April 2012

Gene Therapy: Lookin’ Good!

I happily drove an hour to Westwood, Massachusetts to attend the New England Hemophilia Association’s Springfest, a gathering of local hemophilia families and the companies and medical people who serve them. It was a glorious, sunny day and a wonderful event.
Val Bias, CEO of NHF, attended to present an overview of NHF’s programs and also how funding is raised and where it is spent. Here are some stats:
• There are 57 full time and part time staff at NHF over 10 states
• There are 8 regions for HTCs now, down from 12
• NHF has a PDF you can download called “50 Steps to Cultivating Donors”
• 50% of the NHF’s board of directors are not related to hemophilia
• 52% of its budget comes from pharma
Val stressed to the audience to get involved locally, and to consider joining the NEHA board. He made a compelling plea and is an excellent speaker. Many people told me later they thought Val is a gifted presenter and a wonderful leader for our community.
Next up was a great presentation on gene therapy and new products coming soon by Dr. Ellis Neufeld of the Boston Hemophilia Center. Dr. Neufeld did an amazing job of taking highly complex material and breaking it down for us. Some highlights:
• The St. Jude gene therapy trial that has been in the news: only 6 FIX patients in trial, using “gutted” virus (no capacity to reproduce).
• Of 6, only 1 really took to it but he got higher ALT (liver
function measurement).
• Problems with the study: not enough data, immune response a threat and you can’t be retreated
as you will have an immune response.
 • Long acting factor: many companies working on this. Everyone has different half-lives; children have shorter
ones than adults. So how long is long? New FIX drugs could be 3-5 times longer acting, meaning you might treat once every 10-14 days?
• What will price be of the longer acting drugs? What is the worth for a theoretical improvement of
life? No one yet knows.

(Our next issue of PEN in May examines all these in depth: be sure to download it!)

Dr. Neufeld was very positive about current gene therapy efforts. He made us all laugh by saying that our community has been promising us gene therapy roughly in every ten year cycles, but this time, he truly feels there is a great chance we will find it.
Just before lunch, we had panel presentations from all the pharma and specialty pharmacy reps on their patient assistance programs, delivered all above the growing din from the Bar Mitzvah next door!
These were all great presentations, and we had an attentive audience. I saw so many of my friends in hemophilia, and truly enjoyed myself. I hope you can all attend a local hemophilia event and take part in your community! We had a rap session with the moms, and one among us had an 8 month old. We all remember the feelings of when our babies were diagnosed. We felt for her. Everyone rushed to welcome her, praised her for attending, and offered email addresses and phone numbers. It never fails to amaze me how tight our community is. We are friends, and family, for life.
Great Book I Just Read
Murder in the High Himalaya: Loyalty, Tragedy, and Escape from Tibet
by Jonathan Green
This is a rare book that provides multiple levels of reading, on history, ethics, exposé, culture, politics and an unforgettable story of a young girl’s perseverance, determination and tragic legacy. I love real life adventure and survival books, especially about mountain climbing and/or history, and also books about how one person can change the world. This book has it all. On September 30, 2006 a cruel and thoughtless murder of a 17-year-old Tibetan nun (just a girl truly) by Chinese border guards triggered an avalanche of scrutiny in the press, world agencies, political leaders–and in the consciences of the Western mountain climbers on a paid guided climb who watched in horror. Kelsang Namtso was trying to escape Tibet via the mountain Cho Oyu when she was gunned down in full view of climbers, one of whom videotaped it. The tape went viral—the first time human rights violations against the Tibetans had been filmed—and the rest is history. This book tells this amazing story in gripping and often exquisite prose (much like Jon Krakauer) and provides powerful parallel stories of Kelsang and the mountain climbers, which later intersect dramatically and make history (much like author Erik Larsen). You will be amazed at the courage of this 17-year-old, and at the response by those who witnessed her death. Green’s compelling narrative will teach you much about Tibet, how it has suffered under Chinese rule, and how the West has looked away until one lone video clip, still available on YouTube, shamed us into action. Green raises excellent questions that beg an inward look at our own souls, and portrays Tibetan lives without freedom that make us instantly cherish our own freedom. A must read. Four out of five stars.

Un día para recordar

Blue skies, great food, pulsating music, and surrounded by wonderful people with hemophilia. A day to remember in Puerto Rico.

Puerto Rico—the shining star of the Caribbean. Remember those commercials from the 1980s? Puerto Rico is proud of its status as a Commonwealth of the United States of America; its inhabitants have been US citizens since 1917. How many of use reading this realize that Puerto Ricans are American?

This was part of my quest this week. To return to an island and people I love, to see how hemophilia care has evolved since 1998, when I first visited, and to see how hemophilia care differs from that of the States, especially in light of the Affordable Care Act.

I arrived Thursday afternoon and spent the evening with Osvaldo, a young man with hemophilia, and his girlfriend. We all had dinner together and listened to him share his story of having hemophilia, an inhibitor and limited access to product. Charming and intelligent, Osvaldo has suffered but also persevered: he is college educated and owns his own company!

On Friday I met with the HTC staff at the Centro Medical to get an update on hemophilia treatment. There are an estimated 250 persons with hemophilia on the island… but no one really knows. Like the States, there isn’t yet a national database. The facilities are good and the staff very inquisitive and kind.

On Saturday, Johnny and Tammy Marquez, the husband and wife team who head up the Association Puertoriqueña de Hemofilia (APH), and I traveled about the island to meet a few hemophilia families. We traveled two hours to Salinas to meet one family and another half hour to meet one more in Santa Isabel. Wonderful families; fascinating insights on PR hemophilia care!

We ended our day today by attending the annual meeting of the APH. It was packed, and we had a full agenda with a speaker on dental care, chiropractic care and later an open forum which became quite lively with a debate over whether hemophilia care in Centro
Medico was up to standards. The day ended with a great motivational speaker, lots of hugs, and good feelings all around. People with hemophilia have community, muy fuerte.

So what did I make out of all this? You’ll just have to wait… until August when we publish a full report in our article on Puerto Rico in PEN. Sign up now to receive it. And in the meantime, visit https://www.blog.kelleycom.com/2011/01/la-vida-hemofilia-visit-to-puerto-rico.html to read about our visit last year.

Good Book I Just Read
A Dog’s Purpose by W. Bruce Cameron

Maudlin, unabashedly sentimental, charming, simplistic, sugary sweet… I was at first wondering where this book was going, but then got sucked in by the sentiments, and was in a puddle of tears by the end. This book describes a dog’s life (lives, actually) as told through a dog’s eyes. The main character lives through four lives, reincarnated after each demise as a puppy, aware that he is reincarnated and wondering in each life: what is going on? What am I supposed to be and be doing? When does this end? It makes the reader wonder too, as the book is a bit disjointed at first and it may take a bit to figure out Cameron’s style. If you are a dog lover like me, you will enjoy much of the free association by the dog, especially whenever a cat appears; very clever and funny. The dog is feral in his first life, trying to survive and eventually is euthanized; reincarnated, he comes back as a lab, and his real story begins in earnest when he becomes bonded to a boy. No spoilers here, but the dog becomes smarter and more self-aware, and as a reader you share the dog’s unswerving loyalty to humans and his unending desire to serve and please, despite humans’ baffling behavior. By the end of the book you yearn for your own current dog to hug, or recall your childhood dog with such strong emotions, I can only think of Toy Story 3 as something in comparison. To fairly critique (as a professional editor) you must disregard or overlook the inconsistencies: the dog understands peeing, vomiting, crying but doesn’t have a word for his own natural instinct to mate? The dog refers to chopper, car, truck, cage, various power tools, collar, leash, swing… but when his boy’s mother removes her ring and throws it into the pond he doesn’t know what that little round thing is called? The dog conveniently doesn’t know names of things whenever it’s a Kodak moment. But just keep reading and don’t be critical and you will enjoy it. I hate being manipulated by purple prose but this was overall a wonderful book, because I have loved all my dogs and currently inherited a puppy I’ve only been complaining about. No more. I’ll love him with all my heart too. Three/five stars.

World Hemophilia Day

Today is the birth day of Frank Schnabel, a California businessman who over 60 years ago founded the World Federation of Hemophilia. All across the globe today, the hemophilia community celebrates unity, that we are one family united by a protein deficiency that causes prolonged bleeding and suffering.

We are divided only in access to treatment. Up to 75% of the hemophilia global community has no access to factor. We are trying to close the “gap,” as the WFH puts it, by donating factor medicine to those in need.

Help continue to unite the world hemophilia community. Donate unused or unwanted factor to Project SHARE, or sponsor a child with hemophilia in poverty through Save One Life. Give back to those in need, and to honor those who have gone before us.


A Blessed Easter

For this important holiday, I’m wishing all my Christian colleagues, friends and family a very happy and blessed Easter!

Great Book I Just Read
Holy Ground: Walking with Jesus as a Former Catholic by Chris Castaldo

This is a great book to read at Easter. First time author Chris Castaldo, Director of the Ministry of Gospel Renewal for the Billy Graham Center at Wheaton College, has written a witty, practical and down-to-earth book that simultaneously explains the differences between the Protestant and Catholic faiths, while also providing a bridge to help each side understand and appreciate one another better. Targeted primarily for Evangelicals who wish to convert Catholics, but speaking to anyone wishing to deepen their understanding, Chris offers sage advice for why previous conversion tactics may not have worked, and how Evangelicals can conduct themselves to present their faith more clearly. Chris points out two main differences between the faiths, the direct authenticity of the Bible as the Word of God, and grace versus guilt. As the title states Chris himself was raised Catholic and later became Protestant, so offers both perspectives in this stimulating book. Chris also happens to be the father of a child with hemophilia, and mentions the role that faith plays in his life in dealing with his son’s diagnosis.

I know this well, because Chris and I met just 11 days after his son was born, and have been friends ever since. So yes, my book review is a slight bit biased! I loved it. I found this book a marvelous bridge between two faiths that may have more in common than they think. With the world afire with religious wars of words, in US politics and overseas, it’s refreshing and downright hopeful that such a book comes along. Well done, Chris! Four/five stars.

Aloha Pulse on the Road!

It’s appropriate to start off Pulse on the Road in 2012 near one of our nation’s most historical sites, Pearl Harbor, as the nation this past week holds historical debates on healthcare. But let’s not predict disaster!

Pulse on the Road welcomed 35 families of the newly founded Hawaiian Hemophilia Foundation, headed by Jennifer Chun, mother of five, including two sons with hemophilia. Jenn’s done a super job of bringing the community together to learn, support one another and network.

The day began with a presentation by Jennifer Dumont of Biogen Idec, sharing the different sciences behind long-acting factor and the various clinical studies by various companies. The race is on!

The Bayer Living With Hemophilia program was presented by Kim Shaeffer, nurse at University of Davis, California and covered all aspects of raising a child with hemophilia, including nutrition and dental care.

After lunch came Pulse on the Road, our three-hour insurance symposium. This POTR started with my historical look at healthcare in hemophilia, how the US healthcare system has treated us post HIV-era (good!) and how and why it has evolved to treat us as a generic chronic disorder–not good. Next, Michelle Rice, public policy director at NHF, shared ideas on how to compare and contrast healthcare plans with your insurer, using the NHF Insurance Toolkit. Last, Jim Romano of PSI concluded by reviewing what the Affordable Care Act is, and what Hawaii can expect from healthcare reform.

We had great audience interaction with a very informal format. In fact, so much of everything in Hawaii is informal and laid back–a really nice change of pace!

The weather was great and following yesterday’s program, Michelle and I today were able to see the number one attraction in Hawaii: Pearl Harbor. What an excellent and tastefully done museum, film and viewing of the Arizona. It was breathtaking and sobering. It makes you all at once feel proud to be an American, and we were respectfully silent, knowing that beneath our feet, submerged in the shallow waters lie the bodies of the sailors who perished so quickly that fateful day, December 7, 1941.

If you want to watch a great movie on Pearl Harbor, watch “Tora! Tora! Tora!”

And keep watching the news about the Supreme Court’s hearings on the Affordable Care Act; what is decided will affect us all for a long time to come.

(Thanks to Baxter for funding Pulse on the Road and to the Hawaiian Hemophilia Foundation for hosting it!)

Great Book I Just Read

Issac’s Storm by Erik Larson
Reading about the greatest natural disaster in the US seemed appropriate after seeing Pearl Harbor today. On September 8, 1900, the seaside city of Galveston, Texas was hit with the worst natural disaster that had yet ever hit the US, a colossal hurricane that muscled its way through the Caribbean Sea. Galveston was an up and coming town that wanted to rival New Orleans one day. Monitoring the weather was Issac Cline, a seasoned and educated veteran of the US weather service. But hubris also ruled; despite the many warnings in the sky and on the ground, most notably from the Cubans, the world’s weather experts whom the Americans dismissed as inferior, the storm rolled in with a devastation of Biblical proportions. This story, masterfully told, details the players, the average folks and families, and the hurricane itself in a suspenseful and heartbreaking tale. Over 6,000 men, women and children died that fateful day; Galveston never recovered, and when oil was discovered near Houston shortly afterward, Galveston became the playground of the Houston rich. Why this book has not made it to Hollywood is beyond me: it has everything needed for a blockbuster including Issac’s petty rivalry with his brother, also a weatherman, who rightly predicted the storm would be a killer. Four/five stars.

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