A “Bloody Good” Fundraiser

The Cast of Clue Live! with Ron and Robbin

I travel a lot, and so often miss the events that happen in hemophilia close to home. So I was thrilled to know I could attend the New England Hemophilia Association (NEHA)’s fundraising event this past weekend. And it wasn’t a golf tournament (never touched a club in my life) or a cooking class (disaster! Cooking, me?). It was murder.

Murder? Yes! A real-life game of Clue, based on the board game and movie. The movie is hysterical (thanks to actor Tim Curry) as our evening would prove to be!

Mr. Green and Miss Scarlett, who I am sure are innocent

Doug and I drove to a stunning mansion, just south of Boston, that on a cold dark night could really double as a creepy old haunted house. The massive entrance was crowned not with a statue of an angel, saint or cross, but of a devilish-looking, stone ram’s head, with blank, staring eyes. Inside, a wide-angle view of a spiraling, carpeted staircase, chandeliers, portraits of deceased notables, mounted hunting trophies… and the friendly NEHA staff ready to register us.

There was wine, beer, plenty of great company in seeing the families I’ve known for ages, and meeting some new ones. After a social hour, we had a delicious buffet as members of the cast started to appear, in character, sporting British accents. Professor Plum, Miss Scarlett, Mr. Green, Mrs. Peacock, my favorite—Colonel Mustard, Mrs. White, and the Detective from Scotland Yard. But let’s not forget Mr. Boddy, who unfortunately could not make dinner… because he was dead!

Who did it? That was our task. We had to search the mansion for clues to the murderer, and find the murder weapon: a pistol, lead pipe, candlestick, rope, wrench, or knife. It was fun to be with the cast while we prowled about looking under tables, behind curtains and up fireplaces! Everyone got into the act.

This was a fun fundraiser, but it’s also serious business. There are deep concerns in our community about sourcing funds to keep the nonprofits going. Budgets are tighter as companies wait on new drug developments to see how market share will be affected. At HFA in April, all the talk was about how funding was getting harder to secure. And I just noticed this week that NHF’s agenda for the October meeting has a lot of sessions on fundraising.

Jill with Colonel Mustard: did he do it?

Well, Rich Pezzillo, executive director and person with hemophilia, and team did it right this time. They raised about $10,000 in one evening, all the while giving everyone a great time, a silent auction, and an arrest!

And something new: a couple at my table was unfamiliar to me so I asked if they had a child with hemophilia. No, they knew nothing about hemophilia. They saw a brochure for the event at their local bank, wanted a fun night out, and decided to attend. They were amazed at how much they learned about hemophilia. And they left with new friends and a silent auction item! (But those Red Sox tickets were mine)
So fun + prizes + game (+ murder) + out-of-the-box-thinking + inviting strangers = outstanding event and fundraiser.

Rich Pezzillo, talented ED and fundraiser!

Congratulations to Rich and his team for a great event. So what if it was a murder? It was a bloody good fundraiser and a bloody good time.

Click to see all the Gallery photos of the event!

To learn more about Clue Live!

To Market, To Market

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
Volunteer Award 
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
have hemophilia!
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.

Gene Therapy: Lookin’ Good!

I happily drove an hour to Westwood, Massachusetts to attend the New England Hemophilia Association’s Springfest, a gathering of local hemophilia families and the companies and medical people who serve them. It was a glorious, sunny day and a wonderful event. 
Val Bias, CEO of NHF, attended to present an overview of NHF’s programs and also how funding is raised and where it is spent. Here are some stats:
• There are 57 full time and part time staff at NHF over 10 states
• There are 8 regions for HTCs now, down from 12
• NHF has a PDF you can download called “50 Steps to Cultivating Donors”
• 50% of the NHF’s board of directors are not related to hemophilia
• 52% of its budget comes from pharma 
Val stressed to the audience to get involved locally, and to consider joining the NEHA board. He made a compelling plea and is an excellent speaker. Many people told me later they thought Val is a gifted presenter and a wonderful leader for our community. 
Next up was a great presentation on gene therapy and new products coming soon by Dr. Ellis Neufeld of the Boston Hemophilia Center. Dr. Neufeld did an amazing job of taking highly complex material and breaking it down for us. Some highlights:

• The St. Jude gene therapy trial that has been in the news: only 6 FIX patients in trial, using “gutted” virus (no capacity to reproduce).
• Of 6, only 1 really took to it but he got higher ALT (liver
function measurement). 
• Problems with the study: not enough data, immune response a threat and you can’t be retreated
as you will have an immune response. 
 • Long acting factor: many companies working on this. Everyone has different half-lives; children have shorter
ones than adults. So how long is long? New FIX drugs could be 3-5 times longer acting, meaning you might treat once every 10-14 days?
• What will price be of the longer acting drugs? What is the worth for a theoretical improvement of
life? No one yet knows.

(Our next issue of PEN in May examines all these in depth: be sure to download it!)

Dr. Neufeld was very positive about current gene therapy efforts. He made us all laugh by saying that our community has been promising us gene therapy roughly in every ten year cycles, but this time, he truly feels there is a great chance we will find it.
Just before lunch, we had panel presentations from all the pharma and specialty pharmacy reps on their patient assistance programs, delivered all above the growing din from the Bar Mitzvah next door!
These were all great presentations, and we had an attentive audience. I saw so many of my friends in hemophilia, and truly enjoyed myself. I hope you can all attend a local hemophilia event and take part in your community! We had a rap session with the moms, and one among us had an 8 month old. We all remember the feelings of when our babies were diagnosed. We felt for her. Everyone rushed to welcome her, praised her for attending, and offered email addresses and phone numbers. It never fails to amaze me how tight our community is. We are friends, and family, for life. 












Great Book I Just Read
Murder in the High Himalaya: Loyalty, Tragedy, and Escape from Tibet
by Jonathan Green
This is a rare book that provides multiple levels of reading, on history, ethics, exposé, culture, politics and an unforgettable story of a young girl’s perseverance, determination and tragic legacy. I love real life adventure and survival books, especially about mountain climbing and/or history, and also books about how one person can change the world. This book has it all. On September 30, 2006 a cruel and thoughtless murder of a 17-year-old Tibetan nun (just a girl truly) by Chinese border guards triggered an avalanche of scrutiny in the press, world agencies, political leaders–and in the consciences of the Western mountain climbers on a paid guided climb who watched in horror. Kelsang Namtso was trying to escape Tibet via the mountain Cho Oyu when she was gunned down in full view of climbers, one of whom videotaped it. The tape went viral—the first time human rights violations against the Tibetans had been filmed—and the rest is history. This book tells this amazing story in gripping and often exquisite prose (much like Jon Krakauer) and provides powerful parallel stories of Kelsang and the mountain climbers, which later intersect dramatically and make history (much like author Erik Larsen). You will be amazed at the courage of this 17-year-old, and at the response by those who witnessed her death. Green’s compelling narrative will teach you much about Tibet, how it has suffered under Chinese rule, and how the West has looked away until one lone video clip, still available on YouTube, shamed us into action. Green raises excellent questions that beg an inward look at our own souls, and portrays Tibetan lives without freedom that make us instantly cherish our own freedom. A must read. Four out of five stars.

SpringFest!


It doesn’t feel quite like spring yet in New England, but Saturday was SpringFest, the New England Hemophilia Association’s annual family symposium. I was very happy to be home, so I could attend this event, a mere 15 minutes from my house. Our new executive director of NEHA, Kevin Sorge, said it best: “This is like family.” And it is. We were reunited again, for some of us 20 years now, our sons grown but our desire to see this community continue to progress still burning bright.

Even some of the pharmaceutical and home care reps have been part of our lives for 12-20 years. How fast it has gone by!

Linda Price of Virginia will be thrilled to know I finally met her son Greg, now living in the Boston area. Last time I saw Greg he was about 10. It was a lively time, because of how long we’ve all known each other and how rarely we get to see one another.

The speakers and topics were good, and the turnout was excellent, despite the rain. There were lots of children, and teens. My own son couldn’t make it, and he missed some of his long time hemophilia friends.

The highlight of the day without doubt was a moment that reduced us all to tears: an award ceremony, with our shining star, Leland Smith, age 15. I first met Leland when he was only a toddler. He has hemophilia, and a devastating inhibitor that at times keeps him wheelchair bound, as he was this day. I know his story well, and I can tell you that not many people has endured suffering like this child. The time he has spent in the hospital, the treatments he has undertaken, the missed school, the excruciating and unrelenting chronic pain; and yet he wants to be a doctor, and is an active lobbyist for NEHA. He has traveled to Washington DC and also to the Massachusetts state house to present himself, to help NEHA get bills passed to protect the rights of those with hemophilia. To know how much this kid has gone through, and to see how at such a young age he is active socially, well… we all crumbled inside in admiration of his character and his strength. Leland reminds me of the words: It is in the deepest valleys that the stars shine brightest; it is pressure that turns coal into diamonds. Congratulations to Leland, a true leader of a new generation of young men with hemophilia. Thanks for making such a memorable day for us all!

Great Book I Just Read: Mawson’s Will by Lennard Bickel.
Speaking of pain and suffering, you haven’t read anything till you have read this book. Dubbed the greatest polar survival story ever, this tells the true story of Douglas Mawson, a giant in the age of polar exploration in the early 1900s. Mawson, an Australian, was already a veteran polar explorer at age 29 when he traveled to the Antarctic in 1911 to seek the magnetic pole and to chart unknown territories. Splitting into three groups, he and two other men part with his crew for a few months and enter the incredibly hostile terrain. Mawson loses both men on his team and is left utterly alone, without enough food, shelter or dogs to see him back, in 50 degree below zero weather with gale force winds…. for one month. How he survives is a page-turning, gripping and miraculous tale. Mawson is responsible for Australia’s claim to 45% of the Antarctic. His tale is legend. This was the fourth time I’ve read this book and it gets better each time. Four stars!