Uncategorized

Clinical Trials: In Need of Patients!

Laurie Kelley February 11, 2013

I am getting emails and requests from both pharmaceutical companies and now HTCs to help recruit patients for clinical trials. It’s tough: we have great products out there. What’s the incentive to try something experimental?

Some patients do it for free factor. Others do it to try to help advance science.

There has probably not been such a clogged pipeline for new products coming to market (we hope) in maybe 20 years. Patents have expired, which protected products from competition, and now manufacturers are all competing to get new products into the biggest hemophilia market on earth: the United States of America.

Here’s one I just read about:

CSL Behring has announced that the first patient has been enrolled in the pivotal pediatric phase III study to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) in previously treated children (up to age 11 years). The study site for this first enrollment is the Czech Republic.

CSL Behring, in collaboration with its parent company, CSL Limited, is developing rIX-FP through the PROLONG-9FP clinical trial program for the prophylaxis and treatment of bleeding episodes, including control and prevention of bleeding in surgical settings

in patients with factor IX deficiency.

Results of a Phase I study evaluating recombinant fusion protein linking coagulation Factor IX with albumin (rIX-FP) in patients with severe hemophilia B were publicly presented earlier this year and published in BLOOD 2012 showing that rIX-FP achieved a 91.57 hours terminal half- life, incremental recovery of 1.376 (IU/dL) / (IU/kg), and clearance of 0.75 mL/h/kg. This was an extension in half-life of 5.3 times that of the current recombinant FIX therapy.

We all know that an extended half-life could potentially reduce the number of injections needed in

patients receiving prophylaxis from two or three injections per week to once weekly or significantly less frequently. Several companies and HTCs involved in research, are now in need patient to volunteer to participate.

If you’re interested, go to www.clinicaltrials.gov, where you can read about clinical trials for hemophilia.

Great Book I Just Read
Why is It Always About You? by Sandy Hotchkiss
You know those people who hog the whole conversation, grab the limelight [often from you], make you their personal audience, and don’t listen or even seem to care about what’s happening with you? They could have Narcissistic Personality Disorder. More than just being talkers, they may latch on to powerful or attractive people to make themselves look or feel better; slowly chip away at another’s self-esteem to build themselves up; and go blank even when the person in front of them is in severe emotional pain. They lack empathy. It’s all about them, their agenda, their needs and their ego. We call them emotional vampires because they can leave you depleted and drained. And yet, they are smart and charming typically, and can draw you in, and back again. Run for the hills when you discover someone has this insidious personality, or get professional help to support you through a relationship with them. This excellent book will help you recognize a narcissist, and teach you how to protect your own self against their manipulations. Three/five stars.

A NEW Co-Pay/Co-Insurance Assistance Program

Laurie Kelley February 3, 2013

It’s Superbowl Sunday, but take time to read this important press release: while it’s meant only for Kogenate® FS users, remember that almost all manufacturers of blood-clotting products have some kind of assistance program. I’ll post them as I hear of them, but don’t hesitate to call your manufacturer to find out what they are doing to help you with insurance!

PSI AND BAYER HEALTHCARE INTRODUCE A CO-PAY/CO-INSURANCE ASSISTANCE PROGRAM

Patient Services, Inc. (PSI) and Bayer HealthCare are pleased to announce the Kogenate® FS
Co-pay/Co-Insurance Assistance Pilot Program. The pilot program is funded by Bayer to help qualified Kogenate® FS patients with their out-of-pocket payments.

What is offered through the
pilot program?

PSI will administer and determine eligibility for the Program to help Kogenate® FS patients afford
their copay/coinsurance expenses.

Patients are qualified to
apply for the pilot program* if they:

· have Hemophilia A.

· meet specific financial criteria for enrollment.

· currently have private health insurance.

. Who is not eligible for
assistance?

·Patients on public health insurance, including (but is not limited to) patients insured through Medicare, Medicaid, TriCare, DOD plans, PCIP programs, and High Risk Plans associated with a PCIP.

· Patients enrolled in Bayer Assistance Programs.

Once approved…

·
Financial assistance may be provided for up to 12 months or as long as funding is available. Funds administered by PSI can only be used towards a patient’s co-pay/co-insurance expenses for Bayer’s Kogenate® FS.

·
Financial assistance will be based on each patient’s specific health plan design and annual income, subject to yearly and per payment maximums.

· As a condition of payment, patients and pharmacies must comply with all contractual obligations made with Third-Party Payers and insurance companies, and must provide notice of any benefits received and the value of this program to all payers as required by law, contract, or otherwise.

For inquiries regarding the Kogenate® FS Co-pay/Co-Insurance Assistance Pilot Program, please contact Bayer’s Factor Solutions at 1-800-288-8374. Please remember that applications will be processed and patients will be enrolled on a first-come, first-served basis. Program may be cancelled or changed at any time without notice.

Interesting Book I Just Read

Ice Balloon: S. A. Andree and the Heroic Age of Arctic Exploration by Alec Wilkinson [Kindle]

The story of S. A. Andrée, the visionary Swedish aeronaut who, in 1897, during the age of Arctic exploration, convinced donors to fund an attempt to be the first to discover the North Pole by flying to it in a hydrogen balloon. The book is an easy read, and covers the history of Arctic exploration, failed attempts to reach the pole, a glimpse into Andrée’s past and makeup, his reasons for being obsessed with attempting this, despite many misgivings by experts. If you are new to readings about polar exploration you will love this adventure book. Learning about balloons, how they are handled and how they stocked it for a long haul trip was fascinating! But… the book is a mixed bag. I have been studying polar exploration for over a decade and have an extensive collection of biographies and books. So for me, this book was a bit fluffy: the writing was good, but there was a lot of filler. Six chapters alone were spent on an unrelated story about the Greeley expedition, I guess just to show how tough it can be in the Arctic? But it was disconnected to the rest of the story, and you’d be better off reading Ghosts of Cape Sabine, which is superb reading about the Greeley expedition (which is now legendary). I am glad Andrée’s story was told, but honestly, there isn’t much to his story. He went up, he came down, and his diaries, terse and factual, left little information on what happened. A lot of the book consists on just quoting directly from the diaries. This books gets mixed reviews on Amazon.com. It’s definitely worth reading, but it could have been better. Two/five stars.

Eye on the Summit!

Laurie Kelley January 28, 2013

A very special week in Hemophilia Adventure History! On Tuesday, Chris Bombardier, a 27-year-old Colorado man with severe factor IX deficiency, sets out to climb Aconcagua, a 22, 847 foot mountain located in northern Argentina, near the border of Chile. It is the highest mountain in the Americas, and is part of Chris’s unprecedented Seven Summits
climb. Unprecedented because no one with hemophilia has bagged all seven
summits—the highest mountains on each of seven continents.

Why is Chris doing this? How dangerous is it?

“Obviously I hope to summit,” the Denver native told me in a recent telephone interview. “I also hope to raise greater awareness of hemophilia globally. Most
people in the States don’t even know about hemophilia; think about how little is known worldwide. I think having someone with hemophilia pushing the limits
is a cool story in itself, but I hope it raises awareness of the discrepancy in treatment.”

Chris knows something about that. He sits on the board of the nonprofit I founded, Save One Life, which is dedicated to assisting individuals in
poverty with hemophilia in developing countries. He also has helped establish a blood testing lab in Eldoret, Kenya.

“Physically, I feel good, strong,” says Chris, an avid mountaineer and adventurer. Chris already has knocked off one summit: Kilimanjaro
in Tanzania, when he climbed in April 2011, becoming the first American with hemophilia to summit it. He is using a long lasting factor in experimental
studies currently, which, he says, is working well. He plans to infuse on the mountain as needed.

Chris will be climbing with two guides and eight other climbers. Chris’s climb is being funded by LA Kelley Communications.

“We start the climb on Tuesday, January 29th with a hopeful summit day around February 7th or 8th, says Chris. “I posted a thorough itinerary of the climb on my new website and blog.” Chris also hopes that he inspires people to donate to Save One Life; while his climbing costs are covered, every penny he raises goes to helping run Save One Life, which serves over 1,000 people with hemophilia who live on about $1 a day.

While in Argentina, Chris also hopes to meet the Hemophilia Foundation of Argentina, one of the world’s first hemophilia nonprofits and one of the best run. Carlos Safadi, a lawyer with hemophilia who sits on its board, also serves on the executive committee of the World Federation of Hemophilia. Carlos writes, “It will be my pleasure to welcome Chris to the Foundation.”

Check out www.adventuresofahemophiliac.com to read more about Chris and his momentous climb! And show your support by making a donation in any amount to Save One Life! www.saveonelife.net

Great Book I Just Read

Buried in the Sky: The Extraordinary Story of the
Sherpa Climbers on K2’s Deadliest Day [Kindle]

Peter Zuckerman, Amanda Padoan

The worst accident in the
history of climbing K2, the second highest mountain in the world but known as
the most treacherous, happened on August 1, 2008, when 11 mountaineers from
international expeditions died. What sets this true story apart from other
mountain climbing stories is that it is told primarily from the sherpas’
point-of-view. The authors get inside the mind-sets of the sherpas who brought
the many clients up the mountain that day; their lives from childhood are
replayed, revealing their sterling character, and how most escaped dire poverty
to become rock-stars of the climbing world. But the “goddess” of the mountain
had other plans for the unlucky climbers: reaching the summit too late in the
day, the return became a race against the dark, the cold when disaster struck.
An avalanche buries the lead ropes, scattering the climbers, leaving some suspended
upside-down all night long, others to walk over the edge, and still others to abandon
their fellow climbers. It’s a tragic tale, masterfully told with great
compassion and in-depth focus on each individual. Most fascinating to me were
the many references to the Nepalese sherpas’ faith in the goddess of the mountain,
and the Pakistani guides’ Islamic faith and how their faiths led them to assist
the many climbers and other guides in trouble, putting their own lives at
terrible risk. This story of
heroism and yes, hubris, was a page-turner, and I finished it in two nights.
Four/five stars.

Scholarship in Honor of a Great Lady: Soozie Courter

Laurie Kelley January 22, 2013

Last week I blogged about a great scholarship from Baxter. This week I want to share another from Pfizer.

Pfizer Hemophilia is excited to announce the launch of the new application period for the Soozie Courter Hemophilia Scholarship program. I knew Soozie from long ago, when she actually lived in the next town over from me. A wonderful woman, devoted to her work in the hemophilia community, who left us much too early.

Pfizer Hemophilia awarded more than $50,000 in scholarships for the 2012-2013 academic year to students in the hemophilia community, illustrating Pfizer’s long standing pledge to help patients with bleeding disorders attend college and further their education. Congratulate all of last year’s winners!

These scholarships are intended for U.S. applicants with hemophilia A or hemophilia B who present the best combination of a creative and persuasive essay, excellent recommendations and superior academic standing. Sign up now to be eligible for the Soozie Courter Scholarship. All applications must be received by no later than May 24, 2013. Scholarship winners will be announced in July 2013. For more information or to download an application, please visit: www.HemophiliaVillage.com.

Great Book I Just Read

Explorers of the Nile: The Triumph and Tragedy of a Great Victorian Adventure by Tim Jeal (Kindle) 2011

I’m a huge fan of reading about African exploration, and discovered this comprehensive gem recently. This book covers all the key players in mid-1800-early 1900 who put their lives on the line to explore Africa’s interior. From David Livingstone’s failed Zambezi exploration to Richard Burton and John Hanning Speke’s joint quest to find the source of the Nile, and their subsequent falling out, to Samuel Baker and attempts to colonize Africa, to the brash Henry Morton Stanley–arguably the most accomplished of them all–this 500-page, exhaustively researched book details the horrors, the triumphs, the Royal Geographic Society politics, the men behind the funding. Different than most other books I’ve read, this one greatly details the coordinents and pathways each explorer chose in their search for the Nile’s origins. It was a bit overwhelming at times! I was deeply impressed by the amount of research Jeal did, and how he faithfully records his sources. Less impressive is the axe he grinds against Richard Burton, who was glamorized in the movie “Mountains of the Moons,” making Speke the inferior and petty man. Jeal found just the opposite and lauds Speke, idolizes him really. I just found the comparisons as to why Burton should not be lauded a bit over the top and personal, and detracting from this otherwise exemplary book on adventure, exploration and colonization of Africa. Four/five stars!

Get the Education Advantage

Laurie Kelley January 14, 2013

Did you know that LA Kelley Communications had the very first on-line listing of national scholarships? We started this many years ago, and now update it yearly. Go to our scholarship page to learn more. But this week I want to highlight one right here.

For the fourth consecutive year, Baxter Healthcare Corporation is sponsoring the Education Advantage scholarship program for hemophilia A patients. Baxter has increased its funding of this program year after year.

To date, 104 scholarships have been awarded, totaling $565,000. Students working toward a bachelor’s degree are eligible for up to $15,000 per year. Students seeking an associate’s degree or pursuing a technical/vocational certificate program are eligible for up to $2,500 per year. Scholarships are renewable for up to three years or until the student finishes school.

The program is administered by Scholarship America, the nation’s leading non-profit scholarship administrator. Scholarship America is solely responsible for reviewing all scholarship applications, determining financial need and eligibility, and selecting scholarship recipients.

The Education Advantage program will start accepting new scholarship applications on February 1, 2013. Completed applications are due
to Scholarship America and postmarked by April 1, 2013.

The program goes beyond financial aid with resources to help people with hemophilia A get more out of life, including education planning, career development, health management and community involvement.

For more information on the program, visit www.thereforyou.com/educationadvantage or call Scholarship America at 877-544-3018.

Interesting Book I Just Read

Confessions of a Public Speaker by Scott Berkum (Kindle)

Scott Berkun may be a professional public speaker, but speaking and writing are two different media. This book is a mixed bag. He shares his own career as a public speaker, trying to be part comic and part storyteller, but neither really works at first. The opening chapters are awkward and clumsy, with repeated references to aliens and spaceships for some reason. Lots of the information he shares is info you can get from much better written books. But midway through the book it does get more interesting. It becomes less about his direct experiences (which are kind of lame) and more about the psychology of presenting, listening and delivering. I found the chapter on TV and other media interesting (perhaps because I don’t do a whole lot of that and wanted to know more).

Know that at least one-third of the book is appendices. These contain good condensed information. I didn’t like his use of profanity,
and wondered what kind of a speaker teaches about what to beware of when he himself swears! Unless you know your audience intimately, unless you are on the level of a Steve Jobs or Bill Gates, or unless you are a comedian, never use profanity for risk of alienating your audience. Two and a half out of five stars.