At last! I am not traveling, and I have time to attend the hemophilia events in my own back yard. This one is Fall Fest, New England Hemophilia Association’s biannual event. Held in charming Providence, Rhode Island, we had about 100 people attending to hear speakers like Barry Haarde, who rode a bike across the US four times; Dawn Rotellini of NHF; and Perry Parker, golf pro. I consider all these speakers not only interesting, but also friends!
In fact, and I know this isn’t original, but it really was like a family reunion, to be surrounded by so many friends and families, some of whom I’ve known for 15 or 20 years.
The general session opened the day, with guest speaker Dr. Kapil Saxena, Director, Global Clinical Leader, Global Clinical Development, Hematology, of Bayer Corporation, formerly a pediatric hematologist with the Boston Hemophilia Treatment Center. He spoke about how a drug like factor is brought to market. It was excellent and informative, and stunning. Some highlights:
Every drug on the market starts as one of almost 10,000 compounds. Which one has a chance to be brought to market one day? The Drug discovery journey sorts through those 10,000 compounds, to narrow the field to 259 compounds (this can take up to 6 years). These are furthered narrowed to 5 compounds (another 6 years!), which then are brought to the FDA for review. When approved, they go into clinical studies, which can last up to 1.5 years. Altogether, a drug like factor can take almost 14 years to get to the market.
|Laurie Kelley with Lee Hall, CoRe Manager |
and person with hemophilia
Dr. Saxena explained the stages of clinical studies. There are four stages, and some get killed off in the first stage, Phase I, for safety reasons. Phase II examines efficacy and safety. Phase III, if it makes it that far, involves a large number of patients, from 1,000-3,000 (although that can’t possibly be true for hemophilia). More data is gathered over many clinical centers. For hemophilia, these involve sites now in India, Egypt, China and more. After the drug is brought to market, Phase IV looks for any more problems, now that the drug is in widespread use.
His talk was relevant to our hemophilia marketplace today. With the patents expired on factor VIII, IX and FVII molecules, our R&D and production pipelines are filled with new products. Pharmaceutical companies are banking on selling these, and yet the marketplace is getting crowded. Dr. Saxena’s talk really made us think about the extraordinary effort—and cost--that goes into making any one hemophilia product. On the one hand, these are among the most scrutinized products on earth, and that’s great. On the other, how will the market sustain all these great products?
|Barry Haarde and Christian Mund|
It will be interesting to monitor and to check in a year from now.
The afternoon sessions were a bit lighter! I attended one starring—I mean featuring Barry Haarde! Of course, Barry is pretty much a celebrity by now in this community for his heroic rides across America. Six weeks after completing a 108 mile ride in one day, I still am having severe shoulder pain. Barry does that in a day, and then goes out and does another 100, then another, then another… he’s Superman! He and Christian Mund, a young man with hemophilia, spoke on setting goals, making dreams come true. Christian lives right near me and I’ve known him since he was 12. And he went his first 12 years without being involved in the community at all. Why? Life was good; there was factor, prophy, and no need! But as he got involved, he enjoyed the rich relationships and friends he made. He went through Bayer’s Leadership program, and landed an excellent job at a marketing firm in Boston! Way to go!
Barry shared his story about how he got involved too. Barry went most of his adult life without being involved. Shielding his HIV/hep C positive status from everyone kept him from making connections. But he eventually joined our community when his brother passed away. And boy did Barry join it! No one in the US can compete with Barry on the cycling front. He’s made history at this point, and raised about $170,000 for my nonprofit Save One Life. But his message was simple: find what you can do and love to do. You’ll never know where it will lead.
The day finished off with a dynamic presentation by CoRe managers from Biogen, and also community members: Lee Hall, person with hemophilia, and Lisa Schmidt, former program manager for NEHA. It was inspiring and fun. I’m not going to give away their presentation, because… you should go yourself! I give presentations, and know all the tricks and topics, been doing this for 25 years. But I learned a few really wonderful things at this presentation.
|Jane Smith with her |
We had to hustle out the Omni as the Redskins were approaching for their game with the Patriots! Like Rhode Island, the hotel was kind of small, so we headed home, really feeling happy after spending a day with “family.”
|Heather Case of NEHA, John Bruno and Maryann May|
|The lovely ladies of NEHA!|
|Laurie Kelley dwarfed by golfer Perry Parker and|
baseball player Jesse Schrader, who both
|Patrick Mancini, NEHA president, presents|
Dennis Mackey with an award
|Laurie with Leslie Oygar, RN, who she wrote to 26 years|
ago and now just met!
Good Book I Just Read
The Paths We Choose
Sully Erna [Kindle]
This autobiography by the founder and lead singer of the band Godsmack is not technically a great read, but it's raw and real. The first full 60% of the book is a detailed catalog of Sully's scrappy and difficult childhood, growing up poor in Lawrence, Massachusetts, with street fighting, crime and later on, drugs and alcohol. His breezy writing style is actually sweet, and bittersweet. You wonder how he ever escaped the beatings (received and doled out), the heartbreaks. Music was his driving force, his salvation. When he gets to the bands, and the formation of Godsmack, it all tied in. His survival skills gave him what he needed to take the huge risk to break out on his own, and become a frontman and founder of a great band, and a great sound. The book ends with his advice to all those with dreams and broken hearts to never give up, no matter what you have to go through. A quick, two-hour read, and I really enjoyed it and love the band's music. Two/five stars.