Hooray for the Home Team

A group of researchers at The University of Texas Health Science Center at Houston have developed a modified Factor VIII protein that will help treat inhibitor patients.

From the press release:

With a shortage of the blood-clotting protein Factor VIII (FVIII), people with Hemophilia A typically receive injections of FVIII derived from plasma or produced synthetically to control potentially life-threatening episodes of bleeding. Unfortunately as many as 1 in 3 people with Hemophilia A produce inhibitor antibodies, which attack the externally-administered FVIII and negate its blood-clotting benefits.

To combat this problem, scientists in the laboratory of Sudhir Paul, Ph.D., at The University of Texas Medical School at Houston, developed a chemically modified version of FVIII which during laboratory tests neutralized these inhibitor antibodies, thus paving the way for the correction of the blood-coagulating process. The modification is called electrophilic FVIII analog (E-FVIII).

“It’s a two-step process,’’ said Paul, the senior author. “The E-FVIII permanently inactivates the antibodies that inhibit blood clotting in 20 to 30 percent of patients receiving Factor VIII replacement therapy. Once the antibodies are cleared, additional FVIII can be injected.” The study involved blood donated by eight people with FVIII-resistant Hemophilia A.

Miguel Escobar, M.D., of the department of pediatrics and internal medicine at the UT Medical School at Houston and the Gulf States Hemophilia and Thrombophilia Center, led the clinical aspects of the work.(emphasis mine)

This is great news from just around the corner. Congratulations to Dr. Escobar and the rest of our HTC team. We are so very fortunate that Houston has one of the premier Medical Centers in the nation, if not the world. We are doubly blessed for Jack to be under the care of Dr. Keith Hoots and the whole Gulf States HTC staff (the finest in the nation, and yes we’re biased).

Project DOCC

I helped out with Project DOCC on Friday. What’s Project DOCC? Well, from the Houston Project DOCC website:

Project DOCC, a groundbreaking national program for pediatric physicians-in-training, has a simple goal:

To improve each patient's quality of life by teaching physicians-in training the effect of chronic illness on patients and their families.

Created in 1994 by three parents, Project DOCC's philosophy is that, by understanding how families deal with illness and or disabilities out side the hospital setting, pediatric physicians-in-training become better and more compassionate physicians.

Project DOCC's teacher - parents and guardians of children with special medical needs - provide physicians with a unique perspective on culturally effective, family-centered care.Project DOCC Houston is a non-profit organization that is a collaboration of Houston-area families of children with special health care needs, Baylor College of Medicine's Department of Pediatrics and Texas Children's Hospital. Project DOCC Houston is mandatory for first year pediatric physicians-in-training as one of the teaching modules of Pediatrics 101.

The Project DOCC program is made up of three components; the home visit, the parent interview and the Grand Rounds panel presentation.

Both Allison and I have participated in the home visit portion (check out here for more on the parent interview and Grand Rounds). During the home visit the resident(s) come to the home of a special needs child to get a feel for a day-in-the-life of that family. We go over medications and dietary issues and storage issues for supplies. We get into insurance and Medicaid, home modifications for handicap access, marital and financial stresses and so much more. There’s also a ‘visiting parent’ who interjects their own experiences as well as providing a more global perspective of the challenges of caring for a special-needs child.

I take special satisfaction from being able to tell our story and educate another doctor about hemophilia and challenges that come with it.

So on Friday I was the ‘visiting parent’ and we had a very interested and observant resident. I’ve done home visits where we’ve had two or three residents and, honestly, having the undivided attention of just one is so much better. With three residents or even just two one can shrink back and not engage, letting another resident take the lead.

All in all it was a good visit and hopefully enlightening and productive for the resident.

Check out to see if Project DOCC is in your area and how you can get involved.

FDA approves new NovoSeven product

On May, 13 the Food and Drug Administration (FDA) approved Novo Nordisk's new formulation of NovoSeven(R)RT recombinant factor VIIa product. What makes the new Novo so novel? The RT is for room temperature. RT can be stored at room temperature. According to the press release the new NovoSeven will be available in higer concentrations for smaller infusion volumes.

From the Press Release:

NovoSeven(R) RT has been manufactured to be room temperature stable (from 36 degrees F to 77 degrees F outside a refrigerator). The new formulation does not require refrigeration and can be moved in and out of the refrigerator. In addition, NovoSeven(R) RT has a higher concentration for lower infusion volumes and quick administration. The needed infusion amount is almost half of that needed with the original NovoSeven(R).

Unassisted triple play

No, not Jack in little league,but the Tribe's Asdrubal Cabrera. On Monday night the Cleveland Indian second baseman turned only the 14th such feat in Major League history. To put it into perspective there have been 15 perfect games which makes the unassisted triple play the rarest of baseball achievements. What's really cool about the play-by-play call is that even before Cabrera caught the ball the announcers were calling 'triple play, triple play.' The runners were moving on the pitch and the broken bat liner was so close to second base Cabrera couldn't help but turn the rare three-out play.

Asdrubal Cabrera Turns Unassisted Triple Play
Uploaded by Machochip

'Good night, neighbor'

Veteran Houston newsman, Ron Stone, passed away Tuesday after a bout with cancer. Ron Stone was on the air on Channel 2, KPRC when my family moved to Houston in the late 70's. The team of Stone, weatherman Doug Johnson and sportscaster Ron Franklin were the best, most relaxed, funniest and down to earth news team I've ever seen.

Stone and company were the genuine article. He signed off each night with, "Good night, neighbors." And one felt that he truly meant it and I know he did. He came into our homes each night and gave us the news as a neighbor. When he presented the news it was like you could go outside, walk over to the fence and continue the conversation with him.

For so many years he was a part our Houston life, first on Channel 2 then picking up for Ray Miller on 'The Eyes of Texas' and later with his production company, Stonefilms of Texas.

I also remember staying up as late as I could manage and watching Ron and the rest on the local broadcast of the Jerry Lewis MDA telethon.

I haven't thought about Ron Stone in a while, things change and you move on. But now that he has passed it's hard to forget him. Thanks for years and good night, neighbor.

KPRC report.

Houston Chronicle article.

Channel 11, KHOU report

Channel 13m KTRK report

'Impossible is nothing'

This is a great story about an amazing young woman from the Wichita Eagle. Ngoc Trang Nguyen is the valedictorian at East High School in Wichita, Kansas. That's nice, but so what, you say. Four short years ago, having emigrated from Vietnam, she spoke no English. By junior year, she was doubling up on math and science courses and getting straight A's. Trang plans to attend Creighton University in Omaha and major in biochemistry. But that's not the best part.

"I imagine myself in a lab coat," she said. "One of my wishes is to figure out the cure for hemophilia, which is a blood disorder."

In Vietnam, one of Trang's best friends had hemophilia. "But his family was so poor, they could not afford medication, and he suffered tremendously from that," she said. "This is what inspires me."

This is a story that warms the heart on so many levels. It's fantastic that someone so bright wishes to work on a cure for hemophilia. It also shows that in the meritocracy of this great nation of ours opportunities exist for anyone. With encouragement and dedication, hard work is rewarded regardless of ones background. It's there for the taking. As the Nike slogan says "Just Do It."

Happy Mother's Day

I hope it was a joyful and restful day for all the Mothers and Grandmothers today. These are the ladies in our lives that keep the rest of us from falling apart. Without them we'd be hungry, dirty, smelly and wrinkled.

So it's appropriate to remind everyone of Project Red Flag, NHF's awareness program for Women's Bleeding Disorders. The most common bleeding disorder for women is von Willebrand Disease (VWD).

Earlier this year the National Heart, Lung and Blood Institute (NHLBI), part of the National Institutes of Health (NIH) and the U.S. Department of Health and Human Services issued the first clinical guidelines in the United States for the diagnosis and management of VWD.

Here's the NHLBI press release.

And here's NHF's press release.

Baseball - Jack legging a triple

Jack had a game last night and I remembered to bring the camera (and actually use it!).

Here's Jack hitting a legitimate double and taking third on the throwing error. But hey it's seven and eight-year-old machine pitch, so that's a triple. Jack has severe hemophilia A but that hasn't stopped him from enjoying his natural athletic ability. Ain't factor great!

With two games left in the season, Jack's team is 8-3-1 and in second place.

Genetics and Hemophilia

Congress Sends Bush Anti-Genetic Discrimination Bill - Fox News

Redefining disease, genes and all - Andrew Pollack, International Herald Tribune


Medical science is constantly progressing, stretching the current limits and breaking new ground about what is known about the human body, its genetic makeup and what causes diseases.

The Pollack article is a fascinating glimpse into how advances in the human genome are changing how diseases are classified.

“The research is already starting to change nosology, as the field of disease classification is known. Seemingly dissimilar diseases are being lumped together. What were thought to be single diseases are being split into separate ailments. Just as they once mapped the human genome, scientists are trying to map the "diseasome," the collection of all diseases and the genes associated with them.”

Hemophilia has always been the classic case study of genetic mutations and has benefited from advances in genetic knowledge.

“Some of the earliest work has until now been with inherited diseases caused by mutations in a single gene. Diseases have been subdivided by the type of mutation. Hemophilia was divided into hemophilia A and B, caused by mutations in different genes for different clotting factors. And what was once considered a mild form of hemophilia was later identified as a variant of a different clotting disorder, von Willebrand disease, caused by mutations in a different gene and requiring a different clotting factor as treatment.”

So it is in this light that the Genetic Information Nondiscrimination Act carries even greater importance.

I’m not usually one for expansion of governmental power or governmental intrusion of this type but this is a good thing. I‘m not alone in this opinion. Separate versions of the bill passed the House 414-1 and the Senate 95-0 (Ron Paul the lone dissenter) and President Bush is expected to sign the final version.

It’s important not only for those with a genetic disorder but for those who’s only knock is they show a higher pre-disposition to more common ailments.

Ensuring genetic non-discrimination goes far to alleviating some insurance coverage and medical care concerns for our kids with bleeding disorders.

New VIIa product set for 2009 human clinical studies.

Catalyst Biosciences, Inc. announced it intends to file an Investigational New Drug application and commence human clinical studies in 2009 for its new factor VIIa variant. Catalyst says CB 813 is, “…an improved, second-generation variant of human coagulation factor VIIa…for the treatment of acute bleeding in hemophilia patients.”

From the company’s press release:

“CB 813 is designed to substantially enhance clot-generating activity at the site of bleeding and therefore achieve clinical efficacy with fewer and lower doses than current therapy. In established hemophilia models of acute bleeding, CB 813 has demonstrated a significant improvement in potency compared with the marketed recombinant factor VIIa product, NovoSeven(R) and a competing, second-generation product, NN1731.”

Eventually one of these potential improvements will be realized and raise the bar for the rest of the industry. Here's hoping it's sooner than later.

Hemophilia fact-checking

I saw this article a couple of days ago. It’s about a laid-off employee of Aloha Airlines with hemophilia and his struggles to maintain health insurance coverage. What caught my eye was a glaring mis-statement about hemophilia.

Chun has hemophilia; a bleeding disorder that means his blood does not clot. So he could bleed to death from even the smallest cut. (emphasis mine)

No, he couldn’t bleed to death from the smallest cut.

Part of any advocacy work is providing education about the particular issue of interest. It’s frustrating that loads and loads of work that goes into educating the public about hemophilia can be nullified by one sentence in one article where the research and fact-checking wasn’t done.

Because the bleeding disorders community is (thankfully) small getting free publicity in the form of news articles is extremely valuable. But misinformation like this does nothing to serve our greater goals.

McCain's Health-Care Vision

Republican presidential hopeful, Sen. John McCain had an opinion piece Thursday at National Review Online outlining his vision for the future of the American Health-Care System.

“The problem is not that Americans don’t have fine doctors, medical technology, and treatments. American medicine is the envy of the world. The problem is not that most Americans lack adequate health insurance. The vast majority of Americans have private insurance, and our government spends many billions each year to provide even more. The biggest problem with the American health-care system is one of cost and access, and as a result tens of millions of individuals have no insurance.”

From my standpoint it hit the right notes:

Control of health care choices in the hands of the individuals.

“American families know quality when they see it, so their dollars should be in their hands. When families are informed about medical choices, they are more capable of making their own decisions, less likely to choose the most expensive and often unnecessary options, and are more satisfied with their choices.”

More choices in finding an insurance provider.

“Americans also need new choices beyond those offered in employment-based coverage. They want a reformed system so that wherever you go and wherever you work, your health plan goes with you.

… another option would be available: Every year, they would receive a tax credit directly, with the same cash value of the credits for employees in big companies, in a small business, or self-employed. You simply choose the insurance provider that suits you best.

Increased competition in the marketplace for your insurance business.

“Millions of Americans would be making their own health-care choices again. Insurance companies could no longer take your business for granted, offering narrow plans with escalating costs. It would help change the whole dynamic of the current system, putting individuals and families back in charge, and forcing companies to respond with better service at lower cost."

Competition through reform in the maddening array of states health-insurance rules.

“Right now, there is a different health-insurance market for every state. Each one has its own rules and restrictions, and often guarantees inadequate competition among insurance companies. Often these circumstances prevent the best companies, with the best plans and lowest prices, from making their product available to any American who wants it. We need to break down these barriers to competition, innovation and excellence, with the goal of establishing a national market to make the best practices and lowest prices available to every person in every state.”

Extremely important for the bleeding disorders community provisions to insure preexisting conditions within the marketplace framework.

“But we also need to ensure that those without prior group coverage and with preexisting conditions, who have the most difficulty in the individual market, have access to the high-quality coverage they need.”

“I will consult with the governors to solicit their ideas about a best practice model that states can follow — a Guaranteed Access Plan or GAP — and work with Congress, the governors, and industry to make sure that it is funded adequately and has the right incentives to reduce costs such as disease management, individual case management, and health and wellness programs.”

I don’t agree with Sen. McCain on all issues. But this is a sensible, workable approach to addressing the nation’s health-care needs without the leviathan of government run universal coverage. Universal coverage is well intentioned and admirable in principle but in practical terms would be a disaster for this country. The cost of health-care is already outpacing the ability of Americans to pay it. Involve the federal government (more than it already is) and can we seriously envision those costs going down. On a list of bloated, inefficient government programs health-care would be the biggest colossus of bloat and inefficiency. Rather than sending huge premium checks to insurers the American public would instead send their health-care dollars to the federal government in the form of vastly higher taxes.

One major aspect missing from the senator's proposals is some strong steps toward transparency of medical costs. How can the American public be savvy medical consumers if they can't compare costs because those costs are hidden? How hard is it to get the price of a unit of hemophilic factor out of a specialty pharmacy (negotiated contract price with your insurance carrier be damned).

Senator McCain’s proposal may need work and revisions but it is a sensible, responsible and comprehensive start.

Ffffsssttttt…splash

That was the sound of the air going out of Barack Obama’s sails last week following his Pennsylvania primary defeat and then the sound of Rev. Jeremiah Wright being thrown under the keel today.

The presidential hopeful ship of Barack Obama sailed into Pennsylvania waters last week amid what could have been a political perfect storm. For Sen. Hillary Clinton that is. The Keystone state primary was set against the backdrop of the Rev. Wright controversy, renewed interest in the Senator’s ties to former Weather Underground member, William Ayers and Obama’s association with political fundraiser and wire fraud, bribery and extortion defendant Tony Rezko. Not to mention his own disparaging San Francisco comments about middle-class Midwesterners.

All of this didn’t so much fill Clinton’s sails as take the wind out of Obama’s. Pennsylvania’s more blue-collar electorate set up nicely for the former First Lady in the first place and add to it Obama’s recent woes and victory was all but ensured for Clinton.

In the end it may not matter. By throwing the Chicago pastor overboard today Obama more than likely will have righted his ship. Especially in the eyes and opinion of the fawning adoration of the nation’s media.

But let it be a cautionary tale. Let it serve as a reminder of how much we don’t know about the junior Senator from Illinois. People want him to be something different. People want to feel good about him. He is charismatic and witty and smart and people want him to succeed for so many reasons. But just wanting someone to be what you hope for doesn’t make it so.

Hope and change are universal ambitions. But with nothing behind them they are just words.

And remember he just disowned Rev. Wright a mere six weeks after saying he could no more disown his former pastor than he could his own grandmother. Look out grandma and keep your mouth shut.

Mirror - mirror

I know this just has to have been done before but are Steve Nash (on the right,or left) of the Phoenix Suns and British musician James Blunt (on the left, or right) the same person or at least long lost twins. Eerie.

While we've been away...

O.k. I took off last week from work and thought man, I'm gonna blog every day. This is great!

What did I do? Nothing. Absolutely nothing (in the way of blogging).

Nothing happened though, right? The Pope's visit, primary election in Pennsylvania, nothing.

I'll get back in the swing here shortly.

World Hemophilia Day!

Today is world Hemophilia Day 2008. This year's theme is Count Me In.

The World Federation of Hemophilia describes it better than I could:

This year the theme of World Hemophilia Day (April 17) focuses on registering and identifying patients: Count Me In.

It has often been said that there is strength in numbers. This is definitely true in the bleeding disorders community. As a rare disease group, in order to be heard by governments and others, getting the numbers to back up your case for support makes a huge difference.

Having accurate statistics on the number of people with bleeding disorders identifies how many people are affected and helps ensure that they get the necessary treatment. It also helps to validate claims with ministries of health and build a case for support.

The WFH’s vision of Treatment for All is that all people with bleeding disorders need access to adequate care and treatment. The first step to achieving this is to identify those patients. For 10 years now, the WFH has been collecting data from national hemophilia organizations around the world to quantify the number of people affected with bleeding disorders. Our most recent survey identifies 205,472 people with bleeding disorders in 100 countries.

On April 17 we are launching a new publication on outreach to identify patients. The WFH has also developed a number of tools to help patient organizations, hemophilia treatment centres, and others raise awareness about the need to be counted and help identify patients. These include a poster, news release, factsheets on outreach and national patient registries, as well as educational materials for patients on hemophilia, von Willebrand disease, and rare bleeding disorders.

World Hemophilia Day was started by the WFH in 1989. April 17 was chosen as the official date in honour of WFH founder Frank Schnabel, who was born on that day.

On the day, hemophilia organizations and treatment centres around the world organize gatherings, sporting events, exhibitions, and other activities to raise awareness of the issues affecting people with hemophilia.

We encourage all patient organizations, treatment centres, and corporate partners to celebrate World Hemophilia Day this year.

We want to count you in!

Tax Day

I tend to be a procrastinator. But I had no intention of waiting until the last minute to file our ‘lack of’ income taxes. We’re getting money back for goodness sakes! But still I put it off and put it off. I finally got them done on Sunday only to get an e-mail today from TurboTax telling me the eFile was rejected. I messed up on the 2006 AGI figure the IRS uses as verification for the ‘electronic signature’. So here I am on tax day scrambling to correct the eFile and resend.

Fair Tax anyone? A federal consumption (sales) tax doing away with all income taxes? Doesn’t sound like such a bad idea right now. I know there’s plenty of well-reasoned criticism of the Fair Tax, plan but at least it’s a discussion moving in the right direction. Year after year we hear about simplifying the tax code yet it never happens. Instead it gets more complicated with additional and revised deductions and credits and qualifications pushed by lobbyists and passed by well-meaning but ill-informed legislators.

It’s time for a change. Change we can believe in. Hope we can believe in. I didn’t just say that.

Heparin Problem has not gone away

I posted about the Heparin problem back in January here. Apparently the situation has gotten worse. A recent New York Times article details the latest developments.

Here's the opening graphs:

The number of suspicious deaths in the United States linked to the blood thinner heparin has risen to 62 from 19, with most of them reported this past December, January and February, according to the first detailed analyses of heparin fatalities by the Food and Drug Administration.

The F.D.A. is still investigating whether those deaths and hundreds of allergic reactions were caused by a heparinlike contaminant made in China that was added to the drug somewhere during the manufacturing process.

The drug agency defined suspicious deaths as those involving one or more allergic reactions or a drop in blood pressure. There have been no reports of deaths since the end of February, after Baxter International recalled heparin made with ingredients from a Chinese supplier.

Before we jump to conclusions about Baxter:

In addition to Baxter, companies that have recalled heparin products made with Chinese ingredients include Covidien, formerly Tyco Healthcare, and B. Braun. Both Covidien and Braun said that they had received no reports of adverse reactions from the heparin and that the recalls had been undertaken merely as a precaution.

The common thread between this product and some seemingly unrelated consumer goods is China. I posted about China's exports last year here. Coincidence for the 2008 Olympic Summer Games host? Time will tell.

Cool Perfomance Art

I'm not usually much of a YouTube guy but this is pretty cool. No protest, no motive, just let's see if we can do it. Two hundred people freezing in place for five minutes in Grand Central Station.

The Masters

It’s time. I know you can hear the first smooth strands of the piano music as the images of azaleas, dogwoods, brilliant green fairways, Rae’s Creek, the Hogan Bridge and Magnolia Lane roll across the screen. You are welcomed by the mellifluous voice of Pat Summerall and now Jim Nance. It’s Masters Week at Augusta National.

The only one of the major championships played on the same course every year The Masters holds a special appeal for both players and fans alike.

Both the club and the championship were the vision of golfing legend Bobby Jones and Clifford Roberts. The list of Masters champions reads like a roll call of golf’s greats; Gene Sarazen, Byron Nelson, Sam Snead, Ben Hogan, Arnold Palmer, Gary Player, Jack Nicklaus, Tom Watson, Phil Mickelson and Tiger Woods.

It’s Masters week at Augusta and I plan to enjoy.