August 2013

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.

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!
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.
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!
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.

“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
by John Densmore

Laurie Kelley with Robby Krieger of the Doors

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.
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.

Gears for Girls; Gears for Good

My new addiction!

So this is all Barry Haarde’s fault.

A year ago I was a contented jogger and sometimes mountain climber. Not often, but enough to say I can do it. I also cycled about once a week using an old hybrid bike, which means that it’s not as slick as a touring bike (those skinny bikes with skinny people riding them who have Lance-Armstrong-like ripped muscles and wear cool shirts) but not as bulky as a mountain bike (which have really thick tires and usually young, crazy males riding them through forests). I wasn’t too competitive.
Then Barry just had to go and cycle across America in 2012 (3,700 something miles) to raise money for the nonprofit I founded, Save One Life. And when he hit New York, he emailed me that I was going to ride
the last 50 miles with him to dip our wheels in the Atlantic Ocean, at Rye beach, in New Hampshire, not too far from where I live.
Oh really? I couldn’t refuse; he shamed me into it.
So I did have a top-notch Spanish touring bike, an Orbea Diva, though dusty and stiff, hidden in a closet. It was in storage for the past four years, after I bought it as a consolation prize when I turned 50, fell in the middle of the street on its maiden voyage, and put it away, too scared to ride it. Well, that’s all chronicled here.
Today, I continued what has become an addiction. (Barry, you knew this was going to happen.) I hopped on my baby (the Diva, which used to give me nightmares as it sat eerily waiting for me in my cellar closet, like some haunted, phantom thing from a Guillermo de Toro movie) at 9:30 am and headed out for Route 1A, and cycled along the Atlantic sea coast for over four hours, covering 64 miles, the most I’ve ever done. Yes, it hurt after a while, but you just can’t stop it. Whatever it takes.
Last week, if you are friends with me on Facebook (you are friends with me, right?), you might know I spent four days solo in Sedona, Arizona, where I indulged my new addiction. I checked in last Sunday into my hotel in Sedona, a mere two hours after the Arizona Hemophilia Association meeting, and almost immediately went for my first mountain bike trip. Why? I
was surrounded by majestic Boynton Canyon, with the desert and all its twisting, red-dusty paths calling to me. Something different and new. And kinda scary. Mountain biking is very different than touring biking. It’s like the difference between riding a Mustang with an attitude using a Western-saddle, and sitting all pretty and proper on a fast thoroughbred (well, not that fast when I am on it) on an English saddle.
The day was hotter than I thought, and it would be easy to dehydrate.
I slipped on a new backpack, very small, with a built in “Camelback”
water pouch. This has a tube connected to the “bladder” (I know, sounds gross) from which you can easily drink water.
I set out on the bike, hit “Deadman’s Trail” alone, and pounded my way through red dust, over lots of stones and sand. Sedona sits at 4,500 feet, and my heart was soon thumping in my chest. To my left, the massive sandstone wall of Boynton Canyon, with towering red rocks. Cacti and scrub bushes fringed the trail. After 30 minutes, I felt I was in the middle of nowhere, alone… the path was steep. I stopped, chickened-out (it is DEADman’s Trail, after all), and decided to turnaround the way I came: downhill, rocky, exciting!
My heart was pounding furiously, meaning that I
was overheating. It was trying desperately to circulate overheated blood from
The next day, after four hours of hiking Bear Mountain and Devil’s Trail in the 96-degree heat, in which I drank perhaps more water than in my whole life combined, I actually went for another mountain bike ride, on Cockscomb Trail. Who would be crazy enough to do this after such long and superheated hikes? People with bicycle addictions.
George, the Native American guide from the bike rental shop, gave me excellent tips on biking the trail. It was much better than Deadman’s Trail; more trail than steep rock steps, exciting. I found the trail head off the highway, entered, and was frustrated to stop a few times as I
didn’t have my rhythm down yet. Don’t power up the hills, George had said. That’s what you
“touring” people do. Just sit back, go in your easiest gear, and pretend you are walking “up” the hill. The hill with lots of rocks, you mean?
Yes. When I got through the rocky part, the trail smoothed out and became like the Run Away Train ride at Disneyworld. I marked the trail as I went with the contents of my stomach. I guess I pushed myself too hard and was so spent. But it was fun!
Boynton Canyon
On Wednesday I wasn’t sure at first how to start my last day. Swim in the pool? Attend the yoga classes? Leisurely breakfast? Nope. Up at 6:30 am, made some tea, ate a banana and some berries, checked email, wrote to my daughters to tell them to come and find me if I disappear, threw on my riding gear and headed to the Activities Shop. By 8 am, with some cloud cover thankfully, I pumped down the road, to the Fay’s Canyon trail head, and dove into the
outback again to Cockscomb. I was going to nail it this time, and make George proud of me. No more walking my mountain bike. I did so much better; only had to stop twice as opposed to three or four times on Sunday. At times I felt like I was on a runaway horse, recalcitrant and skittish, as I bounced over the rocks, fishtailed in the red, fine dust, and then rolled up
and down the roller coaster trails. It was challenging, exhilarating, dusty, dirty, sweaty. And did I mention addictive?
After that, I came back out to the road, and then pedaled back towards the hotel, first taking a side tour to Deadman’s Trail again, determined to see it through the end. It was so much harder than Cockscomb, but I did much better this time. I actually pumped up the first very hard incline,
with many rocks, doing just what George had suggested. And in 20 minutes got to the tricky part where I gave up on Sunday. It was better after that, but still challenging. It suddenly got scorching hot as the sun rose, and I was sweating and drinking tons of water. I felt like I had leaks in my skin. Water in, water out. I had to stop now and then, and rest or walk the bike. At one point, the bike seat (which had a loose part to it that stuck out) caught on my shorts, tore a small hole in them and wouldn’t
allow me to hop off when I needed to, so down I went in the dirt, the bike landing on top. The handlebar jammed into my thigh, causing instantly a raised hematoma. Ouch. At that very
second a runner swept passed me (out in the canyon?), a Matthew McConaughey
lookalike, tanned, trim, ridiculously handsome and fit, and half naked. We said
hello… instant painkiller. Back on my bike, finally reaching the road, and aimed for the turnoff for my hotel.

Wipe out!
But I didn’t want to go back, as it meant I had to leave tomorrow for Boston! I still had half an
hour so I swung the bike around, pedaled back to Cockscomb Trail and did it again, and this time only stopped once! It was such a great feeling. I was so incredibly hot, my heart was pounding, sweat
pouring out of me, my legs all banged up and bruised, and I never felt better in my life. (Well, maybe if I saw Matthew McConaughey’s look-alike again)
After the 64-mile ride today, I came back, lay on my bed still in full biking gear, and passed out for an hour. How will I make it to 100? Because I have to do 100. What good is an addiction if it doesn’t keep pulling you to push the envelope?
So here it is: On September 21, I will ride 100 miles to raise money for the Springfield Police Department, in honor of Officer Kevin Ambrose, who was killed in June 2012 in the line of duty, the first officer in that city in 25 years to die in action. My brother Tim, also an officer, was a good friend of his. So he and I will ride together, along with 200 other officers, with a full State Police escort. I might need it.
And, Barry, who got me into this mess to begin with, is going to ride with me, Kimberly
Haugstaud (executive director of Hemophilia Federation of America), Vaughn Ripley, and Allie Boutin of HFA (and a neighbor of mine!) and more, to raise funds for HFA on September 27. “Gears for Good.” Want to sponsor me? Take pity on me and my addiction, and think of a pledge. It’s all for a good cause—for HFA. And it’s only a few days after my 100-miler (my “Century”)—so I will be primed and ready! Or totally passed out somewhere in my gear. What I fear most is not the these two rides, but Barry whispering how I should do the cross-country ride with him in 2014… Barry, it’s not nice to take advantage of someone with an addiction!
Donate here….
Book I Just Read
The Places in Between by Rory Stewart [Kindle]
This book was a best seller, and recommended by my favorite magazine, Outside. In 2002, Rory, a
free-lance writer, decides to walk across Afghanistan just months after the Taliban were deposed, retracing the path of a former and ancient ruler, Babur. He claims to be researching a book about Afghan history and Babur, but this book is mostly about the people he encounters and the extreme physical hardships he endures. The problem is his writing is mercilessly dry, without emotion or sentiment, and seemingly for no purpose. Why does he undertake this trek? Just to see if it can be done. There is no setting up of girls’ schools, as in Three Cups of Tea; there is no bonding of humans who endure outside exposure and
survival. There is barely even a bonding with the faithful and suffering dog he adopts and who accompanies him. While he does have potentially an interesting story, it is told in such a way as to make the reader wonder why he or she is reading it. To what point? There seems to be none. And there is no excuse for mediocre writing; Rory had plenty of time alone to conjure up literary references, flowing of words, rhythm. It’s flat, uninspiring writing. I learned a bit about Afghanistan but didn’t enjoy the trip much. Two/five stars.

Genotyping: Helping You, Helping Our Future Families

Laurie Kelley with Cindy Komar

I attended the excellent annual meeting of the Arizona Hemophilia Association this weekend, invited by Cindy Komar, executive director and mother of a child with hemophilia. (Cindy is an awesome exec director and lady, if you haven’t met her!) It was great to catch up with so many Arizona families I know.

I did the keynote presentation yesterday but I myself didn’t find that half as interesting as a fascinating new program for families with hemophilia: called “My Life, Our Future.” The name is a bit nebulous, but the program is important. NHF has teamed with Biogen Idec, Puget Blood Center and ATHN to create the first genotyping of families and patients with hemophilia in the US. John Indence, from NHF, and Jennifer Dumont, director of scientific affairs from Biogen Idec Hemophilia, presented.
John summed if up first: this is NHF’s most important project. One blood sample per patient will reveal vital information on the patient’s specific hemophilia type, propensity to develop inhibitors, and more. A short movie featured NHF CEO Val Bias, who stated that this will help families understand their personal genetic profile, and also help researchers learn more about the genetics of hemophilia, as the data will be pooled. Best of all, the testing will be offered at no or low cost.
John explained how ATHN currently holds the most information/data on hemophilia patients in the country. Information is supplied from HTCs, who obtain patient consent; privacy of course is key, and ATHN assures complete privacy. And Puget Sound is one of the top HTCs in the country, and one of the top genotyping centers.

Laurie with Rachel Stuart, RN

John noted that everyone in Spain and Ireland has been genotyped, and it’s time for the US too. As early as 1998, MASAC (NHF’s Medical and Scientific Committee) advocated for genotyping. IN 2008, the initiative got a boost when the Genetic Information Nondiscrimination Act (GINA) was passed, making it more reassuring for patients to share their personal health information.

In 2002, the human genome was sequenced at a cost of $3 billion; now, genotyping is much cheaper and the price per family keeps dropping. Genotyping helps uncover the causes of disorders, like hemophilia. It requires an analysis of the population, not just the individual, and the purpose of testing the individual is to aggregate the data to study the entire population. So, this new program requires the data of thousands of participants!
Jennifer Dumont then spoke and gave an excellent description of how genotyping works. She noted that 99.9% of our genetic makeup is the same! It’s the 0.1% that makes us all different. A genotype is “a description of the gene mutation that is caused by a certain condition or disorder.” A mutation is a change in the DNA sequence of a gene. It can happen in different ways: nucleic acids can be inserted, deleted or substituted in DNA, leading to a misreading of information in directing cells how to function.
There are diseases where one mutation leads to one disease, such as sickle cell anemia. Hemophilia is different: there are many possible mutations, such as missense, nonsense, frame shift, deletion and insertion. For factor VIII and hemophilia A, there are 2,513 currently identified mutations; for factor IX, there are 1,094 currently identified mutations.
Jennifer illustrated what each mutation might be like if we used a sentence as a DNA sequence (three-letter words are similar to the DNA sequence of three-letter codes): “The One Big Fly Had One Red Eye.” Each mutation makes the sentence read strangely! The body can’t figure out how to produce factor properly when the instructions are so scrambled.
After the presentation, some great questions were asked, such as will Spanish-speaking services be included in the program to help Latino families? Answer: not at the present but this is something that NHF is keenly aware of.
Another question: If HCPs (Heath Care Providers)  know the risks of inhibitors from the genotyping, what can be done to prevent them form happening? Will it affect choice of product or treatment? Jennifer replied that it shouldn’t affect choice of product since there is no difference in the incidence of inhibitor development with different products, but physicians may closely monitor people in the first several exposure days to factor and possibly change how they dose if there is a known risk of inhibitors.

With the BioRX ladies!
Deena Lapinski and Laurie Kelley
John also mentioned that if you’ve been genotyped before outside this program, you’d need to be genotyped again. And someday NHF hopes to open the program to those with VWD and other bleeding disorders.

The presentation was well done and gave rise to much discussion. The bottom line: to support NHF’s research efforts on the causes of hemophilia and its functioning, something that may help future children by our research now, ask your HTC how to participate in the genotyping program “My Life, Our Future.”

Great Book I Just Read
Gifts from Eykis by Wayne Dyer
In this fictional tale, the narrator somehow visits an identical civilization to Earth’s, in every way, though he is on Uranus. But he learns that the thought processes and psychological lifestyles of these people are strangely different. They experience positive human emotions naturally. They experience negative human emotions only through machines designed to elicit them (very creatively told!). On Earth, we create negative emotions when they don’t really exist, or don’t need to exist, because there is no external “machine” that prompts them. Jealousy on Uranus is caused artificially. The parable is that we create our own misery and can just as easily rid ourselves of it through free choice. Eykis, a beautiful Uranian inhabitant, comes to Earth to observe and then leave us her gifts of observation, in how we can take advantage of our freedom not to engage in negative thinking, such as anger, anxiety, fear, jealousy, and above all, blame. Everyone is responsible for their own emotions. Great story, simply told, easy read, powerful message. Four/five stars.

Capping the Cost of Hemophilia

Here is a coup d’etat for the hemophilia families of Delaware. On July 23, Governor Jack Markell signed a bill into law that caps the cost for drugs to treat chronic conditions like hemophilia. Specifically, the law limits co-payments for prescription drugs.

According to Markell’s office, under the law the co-pay for these specialty drugs will be limited to $150 per month for up to a 30-day supply. Patients will also be able to request an exception to obtain a specialty drug that would not otherwise be available on their health plan.

A news wire cites Executive Director of the Delaware Valley chapter of the National Hemophilia Foundation Ann Rogers:  “The average cost of hemophilia medicine per month is $40,000. So, when insurers proposed high cost sharing at 25 to 33 percent of the actual cost of the drug each month, we were panicked,” said Rogers. “That would be 10 thousand dollars out of pocket each month.”

The law goes into effect January 1 in Delaware and is the triumph of three years of negotiation between insurers, advocacy groups and others.

Rogers adds her group and others are already using the bill as a model for similar legislation in Pennsylvania.

“This model of first a study,” says Rogers, “then a recommendation and then legislation introduced to regulate this [issue] was the process used in Delaware and we found it to be a fair, transparent process that fleshed out the issue and resulted in a law that would protect families.”

Excerpted from

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