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!
family reunion, to be surrounded by so many friends and families, some of whom
known for 15 or 20 years.
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
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
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.
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.
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|
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
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!
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.
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
|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