Saturday, January 12, 2008

A very belated happy New Year

Demands of home and work have hindered regular (or not so regular) posting to the blog. Plus, I've been downright lazy. I don't expect to do much better in the near future, but best wishes in this still-young 2008.

Wednesday, December 26, 2007

Tuesday, December 18, 2007

I bet she doesn't have a Che t-shirt

Elsa Murano will become Texas A&M's new president in January. It's an amazing and inspiring story of a woman who as a girl with her family, fled Castro's Cuba in 1961 and later came to the United States at the age of 14 not knowing any English. Murano pursued microbiology, food science and technology studies earning a masters and doctorate degrees. In 2001 she was appointed undersecretary for food safety at the Department of Agriculture and is now poised to fill the chief executive slot of a major university.

Dr. Murano is profiled in the Houston Chronicle here, the Dallas Morning News here, the Austin American Statesman here.

Monday, December 17, 2007

Study shows extended half-life of recombinant FVIIa feasible

A study by CSL Behring shows that the half life of rFVIIa can be prolonged by fusing it with human albumin.

CSL Behring today announced the results of a pre-clinical study that show for the first time it is feasible to genetically fuse Factor VIIa (FVIIa) to human albumin, prolonging the half- life of this therapeutic protein while retaining its biologic activity. In the study, which was presented at the American Society of Hematology 49th Annual Meeting and Exposition, the half-life of recombinant VIIa-albumin fusion protein (rVIIa-FP) was shown to be extended 6-to-9 fold (emphasis mine) compared to wild type rFVIIa.


Greater half life of rFVIIa would be welcome news indeed. Bottom-line, NovoSeven is expensive. We're glad to have it don't get me wrong. When dealing with inhibitors being able to treat bleeds with a recombinant product is extremely valuable. But it is costly.

When Jack was around 13 months he developed an inhibitor. This was in the middle of the factor shortage for recombinant FVIII (rFVIII) products. Because of the shortage immune tolerance (IT) was delayed for over a year. During which time we treated bleeds on-demand with NovoSeven. We used Novo to treat bleeds for an additional year during IT until it was successful and rFVIII once again had some meaningful efficacy.

For 2 1/2 years we used NovoSeven to treat bleeds from when Jack was around 13 months to 3 1/2 years-old. By the time Jack was four we came very close to the $2 million lifetime cap on the PPO insurance option through my work (and this was with a 5-month period before IT started during which Jack did not have a bleeding episode). Not all of that expense could be attributed to rFVIIa but certainly the lion's share.

Had we had a longer lasting rFVIIa that hit toward the lifetime cap would have been much less. Treating some bleeds every 2-3 hours for 24-36 hours to keep on top of the bleed just ate up a huge amount of insurance. (We've been very fortunate to have a HMO option with no lifetime cap, less choice - but no cap)

So I think there's definitely room for optimism. Noted in some of my previous posts here, here, here, here and here there's a lot of activity in the anti-hemophilic industry. And this is good. Market forces at work. Competition fueling innovation which produces better products and therapies for our community.

Wednesday, December 12, 2007

Surge success causes MRAP slowdown

There's been a whole rash of articles basically saying the same thing. Things are better in theater in Iraq so maybe we don't need all those MRAP's after all.

Like this quote from an MRAP article on Weekly Standard's online site.

But since arguments against the surge are harder to come by these days, the services are taking the first steps in slowing the MRAP freight train. Late last month, the Marine Corps announced it would cut 1,300 vehicles from its order, saving the Pentagon $1.7 billion and removing the logistical headache of moving the weighty vehicles to the field and trying to find something to do with them.


Reading through these articles I notice we get to hear from the big brass now. Before it was the one or two star general run out to take the fire from the press and congress. Both of which were eager to criticize the administration and the war effort but still wanting to appear pro-military by calling for immediate and miraculous production to MRAP's to protect our war fighters. Not exactly a very friendly environment. As with any organization when the news is bad or you expect to take some heat, send out the spokesperson or lower level management. But now that there are reasons for optimism in Iraq we hear from Marine Commandant Gen. James Conway and Army Lt. Gen. Raymond Odierno, the number two commander in Iraq.

In the end fewer MRAP's will be produced but for now the manufacturers are still going full-tilt to field as many of these life-saving vehicles as they can. The front-line folks want them because they save lives.

Here's a recent article from USA Today about the positive effect the MRAP's are having in Iraq.

Sunday, December 9, 2007

Mice cured of sickle cell anemia with stem cells

More stem cell breakthroughs without the need for embryonic stem cells. Life preserved. Win - win.


The strategy should work to treat hemophilia, thalassemia and severe combined
immunodeficiency disease, the "bubble boy" disease, according to researchers, and might also apply to disorders linked a single gene, such as muscular dystrophy and cystic fibrosis.

and

The study is the latest in a string of significant experiments published in the past five months involving a new approach of reprogramming adult cells so they are capable of growing into any type of tissue in the body. (emphasis mine)

Friday, December 7, 2007

December 7, 1941

Never forget.






























Thursday, December 6, 2007

Masterful

Former Massachusetts Governor and Republican presidential hopeful Mitt Romney addressed the issue of his Mormon religion and his bid for the Presidency today from College Station, Texas. Speaking from the George H. W. Bush Presidential Library on the West Campus of Texas A&M University Gov. Romney affirmed his Mormon religion but pledged that his church would not define his candidacy or his presidency

Romney didn’t wade into the tall grass of particulars of his religion nor should he have to. But rather, he engaged the nation in a discussion of religious liberty and tolerance that is embedded in the American tradition by the founding fathers.

Excerpts from the speech:


"Let me assure you that no authorities of my church, or of any other church for that matter, will ever exert influence on presidential decisions. Their authority is theirs, within the province of church affairs, and it ends where the affairs of the nation begin.”

and


"There are some who would have a presidential candidate describe and explain his
church's distinctive doctrines. To do so would enable the very religious test the founders prohibited in the Constitution. No candidate should become the spokesman for his faith. For if he becomes President he will need the prayers of the people of all faiths.”


"I believe that every faith I have encountered draws its adherents closer to God. And in every faith I have come to know, there are features I wish were in my own: I love the profound ceremony of the Catholic Mass, the approachability of God in the prayers of the Evangelicals, the tenderness of spirit among the Pentecostals, the confident independence of the Lutherans, the ancient traditions of the Jews, unchanged through the ages, and the commitment to frequent prayer of the Muslims. As I travel across the country and see our towns and cities, I am always moved by the many houses of worship with their steeples, all pointing to heaven, reminding us of the source of life's blessings.”


and

"Americans acknowledge that liberty is a gift of God, not an indulgence of government. No people in the history of the world have sacrificed as much for liberty. The lives of hundreds of thousands of America's sons and daughters were laid down during the last century to preserve freedom, for us and for freedom loving people throughout the world. America took nothing from that Century's terrible wars - no land from Germany or Japan or Korea; no treasure; no oath of fealty. America's resolve in the defense of liberty has been tested time and again. It has not been found wanting, nor must it ever be. America must never falter in holding high the banner of freedom.”


It was well crafted, well delivered and completely unnecessary. Romney succumbed to the rantings and ravings of his detractors calling for an explanation of his faith. Now that he has addressed the issue on very much his own terms, and done so quite masterfully, they will only use it to further bludgeon him with their vicious and bigoted attacks.

Those people will never be swayed by any one speech (however eloquent it may be). But for those many who were unsure what to think of Romney and his faith this speech should quell their fears and comfort their apprehensions. For here is a man of faith, conviction and integrity who will hold true to the founding principles of this nation’s forefathers and the Constitution they crafted which guides it.

The whole text of the speech can be found here.

Video of the speech here.

Hemophilia in the News

Massachusetts Company looks to jump into the Antihemophilic Factor game

GTC Biotherapeutics Inc., of Framingham, say it's acquired an exclusive license to develop three recombinant human proteins from ProGenetics LLC of Blacksburg, Va.

GTC has been pioneering the production of therapeutic human proteins from the milk of animals.

From the GTC website:

The key enabling science for GTC Biotherapeutics (GTC) is the development of human therapeutic proteins in the milk of transgenic animals. Transgenic animals carry genetic information allowing them to express these human therapeutic proteins in their milk. Once they are produced, these recombinant proteins can be efficiently purified from milk for use as therapeutics.


The company press release is here.

A quick aside: A short blurb in from bizjournals is here. The sentence that caught my eye is this:

With 143 workers, GTC reported a 2006 net loss of $35.3 million on revenue of $6.1 million.

Ain't capitalism grand. The sheer optimism necessary to make ventures like this work. And in the end it make not work.



Non-injectable Factor?...

Not yet, but some initial steps.

Nastech Pharmaceutical Company Inc. of Bothell, Wa has entered into a feasibility study with an undisclosed global leader in the production of plasma products to develop a non-injectable Factor IX product.

Who is the "undisclosed global leader in the production of plasma products"? We could certainly make some well-informed guesses. But really, don't know, don't care. Do it and get it done.

Wednesday, November 28, 2007

What I no longer pray for

I wrote some thoughts down a few days ago, but as real life occupies one’s time it’s taken a while to get them on the blog.

Thanksgiving is past and we move into the Christmas season. It’s a time of reflection for many. A time to take stock of the year past and prepare for the year to come. A time to take account of just what one has, and yes, even what one wants.

I have been truly blessed. I was raised in love by two inspiring parents whose example of life, marriage and parenting is the standard to which I aspire.

I am surrounded by the love of my fantastic wife whose smarts and wit keep me grounded and on my toes.

We have two beautiful boys who amaze us constantly with what they know, learn and do.

I am thankful for modern medical science and safe and effective factor concentrates. Thankful for the hemophilia doctors, nurses and social workers who provide Jack with the care and treatment that allows him to do (most) everything he wants.

I am thankful for the healing gift of time. The time to learn, cope, adjust and accept. The time to come to grips with the challenges of life with a boy with hemophilia. The time to take on that monster of fear and beat it down until that monster is a routine that is managed day in and day out.

And so it is that I pray and give thanks for all that I have. But one thing has left my prayers. I no longer pray for a cure for hemophilia. I want it. I want it desperately. More than I want longer lasting factor, easier delivery methods and cheaper factor. And I want those a lot.

But it is through the grace of God and wonders of time and experience that hemophilia is but one of the many challenges of a busy life. One that we address, manage and overcome.

God knows we want a cure and in time it will come. But there’s something to the old adage that God only gives you what you can handle. Until there is a cure hemophilia we can handle and I will pray for other things.

Saturday, November 24, 2007

Success like this doesn't happen often.

The Aggies pulled out a 38-30 upset over the 13th ranked Texas Longhorns Friday.


Perhaps more surprising is the basketball Aggies improving to 6-0 with a 70-47 win over Ohio State and capturing the NIT Season Tip-Off title that same day. This is a program with no pedigree to speak of in the Big 12 and the Southwest Conference before that. A program that was best known for the witticisms of former coach and SWC legend Shelby Metcalf. The coach who once told (as the legend goes) a player with 4 F's and a D that he was focusing too much on one subject.


But under Billy Gillispie and now under Mark Turgeon the Aggie hoopsters remain a Big 12 and national contender.


Oh, and by the way, so long Fran.

Friday, November 16, 2007

New kids on the block want to play

And they’re bringing their own ball.

Inspiration Biopharmaceuticals hopes to get into the recombinant Factor IX (rFIX) game with a new low-cost manufacturing process.

In a November 13, 2007 press release Inspiration Biopharmaceuticals states having a patented manufacturing process that “significantly increase(s) cell-line productivity.” Which, “significantly lowers the overall production cost for Factor products compared with existing manufacturing processes.”

Inspiration Biopharmaceuticals hopes its cost-effective process will open the door to non-invasive delivery methods and eventually to a therapeutic cure for hemophilia B.

Cheaper, better, easier. All desireable. But first and foremost; safety, safety, safety.

Read the press release here.

Wednesday, November 14, 2007

NHF Annual Meeting Post, Finally

This Post is far overdue.

We went to the NHF annual meeting in Orlando in early November. Allison and I were going for a PEP Program steering committee meeting. So we thought we’d make a long weekend out of it and take the boys and see Disney. Allison’s sister, Heather, joined us for the weekend to help watch the boys while we had our meeting and other obligations. It turned out pretty much as we thought; a harried, hurried, packed and exhausting weekend. But fun.

Friday - after a morning flight into Orlando and the usual baggage claim, rental car, hotel check-in routine we struck out for the exhibition hall to find sustenance, free stuff and people.

Allison and I each had to work an hour manning the PEP booth while the rest perused the other booths, found the pool or rested in the room. The PEP booth was inside the Bayer exhibit. I know PEP is funded by a Bayer grant but I would have been much more comfortable with PEP in its own booth.

We didn’t register for the meeting and didn’t plan on attending any sessions. We really wanted to try to make a mini-vacation. We’ve been to NHF before, attended an On the Road in Dallas, went to an inhibitor summit last year as well as our regular chapter events. So we didn’t feel too guilty about missing the sessions.

Saturday - we had our PEP breakfast meeting then off to the exhibits for a few last-minute freebies and chats with old friends. Then it was off to The Magic Kingdom. (Thank you so much Matt Angell !). Needless to say the boys had a blast. We quickly learned the strategy of the Fast Pass and hit as many rides as we possibly could. We stayed until late and got to see the electric light parade and the fireworks show. Once again I turn to James Lileks for humorous and insightful descriptions of the Disney experience (see them here, here, here and here).

Sunday - was even more hectic. We had a late flight that evening so we could do more Disney during the day. So after checking out of the hotel and a Waffle House breakfast we trekked off to Animal Kingdom. Not impressed. Mind you, our time was short so we didn’t do shows. We pretty much stuck to the rides and activities. A number of the rides were similar to Magic Kingdom rides if not out and out the same ride with a different theme. So finally after the “spin around on a raft and get soaked ride” we decided to catch a bus to the Disney-MGM park.

The boys loved the Star Wars flight simulator ride and Heather and I laughed our heads off on The Twilight Zone Tower of Terror. But all too soon we needed to head for the exit and the bus back to Animal Kingdom and our car. We had plenty of time to top off the tank, return the rental car and make our flight. Well, except that we had to make some makeshift packaging for the long Frontierland toy rifle we bought for Jack that didn’t fit into any of our luggage. That, and I also got slightly lost.

After some starts and stops, u-turns and some colorful language we found a Walgreens and fashioned some packaging from a cardboard tri-fold project board. The fact that we had to do this to get the toy rifle home was a bit dismaying. I think it’s safe to say that most people coming to Disney are coming by air. So the lack of available packaging for an undoubtedly popular item that doesn’t readily pack well for air travel (unless you brought golf club, that I alas did not) is an oversight by Disney (and they don’t overlook much). We tried to get help at several Disney shops and at customer service. They would be happy to ship it for us but no dice on ready-to-go packaging. A niche the Disney folks, I think, would be happy to exploit.

We eventually made it to the airport, returned the car, changed into fresh clothes and got to the baggage check – 5 minutes late. The bags were marked ‘Late Check In’ but were told they could still get on the plane. Next, a quick dash to wait in the security line for the obligatory screening. At some point in which we came up one driver’s license short. But because of time constraints we resigned the i.d. to being lost and just barely made the boarding of the plane.

Both boys fell asleep on the flight and we arrived in Houston without further incident. We were happy to discover that our bags made the flight with us. All except, of course, the toy gun. But even that was on the first flight the next day and it was retrieved in due course.

It was a quick, frenzied, fun weekend with a little NHF, a lot of Disney and few pics which I’ll add below. If we never make it back to Orlando I’m glad for the boys that we made the trip. If we do venture back we’ll be that much better prepared for the Disney immersion.



The Boys: excited to ride the 1970's idea of futuristic transportation - the monorail.













After too many bad jokes and puns Carrick decided to take over direction of the Jungle Cruise.












This looks like a good place to sleep after a full day at Disney.















Gosh fellas he can read.















The bury a kid attraction was quite popular.












You gotta have one with the Castle.

Monday, November 12, 2007

Bad Weekend for Buckeye State football

In a weekend that saw the Buckeyes likely drop from national title contention; the hapless, but improving, Browns couldn't hold onto a haftime lead over the Steelers. The Steelers continue to play well and fight for recognition in a conference dominated by news of the Patriots and Colts.


Veterans Day

Many people serve our great country in many ways. But it is on this day that we pay honor to those who serve and served in our armed forces. Those in uniform who stood ready, prepared to make the ultimate sacrifice for our country. God bless our Veterans.

Thursday, November 1, 2007

Halloween revisited

The boys had a fine time trick or treating. Jack had a massive haul of goodies, enough to maintain a sugar high for a month. Now the question of what to do with all that chocolate and candy. Let him eat as much as he possible can in one sitting then get sick and swear off candy for good or ration it out, extending Halloween well into next year? Or, eat it myself then cross my heart, spit in the dirt and pinkie swear I'll start going to the gym again.

Ah well, who knows?

Some better reflections on the Halloween just past come from the indispensable James Lileks. Lileks is a writer for the Minneapolis Star-Tribune. Sort of like Dave Barry or if in Houston, like Ken Hoffman but better, much better.

Here's a taste:


I had a small light in my pocket that made ghastly moans when the motion sensor was activated, one of those cheap Chinese devices that probably broadcasts lead through sound waves. It turned itself on as I walked home, and a thin weird howl came from my pocket. There’s always a moment on Halloween when you get a faint fast flash of what it was like to be six on the spooookiest of nights, when the dark and the breeze and the sudden silence – followed by a crowd of leaves fleeing down the street – reminds you what it was like, how delicious it felt to laugh at the things you feared. Even if you didn’t really fear them. Even if you didn’t know just what it was you feared.


Read the whole thing here.

Wednesday, October 31, 2007

Happy Halloween !!
















Hope it's a fun and safe night for all.

Sunday, October 28, 2007

Medicine that stops bleeding invented in Turkey

Various news agencies including Russian news agency RIA Novosti are reporting that a team of Turkish scientists have created a drug that will stop any bleeding within a few minutes. The article states the drug could be used to treat hemophilia.

I will remain, for the time, skeptical.

From the article:
"The drug, already approved by the Turkish Health Ministry, was made from a mix of herbs and plants, in particular, nettle, licorice, thyme, and grapevine."

The scientists say its an "old recipe."

Like I said, I'm skeptical and will wait for further proof.

Wednesday, October 24, 2007

Hemophilia Athletes

Here's a nice article about two boys who haven't let severe hemophilia slow them down. Its from the London Free Press in Ontario, Canada.

The opening graphs:

They play soccer and run long distance races. One's even a competitive
diver.

They ran in this year's Toronto Marathon as part of a family relay
team.

They act like normal 12-year-old kids.

Paul Travaglini and Emrik Burrows are about as normal as you can be, considering what they have to live with.

Both have severe hemophilia, a genetic disorder that results in the blood not clotting normally and making a bruise or cut a serious problem.

Read the whole thing