different and such things are to be expected with medications.

2. There are a number of countries overseas where the drug is approved, and thus legal, but the health care system isn't approving payment for the drug. These positive findings will help to further the case for coverage everywhere.

Again, this is not the cure. But, it does hold some promise to slow down the disease. We still have two patients from the phase II study who are living, taking the drug, and doing well.

It is likely that if the cure involves medications, it will eventually be several medications working together and not just one.

Still, I am on cloud nine! There are so many dark and disappointing days in this journey to the cure that you've got to celebrate the days when something moves us forward!

If you want to listen to the results yourself, go to the investor relations section of the Intermune website. The info they put out was designed for the investor market, not patients.

They also had more data they didn't share because they are planning on presenting it at the upcoming American Thoracic Society meeting. They are also trying to get the data into a medical journal. They didn't want to throw cold water on that process, which I totally understand. Grin!

Off to do another happy dance!

Comments

Popular posts from this blog

Nobody likes rejection

Me posing for a photo with Finley. This is my swollen steroid face. Thankfully, it is starting to go down.


Rejection – it’s a word I’m hard pressed to find a good context for – and in transplant land, it certainly isn’t a good thing, but perhaps it’s a constant companion. Once you get an organ that you weren’t born with, your body is intent on seeing this life-saving gift as a foreign invader it must destroy. It’s a battle that is constantly going on inside you, and it’s a battle you and your doctors constantly fight. The only good news is medicine has created a number of weapons to hold rejection at bay.



In June I had my first setback because of acute cellular rejection. They tell me it’s the good kind of rejection to have because it is treatable with an onslaught of steroids and medication adjustments. Oh goodie!



It was amazing how quickly it came on, and really brought home how important it is to do those vitals and spirometry every day. When I thought I might be in trouble, I had m…

Planning for Armageddon

Sometime in January I had a transplant clinic appointment. I can’t exactly remember the date now, but I remember the appointment very well. It was the beginning of what could have been the end.



I did the usual tests – spirometry, a six-minute walk etc. I was now not just on eight to 10 liters of oxygen. I needed 10 liters of oxygen pretty much all the time; even to walk the maybe 15 to 20 feet to my bathroom. To go for a slow walk required 15 liters.



The funny thing about increasing oxygen, especially when it happens over years as it did for me, is that you adjust to it. It just becomes your normal. You might get annoyed by it, but somehow it becomes this thing in your life that just is there. So, although I was clearly very sick at this stage, I didn’t feel sick. I felt tired a lot, but not sick. I didn’t feel pain (thankfully). I didn’t feel weak (even though I needed a lot of help with household activities so clearly I was). I just adjusted. I coped with the waiting for a new lung …

The next generation with Hermansky-Pudlak Syndrome

I'm so behind on posting about the trip to Puerto Rico. Since the episode of Mystery Diagnosis on Hermansky-Pudlak Syndrome ran right after we got home, it's been a little busy. These, however, are my favorite pictures from Puerto Rico. I know, not pretty senery etc - but these little guys and gals inspire me. They are the next generation of folks with HPS, and if we keep up the hard work, they will live better lives because of it. They motivate me.