Local Hemophilia Families Meet the Manufacturers

Local Boston hemophilia families
tour a manufacturing plant

I was thrilled last week to invite a group of local hemophilia families to the Pfizer Andover manufacturing plant, where BeneFIX,a  recombinant, third-generation blood-clotting medicine, is made. Andover is only 40 minutes from my house; imagine that this amazing medicine is practically made in my neighborhood! It was a wonderful opportunity for the scientists and researchers there to meet real families, and for the families to see just how blood-clotting products are made.

Carolyn shares her story
We were greeted by Bob Duane, Senior Director, Site Quality and Compliance. Families were briefed about their tour, which would be a closed off tour–we didn’t need to get into special outfits to protect the manufacturing process from us and any germs we may have been carrying. But we would get to see how factor is made!
Tom Porter of Pfizer with families
First, we met with a large group of employees, and heard a presentation by Tom Porter, PhD, Senior Director, Analytical Research & Development, Global Biologics, who gave an overview about how factor was produced. He was so enthused about his work; it was refreshing and delightful! He reviewed the history of hemophilia innovation of products from Pfizer, including creating the first recombinant factor VIII product in 1992, which was then licensed to Baxter. I had actually forgotten that little piece of history. 

Manufacturing vats
Then each family was invited to stand up and speak a little about their experience with hemophilia. Bob wondered if the families would be comfortable with that and we all laughed—hemophilia families usually have no problem speaking up!
Shane is fascinated by the equipment!
Wendy spoke about having an 18-year-old; Allie spoke about having two with hemophilia (“I win!” she quipped!); I spoke about having a transitioning 25-year-old and our insurance concerns for all in his age group; Carolyn shared about having an 18-month-old; and Diane informed the group that her baby was just diagnosed with an inhibitor. Most special was the speech by Shane, a 12-year-old with hemophilia A, who related that he is learning how to infuse better to save wear and tear on his veins. 
Explaining the process

After a lovely lunch, during which the families were able to sit casually with the researchers and scientists, the group split into two and began their tours. We saw the vats containing the medium in which the Chinese hamster ovary cells produce human factor; learned how it is separated from the medium and then harvested. In the end, product is shipped to Spain to be finished, lyophilized and packaged, ready to be sold. The sheer level of intricacies involved in each step was a bit overwhelming. We walked by many laboratories where staff worked diligently. Everything is breathtakingly clean; most tasks are automated to reduce the risk of human error. We walked on many floors, levels and peered in through large glass windows to see the giant vats where product is made. The families were stunned, at the number of employees required to make product, at the level of safety controls and at the amount of money required to make product.

“It is really fun to see how factor is made and all the work that goes into it. I will definitely think of that when I infuse now,” Shane comented.  

Bob Duane of Pfizer explains
how factor is made

“The tour of the Pfizer facility opened my eyes to the extremely long process involved in making factor,” said mother Kathy Secinaro. “It became clear that the staff there are dedicated to research and product safety. In addition to that, they truly showed they care about our community and want to know more about living with hemophilia. I never expected such a genuinely warm welcome.  Others should be strongly encouraged to do a similar tour. I hope to do it again.”

And what did the employees think about the families?

“Yesterday was the best day I have ever had at Pfizer.  I received far more than I gave.”    “I was touched and was amazed at the strength that was displayed by [the families].”   “I was truly inspired by their stories and had such a good feeling all day. I’m looking forward to the next visit and other visits like this.”   “Having patients and their families on site really drives home the importance of our job. It never fails to touch me when I hear them speak. We do great work here. Work that keeps people alive. We sometimes forget.”

Other manufacturers offer tours of their facilities, too. I’ve toured the Bayer plant in Berkeley, and know other families who have seen the Grifols plant in Los Angeles, and the CLS Behring plant in Kankakee, Illinois. Ask your local rep if you can see their plant, and learn how factor is made.

And read the August issue of PEN, coming to your mailbox soon; we review all factor products and manufacturers, and chart out the new products coming in the pipeline.

Thanks to Gail McCarthy, our local Pfizer rep, for this invitation, to the families who took a day off to join us, to the Pfizer staff who made this such a pleasant and educational day and who care enough to meet and learn about our very special community.

Good Book I Just Read
The Doors Unhinged: Jim Morrison’s Legacy Goes on Trial
  [Kindle] by John Densmore


An unflinching account of the 2002 lawsuit by Densmore, former drummer for the 60s band the Doors, against his former bandmates, keyboardist Ray Manzarek (who just passed away in May) and former high school best friend guitarist Robby Krieger. His suit was primarily to stop the use of the name and logo of the Doors by Manzarek and Krieger, who were touring (without Desnmore) as the “Doors of the 21st Century.” He did not seek to stop them from playing or from calling themselves “Of the Doors.” He did not seek money from this lawsuit. Manzarek and Krieger countersued for $40 million, claiming that Densmore vetoed a $15 million commercial with Buick… and then Densmore’s real beef is revealed. Densmore goes to court to preserve the noncommercialsm of the original Doors, particularly frontman and cultural icon Jim Morrison, by not “selling out” to corporate America; and to honor the Doors agreement that all bandmates had equal weight in determining decisions with equal veto power. Indeed, Densmore was backed throughout the trial by the estate of Jim Morrison, and even Morrison’s father, a former rear admiral in the Navy, testified. Densmore further questions the need of Americans, and in particular his bandmates, who are all wealthy, to accumulate millions more at the risk of losing their creative purity. It’s an interesting question; Densmore makes many good points, even though at times he unnecessarily takes uncomfortable jabs at Manzarek in particular. Yet Densmore himself stands to earn a lot by being a former Doors member, and writing these books! It’s a head-scratcher at times, as you wonder from what angle Densmore is coming sometimes, as there are many; his arguments don’t always seem clear or follow logically. It’s a quick read, raises good questions and will generate a lot of discussion. From an insider, I have heard that the book is quite accurate.

Laurie Kelley with
Robby Krieger of the Doors



For devoted Doors fans, this is a hard book to read, to learn what has become of their legacy. I’m a bit biased as I love the Doors, and appreciate each musician for their talent and dedication, and just met up with Robby Kreiger on Saturday night at the Hard Rock Cafe–he’s a nice guy, by everyone’s account. He wrote my favorite song ever, “Touch Me,” which I admit would have a different meaning and feel entirely if it were used as a commercial for say the iTouch. For anyone in rock and roll, it is a fascinating legal read. Two/five stars.

This Time for Africa!


One month from today I will be in Africa, about to reach new heights, literally. I’m planning a huge mountain climb, up Mt. Kilimanjaro, the largest mountain in Africa and largest freestanding mountain in the world. At 19,340 feet, it’s colossal. We will start at tropical temperatures, and gradually progress up to Arctic temperatures, below freezing. It will take us six days: five up and one down!

Who are we? The nine other climbers are: my 17-year-old daughter Mary; Eric Hill and son Alex; Neil Herson and daughters Britney and Kelly; Jeff Salantai, who has hemophilia, and Julie Winton, a nurse with a son with VWD. Jeff and I just met for the first time last week in San Antonio! He was the first person with hemophilia to climb Mt. Ranier last year!

Eric is president and founder of BioRx, a homecare company. Jeff and Julie are his employees. Neil is president and founder of ASD Healthcare, one of the largest distributors of plasma derived products and pharmaceuticals in the US.

Our goal is to raise money for Save One Life and its Africa programs. Save One Life is the child sponsorship program for impoverished children with bleeding disorders in developing countries. I’ve been traveling to Africa since 1999, and was the first person from our international community to travel to Kenya, Ghana and Tanzania. I’ve also been to Zimbabwe four times and hope to return again soon. We started outreach programs in Tanzania and Zimbabwe to help locate more patients. We also want to start a scholarship program for college age men in Kenya. They are all so lacking in funds, and it doesn’t take much to get them an education. Once they are educated, they have a chance at a better life.

Eric is a sponsor of two children through Save One Life Neil is one of our biggest sponsors at 49 children! Obviously they are deeply committed to our cause.

They must be to tackle Kilimanjaro. Long the focus of lore and legend, Kili is very special. And tough. While not a technical climb, meaning there will be no ropes or climbing gear, it is strenuous. The biggest worry is altitude sickness: migraine headaches that lead to vomiting and lack of appetite. Oxygen levels will be at 50% of sea level.

But I have hope. Chris Bombardier, a young man from Colorado, just returned from Kenya and I think is the very first American with hemophilia to summit Kili! His climb also raised money for Save One Life.

If you’d like to sponsor a climber, please go to our donation page. Please note that 100% of your donation goes to Save One Life and its African programs, and not to cover the costs of the climb, or airfare or anything related to the trip. Each climber pays for his or her own expenses. All funds raised are donated to Save One Life. It’s a huge commitment in terms of time, energy, and money for these climbers. Please give them motivation and support by pledging today!

http://www.saveonelife.net/mt-kilimanjaro-climb.phpa

Interesting Book I Just Read
Break on Through: The Life and Death of Jim Morrison by James Riordan and Jerry Prochnicky

Shaman or madman? Forty year ago today Jim Morrison died of “heart failure” in a bathtub in Paris, of a suspected overdose. His death is being honored around the world by tens of thousands who loved his music and somehow identified with this tortured poet-turned-rock star. It’s hard to see the poet when you read the account here which can make you wince: the drinking, the juvenile antics, destruction of property and disregard for the rights of others, including his own band mates who suffered six long years with their front man. Gifted with a gorgeous voice and even better looks, Morrison relished the role of star, but also sought solitude personally, and respect for his poetry. “The Lizard King” brought the Doors fame and a legacy in rock and roll history, but as much for pushing the envelope as for his lyrics. His on stage antics made him the first rock star to be arrested on stage; he had 20 paternity suits pending at the time of his death; he was banned forever in Phoenix. Now his antics look tame, but in the 60s, this was all new, and dangerous. Morrison took rock where it had never gone before.

Riordan was a Rolling Stone contributor, and interviewed Morrison. But the writing is choppy, perhaps reflecting two different authors’ styles, and references to current events—Vietnam, Charles Manson, Apollo 11—are stuck in the middle of the story, often without a connection, or when there is a connection, it’s contrived. Some of the statements are just ridiculous (No matter how he tried to avoid it, legal trouble followed Morrison [as though he were an innocent bystander] and then in the next paragraph, they relate how he was arrested by the FBI and held in jail for disturbing the staff and passengers on a commercial plane ride). The attempt to view Morrison as a shaman is a bit too serious, verging on hero-worship. Much of the material here seems to be pinched from other sources, though sources are often not cited. So this is not a well written or researched book, certainly not as good as No One Here Gets Out Alive. I prefer drummer John Densmore’s Riders on the Storm the best, for its candid and sympathetic view of a man, Jim Morrison, who vented his deep-seated angers at his audiences, and muted his insecurities through alcohol. What a waste: judging from the Celebrations of the Lizard today, he is still cherished as a star, marveled at for being truly innovative, and listened to with pleasure. He appalled and attracted. The world is ever fascinated by him. Morrison has influenced many rock stars since his brief life ended. And members of the Doors are still touring, as much as ever (I almost went to see Ray Manzarek when he was in Massachusetts in May–so sorry I missed it!) He always said he would be a comet: a brilliant flash, here for a short while, but leaving a lasting impression. How true! RIP, Lizard King. Two stars.

Lucky Number 13 part 2

Just last week I blogged about CSL Behring’s new factor XIII concentrate Corifact. Now I’ve learned that Novo Nordisk announced that a Biologic License Application (BLA) has been submitted to the US Food and Drug Administration (FDA) requesting the approval of a recombinant factor XIII compound for those with congenital Factor XIII (FXIII) deficiency.

Corifact is made from human blood plasma. This new drug is made from recombinant technology, which uses genetic engineering.

Factor XIII deficiency has a prevalence of one case per two million people, with an estimated 600 diagnosed patients worldwide, making it one of the rarest bleeding disorders.

A press release from Novo Nordisk states: “Positive results from a phase III trial examining the efficacy and safety of recombinant factor XIII for the prevention of bleeds associated with congenital FXIII deficiency showed that when compared to a historic control group of individuals who did not receive routine FXIII infusions, preventive treatment with monthly recombinant FXIII injections significantly decreased the number of bleeding episodes requiring treatment. These data were presented at the American Society of Haematology (ASH) meeting in December 2010, and marked the first completed phase III study conducted to study the use of a recombinant FXIII treatment to prevent bleeding episodes in congenital FXIII deficiency patients.”

It’s all great news for those who have factor XIII deficiency, and with the sad news from around the world, especially Japan, we need good news.

Good Book I Just Read

No One Here Gets Out Alive
Jerry Hopkins and Danny Sugerman

I’m continuing my reading streak on The Doors, the 1960s band that took America by storm. In this book, Jerry Hopkins (Rolling Stone) and Danny Sugerman (then teen office boy for Doors) profile the madness and magnificence of Jim Morrison, front man and silky-voice singer, whose depravity and self-destruction eventually upstaged the music itself and his three talented band members. The book details Morrison’s schizophrenic adolescence in which he, with an IQ of 150, devoured sophisticated philosophy and poetry books, and wrote poetry himself, but yet immaturely and cruelly harassed his younger brother, scared the elderly and openly mocked cripples. The first few chapters alone might end any hero worship of The Lizard King. The book recounts chronologically how the Doors formed, their skyrocketing rise to fame, and the toll of fame on the unstable Morrison. They were only 21-23 years old, in a music industry and culture awash in drugs, but why did only Morrison succumb to the pressure? The book is insightful into the music industry of the 1960s. It’s incomplete in that no other character is truly explored: Manzarek, Densmore and Krieger are barely mentioned. They were together for six intense years: how did they cope with Morrison’s descent into madness? The book also sheds little insight into Morrison’s state of mind, or why he acts as he does other than the drugs or alcohol, but factually states a portrait of a seeming sociopath: the dozens of paternity suits, his wanting his child to be aborted because he simply didn’t want one, his cruelty to his mother when he performed, his promiscuity and debauchery.

It’s hard to reconcile the deep thinker with the raging drunk. Perhaps the book doesn’t go deep enough into Morrison’s psyche, but then, Morrison didn’t seem to let anyone in. He internalizes his pain, which seeps out in frightening rages, and then is dampened by alcohol. One only needs to look at his photo progression in just six years: from sleek, sexy rock star to bloated, bearded drunk. I don’t think the pain was from being a frustrated poet or even a rock star; that’s putting the cart before the horse. His pain was chronic, malignant. Poetry was one expression; rage and cruelty was another. Adoration from the masses was one treatment; alcohol, maybe heroin, was another, and became the final exit.

Morrison helped put the Doors on the map, and he destroyed the Doors by destroying himself. Forty years later, it seems we will never know what drove Morrison to the edge, and then over. The authors skim over any analysis by comparing his angst to being like the Greek god Dionysus, or part of the Beat generation, or expressing himself like the Indian shamans he revered. But this is shallow. Looking back today, Morrison was an emotionally disturbed artist who sought to medicate his pain through alcohol, and expressed himself through rage. Incredibly, he left a legacy of beautiful, mystical music that captured a unique time in America, and the black hole of his inner life. And we are drawn to such people, scared of them, and yet worship them. Despite the incomplete picture the book paints, it was a great read and I could not put it down. Three stars.

Lucky Number 13


When is the number 13 lucky?

We now have a factor concentrate for people with factor XIII deficiency. CSL Behring, makers of Helixate FS, Monoclate P and Mononine, was granted FDA approval for their factor XIII concentrate Corifact, indicated to treat congenital factor XIII deficiency. This is the first and only U.S. approved treatment for factor XIII-deficiency and the fourth new product that CSL Behring has brought to the U.S. market in the past two years. Corifact is already available for use in 12 countries throughout the world under the trade name Fibrogammin- P.

Factor XIII deficiency, also known as fibrin-stabilizing factor deficiency, is rare, affecting one in two million, with an incidence in the U.S. of approximately 150 people.

Symptoms include bleeding from the umbilical cord after birth, poor wound healing, miscarriages, subcutaneous bleeding, and excessive bleeding in joints and muscles following trauma. Patients lacking the FXIII protein are also at high-risk for intracranial hemorrhage (ICH), bleeding inside the skull that can be life threatening. Studies have shown that between 25- 60% of factor XIII deficient patients will experience an ICH at least once during their lifetime.

This is great news for those suffering with factor XIII deficiency. For more information, please see www.corifact.com and contact your HTC staff.


Great Book I Just Read
Riders on the Storm by John Densmore

“There’s the Beatles, the Stones, and the Doors,” says Paul Rothschild, producer of one of the greatest bands of the sixties. Drummer John Densmore was only 21 when he joined with three other California students to form a band that would soon skyrocket to the top at a time when the US was a nation at war in Vietnam, and with itself. With quiet Robby Krieger on guitar, methodical and rational Ray Manzarek on keyboard and Adonis-look-a-like singer Jim Morrison, the band was like no other. With no bass player, they combined blues, jazz and psychedelic rock, with some Indian strings and often very dark lyrics, about death, murder and rage. And no wonder: Not long after forming, Densmore writes about their iconic lead singer, “I’m in a band with a psychotic.”

The book is captivating. It starts in Paris, 1975, with Densmore visiting Morrison’s grave, four years after his death at age 27 on July 3, 1971 from an overdose. Returning to the hotel, he pours his feelings out in a “letter” to Morrison on the hotel stationery, which then gets interspersed throughout the book with his recollections. His memoir details his time in the band, returning now and then to the ongoing letter, and towards the end, “updates” Morrison on events of the 1980s and 1990s. Interspersed are appropriate lyrics from their songs, so that the book itself becomes quite artsy. The book gives excellent insight into what the 60s were like, what it’s like being in a band on the rise and what it was like to survive the onslaught of Jim Morrison. What’s missing, though, is what “The Lizard King” was really like. We get snapshots, snippets, and stories, but it’s as if Densmore viewed Morrison at arm’s length, even though he intensely shared six years with him. He was in awe of Morrison—who wasn’t?—and scared of him, and rightly so. Though he feels guilty at not being able to help Morrison, we have to remember he was only a young man from a humble Catholic background, ill equipped to cope with sudden stardom, wealth and the phenomenally complex, creative and self-destructive Morrison.

As if to highlight the detachment Densmore had with Morrison, Densmore dedicates the book to John Lennon of the Beatles, one of Densmore’s heroes, and also mentions Lennon’s assassination in his “letter” to Morrison, yet doesn’t mention or maybe doesn’t even know that Lennon was assassinated on Morrison’s birthday, a fact he should know as Morrison and Densmore were born only a week apart.

“We sensed rage and a possible explosion too near the surface to mess with in dealing with you,” Densmore writes in his letter to the now dead Morrison. “It seemed to have a lid on it—Pandora’s box with all the demons that wanted to be released. We never opened the box… we had to deal with your demons seeping out the side.” They seeped out and poisoned everything; the Doors had to cancel a 20-city tour when Morrison was arrested in Miami for indecent exposure at a concert (he was just pardoned this past December!). The charisma, lyrics and stunning voice of Morrison helped make the Doors a success; his addiction, rage and irresponsibility destroyed them. The book was published in 1990 and Densmore writes, “Well, we’re going on 20 years and there’s no end in sight” of the fascination people have with the Doors. On July 3, it will be 40 years since Morrison’s death, and the Doors are almost as popular as ever. I picked this book up and finished it 5 hours later—it’s compelling, honest, shocking and eye opening. A great book about one of my favorite bands and my favorite singer—for no one could sing like Jim Morrison. Three stars.

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