Novo Nordisk

Lucky Number 13 part 2

Laurie Kelley March 14, 2011

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.

iPhone App Helps in Diagnosis

Laurie Kelley February 20, 2011

Here is the latest in very cool applications for the iPhone. I don’t think I have yet seen one that helps in the diagnosis; I believe this is the first. This is from Novo Nordisk. This app does hematologic testing, including prothrombin time (PT) and activated partial thromboplastin time (aPTT) tests. This resource has been developed to help you diagnose coagulation disorders. Here, you have access to the following features:

Lab Value Analyzer: Input the patient’s coags, and the clinician receives a list of possible diagnoses
Diagnostic Algorithm: this walks a clinician through step by step to narrow the possibilities of the diagnosis
Acquired Hemophilia

Check it out at http://www.coagsuncomplicated.com/Home.aspx

Great Book I Just Read
Annapurna: A Woman’s Place by Arlene Blum

Annapurna is the 10th highest peak, and renowned for avalanches. It takes extreme mental toughness and physical endurance. Of the 4 successful ascents of Annapurna only 2 men had reached the top each time. Arlene Blum became the first woman to ever summit it, and led an all woman team. She was met with skepticism and ridicule, and also great enthusiasm. This book details the preparation and the journey, and her struggles as a leader. It’s a great book about leadership. I had my own doubts reading it, as Arlene seems so insecure. But she is candid, and in the end, her leadership really shines. “My scientific training contributes to a natural tendency to see all sides of an issue. I tend to agonize about the full range of possibilities, and even after I’ve made a decision, to worry interminably about whether it was the right one.” Sadly, two women climbers on the team died when they chose to strike out on their own, against the wishes of the others to summit after Arlene.

Arlene Blum has played a groundbreaking role in US women’s mountaineering. She led the first women’s team up Mt. McKinley; was the first American woman to attempt Mt. Everest; and has played a leading role in more than 20 mountain expeditions worldwide. Three stars.

From Diabetes to Hemophilia

Laurie Kelley August 23, 2010

Novo Nordisk is best known throughout the world as the leading manufacturers of insulin to treat diabetes; in the hemophilia community, it is best known for NovoSeven®RT, an inhibitor bypassing agent, a miracle drug to many. (Photo: with Lars Sørensen and Stephanie Seremetis)

I visited Novo Nordisk’s headquarters in Copenhagen, Denmark on Friday, to speak with CEO Lars Rebien Sørensen, arguably the most famous businessman in Denmark. The country is very small, about the size of my home state Massachusetts, and Novo Nordisk is its biggest company. Denmark is very pretty, a haven and heaven for cyclists, as everyone seems to bicycle everywhere. It doesn’t take long to get to Novo Nordisk from my hotel; indeed, it wouldn’t take long to get anywhere in this diminutive and charming country.

I am struck by how understated Novo Nordisk is. The buildings, designs, interiors are all minimalist, low key, humble even. Entering the executive suites, I am surprised to see that no one has offices per se; just desks, no walls, no glass, only hip-high borders to segregate one work area from another. Quite the opposite of the US and most other places, where executives usually prefer to have corner offices with glass, big cherry furniture, and doors for privacy. Here, everything is open, transparent.

Lars Sørensen came to the US in March (see blog.kelleycom.com/2010/03/spotlight-on-inhibitors-novo-nordisk.html#comments) to meet for the first time the key opinion leaders in the hemophilia community, and to also meet some patients. This meeting now was to follow up on points we touched upon, including products in the pipeline and the company’s policies for international humanitarian donations of product.

I first had lunch with Lise Kingo, executive vice president, Corporate Relations, and Charlotte Ersbøll, corporate vice president, who graciously filled me in on their programs. Next, a meeting with Charlotte and Trine Thomsen; Trine is heading the HERO project, a massive multicounty research that will attempt to quantify and publish responses about the psychosocial aspects of life with hemophilia. (Watch our blog for more news about this)

Last, a meeting with Lars and Dr. Stephanie Seremetis, formerly with a New York HTC. I had heard of Stephanie for so many years and each of us couldn’t quite believe we had never yet met! Lars is an energetic and welcoming executive, and we spoke for an hour about:

1. New products. We spelled out some of Novo Nordisk’s new products in research in the February issue of PEN: long-acting FVIIa; recombinant FVIII (to take on Advate and now Xyntha); a long-acting rFIX product; and a FXIII (13) product. We spoke about causes of inhibitors, patients’ personal experiences and what physicians are requesting (nonimmunogenic factor VIII—in other words, factor that does not cause inhibitors, although it’s not clear yet if how and why factor causes inhibitors)

2. Hemophilia in developing countries. One of my passions is to increase product donations to developing countries, and Novo Nordisk as of yet is not a big donor—although they donate a lot of product in the US, its main market. One problem is production itself: a specialty product for such a rare complication of such a rare disorder means not a whole lot of product sits on the shelf anywhere in the world. In other words, there isn’t much to donate. At Project SHARE we are over the moon when a rare donation of NovoSeven reaches us from some patient, HTC or homecare company, and usually it’s only a few boxes. It will be a while before donations become a routine thing and for now we will be dependent on third parties who donate NovoSeven to us. Novo Nordisk supports Project SHARE’s operating expenses, as do quite a few other pharmaceutical companies and others, and for this we are glad, but we look forward to the day when we and others can also expect product donations.

One interesting insight is Novo Nordisk’s take on product donations. As you can imagine, donating tons of products (just like doling out money to charities without expectation of anything in return) into a poor country can lead to dependency, which only perpetuates a bad situation; this is a constant threat to donations of anything in the humanitarian field. Lars explained that right now, the Novo Nordisk diabetes model has been extremely successful throughout the developing world, and perhaps this is the model to be followed in the future with hemophilia, when emerging markets like India and China start to purchase products. In diabetes, Novo Nordisk’s biggest single financial commitment is its annual donation to the World Diabetes Foundation. At the company’s Annual General Meeting in 2002, the shareholders made an agreement obliging the company over a 10-year period to make an annual donation of 0.25% of the net insulin sales for the preceding financial year (not to exceed approximately $12 million or 15% of taxable income).

In diabetes, the company’s donation policy is tied to product sales: higher sales mean a higher donation to the World Diabetes Foundation, and you can see where this would make good business sense. Also, clinics get established in this model; I saw some of these while traveling in Africa this year. Novo Nordisk is big in Africa, and does a lot to help those with diabetes.

In hemophilia, Novo Nordisk donates about $3 million to the Novo Nordisk Haemophiia Foundation (NNHF). NNHF supports sustainable local and regional projects as well as the advancement of relations between healthcare professionals, patient associations and authorities within the hemophilia community.

In hemophilia, a model like the diabetes model will take a long time to develop. But there are signs: patients in developing countries are integrating and speaking up like never before, thanks to information’s and resource exchange on the internet, and entities like the World Federation of Hemophilia, which brings patients, physicians and nonprofits together to learn and network, and to institute programs that train medical staff. Even at Project SHARE we sometimes, often, dangle product donations like a carrot to get patients organized and to take action. It works, no doubt. But people are still dying, weekly, and we will always need to donate life-saving products with no strings attached sometimes.

Hemophilia is a rare disorder, an expensive one, and we are a small community. Changing the current situation in poor countries will take a long time, and I plan to stick around for as long as humanly possible to ensure that change happens. Just so happens that Novo Nordisk’s slogan is “Changing Possibilities in Haemophilia®” and that needs to be believed no where more importantly than in hemophilia, by the patients, governments, humanitarians and very companies that provide the product. (Photo: Hartnell of Jamaica, who died of inhibitor complications in August 2009 at age 20)

Thanks to Lars and his team for welcoming me, for partnering with us, for sharing so much of their time and ideas. Let’s count on changing possibilities in the near future and make some dreams come true for those patients in countries where no treatment is available now.

Great Book I Just Read
Lord Jim
By Joseph Conrad
This classic was the basis for the movie “Apocalypse Now.” A young sailor, Jim, runs from his past by taking job after mundane job in far ports in the 1800s. He had served as first mate on the Patna, a ship carrying 800 Muslim pilgrims, which hit something in the dark waters one night during his watch and threatened to sink. Jim and the rest of the crew abandoned ship, thinking there is no hope of saving the passengers. The ship does not sink, and when Jim and the crew are rescued, their reputations are ruined. This story within a story is narrated by Marlow, an old sailor, who is fascinated by Jim, and spends the rest of his life trying to help the young man find redemption. Eventually Jim is offered a job to run a trading post in the far reaches of Southeast Asia, so remote few white people have ever visited. Eventually, his leadership and good deeds earns him the title “Lord Jim,” and he is beloved by the people. Soon his honor and character are tested as never before, earning him a last chance at redemption. The writing is amazing, the structure complex, superb, but difficult: some paragraphs are three pages long, and the story within a story can be confusing at times (think of the movie “Inception”!), but a great read; not to be missed. Four stars.

Spotlight on Inhibitors: Novo Nordisk CEO Visits US

Laurie Kelley March 7, 2010

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.

Inhibitor Summit 2008

Laurie Kelley July 28, 2008

The very successful Inhibitor Summit meeting took place this past week and weekend in Denver, Colorado. Over 80 families from all over the US attended to learn about inhibitors, current research, and treatment and insurance information. If you’ve never been with a group of inhibitor patients, you’d be amazed at their level of knowledge and expertise. I guess they have to learn fast, given the nature of the disorder, which is a severe complication of hemophilia.

I arrived a day early to help facilitate the Novo Nordisk Consumer Council, which is comprised of patients with inhibitors and parents of children with inhibitors. It’s a wonderful chance for the patient community to give direct feedback to Novo Nordisk, and to learn in detail about the company’s philosophy, products and ideas. The Council actually helps to shape some of the educational material coming from Novo Nordisk. Working from 8 am to 4 pm, with only a ten-minute break, we really accomplished a lot and learned a lot!

The Summits just keep getting better and better. Dr. Guy Young from Children’s Hospital, Los Angeles, moderated the panel speakers and also gave an excellent overview on inhibitors. He focused on differences between FEIBA and NovoSeven, the two main treatment options for inhibitor bleeds, their efficacy and safety, dosing regimens. Most exciting were the slides showing how effective it is to use these products prophylactically to prevent bleeds. Dr. Steven Pipe gave a very insightful yet understandable presentation on the future of inhibitors: what’s being worked on now around the world–very exciting things. I’m saving what I learned to publish in PEN soon.

I attended a few other sessions: Regina Butler, RN of Philadelphia, and Angela Forsythe, PT, teamed up to give a dynamite presentation on joint disease. This is especially important for those with inhibitors due to the excessive bleeding they face. Angela stressed the important role physical therapy and exercise play in rehabilitating joints. Stopping the bleeds is not the only thing we have to worry about; we need to ensure the joints are cared for to help prevent future bleeds, and future deterioration. Excellent presentation.

There were other talks on pain management, prophylaxis, and central venous access devices. Again, CVADs are very important to inhibitor patients because when they have Immune Tolerance Therapy, they are being infused daily, sometimes for months! Vein access is extremely important.

I was honored to give a presentation along with Glenn Mones, VP Advocacy for the National Hemophilia Association, and Val Bias, colleague, friend and new CEO of NHF. We spoke about the changing insurance scene, what’s causing the changes, how we can advocate to slow down change and ensure we get the insurance coverage we need for these high cost, but life-saving products. (You should know that NHF is working hard to get a bill introduced to raise life-time maximums; go to www.hemophilia.org to learn more. They need your help!)

Novo Nordisk provided a grant to support the Summit, and it was great to see so many families returning from previous Summits. The event was planned and carried out by the amazing staff at CBCE. Thanks to everyone for making this event successful, useful and possible. If you have inhibitors, come to a Summit! The next one is in October in Birmingham, Alabama. If you know of someone with inhibitors, encourage them to register. The trip is free, and the knowledge gained is priceless.

(Photos: Rich Pezzillo and Sasha Cheatham; Dr. Manco-Johnson with the Wilkes family; Glenn Mones of NHF gives presentation; Kari Atkinson of Iowa and Laurie; Val D. Bias)

Great Book I Just Read: Miracle in the Andes by Nano Parrado
I had already read the book Alive by Piers Paul Read, and seen the movie, but they pale in comparison to this account, published 35 years later, in 2007, by the young man who actually walked out of the Andes, after 72 days in the most horrific conditions you can imagine. I could not put this book down. In 1972, a chartered plane crashed in the Andes, with a rugby team from Uruguay, which had been en route to Chile. The players were just boys, aged 17-21 mostly. How they survived and how they escaped is one of the greatest survival stories of all time. But this is a story of leadership, primarily, teamwork, and faith. After you read this, you might believe anything is possible. An enthusiastic four stars!