My Life, Our Future Enrolls 1,000!

Last summer, I blogged about a new program called My Life, Our Future, a nationwide initiative between NHF,
ATHN, Puget Sound Blood Center in Seattle and Biogen Idec that offers free
genotyping to people with hemophilia in the US. As you may have seen around NHF’s Twitter chat on 7/23, the
program has since successfully enrolled more than 1,000 participants! This is a
significant milestone for the program and a great example of our community
joining together and taking action to help advance scientific breakthroughs.
During the chat, the community showed great interest in the
program and how it works. There are two components to My Life, Our Future (or “MLOF”). The first is a blood test that
enables people to learn more about the specific genetic mutation that causes
their hemophilia. Already, 61 new mutations have been discovered through the
program. Participants also have the option to contribute their data and samples
to a central repository. Once 5,000 people contribute to this research “bank,”
scientists will be able to study the data and samples, potentially leading to
improvements in care and treatment.
Getting Involved for Future Generations
Guy Law of Erie, PA, participated in MLOF for just that
reason. “Hope comes to mind when I think of MLOF,” says Guy. “I hope my participation
in the program can positively impact families with hemophilia in the future.”
Guy, who has severe hemophilia A and has long been an advocate in the
community, learned through MLOF he has a unique mutation. “I know the word
“cure” gets thrown around and I’ve accepted the fact that in my lifetime there
may not be one,” he said. “But if I can contribute to a research project that
may eventually lead to a treatment breakthrough, I’m happy to.”
Dr. Barbara Konkle, director of clinical and translational
research at Puget Sound Blood Center, hopes that everyone with hemophilia A and
B participates in MLOF because of its value today and tomorrow. “Genotyping
provides meaningful information about a person’s hemophilia,” said Dr. Konkle.
“By building a robust bank of data and blood samples for researchers to study,
we can deepen our understanding of the disorder and hopefully advance the
science.”
Interested in participating? The program is currently
available at 42 HTCs across the country, and expanding to additional locations
regularly. Visit the MLOF site to see if your HTC is offering the program.
MLOF By the Numbers
•      
MLOF is now available at 42 hemophilia treatment centers in
26 states
•      
80% of participants have opted to participate in the
research repository
•      
61 new mutations have already been found
•      
Once 5,000 people have consented to participate in research,
scientists at academic institutions or companies can apply to study the data
and samples




Great Book I Just Read

Flight 232: A Story
of Disaster and Survival  
[Kindle
Edition]
by Laurence Gonzales
Sioux City, July
1989: one of the worst aviation disasters ever. A DC10 cart wheeling on the tarmac
after the hydraulics completely fails. Gonzales tells the story of the crash
over and over, through different angles by probing the different experiences of
many involved: survivors, crew, rescue workers. His excellent research in interviewing
so many who lived through this puts you there; he also delves deeply into the engineering
flaws that led to the crash. You will learn a lot about titanium. And that so
much of survival in an airplane is sheer luck. It is amazing that so many
survived the crash; the pilots were absolutely heroic. Gonzales is an expert on
survival, and follows the survivors just after the crash and then a year later.
The testimonials are surprising—what goes through someone’s mind, as a plane is
disintegrating—and poignant. The most important thing in our lives is
relationships, most conclude. Four/five stars.




News from Pharma

So much has been happening in the hemophilia community lately, I decided to post some news releases about new products and new services from three different pharma manufacturers. Last week we shared the news about Biogen Idec’s Eolctate approval; below are more interesting items, straight from the horses’ mouths.

From CSL Behring
New–CSL Behring’s world-class biotechnology manufacturing facility in Broadmeadows, Australia. The facility is for late-stage production of hemophilia therapies and is one of the largest and most advanced of its kind in the world. Leading-edge science is at the core of the $250 million expansion, which is expected to drive long-term growth in CSL Behring’s promising bleeding disorders portfolio.
In particular, the facility will produce novel recombinant therapies on a large scale for international clinical trials. Our recombinant technology uses a unique recombinant-albumin-fusion platform, which we believe could offer distinct clinical advantages and improve patient experiences.  Suffice it say, CSL Behring’s steadily-rising research and development spending has played a key role in developing these and other new technologies. For example, in 2011/2012 we spent $368 million on R&D (about 8% of total sales). And over the last 5 to 10 years, we have advanced an enviable pipeline with exciting product candidates that may offer enormous treatment benefits. 



From Baxter Healthcare

Mobile video gamers looking for the next app for their smartphone or tablet now have an option developed specifically for the hemophilia community. Baxter Healthcare Corporation has created a free multi-game app, called Eco-Trek, primarily for people between the ages of 15 and 32. Yet based on the reactions of pre-release product testers, Baxter expects that Eco-Trek will appeal to people of all ages, from adolescents waiting to be seen at hemophilia treatment centers, to older adults that enjoy competing in video games. Eco-Trek, which is available to everyone regardless of their treatment, is the first socially connected video game designed for the hemophilia community. With hemophilia-specific content and Facebook score-sharing built into the game, Eco-Trek is a first-of-its-kind mobile app. A national leaderboard will create competition among players in Eco-Trek’s three online adventures, each of which have 10 levels:
·     Bushwhacked! – Find your way through a forest maze without losing supplies from your backpack
·     Alpine Summit – Compete in an uphill race against the clock, dodging obstacles along the trail
·     Roughin’ It – Set up camp and complete specific tasks while pesky critters try to get in your way

“We are really excited about Eco-Trek because it’s simple, interactive, and offers important educational information about hemophilia A and ADVATE [Antihemophilic Factor (Recombinant)], while you’re competing in fun activities,” said Jeff Schaffnit, senior director of US Hemophilia Marketing. “For teens and young adults with hemophilia, this type of format can make it a lot more fun to gain knowledge on hemophilia.”

Eco-Trek is a free app available for download from the Apple App Store and Google Play. For more information, contact your Baxter representative.

From Pfizer

Pfizer understands that health care costs can be a challenge for those with hemophilia and their families, and strives to provide access and patient-centered support. Therapy is just one part of the equation, which is why we offer financial support for eligible patients as well as educational programs for patients with hemophilia who infuse with Pfizer Hemophilia Recombinant Factor products.

We’re pleased to share with you three program updates and hope that you will help us share this news with the hemophilia community. As of April 1:

•    Pfizer increased the maximum cap on its Pfizer Factor Savings Card
Program. Eligible patients can now save up to $5,000 annually on copay, deductible and coinsurance costs. Terms and conditions apply. Visit PfizerFactorSavingsCard.com to learn more and fill out a brief registration form. If you have any questions about the use of this Pfizer Factor Savings Card, please call 1-888-240-9040 or send questions to: Pfizer Factor Savings Program, 6501 Weston Parkway, Suite 370, Cary, NC 27513.

•    To continue to meet the changing needs of today’s patients, Pfizer is consolidating its various prescription assistance programs, formerly known as Pfizer Helpful Answers, into a new comprehensive program called Pfizer RxPathways. Pfizer RxPathways is a family of prescription assistance programs that provides eligible patients with financial support and reimbursement support services. Access the program today by visiting PfizerRxPath.com.

•    The Soozie Courter Hemophilia Scholarship Program, a tuition assistance program
for students with either hemophilia A or hemophilia B, is now accepting applications for the 2014-2015 school year. To apply, please visit http://www.hemophiliavillage.com/resources-support/scholarship-assistance 

Another Product Debut: FVIII This Time!

We were hoping for an August approval, but it seems the FDA is making Biogen Idec’s day. Two products approved in two and a half months! Both with extended half-life. Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] is the newest factor VIII product. 

A new era of hemophilia treatment has begun. I read somewhere that the last truly “new” product that was introduced was Advate, in 2002. True?

The hemophilia industry is big, with much at stake. The introduction of two new products, with distinct and so far unique,  differences, may cause a shake up. And more products, with longer half-lives included, are coming.

More products give us more choice.  But you have to know what it is you are choosing between.  I have to say I don’t think hemophilia parents and patients are as well informed as they should be. I just conducted a recruitment of patients and caregivers this past week, looking for those who use “plasma-derived” products ONLY. I couldn’t believe the number of people who called to participate who use recombinant. They asked: what does plasma-derived mean? Isn’t the product I am using plasma-derived because it contains Albumin? Or worse, arguing with me that a recombinant product is plasma-derived.

We’ve written so many articles describing the difference between the two categories. All factor is either plasma-derived (originates from human blood) or recombinant (from human genes in the lab). The new product is a recombinant product manipulated to last longer in the blood—basically through a longer half life.

One savvy mom I’ve known for year, who has a son with the World’s Most Stubborn Inhibitor, wrote this to me:

It’s important for Biogen Idec to make clear that this product has not been tested in Previously Untreated Patients (PUPs), and the risk of inhibitors is highest in this group.
Biogen Idec will be opening up a clinical trial for PUPs and I am encouraging anyone who wishes to use the Alprolix (long-lasting recombinant FIX) in a previously untreated patient, to wait and enroll in that trial, so Biogen can gather the appropriate data to understand how safe the product will be in PUPs.
Please always speak with your HTC hematologist to learn which product is best for you or your loved one. (And I don’t usually use underlines in my writing! Yeah, it’s that important)

To learn more about Eloctate, visit:

http://www.biogenidec.com/therapies_eloctate.aspx?ID=20259
Good Book I Just Read
The Right Stuff by Thomas Wolfe [Kindle]
The gripping story of the origins of the space race, and the American men who volunteered to become the first Americans in space. The Mercury astronauts, made heroes by the press and NASA, possessed the character and qualities that made America proud. The book covers a lot of ground, from what it’s like to sit in the cockpit, going to Mach 1, the speed of sound, the tough men who were test pilots, the culture of the test pilots, and eventually the program to put a man in space. It’s a great story (and the movie is superb) and you’ll learn a lot. Wolfe’s flamboyant, hyperdramatic and  over-the-top style just wore on me terribly and I had a hard time eventually getting through this. Way too many exclamation points per page! Great story, odd writing though many consider Wolfe a national literary treasure. Three stars/five.

The [Infusion] Shot Heard Round the World

Unless you were watching the new streaming releases of Season 6 of Breaking Bad on Netflix (for which I might excuse you) or were abducted by aliens (for which I will not) you must have heard the news….. the first extended half-life factor has been approved for commercial sale. It’s the hemophilia B drug APROLIX. The news was announced at the HFA meeting in Tampa last Saturday, and it truly is the Shot Heard Round the World (an appropriate reference to the American Revolution). This ain’t just a new product, folks; it could shake up the industry. Physicians are watching; patients are watching; Wall Street is watching. What will the “uptake” be? 

Uptake is how many patients will switch to using a new product. The hemophilia community tends to be a “wait and see” creature, perhaps due to our devastating experiences of the 1980s. When Advate was launched in 2002 (yes, drugs are launched, like ships), investors predicted a rosy 20% uptake, but initially it was more like 12%. As the risk-takers who switched early reported good results, more and more patients switched. But Aprolix is truly a different kind of drug. Not just a new generation, it is a new type of hemophilia drug: an extended half-life, meaning fewer infusions for the same results. 

And Hemophilia B has not had a buffet of drugs to try like hemophilia A. There was only one recombinant (you all know what that means, right?) FIX for years, until October 2013, when Rixubis was approved. With extended half-life factor, prepare for some major shifts in our landscape. Much may depend on the price, which no one is talking about. If it’s too high, will insurance cover it? 

And what about individual results? Will each patient respond well? Advate had major hiccups when there appeared to be an increase in inhibitor formation in users. Was this due to higher scrutiny of inhibitors with a new product? The initial scare seemed to be due to increased scrutiny. And these are among the most scrutinized things on the planet.

And lest we get too carried away, don’t forget there are other products in the pipeline by other companies, some extended half-life (the products, not the companies, though we hope they are long lasting too). Basically, the news about Aprolix marks the beginning of a new era in hemophilia. Maybe a Revolution, maybe the Renaissance. A Hemophilia Renaissance. 
Below, an article in its entirety about the news….
U.S. FDA approves Biogen’s hemophilia B drug
Alprolix
Biogen Idec Inc has won U.S.
approval for its long-acting hemophilia B treatment Alprolix, the U.S. Food and
Drug Administration said on Friday.
Hemophilia B is a rare, inherited disorder in
which a person’s blood does not clot properly, which can lead to prolonged
bleeding and bruising. It affects about 4,000 people in the United States and
25,000 worldwide.
Alprolix is a bioengineered version of the blood
coagulation factor IX, a protein needed for normal blood clotting. Biogen is
developing the drug in partnership with Swedish Orphan Biovitrum AB. It
received approval in Canada a week ago.
Hemophilia drugs in general must be infused two
to three times a week to prevent bleeding episodes. Biogen’s drug needs to be
administered once a week or once every 10 to 14 days.
The company is also developing a long-lasting
drug, Eloctate, for Hemophilia A, a more common form of the disease, that
affects about 20,000 people in the United States. Patients with hemophilia A
lack or have reduced levels of coagulation factor VIII.
Current treatments for hemophilia B generate
about $1 billion a year, according to Biogen. The market for hemophilia A
therapies is worth about $6 billion.
Eloctate would compete with Baxter International
Inc’s drug Advate. Baxter is seeking to develop its own long-acting version but
Biogen is expected to be first to the market.
Alprolix is expected to generate sales of $286
million by 2019, according to Thomson Reuters data. Eloctate is expected to
generate sales of $1.1 billion.
(Reporting by Toni Clarke in Washington; Editing
by David Gregorio)  Fri Mar 28,
2014 5:11pm EDT
For more info:
http://www.alprolix.com/

United by Blood: HFA Meeting in Tampa

Laurie Kelley, Jeff Johnson, Barry Haarde

Grey skies and a chilly breeze couldn’t dampen the spirits of those attending the 20th anniversary meeting of Hemophilia Federation of America in Tampa, Florida this past week. A record number attended, estimated between 600-900, from all parts of the US. Zoraida and I arrived on Wednesday, a day early, to meet with some of our colleagues, for this is a prime meeting for business networking, fundraising and brainstorming.

Central to the meeting, like its heart beat, was the History Room. This stunning display of our past 70 years in all its pain and triumph, was a somber reminder of how far we have come in the war against bleeding, and the sacrifices of our fallen. Following a poster timeline, in which each era was clearly defined, led to the room, where dozens of resources were provided (hey, including my own stuff), a community poster board with hundreds of photos, and the Ryan White section from the AIDS Quilt. Kudos to Rich Pezzillo, Ray Datolli and Barry Haarde (and their helpers) who masterfully compiled with painstaking detail this amazing tribute to our community, to our fallen.

Selfie-time!

Symposia included a variety of topics. One on advocacy and the ACA, called Making Advocacy Personal, featured Jim Romano of PSI and Wendy Owen who answered a slew of questions on advocacy and health care policy. Another on just inhibitors, a new feature at HFA–my only concern was that is was closed only to families with inhibitors. Huh? Everyone could benefit from attending, as there will be families this year who will develop inhibitors, and there are those of us who help educate them. (Anyone want to explain that policy to me?***)

Baxter-Sponsored Dinner Friday Night

A Baxter-sponsored dinner Friday night provided talks from two young men with hemophilia who shared their stories of growing up feeling different, and who now are talking life by the horns–very inspirational!

Ray Datolli, Emily Haarde, Rich Pezzillo,
Laurie Kelley

After that dinner, at 9 pm, I attended the Committee of Ten Thousand (COTT) meeting, led by the legendary Corey Dubin. We discussed the Living Memorial, a gorgeous “Vietnam Wall” style, stone memorial, to be placed in San Francisco, with the names of all who died of hemophilia/HIV inscribed on it. The artist’s rendition is spectacular, and prompted Jane Cavanaugh Smith, executive director of the Coluburn-Keenan Foundation to donate $10,000! And to pledge matching donations up to $50,000! Nathan and Sonji Wilkes, parents of Thomas, who has hemophilia and inhibitors, immediately pledged $1,000. Corey was touched and grateful, and we all look forward to learning more about the Memorial’s fundraising and financing so we can begin to help fund this, and at long last, close the wound in our community while the survivors are still with us.

The final night was a wonderful buffet dinner sponsored by Biogen Idec, complete with games for the kids and dancing. And what timing. That very day it was announced on the newswires that Alprolix, Biogen Idec’s long-lasting recombinant factor IX, was approved for sale by the US FDA!

So in addition to congratulating HFA on 20 years of service, Biogen Idec also announced to the crowd the news about this game-changing drug. And don’t forget there are many other drugs in the pipeline coming on line soon from many of the manufacturers….

It’s going to be an interesting year, folks.
Thanks to Kimberly Haugstad and her team for a fabulous meeting!

*** Policy explained, April 1 from HFA: “We closed it because inhibitor families asked for it to be closed.”

History Room: 1950s…

History Room: 1950s…

Why Should We Care?

Laurie Kelley and Guy Law: Friends for 20+ years!

AIDS Quilt

Andy Matthews and Laurei Kelley: friends for 20+ years

Laurie Kelley and Sarah Workman

John Parler and Laurie Kelley
Laurie Kelley meets author Shelby Smoak
Julie Heinrich and Laurie Kelley
Laurie Kelley and Juanita Fish!

Dawn Rotellini of NHF!

Sunday morning: EJ, Matt and Jeff
Zoraida and…
… Laurie at the Dali Museum