Inhibitors

Got Rabbits? Their Milk May Treat Inhibitors Someday

PEN has printed in the past articles about coming products, like long lasting and human-cell line products. We’ve also mentioned transgenic animals—which express proteins in their milk that can be used for human treatment of certain disorders. Hemophilia is one of the therapies being researched to create products from transgenic animals.

Charlton [Massachusetts] farm to raise rabbits for medicine

By Lisa Eckelbecker TELEGRAM & GAZETTE STAFF

A French biotechnology company that turns milk from genetically engineered goats into medicine plans to expand operations at its farm in Charlton by raising rabbits that produce a blood-clotting agent for patients with hemophilia.
LFB SA and its Framingham-based subsidiary rEVO Biologics plan to build a colony of 1,000 to 1,200 rabbits making a protein called Factor VIIa at the farm, said Dr. William Gavin, a veterinarian and senior vice president of operations for rEVO.

“We’re going to have the first shovel in the ground in August,” Dr. Gavin said. “About one year later we will be producing milk here from the rabbits that produce the Factor VII in their mammary glands.”

The plan represents the first potential product expansion at rEVO, previously known as GTC Biotherapeutics, since it launched a clot-busting drug called ATryn in 2009. ATryn was the world’s first drug made in the milk of genetically altered animals.

LFB has been producing limited amounts of Factor VIIa in the milk of rabbits in France while also testing the protein in humans. The company said Monday it expects to launch the third and final phase of human studies this year.

If approved, LFB would market its Factor VIIa product as a treatment for hemophilia A and B patients who have developed inhibitors, or antibodies, to other clotting proteins known as Factor VIII or Factor IX.

The global market for blood disorders, including hemophilia, is estimated to reach nearly $64.7 billion by 2017, according to analyst Usha Nagavarapu in a market research report published last year by BCC Research of Wellesley.

NovoSeven, a Factor VIIa product sold by Novo Nordisk of Denmark, posted worldwide sales of 8.9 billion kroners in 2012, or about $1.6 billion in current dollars.

Founded in 1993 as part of Genzyme Corp., rEVO has offices and laboratories in Framingham. The company developed transgenic animals as an alternative to traditional biologics manufacturing.

Transgenic animal production generally starts in a laboratory, where scientists inject human genes into an early animal embryo. The embryo then gets implanted in the womb of a surrogate mother. If the procedure is successful, the animal born will carry code for a human protein in its genes. Then the animal can be bred normally to produce offspring with the human code.

That is how rEVO built its herd of goats on its 383-acre Charlton farm. Transgenic females in the herd produce milk carrying antithrombin III, a protein involved in blood clotting. The company processes the milk to a sterile powder form of antithrombin III for sale.

Dr. Gavin said rEVO plans to bring transgenic New Zealand White rabbits from France to build a new Charlton colony. The company chose rabbits rather than goats to produce Factor VIIa because rabbits can produce the key protein with certain sugars needed for the best therapeutic results.

Rabbits can also produce 200 milliliters of milk per day, or nearly 7 ounces, and they lactate for about three weeks.

Contact Lisa Eckelbecker at lisa.eckelbecker@telegram.com. Follow her on Twitter @LisaEckelbecker.

So I just saw this in the newswires… and go here to read about my visit to this farm a few years ago, and to see pictures of the goats mentioned in the articles.
https://www.blog.kelleycom.com/search/label/GTC%20Biotherapeutics
You can also learn more here: http://www.transtechsociety.org/livestock.php
Great Book I Just Read
Bonk: The Curious Coupling of Science and Sex [Kindle]
by Mary Roach
This best selling author examines the history of the scientific study of
sex, and researches the sex researchers. It’s at once funny, interesting, witty
and head-scratching. From examining artificial insemination of sows in Denmark,
to examining her own physiological reactions in bed while participating in a
scientific experiment she reveals myths and facts about procreation. It’s
science, but with a flair for the funny. The author’s style is dead pan and
spot on. Four out of five stars

A Camp to Call Their Own

By Janet Brewer
The bleeding
disorder community is small, comparatively speaking. Smaller still is the community of families and individuals affected by an inhibitor. But what we lack in size, we make up in our voices.
The hemophilia community has advocated for better care, better products and better programs to support our daily needs. As a result, we have arguably the best treatment model in the world. In the last 20 to 30 years, hemophilia has realized a standard of care that involves routine prophylaxis. This 2-3 day per week regiment enables most affected by hemophilia to lead a relatively “normal” life. Annual chapter meetings, national education conferences offer a plethora
of information specific to those living with hemophilia. Week long summer camp programs where self-infusion is taught and opportunities to meet peers in an environment that promotes leadership are a right of passage for our community.
Yet the smaller subset of individuals and families affected by an inhibitor continues to struggle. An inhibitor diagnosis changes the entire landscape of hemophilia treatment. Routine prophylaxis becomes a thing of the past until the inhibitor is tolerized. Immune tolerance treatment (ITT) can take years of daily, sometimes twice daily infusions and even then, not every child becomes tolerized. There is no single factor product that works to stop bleeding consistently. Bleeds can take days, even weeks to stop. Hospitalizations are frequent and family life is turned upside. Attending a weeklong hemophilia summer camp often becomes a wish. When attendance is possible, there is a high probability that there won’t be anyone else there with an inhibitor. Participating
in many of the activities that other blood brothers can do with prophylaxis is impossible.
As one camper with an inhibitor states, “How I envied them and wished I could
do what they could do.”
Jane Cavanaugh Smith and Janet Brewer conceived the idea for Inhibitor Family Camp. As long standing members of the hemophilia community and mothers of sons with an inhibitor, they endeavored to bring families affected by an inhibitor together in an activity filled, intimate environment and where better than camp? The very nature of a camp setting provides opportunities to try things for the first time, stretch ones limits and create peer relationships.
In 2010, Comprehensive Health Education Services (CHES) sponsored the very first Inhibitor Family Camp at Victory Junction in North Carolina. For the first time, children with an inhibitor and their families had a camp to call their own. Supported by an educational grant from Novo Nordisk, its goal then and still is to bring together families who continue to struggle with the challenge
of an active inhibitor. Families of a child whose inhibitor has tolerized are welcome if space allows. With camp now in its third year, CHES has partnered with Serious Fun camps (founded by Paul Newman), The Painted Turtle in California and Victory Junction in North Carolina to bring two Inhibitor Family Camp programs to the inhibitor community. In response to participant requests, this year’s camp program will offer three nights of education, activities and fun!
Inhibitor Family Camp is limited to 25 families per camp and slots fill up quickly. The Painted
Turtle session will be held Friday April 19 to Monday April 22, 2013 in Lake Hughes, California. Registration opens on January 4, 2013. The Victory Junction session will be held Thursday October 17-Sunday October 20, 2013 in Randleman, NC. Registration will open on July 1, 2013. Each of these programs are offered totally free of charge to eligible families.
Families response to what Inhibitor Family Camp means to them include:
“I loved all the activities. Lots of things to do. The boys had a wonderful time. I have no
complaints except we didn’t want to leave! Thank you!”
“Hanging with the other kids and connecting with them through similar experiences.”
“This program has been fully instrumental to my family in helping us deal with hemophilia and inhibitors.”
For additional information,
please call Comprehensive Health Education Services at 781-878-8561. 
Click below to view Comprehensive Health Education
Services’ 
Inhibitor Family Camp Brochure http://attachment.benchmarkemail.com/c51644/2013_Inhibitor_Family_Camp.pdf

Inhibitor Camp This April

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.

World’s First Inhibitor Book Published


Three years in the making, and it’s finally here–Managing Your Child’s Inhibitor, the world’s first book on inhibitors. At 279 pages, this is a comprehensive look at everything parents and patients need to know about so many aspects of inhibitors. From ITI to pain management, from insurance to attending school, this book weaves together advice from over 40 families with hemophilia, and includes the latest research and knowledge to give you a guidebook through every stage of inhibitor management.

The book is authored by me and my friend and colleague Paul Clement, who also has a son with hemophilia. The idea for the book was born during the Novo Nordisk Consumer Council meeting of 2005, after I heard the anguish, the suffering and saw the tears of so may families with inhibitors. I couldn’t believe what I was hearing. It was as though inhibitors were a separate disorder. I didn’t know people with hemophilia still suffered like that in the US.

As you may know, an inhibitor is an antibody the blood makes, directed against factor VIII or IX. The body doesn’t recognize the infused factor as belonging to the body–it sees it as a foreign invader and mounts an attack. The body produces antibodies which latch on and neutralize the infused factor. It renders it useless, and the blood continues to bleed. Very scary.

I learned so much creating this book: about pain management, different protocols, and the different products. But most of all, the incredible strength of people with inhibitors and their families. They are truly heroic people. I hope the book is a testament to their courage, and also helps give guidance to families new to inhibitors, so they can learn from their peers, and find the best route to elimination or at least management of the inhibitor.

My thanks to Novo Nordisk, which gave us an unrestricted grant to research write and publish this book. Novo Nordisk is the manufacturer of NovoSeven® RT, a bypassing agent used in the treatment of acute bleeds in patients with hemophilia and inhibitors.

You can order the inhibitor book via our website at https://www.kelleycom.com/books.html

Spotlight on Inhibitors: Novo Nordisk CEO Visits US


Last week I had the unique pleasure to meet, along with three other community patient representatives, the CEO of Novo Nordisk, Lars Rebien Sørensen, in the Princeton, New Jersey offices of Novo Nordisk US. To my knowledge, this is the first time he has met with patients from the American hemophilia community. Novo Nordisk is a leader in diabetes therapies, and also the only provider of recombinant factor VIIa for the treatment of inhibitors in hemophilia.

(Photo, left to right: Eddie Williams, Ashley, Schlander, Jurek Gruhn, Laurie Kelley, Mike O’Connor, Lars Sørensen)

With me were Mike O’Connor, chair of NHF and former person with hemophilia—Mike is one of only a handful of people cured of hemophilia through a liver transplant. I’ve known Mike for many years. Also attending were Schlander and Ashley, two great ladies who both served with me on the Novo Nordisk Consumer Council.

From the Novo Nordisk side, we also had quite a few attending, including Jurek Gruhn, President, and Eddie Williams, Vice President, Novo Nordisk US.

So we had a nice reunion as colleagues and friends, and then sat down to a working lunch.

I had learned a few things about Lars before meeting him, such as his passion for cycling. He competes in the Death Valley biking race each year, which sounds terrifying. I’ve read about the infamous ultramarathoner Dean Karnazes and his running Death Valley–his sneakers literally melt on the tar in the 120 degree heat so he runs on the white strips on the highway, in a white reflective space suit to deflect the heat. Hard core athletes. I had just bought my first racing bike two years ago, and we chatted about biking, though I admit I am a bit intimidated by its engineering, and after taking a spill on a major road.

After introductions, I offered some statements from inhibitor families who wrote in a few days before to express comments directly to Lars:

Doris wrote: “Thanks to NovoSeven, my husband was able to have successful hip replacement surgery last September 1. Not in his wildest dreams would he have ever imagined that he would be able to have surgery and live through it. We just want to thank you for developing this wonderful product.”

Karen wrote: “What would I say to the CEO of Novo Nordisk, Lars Sørenson? Well, first and most importantly, I would say thank you from the bottom of my heart. Our son, Michael, now 17, wouldn’t be here without NovoSeven.” And John wrote, “You might tell Mr. Sørensen how grateful our family is for the $2,500 scholarship our son received from his company this year. When you have a bleeding disorder and are used to physical trials and disappointments, receiving a scholarship is a huge lift.”

Some also wrote and asked about the high cost of the product. We had a discussion about healthcare reform, as Mike was just back from NHF’s Washington Days, where 350 of our community attended. Mike reported that the discussion was all about eliminating lifetime caps and pre-existing condition discrimination. With no caps, of course, cost of product would not be such a problem for so many.

Lars and his executive team listened intently, especially about the daily realities of living with inhibitors, which Ashley shared. Infusing every two hours, as prescribed, is very disruptive. He shared a slide that showed new Novo Nordisk products in the pipeline, and one of these is a long acting version of NovoSeven. (See also our latest issue of PEN which details all the new products being explored: https://www.kelleycom.com/newsletter.html)

We wish to thank Lars Sørenson and his team at Novo Nordisk for the privilege of meeting, and applaud his spending time with members of the community, to hear about their needs.

Of our meeting, Lars and his team writes:

We had the privilege last week to meet with members of the US hemophilia community. For those who follow Laurie’s blog regularly, you probably know a bit about this meeting. Novo Nordisk is listening….listening to the wishes, needs, concerns and challenges of people who take our medicines and their loved ones, the physicians who care for them, advocacy groups who drive for change, policymakers who govern how care is delivered, and our employees who make it possible for us to do what we do. We are doing this because we want to be a better company— a better healthcare partner.

It was a very enlightening discussion with Laurie, Michael O’Connor, chair of NHF; Ashley, a mom of two young boys with hemophilia and inhibitors; and Schlander, a woman who cares for her older brother with hemophilia and inhibitors. We were very moved by the discussion, and reminded why it is so important to stay connected to those we serve. Our only regret was that we couldn’t meet personally with more of you. But thanks to Laurie and her network, we did hear from many of you. Laurie shared letters from several of you and the “wish lists” you provided. We know that the challenges and decisions you face are extraordinary and even overwhelming at times. We heard you. You have our promise to strengthen our commitment to you and do our best to expand it where we can.

Sincere thanks and warm regards,

Lars Rebien Sorensen, CEO and President, Novo Nordisk A/S
Jurek Gruhn, President, Novo Nordisk US
Eddie Williams, Vice President, BioPharmaceuticals, Novo Nordisk US

Great Book I Just Read
Ada Blackjack: A True Story of Survival in the Arctic by Jennifer Niven

Another polar survival story (I have a huge collection) and this one is brilliant. But I’m a fan of Niven’s anyway. We pick up after the disastrous Karluk expedition of 1913. Explorer and unethical, shameless self-promoter Vilhjalmur Stefansson is planning a new attention-getting scheme in 1921: to sail to, explore and claim Wrangle Island for Canada. He lures four men into his service (two who have never stepped foot in the Arctic!), with nebulous contracts, inadequate provisions, sickly sled dogs and one impoverished, 23-year-old Eskimo woman named Ada Blackjack, to serve for a year on Wrangle Island. The young men are starry-eyed and eager to make their names; Ada simply wants to earn enough to help get her five year old son treatment for his tuberculosis.

Despite Stefansson’s assurances of plentiful game, the uninhabited island is almost barren. When winter sets in, they are tested as never before in their lives. And Stefansson, who didn’t even go, simply puts them out of his mind as he continues to do speeches and scheme new schemes. Eventually starving, three of the men head out to get help, leaving one behind seriously ill with scurvy, and Ada. The story of how she fended for herself should give anyone courage; she survives, but only to face a media circus surrounding the events of her two-year stay. She is hunted, harassed and used. The aftermath of the trip is as amazing as the survival story itself. Survival of a different kind, from the blood-thirsty public and money-hungry rescue operators. This is an inspiring story of a dignified woman who survives horrific circumstances, and is immortalized forever by Niven. And rightly so. Three stars.

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