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If you’ve ever toyed with the idea of doing your own Hermansky-Pudlak Syndrome fundraiser, now would be a good time. We’ve had a very busy spring and summer at the HPS Network. We’ve attended multiple medical meetings, spoken to thousands of researchers and physicians, marched in the New York Puerto Rican Day Parade, helped dozens of people connect with the NIH for research-oriented treatment, hosted the New York conference, hosted a major outreach effort in Florida and attended NOAH as an outreach.
We accomplished a LOT! We have a lot to accomplish as we finish the summer and move into the fall. Among the items on the list – the annual Puerto Rico outreach and a number of additional medical meetings just to name a few things.
But, nothing is for free in this life. Sending packets of information to new families and doctors takes money. Printing brochures takes money. Maintaining the 1 (800) number at the office takes money. Running the computers and our server takes money. Making copies takes money. Translation fees cost money. Going to medical meetings means paying registration fees, booth fees, shipping fees, hotels and airline tickets. Organizing the conference in a way to try to keep the costs down as much as possible for families isn’t cheap either. This past New York conference I think ran around $50,000 (and that was with a lot of items we needed given to us for free or at cost.) We’re currently feeding a colony of HPS mice being studied to help us find new treatments and we’ve been funding our own research for the first time.
We can’t keep doing those things without good old fashioned cash. And just like the rest of the country, our expenses are going up. And that’s just to keep doing what we’re doing now. It doesn’t address the possible need to hire future staff, or to fund additional research outside of the NIH. It doesn’t address serving the needs of more and more families every year. We’re growing all the time, and as we grow, we have more and more needs.
Treatments and cures don’t happen on their own. They especially don’t happen without patient involvement for ultra rare disorders like Hermansky-Pudlak Syndrome.
I hate fundraising. Boy do I hate it. I frankly hate asking my friends and family for money. I’m not much of a sales person to be honest. But I look at it this way. I buy girl scout cookies. I buy cookie dough for the local high school band trips. I’ve bought candles and wrapping paper from church youth groups. Thus, asking friends and family to buy an HPS Christmas CD, or whatever else we happen to be selling, is no big deal. What comes around goes around – and I’m not asking so I can go on a trip or go to camp. I’m asking the people that love me to help find a cure.
There are a number of you out there that do more than your fair share of fundraising. We’re so grateful to you. Some of you are able to bring in thousands of dollars, and we treat each one of those dollars so carefully! They are so precious.
But to help you don’t have to raise thousands of dollars. If everyone affected by HPS pitched in just a little we could make a big dent in paying for the things we need.
The patient registry is growing all the time. I don’t know what it was at last count – but lets say for the sake of argument (I know it is more than this now) it’s 800 HPS’ers.
If every person with HPS raised $50 a year that would total $40,000 a year. Now, let’s say that everyone has relatives that want to help. Let’s say those 800 HPS’ers got two other people to raise $50. That would be another $80,000. (Sandy, check my math – remember, I’m numerically challenged – and I haven’t been kidding about that!)
In the course of a year, what do you spend $50 on?
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This week has been a tough one from the fatigue point of view. I’ve been taking my daytime medicine and trying to use the CPAP at night, but I find myself waking up six to seven times a night needing to go to the bathroom for varying reasons. Or, I’ll wake up because a neighbor has turned their car on in the parking lot and shined their bights in my window – or the neighbor’s gaggle of dogs is out again barking and howling. Usually I can sleep through these things so I must not be getting any deep sleep.
My joints have also been more achy than normal this week, and I don’t know why. My GI is fine (except for some extra gas that gets me up at night). I don’t know what that’s about, but my fingers and wrists have been especially bothersome – a crisis for a net addict like myself. I haven’t been online as much partly because mom is here, but partly because typing hurts.
Mom thinks my fatigue is because I go to bed too late. What she doesn’t understand, being the bright-eyed morning person that she is, is that if I go to bed at 11:00 pm and get up at 7:00 am, I should have had eight hours of sleep. That isn’t the problem. It’s all this waking up in the night that’s the problem.
Mom and I have had a really great visit so far. No arguing etc. although last night we came close. I was taking my nightly meds, and there are a lot of them, and my mom asked if my different doctors were all aware of all the medications I was on. She meant the question out of concern, but in my brain my response was – what do you think I’m an idiot? Do you think I don’t understand the concept of drug interactions? Or do you think I’m intentionally drug seeking? For peat sakes – of course they all know. And if there’s any doubt just look at my records. I carry the entire pharmacy with me to each and every medical appointment just to be sure that everyone has an accurate list with dosages. I’m not hiding anything from any of my doctors. The comment was probably purely innocent, but it bugged me.
It ringed of the attitude that has been the heart of a lot of discord between us – that somehow this isn’t as serious as I make it out to be, that somehow I’m just being “dramatic.” It’s an assertion I highly resent. I may be doing fine. I’m even doing much better than expected – but I have a number of friends who haven’t done as well. Because of that, I do have a tendency to instantly get irritated at innocent comments. I’ve been discounted so many times that I’m instantly defensive.
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The following article appeared on the Coalition for Pulmonary Fibrosis Web site. I thought it was a very nice summary of some of the major developments in pulmonary fibrosis research in the last year as well as what's on the horizon in the next year or two.
Sometimes it amazes me how far we've come just in the six years since I learned I had HPS.
CPF Chairman Marvin Schwarz, MD Discusses IPF Research with BioWorld Today Reporter
Rare Pulmonary Fibrosis may Have Blockbuster Potential By Catherine Hollingsworth, BioWorld Today
A handful of companies are working to develop a treatment for a rare lung disorder called pulmonary fibrosis that, by some estimates, could present a $500 million to $1.5 billion market opportunity.
"It is probably a commercially significant market," said Michael Gilman, CEO of Stromedix Inc., whose company has a compound for pulmonary fibrosis in Phase I.
Aside from the sales potential, a drug targeting the fatal lung-scarring condition also would improve the care of patients who currently have no approved treatments.
Pulmonary fibrosis is a disease that leads to worsening shortness of breath, until patients eventually suffocate. Patients suffering from the disease usually live an average of three to five years, according to the California-based Coalition for Pulmonary Fibrosis and other sources. In fact, many people with cancer often live longer than those diagnosed with pulmonary fibrosis.
An estimated 128,000 Americans are affected by pulmonary fibrosis, and an estimated 48,000 new cases develop each year, according to the coalition.
A few companies - Japanese drugmaker Shionogi & Co. Ltd., U.S. biotech InterMune Inc., and Swiss biotech Actelion Ltd. - are out front in developing potential new treatments for patients with idiopathic pulmonary fibrosis (IPF), a form of the disease in which the cause is unknown.
Shionogi has filed for approval of its IPF drug candidate pirfenidone in Japan and is awaiting a final decision. Brisbane, Calif.-based InterMune Inc., which holds rights to pirfenidone in the U.S. and Europe, has completed two large Phase III studies and plans to initiate an extension study. Swiss biotech Actelion also has an experimental IPF drug, bosentan (Tracleer), in Phase III testing, with results expected in 2009.
Several other companies - Amgen Inc., Wyeth, Boehringer Ingelheim GmbH, Novartis AG, Genzyme Corp., and Pipex Pharmaceuticals Inc. - have earlier stage compounds intended for the treatment of pulmonary fibrosis.
Currently, InterMune's Actimmune is prescribed off-label to treat IPF, though the off-label use has steadily declined in recent years in light of two negative studies, said analyst Howard Liang, of Leerink Swann & Co.
According to Liang's figures, U.S. sales of Actimmune were $107 million in 2005 and declined to $90 million in 2006 due to the unfavorable IPF data. In 2007, sales slid further, totaling $53 million, due to a second negative trial in that indication, Liang said.
In March 2007, InterMune halted further Phase III testing of Actimmune in IPF after an independent data monitoring board found a lack of survival benefit compared to placebo.
To date, the most successful treatment available for IPF patients is lung transplantation, said Dr. Marvin Schwarz, board chairman of the Coalition for Pulmonary Fibrosis. But he said many patients who are eventually diagnosed with IPF are older and often are not eligible for lung transplants due to comorbidities such as diabetes or cancer.
Other drugs that have been tried in the past for IPF, such as corticosteroids and immunosuppressants for lung cancer, have not been sufficiently effective, Schwarz said.
However, he noted that many drug companies now are taking an interest in the disease and are exploring a number of pathways to target the disease. Some are focusing on interferon gamma and tumor necrosis factor-alpha to address the uncontrolled scarring in the lungs, while others have focused on the beta pathway to slow the progression of the disease, said Schwarz, who recently received the top honor given by the American Thoracic Society and the American Lung Association for his career accomplishments in pulmonary medicine.
In addition, the National Institutes of Health established an IPF network of medical centers that are focused on IPF research.
InterMune spokesman Jim Goff said high dropout rates in IPF patients and tolerability issues have been a challenge for conducting studies in that population. In addition, he noted that it is a tough disease to study, on the level of some forms of cancer. But he said InterMune was encouraged by the Shionogi Phase III trial results that showed a 44 percent decline in the rate of lung function. InterMune's Phase III program is similar to Shionogi's in that both companies are seeking to slow the decline in lung function. However, Goff said, the InterMune study is of longer duration and has a larger patient enrollment.
Goff agreed that IPF represents a "potentially large market opportunity from a financial standpoint."
Schwarz said it is possible that a treatment for IPF likely will have broader applications in treating other forms of fibrosis affecting the liver, kidney, eye and skin, for example. The final pathway in uncontrolled fibrosis, he said, appears to be similar across those different forms of fibrosis.
It is not unheard of for a rare disease that affects a relatively small number of people to approach blockbuster status, Liang said. He pointed to Novartis' Gleevec for chronic myeloid leukemia, a drug with $3 billion in sales; and Genzyme, a company with products focused on rare diseases.
Any IPF treatment is expected to have a high price tag, Liang said, based on the cost of other pulmonary drugs and the $50,000 cost of Actimmune.
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The following is a press release that came from the American Thoracic Society. It involves two issues of importance to us. First, without this action the amount of payment doctors recieved from treating patients on Medicare would have been cut. Trust me - it isn't the docs getting rich off of Medicare patients. And to penalize them for treating Medicare patients by not paying market rates would mean that Medicare patients would find it harder and harder to find a doctor willing to take them on.
For us, throw in an ultra rare syndrome (thus more time consuming) and this would have been a problem for many of those with HPS.
The second issue has to do with pulmonary rehabilitation. By getting Medicare to cover pulmonary rehabilitation, hopefully other insurers will join in as well. The benefits are real from what I've seen first hand. It's an issue the HPS Network has worked on with the ATS in the past so it's another victory for the year.
American Thoracic Society Applauds the Enactment of the Medicare Improvements for Patients and Providers Act (H.R. 6331)
Today, Congress voted to override President Bush's veto of the Medicare Improvements for Patients and Providers Act (H.R. 6331). Congressional action today averted the scheduled 10.6% cut in Medicare physician payments. This represents an important victory for doctors and their patients.
Congress also enhanced the Medicare program in important ways, most notably adding pulmonary rehabilitation services as a covered benefit under Medicare. Along with fighting for fair reimbursement for our members, the Society's Government Relations Office, in Washington, has long worked with individual ATS members, PAR, the Council of Chapter Representatives, the Clinicians Advisory Committee, and sibling organizations to have this important therapy for COPD and other respiratory diseases covered uniformly by Medicare.
I know I speak for our members, as well as their patients, in thanking our Congressional champions Sen. Mike Crapo, Sen. Blanche Lincoln, Rep. John Lewis and Rep. Chip Pickering for their tireless efforts to secure Medicare coverage of pulmonary rehabilitation for patients with COPD. Their leadership was crucial in ensuring Medicare coverage of pulmonary rehabilitation.
Jo Rae Wright, PhD
President
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 Regular readers have seen this egg before. I'm posting it because we've been talking about how people with low vision can indeed do art on the HPS adults listserv lately. I don't have a lot of artwork around as I can't paint as much as I once did. But, I do these eggs around Christmas time. They end up being presents and then this year I donated a few eggs to the HPS Network Craft Bank and they were sold at conference with the funds benefiting the HPS Network. The egg is made of paper mache (you can buy them already made at the craft store). I then put a sealer on the egg so the paint doesn't soak through the paper too much. Then I paint the egg. When it's dry, I put several coats of varnish over it and attach a little dodaddy thing to the top so it can hang as an ornament. I'll be doing some of these again for the HPS Network. Actually, I have one that is a larger size than this one about half finished now.
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Regular readers might notice that the ad that regularly appears in the top right corner of the blog has disappeared.
This evening I got a letter from Google saying that my Google ad account had been shut down. Apparently they detected some sort of thing where someone was clicking over and over and for the protection of their advertisers they shut down the account.
I’m having mixed feelings about the letter. On the one hand I totally understand where they are coming from. I have also been a google ad advertiser and you certainly don’t want to pay for bogus clicks. On the other hand, it’s irritating that the system punishes the publisher for something I have no control over. It’s not like I’m sitting here clicking over and over and over.
Many people know that I donate the revenues from my google ad to the HPS Network. I’ve never been out to make a buck off of HPS. Trust me, it drains my wallet far more than it has ever added to it. Grin! It could be that someone was thinking they were helping, although I’ve always told people not to do that. One click is a trade - your pennies for my reader's time reading your messsage that has been targeted to them - more than that is something different.
The part that does have me very irritated, however, is that they owe me a check which they may have stopped payment on. The ads that have been running lately were correctly focused to my audience. It’s been a lot of low vision stuff and I’d estimate 80 percent of my readers are low vision. To do that to me is crooked, although cleverly they’re contract gives them the right to do it. But the advertisers have already had the exposure to my audience, and now they don’t have to pay for it? That does rub me the wrong way – especially when I’m held responsible for something I don’t even know about.
I will file an appeal, but doubt very much I’ll get anywhere with it. Beyond that, what can I say? Oh well….I’d have to consider the time investment to fight about it versus the many other things the Network needs me to be doing right now.
I am perturbed though. Grunt!
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 Here's a pic of a barn cat at Jessica's. He's so cute!
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My mom is visiting me right now. So far it’s been a good visit. Yesterday we went to see the new expansion of the Nielson-Atkins Museum of Art. It’s been open for quite a while, but I’ve never had anyone that wanted to go. My mom was an art major for her undergraduate work and knows a lot about art history, so she’s fun to go to museums with because she can often tell you a lot about the artists etc.
I really liked the new section. The first part was a lot of modern art and pop art. That isn’t so much my taste. But the second part had a wonderful photography exhibit. It talked about the history of photography and how the different methods worked and developed over the years. Then they had a section of artwork that people had done with digital photography. It was so cool!
There was one in particular that I found fascinating. I wish I had written down the name of the artist. He took his digital camera and took pictures of trees staring straight up at them into the branches. The photos were taken at all different times of the year. Then he edited out anything in the background except branches or leaves so that the backgrounds were white. Then he put about 40 of them together in one piece of art.
From a distance the way they were lined up it looked to me (keep in mind I don’t see very well) like those pictures of chromosomes all lined up. But as I got closer, it looked like a collection of images of something crystallized. And then when we read the description and discovered these were images of trees I was blown away.
There was another image of a little girl in a swimming pool. It looked up from under the water at her (but in a respectful way – nothing not decent or anything). You couldn’t see her face because of the ripples of the water and her swimsuit had the most beautiful colors all sort of distorted the way something looks when you look at it through water. I thought it was a photograph, but when I read the label, it was actually a printmaking project. Having done some printmaking and found it difficult when working with highly detailed images, I was blown away.
After the museum we went to the mall. I’m not much of a shopper but I did find a new pair of pants and some blouses. Yeah! Score! Grin.
So, so far it’s been a nice visit.
One thing that is kind of fun and entertaining about having my mom around is to see how she reacts to things having lived in Germany so long.
I’m sure that people in Germany use the internet for phone service as well. But my mom was delighted to hear that she could make long distance calls to her heart’s content on my phone and it wasn’t running up my bill. She keeps asking me if I’m sure she’s not affecting my bill as if she can’t quite believe it. She’s been calling friends she hasn’t spoken with in years.
I called home at lunch to see how she was, and she was having fun watching television. Think of all the nonsense we have on TV, and think about how funny/odd/grotesque it might seem if it was all new to you. She’s quite entertained.
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 I'm working on the HPS Newsletter and was trying to find an image of the signing of GINA by President Bush. I found this one on the White House Web site, although the resolution is crappy and they don't make high res pics available, not even in the media section. I have to say, the Clinton administration had a much better online media room - but that's a story for another day.
I saved this pic, and then I had a wacky Friday thought. I looked around at all the piles of paper, all the things to do, sitting around on my desk at work. And then I looked at this picture - note how clean the President's desk is! There's nothing on it! It's spotless!!! Do you suppose some secretary comes by before the photographer shows up and clears it off for him? Or do you think it's always that spotless? I'd like to think that it's usually covered in work like my desk.
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 Grandma and Aunt Phyllis enjoy talking at Jesse's farm on the 4th of July.
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