Our Brave Explorers

I recall watching the movie “The Lost City of Z” this summer, about Percy Fawcett, a British soldier turned explorer who several times went deep into the heart of the Amazon to locate what he thought would be Eldorado, the city of gold. The movie clearly showed what he gave up for years at a time: beautiful, intelligent wife, three adorable children, a home, relationships.
What it didn’t really tell you was why. Why abandon all this, leaving your children to grow up fatherless, your family to slide into poverty, to suffer the terrible depredations in search of something that might not even exist?
 
I asked that this week when I learned of the death of a patient with hemophilia in a clinical trial for a new factor product. We have good treatment here in the US; we have safe and efficacious products, dedicated HTCs. Why risk your life for a product that may or may not work?
 
To me, the patient was himself an explorer. Willing to risk all for something greater than himself. A hope of a new treatment, an advancement in science and medicine. We don’t know who this explorer was. Surely someone in our community knows who is it, and eventually it will be revealed, but for now, until an investigation is complete, he will remain known as an Explorer.
 
Facebook has lit up with comments, some cautious, some not so cautious, about the risk involved in clinical trials. Is it worth it? Did the patient really understand what he was undertaking? Most likely he did. Like Fawcett, explorers research, read, learn and
understand what could happen. They know the risks. But they still seek to make a lasting contribution. I’ve studied for decades the lives of the explorers: what makes a person a Columbus, an Earhart, a Livingstone, a Scott, a Shackleton, a Mawson, a Magellan, a Hillary? True explorers are in a class of their own as they forge a path through life.
 
George McCoy of North Carolina
While this news is making headlines throughout the country
and world, more quietly, another explorer just died on August 23, after
fighting pancreatic cancer. George McCoy was born in 1947 and had severe
hemophilia A; he contracted HIV and hepatitis C. He earned a master’s degree in
education from the University of NC, and worked for 31 years for NC Department
of Health and Human Services, helping people with disabilities. He helped found
Hemophilia of North Carolina, which is today one of our best run patient
organizations. He never stopped working for the community, from stuffing
envelopes to public speaking.
 
His amazing claim to fame is that he was the first person dosed with recombinant factor VIII in 1987.
George McCoy and Laurie Kelley 2012
He was an explorer, volunteering for clinical trials with these new, biotech drugs. It was considered risky then; these were genetically created drugs, a new field in medicine. And these drugs revolutionized hemophilia treatment at a time when the community was suffering through the devastation of HIV.
 
George was a daring explorer and public servant, who dedicated his life to serving those in need. I met him in 2012 at the World Federation of Hemophilia Congress in Paris, where he gave a profound speech about his volunteering for science.
 
Read “MadMan or Guinea Pig?” About George McCoy
 
It’s human nature to point fingers when tragedy strikes; we feel someone must be held accountable. An investigation is underway to find the exact cause of the patient who died, but there is speculation and some information; a note on the Facebook page “BioSpace” says, “The patient was suspected of having viral meningitis and died from a thrombotic event.”
 
 
And one of our popular advocates in the community on Facebook wrote: “He gave his life so that we may see better treatment… Please think carefully and fully understand the risks entailed when your HTCs ask you to join a study…If you ever find someone pushing you to participate in a drug study, pay attention to their motives… Thank you to whoever it was that lost their life in this drug study.”
 
Whoever it was, like George McCoy, he was one of our bravest explorers.
 
 
 

 

 

It’s Just a Phase: Clinical Trials of New Products

We’ve witnessed several new products come to market in the past few months; it’s pretty astounding, given that years went by without any new products. You’ve probably heard about others that are in clinical trials. What does that mean, exactly?
The FDA requires that new drugs and treatments be tested for safety, and also to demonstrate that they effectively treat the disorder or disease for which they are being developed. Drugs always start with a
phase I clinical trial, and then may move to phase II and phase III trials. If a drug is found unsafe or ineffective at any stage, then the clinical trial will be ended, and research on the drug will be either modified or abandoned.
Phase I: After a new drug or treatment is initially tested on animals, researchers test it on a small group of people (20–80) to evaluate safety, determine the safe dosage range, and identify side effects.
Phase II: If a drug or treatment appears safe at the end of the phase I trial, researchers give it to a larger group of people (100–300) to evaluate its effectiveness in patients, and to further assess safety.
Phase III: At this stage, the drug or treatment is given to an even larger group of people (1,000–3,000) to confirm effectiveness over a broad range of patients, monitor side effects, compare to commonly used treatments, and collect information to promote safe use. For rare disorders such as hemophilia, it’s almost impossible to enroll 1,000 patients for a clinical trial. In these cases, the FDA will approve
phase II or III trials with much smaller populations.
Phase IV: Post-marketing studies provide more information, including the drug or treatment’s risks, benefits, and best use.

Clinical trials for new bleeding disorder products can take place only when patients volunteer to be in them. This may seem risky, trying a new product that hasn’t been FDA-approved yet. Yet you can participate in a clinical trial, to help research, to take advantage of a new product that is needed, or simply for free factor. You’ll need to speak with your HTC about joining but k
now as much as possible about the clinical trial you are interested in, 

and feel comfortable asking your healthcare team 

about it. Use these questions as a guide:



• What is the purpose of the trial?

• Who is going to be in the trial?
• Why do researchers believe the experimental treatment being tested may be effective? 
• Has this treatment been tested before?

• What kinds of tests and experimental treatments are involved?

• How do the possible risks, side effects, and benefits compare with my current treatment?
• How might this trial affect my daily life?
• How long will the trial last?
• Will hospitalization be required?
• Who will pay for the experimental treatment?
• Will I be reimbursed for other expenses?
• What type of long-term follow-up care is part of this trial?
• How will I know that the experimental treatment is working?
• What happens if I am harmed by the trial?
• Who will be in charge of my care?
Patients who volunteer to be in clinical trials are truly heroes and trailblazers. Perhaps you will be one of them someday!

Adapted from clinicaltrials.gov/ct2/info/understand


Great Book I Just Read

Deep Down Dark: The Untold Stories of 33 Men Buried in a Chilean Mine, and the Miracle That Set Them Free [Kindle]


Hector Tobar

Astounding survival story of the 33 Chilean miners trapped over 1,000 feet underground for 69 days. Tobar skillfully introduces each man, his home life, aspirations, character flaws… all which would impact each’s reaction and perspective during their ungodly entombment. As in many crises, the positional leader does not always become the situational leader. This is as much a story of survival as it is leadership. Their time underground is gut-wretchedly and vividly portrayed. It seems a true miracle that they survived. The efforts to help the men spread internationally, and forever changed the Chilean mine industry. Five/five stars. (The movie “The 33” is good but not nearly as good as the book)

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.

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