January 2012

What on earth are you doing for heaven’s sake?

Laurie Kelley January 30, 2012

Remember this slogan? It was found on bumper stickers in the 70s, from what I recall. Maybe this was my first attempt at editing: were they trying to say, “What on earth are you doing, for heaven’s sake?” Like, “Are you crazy or something?” Or maybe, “What on earth are you doing for Heaven’s sake?” as in, what’s your purpose here on earth before you go to Nirvana, Heaven or get reincarnated as an insect?

I thought of this bumper sticker when I read the essay submitted to me by friend and colleague Adriana Hendersen. She is a one-woman agent for change in Romania, and has changed the lives of dozens of boys forever. This is excerpted from the November issue of my newsletter PEN. In case you missed it, read on…

Why Am I Here?

“Why are we here?” is a philosophical question concerning the purpose of life.

“I have asked myself many times why I am here, but with the emphasis on here, in the US.

“In 1970, I seemed destined for a different life, in Romania. My father was sentenced to spend his life in a communist prison for criticizing the government’s decision to deny the family the right to emigrate; and we, his children, were supposed to be sent to reeducation school. But we didn’t know that our situation had been receiving international attention. Following pressure from the United Nations and various churches, the Romanian government asked us to leave Romania immediately. It was a magnificent, magical, miraculous exit. We were the first family in Romania to leave legally, not only with a passport for emigrants, but also carrying an American green card.

“As a young girl I never once looked back, or even thought about what I had left behind. I thought I would forget Romania, and hoped I would forgive. I pursued everything the US offered: freedom and opportunity. I had a wonderful family, a big house in the suburb, cars, trips around the world, a closet full of designer clothes. Most important, my family was healthy. Yet it felt like something was missing.

“When the Iron Curtain fell in 1989 and images of the harsh realities in Romania besieged the world, I could not ignore my country and people anymore. The question “why am I here” started to nag me.

“As a child, all I could think of was survival. Now, with my newfound freedom came a desire to succeed. When success wasn’t enough, I started to look for significance. I wanted to give back, to make a difference, to acknowledge the blessings that were bestowed on me and possibly see if there was a reason for my being here.

“I was at a loss about what I could do, and for years I looked for some cause I could identify with. Then I met a woman who was looking for medicine to help a boy with hemophilia in Romania; he needed corrective foot surgery to walk. I barely knew anything about hemophilia, and the little I knew was mostly inaccurate. After a quick and shocking lesson on hemophilia, I began a quest to find the miracle medicine. It wasn’t easy, and the more I searched, the more I lost hope. I made hundreds of phone calls, all over the world, trying to source any kind of donation. It was a test of endurance and tenacity. I had one phone call left to make, the call to Project SHARE. They immediately shipped the factor, and the rest is history. That was more than 10 years ago.

“Since then, I know why I am here. The boy had surgery and his wish was granted: he is now walking. That’s when S.T.A.R. (Start Thinking About Romanian) Children Relief was born. S.T.A.R. is a multipurpose organization with an emphasis on healthcare and a focus on blood disorders. Through S.T.A.R.’s efforts and donated factor concentrate, many Romanian boys and adults with hemophilia have had their lives improved or spared. On World Hemophilia Day, April 17, 2004, S.T.A.R. organized the first-ever hemophilia symposium in Romania. And S.T.A.R. organizes and hosts Camp Ray of Hope, in its sixth consecutive year this past summer, the only camp for children with hemophilia in Romania.

“S.T.A.R. is also Save One Life’s partner for Romania. We have 59 children and adults with hemophilia sponsored through Save One Life. I know most of the beneficiaries personally, and have visited them at home or seen them at camp where they play with carefree enjoyment. I get to see them smile and hear them laugh. It feels awesome to be so intimately and personally involved.

“I never thought I would be involved in charity or volunteer work. I don’t have the personality. I am shy, withdrawn, introverted, not the type that would organize international conferences and fundraise for summer camps. It’s said that we, in the nonprofit world, change other people’s lives. That’s true, but in the process, our lives change too. We have a purpose, our life has meaning, we do things we thought we could never do. That’s a terrific feeling! We give a little, but we get a lot back.

“I know—Romania is close to my heart, and I have a vested interest in helping my people. But to anyone who, like me, is searching and wondering if there is something more than just the fleeting pleasures in life: if you want to make a difference or improve a life, while you improve your own, consider sponsoring a child. Look at the Save One Life website, where many with hemophilia are waiting to be sponsored. Pick a country, pick a child. Put a sparkle in those eyes that look so hauntingly and sadly at the lens. Bring a smile and a chance for a better life. It’s a small gesture that will bring priceless rewards.
I know why I am here. Do you?”

Maybe the bumper sticker needs to simply say: “I know why I am here. Do you?”

Adriana Henderson is founder and president of S.T.A.R. Children Relief, a nonprofit dedicated to helping Romanian children in need. She was born in Romania and immigrated to the US, where she has lived for the past 40 years. She is a graduate of UCLA, and lives in North Carolina with her husband Tom, who often helps with her charitable work. They have two daughters. Visit www.starchildrenrelief.org

So Long! Extended Haf-Life Factor Sets the Stage

Laurie Kelley January 23, 2012

You’ve probably heard about Biogen Idec’s clinical studies of factor with extended half-lives. They aren’t the only ones investigating. CSL Behring just announced that they are starting a global phase II/III, multi-center study to assess the safety, efficacy and pharmacokinetics of recombinant fusion protein.

This recombinant fusion protein links coagulation factor IX with recombinant albumin, called “rIX-FP.” rIX-FP is indicated for the prophylaxis and treatment of bleeding episodes in patients with factor IX deficiency.

Of course, extended half-life factor means potentially fewer injections for patients, and may enable or enhance prophylactic treatment, improving quality of life for patients.

So many patients worldwide have been hoping for gene therapy–a “cure”–but it seems that the next great thing that will come along just might be extended half-life factor. We’re not sure yet which company will deliver first, but keep watching. These are exciting times and the long acting factor race is on!

Book I Just Read
The Hunger Games by Suzanne Collins

This best selling novel takes place in post-apocalyptic America, now called–for no reason disclosed– Panem. In fact, there are a lot of things that happen for no apparent reason in this book. Quick synopsis: the powers that be dictate that one teen from every district must compete to the death in the Hunger Games, in retribution for an uprising that happened a long time ago. To the victor goes fame and food to their district. The whole event is televised–reality TV where kids get killed. The heroine narrates the story, and has a pretty flat tone throughout. Think “The Truman Show” meets “Mad Max Beyond Thunderdome” meets The Olympics meets “Rollerball” and you get a great idea what the book is about. It’s not terribly original; the writing is mediocre and the story pretty predictable, characters shallow with no depth of thought or emotion. After killing her first kid, arrow through the throat, the 16-year-old narrator with the flat affect seems more concerned about whether the boy she is with loves her. Someone recommended this to me, but neglected to tell me it’s teen romance fiction, with a lot of killing. Read it if you want to kill time; it’s a few steps up from a comic book. If you want fantasy with substance and depth, try Tolkein. The book seems to be meant for a movie, and reads like a screenplay; I somehow think the movie will be better as a result, coming in March. Two stars/five.

Factor: Lost in Space

Laurie Kelley January 16, 2012

My friend and colleague, Richard Atwood, is somehow always able to muster up some amazing research on hemophilia. Here’s one I bet you never heard of…. “Space AHF: A Treatment of Hemophilia.”

Richard writes: “In 1981, Frank Schnabel, President of the World Federation of Hemophilia in Montreal, Quebec, Canada, reported on a letter sent by Dr. Weiss at McDonnell-Douglas. The company had developed facilities to reproduce certain medicines in space that could not be perfectly produced on earth. The space environment was characterized by weightlessness, vacuum, isolation, and almost no thermal convection.

“McDonnell-Douglas conducted a feasibility study exploring the potential production in space of anti-hemophilic factor VIII. Blood products could be improved by removal of noxious contaminates using electrophoresis (CFE). The space electrophoresis process allowed continuous purification and collection of plasma proteins, thus reducing total blood needs and increasing quantities of biological materials available for treatment. The extraneous protein in commercially available AHF could be removed and the amount of AHF processed in space was not limited as it was at ground level. The electrophoresis hardware was scheduled to be tested on a shuttle Spacelab flight in early 1983.”

Well, that was then, this is now. We have terrific products that help our hemophilia patients stop bleeding instantly. Back in 1981, there were a limited number of products, no recombinant, and of course, a deadly virus about to strike.

I just visited last week the Titan II Missile Museum, and had a close-up look at the very last Titan missile (minus the nuclear warhead) in its silo, which got me thinking about all things space. We have exciting new hemophilia products coming down the road, and it’s unlikely any will be produced in space!

Reference: Schnabel F., 1981 Introduction: The promise and potential of the eighties. Haemostasis 10 (Suppl 1): 109-11.

Great Book I Just Read
Soul of a New Machine by Tracy Kidder (hardcopy)

This was a great compliment after having just read Steve Jobs. In 1978, super and mini-computers were the mainstay of the aerospace and defense industry; there was no such thing as a “personal computer” (microcomputer) or laptop. This book, for which author Tracy Kidder won a Pulitzer Prize, chronicles, like a great novel, the development of a 32-bit mini-computer Eclipse, that would be the fastest on the market. With the gruff and brittle Tom West at the helm of an almost secret Data General crew working on a project code-named “Eagle,” this is an insider’s look at project development, the birth of a new computer. You feel the tension, the sacrifices, the work ethic; it is agonizing. This is back when high-tech workers could say “I’m not in it for the money,” and would freely work 80-hour weeks, so devoted to their craft are they. The section where the team is debugging the computer takes you through their seemingly endless nights—and nightmares. The book is dated for sure, and goes into more detail than you could ever want about how computers work, but it truly is fascinating! How far we have come. You watch as these men almost self-destruct in their heroic efforts, and now we take for granted the ease and simplicity of our laptops and wireless creations. This all takes place in Westborough, Mass., a place I know well, as I worked not far from it in the mid-1980s. Tom West, the enigmatic project leader, just died in May 2011, and though he was reclusive, is immortalized in this gripping book. Four/five stars.

Inhibitor Camp This April

Laurie Kelley January 9, 2012

Camp is coming early this year!

US hemophilia families with inhibitors are invited to apply for the third Inhibitor Family Camp weekend to be held April 13-15, 2012 at The Painted Turtle Camp in Lake Hughes, CA.

This program, run by Comprehensive Health Education Services (CHES) and sponsored by Novo Nordisk, is designed for families with a child age 6‐19 with an active inhibitor. These camps are special: children with inhibitors often report that they cannot participate in many of the activities offered at a traditional hemophilia camp due to the threat of injury. Or that if they do choose to participate, they may spend the rest of the week in a wheelchair watching from the sidelines.

Inhibitor Family Camp provides these children and their families an opportunity to come together with their true peers in the hemophilia community.

Space is limited so all registration materials must be completed in full by February 17, 2012. There is no cost to families associated with Inhibitor Family Camp. For additional information, please call Comprehensive Health Education Services at 877-749-2437 or visit www.inhibitorfamilycamp.org.

Great Book I Just Read
Steve Jobs by Walter Issacson (Kindle version)

This is an incredible read, quite possibly the best book I read in 2011. Not a perfect book, however, because there are gaps, almost as if Issacson rushed to get it out following the death of Jobs on October 5. Issacson was asked by Jobs to write his biography, and despite Jobs’s well known control issues, gave full control to Issacson, and engaged in 40 interviews with him. Issacson also interviewed Jobs’s family, coworkers, enemies, friends and wife.

This 600 page tome traces Jobs from his birth, adoption, and early childhood years all the way through his stint at Reed College, dropping out, founding Apple in his father’s garage, and the impact his adoptive father had on his curiosity about all things electrical and his father’s love of perfect workmanship, as we see in Apple products today. It’s almost two books in one: a fascinating account of the rise of the personal computer industry in Silicon Valley, and the infamous competition with Bill Gates and Microsoft (hard to believe Jobs and Gates were born in the same state, same year!). For those of us who lived through these years, it brings back memories and completes a story. For the new generation who takes these marvelous machines for granted, this history is a must read.

The other “book” is a searing account of Jobs’s psyche: his infantile approach to managing people, his tirades, temper tantrums, his eating disorders, control issues, his obsessive perfectionism, his total lack of empathy, his need to squash others’ self-esteem. If there is one psychological term you can hang on him, it’s narcissism. He was a genius, a visionary who saw things before they were built, who knew exactly what he wanted because he could already see it before him when no one else could. He invented an industry that has completely changed people’s lives and the course of history. He didn’t do market research to find out what the customer wanted; he knew what people wanted. He created need. But he left a scarred and brutalized wake, and no doubt his children will tell all one day. I have been an Apple customer since the 1980s, from day 1. I love the products, the marketing, the beauty of the machines, the integrated approach to everything Apple makes. They make intuitive sense, and Jobs knew this. He knew so much, saw so much, and had he lived, his impact would have been even more tremendous. I couldn’t help but feel sorry for his children, and have no doubt that his cancer originated from his chronic high stress levels and internal obsessions. When you go into an Apple store next time, look at the Restroom sign. The grey color took a team, including Jobs himself, 30 minutes to decide what shade of grey. Jobs involved himself at every level. When you live your entire life to that extreme, it takes its toll internally. What price genius? This book seems to mournfully ask that, while at the same time, marveling at an extraordinary man who led a great team that changed the world. The frustrating part comes when even Issacson, for all he interviewed Jobs 40 times, still could not but scratch the surface was what drove this man. No doubt, Jobs wouldn’t let him in; maybe no one got in. Five/five stars.

Happy New Year With Prophy News

Laurie Kelley January 2, 2012

All is quiet on New Year’s Day here, with the warmest winter in history in the Northeast. My grass needs to be cut!

Here’s some good news to start the year off…. Baxter’s Advate has been FDA-approved for routine prophylaxis in both children and adults, the only factor product to be so licensed.

In a Phase IV prophylaxis study, funded by Baxter, researchers, led by Dr. Len Valentino of RUSH University, found that prophy on Advate reduced bleeding episodes from 44 to only 1 in a year: great news and findings.

From its press release, Baxter reports that “for the prophylaxis regimen to prevent or reduce frequency of bleeding episodes, Advate dosing of three to four times weekly (between 20 to 40 international units of factor VIII per kg body weight every other day) may be used. Alternatively, an every third day dosing regimen targeted to maintain FVIII through levels greater than or equal to one percent may be employed.”

Of course, most parents and patients know that prophy with many products has been around for a long time. But it’s important for the prescribing doctor to feel comfortable prescribing a product with FDA approval. It takes a long time and money to investigate these products and their treatments, so it’s a cause for celebration when a Phase IV study is complete.

And so is New Year! Wishing you all a great year!

Great Book I Just Read
Lucky Ears: The True Story of Ben Kuroki, World War II Hero

Written for young readers, you can easily polish this book off in 30 minutes, but the images will stay with you a lifetime. Ben Kuroki was born a Nebraska native, of Japanese descent, and suffered through challenges like the horrible Dust Bowl, the Great Depression, and poverty. But he was deemed “lucky” by his parents for the little dimples he had on his ears. He never experienced racism until he enlisted in WWII following the attack on Pearl Harbor. He had no sympathy for the country of his ancestors yet was suddenly treated as inferior. He finally was allowed to enlist, and became one of the most famous bombardiers in US history! He had more bombing runs than almost any one else, so great was his love for the US. Despite the racism, he became a US hero, and was honored by President George W. Bush. He escaped many close encounters with death, and you close the book marveling indeed at his luck, and his wonderful career and patriotism. Five/five stars!

And this makes 35 books I read this year, achieving my goal!