clinical trials

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.”
Read the official statement from the company here.
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

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)

Clinical Trials: In Need of Patients!

I am getting emails and requests from both pharmaceutical companies and now HTCs to help recruit patients for clinical trials. It’s tough: we have great products out there. What’s the incentive to try something experimental?

Some patients do it for free factor. Others do it to try to help advance science.

There has probably not been such a clogged pipeline for new products coming to market (we hope) in maybe 20 years. Patents have expired, which protected products from competition, and now manufacturers are all competing to get new products into the biggest hemophilia market on earth: the United States of America.

Here’s one I just read about:

CSL Behring has announced that the first patient has been enrolled in the pivotal pediatric phase III study to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) in previously treated children (up to age 11 years). The study site for this first enrollment is the Czech Republic.
CSL Behring, in collaboration with its parent company, CSL Limited, is developing rIX-FP through the PROLONG-9FP clinical trial program for the prophylaxis and treatment of bleeding episodes, including control and prevention of bleeding in surgical settings
in patients with factor IX deficiency.
Results of a Phase I study evaluating recombinant fusion protein linking coagulation Factor IX with albumin (rIX-FP) in patients with severe hemophilia B were publicly presented earlier this year and published in BLOOD 2012 showing that rIX-FP achieved a 91.57 hours terminal half- life, incremental recovery of 1.376 (IU/dL) / (IU/kg), and clearance of 0.75 mL/h/kg. This was an extension in half-life of 5.3 times that of the current recombinant FIX therapy.
We all know that an extended half-life could potentially reduce the number of injections needed in
patients receiving prophylaxis from two or three injections per week to once weekly or significantly less frequently. Several companies and HTCs involved in research, are now in need patient to volunteer to participate.
If you’re interested, go to, where you can read about clinical trials for hemophilia.
Great Book I Just Read
Why is It Always About You? by Sandy Hotchkiss
You know those people who hog the whole conversation, grab the limelight [often from you], make you their personal audience, and don’t listen or even seem to care about what’s happening with you? They could have Narcissistic Personality Disorder. More than just being talkers, they may latch on to powerful or attractive people to make themselves look or feel better; slowly chip away at another’s self-esteem to build themselves up; and go blank even when the person in front of them is in severe emotional pain. They lack empathy. It’s all about them, their agenda, their needs and their ego. We call them emotional vampires because they can leave you depleted and drained. And yet, they are smart and charming typically, and can draw you in, and back again. Run for the hills when you discover someone has this insidious personality, or get professional help to support you through a relationship with them. This excellent book will help you recognize a narcissist, and teach you how to protect your own self against their manipulations. Three/five stars.
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