Blog Archive

Monday, March 31, 2014

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






Sunday, March 23, 2014

Insurance Workshop in Nebraska: Pulse on the Road 2014


Kristi Harvey-Simi and Laurie Kelley

Warm air greeted greeted us in Omaha, Nebraska on Friday as Zoraida and I arrived from Boston to set up for our first Pulse on the Road workshop of 2014. POTR is a three our workshop, sponsored by Baxter Healthcare and held in tandem with the National Hemophilia Foundation, which so generously lends us the incomparable Michelle Rice, mother of two adult sons with hemophilia, and director of public policy at NHF. Last year she hired more staff to help her, as she is stretched thin visiting chapters, patients, insurance companies and governments in all 50 states. On this trip we were thrilled to have Nicole Quinn-Gato of NHF, also a policy specialist, accompany Michelle.


Kim Isenberg of Baxter Healthcare
The warm welcome by Nebraska Chapter of NHF executive director Kristi Harvey-Simi was followed by an update on the Affordable Care Act by Kim Isenberg, Senior Manager, Reimbursement and Advocacy, Baxter Healthcare. She covered the ACA in general and as it affects hemophilia, noting exceptions for some groups. Nevada has elected not to expand state Medicaid, an important policy to note.

Michelle Rice of NHF
I then presented important points on the importance of choosing healthcare, since it is now mandated that almost everyone have healthcare. We need to still watch out for certain variables, such as out of pocket costs, which may increase as a result of changing policies and plans.

And finally, Michelle and Nicole did a great job proving a 90 minute, hands-on workshop to access the healthcare.gov website, using laptops and iPads, and actually logging on to scope out choosing a plan! It was interesting and frustrating at times! Sometimes it’s hard to find your HTC, was the most common problem.

After lunch there was a Q&A, where the audience could ask questions of the experts.

Though I offered everyone a chance to go home an hour earlier, nobody took advantage: I think they were really soaking up all the information Michelle, Nicole and Kim had to offer!

Next stop for POTR? Philadelphia in June!                 
Nicole Quinn-Gato of NHF
 



Sunday, March 16, 2014

Inhibitor Summits are Coming!


I was present way back at the first ever inhibitor summit meetings, brainchild at the time of George McAvoy of Novo Nordisk, and funded by Novo Nordisk. Now run by National Hemophilia Foundation (NHF) with funding from Novo Nordisk, NHF is pleased to announce the 2014
 Inhibitor Education Summits, designed to specifically cater to your inhibitor educational needs. Come join this dynamic event and interact with expert healthcare professionals as well as other patients and their families for a weekend of education designed to improve your overall health and quality of life.

The Summits provide:

• Travel and lodging financial assistance provided for eligible patients and their caregiver(s)
• Both locations accessible to wheelchairs and other mobility devices
• Four different educational tracks tailored to suit your needs as a patient or caregiver
• An Interactive Education Camp for Youths, including an off-site activity (Ages 4-12)
• Childcare for infants-3 years old

To learn more, contact NHF:  877-560-5833 or inhibitorsummits@hemophilia.org
or go to https://www.nhfinhibitorsummits.org/register.aspx

Great Book I Just Read
Minus 148 Degrees: First Winter Ascent of Mount McKinley
 by Art Davidson [Kindle] (1969)

This is a classic in mountaineering books. Young, impetuous and even reckless climbers, all skilled, set out to "conquer" Denali in the first winter assualt. The mountain humbles them in ways too painful and detailed to spell out. It is an amazing testament to leadership, teamwork, lack thereof sometimes, hubris and human spirit. When a raging winter storm barrels in, only a snow cave stands between the climbers and death. With virtually no food, frost bitten, and disorientated, they wait out the storm in 148 degree below zero weather, and struggle back to freedom and go down in history. What keptthem going? A fascinating read on a cold winter night. Four/Five stars.



Sunday, March 09, 2014

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. 
http://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

Sunday, March 02, 2014

Hemophilia Awareness Month

In the US, this is Hemophilia Awareness Month, a chance for us to let others know who we are and what we need. Our community just did that last week on Capitol Hill, storming the hallowed halls and meeting with state representatives and senators when possible, to inform them about bleeding disorders and mostly about our funding needs.

I participated for the first time last year and loved it. An illness kept me away this year (all better now!) and how I missed it! I was so impressed with the event. It's a chance for all of us, well known and not so well known, to share our stories. In fact, it's most impressive when the average mom and dad, and even their children, meet with Washington folks to tell them about life with hemophilia.

Visit the NHF website (www.hemophilia.org) or HFA website (www.hemophiliafed.org) to learn more.

Speaking of HFA, what a fabulous website they have! And their annual meeting is coming up on March 27 in Tampa, Florida. While there are no scholarships left to support attending, if you can at all try to attend, please do. It's a very different flavor than the NHF events. Both are great, but different.

What can you do for Hemophilia Awareness Month? First, learn more about the disorder yourself. It's hard to have others appreciate what you endure if you don't have a ready and coherent "script." I was on Facebook most of the weekend contacting the many people who have reached our to me this past year, sorting out who is actually related to hemophilia, to keep them as friends. Some are first-time parents of a child with hemophilia and I am delighted to send them our educational materials.

Second, check in on our website (www.kelleycom.com), as well as NHF, HFA, PSI (www.youneedpsi.org) and your local hemophilia organization. Make it a habit every couple of weeks to check out a new website. There are so many!

Try to attend a hemophilia event. It might be the HFA one in March, the NHF one in Washington DC in September, or maybe a local one near you. Meet families, meet physicians, meet factor manufacturer reps. The bottom line is--get active and involved! We need you and the world needs to know about hemophilia. Help pave a great future for your child starting this month!



 
Bayer