Thursday, August 06, 2009

Urgent: Update on the Pirfenidone protocol

As many of you know, this summer there was to be an interim analysis of the results thus far in the Pirfenidone trial at the NIH.

The statisticians have met, and the news is mixed.

The statisticians declined to review the data thus far because we haven’t met our recruitment goals for the protocol and we’ve had several people drop out of the trial. As a result, the statisticians believed that any results they came back with would indeed not be statistically relevant or accurate.

Those of us who have been in the trial for a long time were hoping to get some answers about how effective Pirfenidone has been in this trial, and we were hopeful that they’d decide to put us all on the drug.


Unfortunately, that isn’t going to happen. I think we can’t help but feel disappointed. The trial will continue on as it is now, although some of us who have already been in the study for more than three years might not have to return to NIH as often.

This news is disappointing to say the least, but it could have been worse.

The good news is the NIH could have simply shut down the study and they didn’t do that. They also could have found that the drug didn’t work, and no one is saying that either. In fact, they must have some hope that this is worth additional time and investment. That’s not a promise that this drug will work, but it is hopeful.

Besides this mixed news, the NIH has also issued us an incredible challenge. We need to wow the socks off of the statisticians and those who evaluate these trials. Thus, the NIH has decided we need a minimum of 50 patients instead of 40. That means we MUST find 17 more participants, and those participants must be in the trial three years before we can approach the FDA about approving the drug. Considering that as of this writing, we have 33 people, this is an incredibly tall order.

But we can do it! It’s all hands on deck! We have to do it!

We all have times in our lives that are pivotal – do or die moments. For the Network, and indeed for the future of HPS research, this is one of those moments. If we can’t meet this challenge, it could make it difficult to convince researchers to do further drug studies on HPS. Not meeting this goal is simply not an option.

I don’t mean to depress everyone, or to make you feel stressed or hopeless. I simply want everyone to understand how important this is, and how much we need everyone’s help. The office in New York simply can’t do this alone. We simply can’t all sit back and assume the “office” will take care of it.

We need your help. That means you.

Everyone can do something to help. Don’t wait for the HPS Network to come to your area for an outreach trip. We are pleased to try to help you become your own outreach expert. Here are some of the things you can do to help make the cure possible.

Do you have a Puerto Rican community center in your area? Visit them. Find out if they know about HPS. Take them some brochures for their lobby.

Do you have Spanish news outlets in your area? Try to approach them about doing an interview or story about HPS. If you can get them interested, we can supply an expert for interviews.

Do you go to a church or belong to a civic group in a Puerto Rican area? Get a Presentation in a Bag from the New York office and ask your church or group if you can give a presentation on HPS. Tell your audience you’re recruiting them in our Hundred People Search army. Ask them to pass the word about HPS along to family, neighbors and friends.

Do you live in a Puerto Rican neighborhood? Get brochures from the Network and ask local businesses to keep them by their cash registers.

Do you participate in other albinism-related groups? Get involved with regional NOAH conferences, or albinism-related online groups. Share information about HPS. Everyone with albinism should know about HPS. We need to change the perception that HPS is something “so rare you don’t have to be concerned about it.”

Get a stack of brochures from the Network and share them with any low-vision specialists, lung docs or GI docs in your area.

Do you participate in any other low vision groups? Share information about HPS with your local Voc. Rehab. counselor, vision teachers, school for the blind, Lighthouse or vision-related consumer group.

Time is critical. We need to prove to NIH that we can do this. We need to have a flood of HPS’ers knocking on the doors of the NIH. We can do it!

There’s a folk song I love called Step by Step. It says, “ Step by step, the longest march can be won. Many stones can build an arch, singly done.” That’s us – we need every one of you to chip in and help out. The cure won’t be found by the work of the “office.” It will be found by piling up everyone’s single acts of outreach. We need the cure, and we don’t want to leave anyone with HPS behind when we find it.

I’m going to be compiling a journal of acts of outreach, no matter how big or how small, and publishing it on the Website and in the newsletter. I also want to share it with the NIH so that they know how hard we’re working to meet this challenge. As you work for the Network, no matter what you’re doing, please, please let me know about it. Pictures are also helpful.

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