A Christmas Carol

Save One Life’s Gala: Honoring the Massies

Save One Life honored the Massie family Thursday night at its second annual gala. About 150 guests arrived at the Commons, in Topsfield, Massachusetts under sunny skies to enjoy cocktails, dinner, business networking and to honor the people who have helped make Save One Life so special.
Nelson Escoto, president of Bleeding Disorder Resource Network, received Sponsor of the Year award, for supporting a sponsorship for each new patient he receives on his service. The lobby wall at his office is covered with photos of their beneficiaries. Before the night was over, Nelson would sponsor 5 more children with hemophilia!

The elegant dining room
Laurie Kelley as host
Guests enjoy the good New England weather
The Massie family
Bob Massie accepts his award

Adriana Henderson, founder of STAR Children Relief, won an award for Program Partner of the Year for her work in Romania, where Save One Life has many beneficiaries. Her photos of the children brought tears to the eyes of many in the audience. Laurie Kelley made a special plea to the audience to help raise an additional $2,500 to meet the cost of camp this year, only a few weeks away. By evening’s end, all the funds were raised. Sasha Zatyrka, director of the Colburn-Keenan Foundation, presented Adriana with a $1,000 check.
The highlight of the evening was duo awards presented to mother-son team of Suzanne and Bob Massie. Bob Massie was given an Inspiration Award first, for his life of public service, particularly in representing hemophilia to the public. Bob’s newest book, A Song in the Night, a Memoir of Resilience, was just released and Doubleday Books generously donated copies for each guest. Bob, a gifted orator, share a story about his childhood to highlight how his mother Suzanne kept his faith and hope alive that he would be okay despite his hemophilia. Bob has a long list of accomplishments that would exhaust an average person, let alone one with multiple chronic illnesses and frequent setbacks. He most recently ran for US Senate, and now is president of a nonprofit.

Attendees enjoy dinner
Nelson Escoto, president of BDRN, accepting his award as Sponsor of the Year
Sasha Zatyrka presents a $1,000 check from the ColburnKeenan Foundation for the Romanian Camp Ray of Hope
Adriana Henderson accepts her award
Bob and Suzanne Massie autograph their books
Suzanne Massie is co-author of Journey, the first book about hemophilia in America, which poignantly details life with a child with hemophilia in the 1960s. She became an expert on Russia after helping research and write Nicholas and Alexandra. She went on to author several books about Russia, still in print today. She created the Firebird Foundation to help youths with hemophilia in St. Petersburg, Russia, and as Laurie pointed out, was the inspiration for Save One Life. When Laurie worked with Suzanne on the Firebird Foundation, she saw first-hand what one woman could do to change the lives of so many in need. Suzanne has a new book in the works, about her years advising president Ronald Regan on Russia as the end of the Cold War was coming.
In a warm and gracious speech, this elegant lady still showed the passion which drove her to accomplish so much and change the lives of so many. The audience was mesmerized and gave her a standing ovation at its end. It was a great privilege to have the ever-active Massies with Save One Life for the evening, and to present such well-deserved awards. The evening ended with book signings, and the silent auction, and many, many happy memories
Laurie presents the Inspiration Award to Suzanne Massie, author, humanitarian, presidential advisor
and pioneer in hemophilia

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.

Happy New Year With Prophy News

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!

Gene Therapy… at Last?

I spent part of last week in Europe with members from our global hemophilia community, and before our meetings, the talk was about the news release concerning gene therapy for hemophilia.

Remember that?

We all want to roll our eyes when we hear talk about gene therapy. Those of us old enough to remember heard that gene therapy was right around the corner back in the 1990s. There were a lot of clinical studies and experiments happening, and it did seem feasible. In fact, PEN (my newsletter) included the first article on the world’s first gene therapy trial, by TKT right here in Cambridge, Massachusetts.

Then in 1999, Jesse Gelsinger, an 18 year old, died in a clinical trial for gene therapy. It wasn’t for hemophilia, but it sent shock waves to all gene therapy trials. Fearing lawsuits and other deaths, many shut down. And gene therapy has gone sort of underground since.

So it takes guts to come out and declare that a single treatment with gene therapy has been shown to raise factor IX levels blood clotting factor. But it’s been declared in the prestigious New England Journal of Medicine, and reported at the American Society of Hematology in San Diego last week.

The study is taking place at the University College London Cancer Institute and St. Jude Children’s Research Hospital in Memphis, Tennessee. How does it work? It uses a viral “vector,” designed to target the liver specifically. The human gene that makes factor IX is inserted into a virus (viruses are very, very good at reproducing quickly in the body, as we all know). The viral vector containing the human gene is delivered to the patient’s liver cells. These are the cells capable of producing clotting factors.

The results have been promising. It’s reported that four of the six trial participants have stopped routine treatment and remain free of spontaneous bleeding. The other two infused once every 10 days to two weeks, and now from two to three times a month!

Right now researchers must look for any side effects: we are dealing with a virus, after all. And indeed, one high-dose patient developed elevated levels of “transaminases,” which could indicate possible liver damage.

Researchers plan to enroll more patients and see where this goes. While nothing is certain yet, this looks promising and has the whole community watching.

Great Book I Just Read
Ethan Frome by Edith Wharton

Nature abounds in this book, and as a backdrop, Wharton uses its lighting, temperature, seasons and harsh beauty to mirror the events in the lives of a few humans in an 1800s Massachusetts town. This is the portrait of a man in constant emotional pain. Ethan is baffled by his life’s path, doesn’t know how to improve his life, and plods through his daily chores waiting for something, eventually torn by desire and duty. Ethan seems resigned to eke out a living on a failing farm, with his hypochondriac wife Zeena. For the past year, hope blossoms like spring in the form of Zeena’s younger cousin Mattie, sent to aid the ever-ill Zeena. Her sparkling demeanor and warmth contrast starkly to Zeena’s self-absorbed and cold manner. Ethan finds himself slowly captivated by Mattie, in ways he does not quite fathom, adeptly documented by Wharton, and then falls totally and irrationally in love. With Zeena’s constant interference, and manipulative presence, it’s a doomed love. Eventually, pressure builds: Zeena’s imagined health concerns worsen as she suspects the growing bond between her cousin and husband. Mattie is forced to leave, and Ethan feels life, love and meaning slipping away. The story is simple yet extremely sophisticated psychologically, the characters powerful. This quick read is a complex and deft weaving of personalities, hopes, dreams, longing, regret and circumstances that eventually force a decision—though the ending is shocking, almost gothic. And the pervading sense of nature helps to create a foreboding and destiny, all too terrible to conceive. Five stars.

PS: Edith Wharton was the first woman to win the Pulitzer Prize for literature. And the movie, starring Liam Neeson, is excellent.

Recombinant VWD Product on the Horizon?

I just received word that Baxter has started Phase III of its recombinant VWD product. The investigational product is BAX 111, the first recombinant von Willebrand product in clinical development.

Currently, we have Humate-P (manufactured by CSL Behring) and wilate (manufactured by Octapharma), which are both plasma-derived. Wilate was the first product indicated for the treatment of bleeds in VWD patients. Humate-P has been the standard for years in the US. But don’t forget Alphanate (from Grifols), originally for treating hemophilia A bleeds, and now indicated for treatment of VWD patients (FDA-approved for surgical and/or invasive procedures in certain patients with VWD). Not FDA-indicated but sometimes effective is Koate-DVI (Grifols, distributed by Kedrion in US), because it has VWD in it, along with FVIII.

No one can say that America doesn’t have choice!

It’s always exciting when there are new products on the horizon. Please remember too that all US FDA-approved drugs are considered both safe and effective for treatment of bleeding for VWD patients.

The Baxter study will assess a minimum of 36 patients in trial sites in the United States, Canada, Europe, Australia, Japan and India. Information about the trial including enrollment is available at www.clinicaltrials.gov or by calling 1-805-372-3322.

I am quite sure, that when and if the product hits the market, Baxter will think of a catchy name for BAX 111!

For more info, call Marie Kennedy, (805) 372-3543– I know her and she is a very trusted source and nice person!

Interesting Book I Just Read
The Nine Rooms of Happiness
by Lucy Danziger and Catherine Birndorf

This perky, feel-good book uses a clever metaphor for getting women to think about their life, stress level and aspirations: think of your life as a house, and each segment of your emotional life is a room. Basement (memories, childhood), Family room (family), living room (friends, relationships), bathroom (self-esteem, health) etc. In which room are you? Where do you have the most problems? Is your bathroom too close to your family room? In a sense, the authors are asking us to compartmentalize, ironically a task usually associated with men. The authors use real life vignettes from women of varying backgrounds and situations to reveal some universal anxieties women share, and then applying the “house” metaphor to show how they can get unstuck from their unhappy situation, and move into a different room, and therefore happiness.

Sound simplistic? It is. The book is okay for those new to the self-help genre and studying relationship and introspection; I think they will read it and come away feeling understood, optimistic and less alone with their unhappiness. But to someone who is well versed in more serious relationship books (Harriet Learner has excellent ones), this is psycho-lite. The book is based on many presuppositions: woman must have female friends, you must keep old friends in order to be happy (even if they drive you batty?), women are prone to anxieties, women are unhappy. I found the book putting much blame on women for their condition, which I am sure the authors didn’t intend! For example, why should a woman be made to feel in “denial” about getting older, or narcissistic because she tires of her long-time friends who are aging, speaking about grave plots, and do nothing to engage in life, while she is full of spirit and wants to take on life and adventures? Why is that a “problem,” as the authors clearly state? They insist she needs to keep these stick-in-the-mud friends while cultivating new friends. What is she doesn’t have time? What if some people really are just jerks and not just a result of a relationship problem stemming from “regression,” or “transference” or “denial”? The authors seem to have a psycho answer for everything, and not a street-smart, pragmatic way of viewing relationships.

A cookie cutter response to each vignette wears thin, as do the clichés, which run rampant (oops, there I just did it) in the book. “To have a relationship, you first have to relate” –really? The model is a clever idea, using a house, and no doubt some will feel helped by this book. It’s gotten pretty mixed reviews. Maybe good for novices, but when you finish this, move on to some serious relationship books, as this one is pop-psychology, cutesy and sends mixed messages. If you challenge the presuppositions, half the book is sunk. Two stars.

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