I (Went) Back to Michigan

That’s the name (sort of) of a Judy Garland song from the movie “Easter Parade.” And I went back to Michigan this past weekend, for the annual Springfest! The Michigan Hemophilia Foundation invited me to attend the showing of “Bombardier Blood” on Friday night. As I walked the long Marriott hallway to the conference room, I saw the stunning visual timeline, created by Shair Luckey, and realized that Ypsilanti, Michigan was also my first professional appearance in the hemophilia community, in 1991, six months after my book Raising a Child with Hemophilia was published (and my second child was born). How incredible was that! I promptly add my sticker to the wall on 1991, to become part of this rich history.

And the Hemophilia Foundation of Michigan has secured its place in history: it was the first hemophilia organization in the world to have a summer camp, Camp Bold Eagle! How about that for vision?

The movie was great–again! The audience was enthralled, and afterward we had a Q&A all about Save One Life. Of all the comments I made, the one that seemed to stick was the one about family life in developing countries. How strong families often seemed because of survival. There is typically no social welfare, no nursing homes; so family is all you have for a safety net. The eldest son is usually responsible for the survival of the family, and when that child has hemophilia, the entire survival of the family is cast into doubt. Save One Life, in helping one child, can then save an entire family. In the US, we are so used to being independent, that this concept seems rather foreign to us. So it was a revelation and I was asked about it again and again the next day.

On Saturday, Pat Torrey gave a lively and inspiring talk about finding your passion and dreams. Rick Starks gave Tai Chi demonstrations and classes. Dr. Steven Pipe, chair of NHF’s MASAC, gave his talk “Shifting Paradigms,” which I’ve known seen for the second time. Absolutely fantastic; all about understanding current therapies, new therapies and coming therapies. He is gifted in being able to explain very abstract and difficult scientific concepts into simpler terms, using analogies like Amazon.com and eHarmony! The audience rippled with laughter when Pipe described Hemlibra as being the “eHarmony” of hemophilia, bringing factor IX and factor X together to mimic factor VIII, and thus clot the blood. Who would have thought of blood clotting as a love story? The audience sat still and attentive for an hour, completely immersed in this great presentation.

And the positive talk continues about gene therapy. Present at the booths were Spark and uniQure, two companies with gene therapy in clinical trials. Exciting times ahead!

I was just content to reconnect with so many friends who I often do not have much time to speak with: Deena Maki, Vaughn Ripley, Rick Starks, Suzan Higgins, Dave Luckey. Great people doing great things. We are a community of true superheroes!

Gene Therapy Clinical Trials and You!

A special request and letter to the hemophilia B community!

As you may know, since 1990 LA Kelley Communications has been educating the hemophilia community about hemophilia. In the February 1998 issue of PEN, we actually were the first to break the news to the world about the first hemophilia gene therapy trials. Gene therapy is a subject we care deeply about.

Gene therapy for hemophilia is moving closer to becoming a reality, but it can’t become a reality unless patients participate in clinical trials.

uniQure is one company that has reported encouraging data with respect to factor IX levels in its recent phase 2b Dose Confirmation trial for a hemophilia B gene therapy. Discover more about uniQure’s current gene therapy trial for hemophilia B by reading a message from uniQure, and clicking through the link below. And please consider whether you’d be interested in participating in a trial!

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My name is Dan Leonard, I am the Senior Director of Global Patient Advocacy at uniQure. uniQure is a company developing an experimental gene therapy to treat severe and moderately severe hemophilia B. We are currently conducting a phase III clinical trial – known as the HOPE-B trial – and wanted to make the community aware. You can find more information about the clinical trial at the website www.hopebtrial.com . You can also find more information at a government-sponsored website, www.clinicaltrials.gov . Simply enter NCT03569891 in the “Other terms“ search field, and you will see where the clinical sites are located along with other important information.

The most important step to take when considering a clinical trial is to speak to your doctor. Your doctor can help you understand if a particular clinical trial is appropriate for you and if you meet the criteria for the trial.

Sincerely,

Daniel Leonard, Senior Director of Global Patient Advocacy, uniQure, 339-970-7068 . 339-970-7068

This is a paid public service notice from uniQure

HFA: 25 Years of Advocacy

Hemophilia Federation of America (HFA) celebrated its 25th anniversary this past week in San Diego. I remember HFA in its infancy, when its annual meeting drew about 100 attendees. This past week over 1,200 people, mostly consumers, gathered from all parts of the country to learn, socialize, hold remembrances, and give awards.

Laurie Kelley with Carri Nease and family

An HFA meeting is also a chance to network, to brainstorm new ideas to meet the ongoing needs of the bleeding disorders community. Three hot topics? Women and bleeding disorders: how to include them in our data, discussions, treatment and future. Gene therapy: what will it look like, cost and be available? And perhaps the most urgent: mental health issues and how to identify and treat them.

This last issue for me is paramount. A year ago we lost Barry Haarde, who was a prominent contributor and attendee at HFA. His absence was very much felt. Since his passing, so many community members have opened up about their own mental health issues, including depression and anxiety. To address these issues, I was privileged to listen in on some exciting programs and ideas that could help not only patients but caregivers better deal with stress, sadness and overwhelm.

My good friend Debbie de la Riva, who knew Barry, a fellow Texan, started “Mental Health Matters Too,” following his death, to bring the issue forth in meetings across the country. Her program is appearing everywhere it seems. Visit Debbie’s website, www.MentalHealthMattersToo.com, to see if it will be coming to your state. I know it’s coming to New England in October, and I hope to attend.

HFA CEO Kimberly Haugstad
at Award Ceremony

The three days of meetings felt like a week, and I mean that in a great way! It’s always a wonderful feeling to spend time in this community, which is like family. I’ve been in this community for 31 years, and one of my highlights of the week is to bump into people like Sam Doughty of Tennessee, who I have known since he was a child. Age 34 now, and president of the Tennessee chapter, Sam is a fine example of young men who have taken the reins not only of their disorder but also of the future of the hemophilia community.

The conference was capped off with an incredible final night aboard the USS Midway. Above the sea and under stars we had drinks, food, dancing and fireworks! Check out photos here to see more! Thank you, HFA, for making such a huge difference in so many lives, and for giving us the chance to have a reunion; it strengthen the bonds and brings us joy. Bleeding disorders can never defeat us!

Remembering a Visionary

March is Hemophilia Awareness Month, and this year, also the 15th anniversary of the passing of a visionary in our community: Dave Madeiros. Dave passed away from complication of hepatitis C at age 47 in Boca Raton, Florida, just two days after I had visited him. He was a friend, mentor, businessman and amazing visionary.

If you ask what the greatest complication of hemophilia is in the US now, many will say insurance. It was Dave who first sounded the alarm in 2003 that insurance was undergoing massive changes that would eventually impact hemophilia care. Each quarter, Dave would invite a select group of people to his headquarters in Boca, to share and brainstorm about what we called “The Coming Storm.” Included in this group was Dana Kuhn, founder and president of PSI, very knowledgeable about insurance issues. And Ed Burke, who worked for Dave. Dave was founder and president of Factor Foundation of America, a specialty pharmacy that gave away profits back into the community. Dave’s dream was to become the number one specialty pharmacy serving the bleeding disorders community, where 70% of profits would be channeled back into the community.

Dave predicted the coming changes in insurance amazingly well. He told us as early as 2003 that insurers would: start restricting access to certain therapies; would challenge whether patients needed so much factor; would start step therapy; would focus on the bottom line more than on patient needs; that PBMs would become megaplayers in the industry.

Of course, he was right. All that he predicted came true. And he predicted this at a time when no one else in our community seemed to know anything about what was to happen. NHF had no clue; they were busy undergoing an internal restructuring. Based on what Dave shared, I wrote an article called “The Coming Storm” in PEN, outlining the predicted changes. It became one of our most popular articles, and indeed, the community started referring to the rapidly changing landscape as the coming storm. These changes continue even today. NHF and HFA got on board after the article was published, and now are the foremost advocates for preserving access to care nationally. (Download the article here)

I feel we as a community owe a lot to Dave. Myriad presentations, meetings, marches, and more are all due to his early efforts to be like Paul Revere, and warn us that the “Payers are coming!” He had insight and shared it; he changed the way we thought about our community, roused us from complacency, and put us on the right path to protect our needs and interest.

We lost a great man when he passed away February 29, 2004. He left behind a wonderful wife, Kim, who you might meet these days at a hemophilia meeting. He left behind his prized car, a Mustang Shelby. But mostly he left behind an incredible legacy. Let’s remember him and honor him for eternally helping our community, as a visionary leader.

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