No man is an island,
Entire of itself,
Every man is a piece of the continent, A part of the main. —English poet John Donne
I stepped off the plane in Kingston, Jamaica on Thursday evening and the sultry, warm air enveloped me like a thick blanket. This tropical island has great music, savory food, friendly people, world-class resorts, a raging drug problem… and hemophilia. I was here this past weekend to speak at the first-ever conference for the English-speaking countries of the Caribbean. The Jamaica Haemophilia Committee hosted this landmark World Federation of Hemophilia conference.
I’ve been to Jamaica twice before to help the patients organize, and numerous times over the past 20 years to a few other English-speaking countries here, including Barbados, Bahamas, and St. Vincent and the Grenadines. All were represented here, at long last.
The emerging theme of the conference became isolation, and how
to break it. The poetry of John Donne expresses this so beautifully: so many people with hemophilia feel isolated, as if they are on an island, cut off from the world. Indeed, these people really are on islands! Our challenge is to unite patients and families with one another in their own countries, and then link and connect country with other countries, to learn from one another and to support each other. To make a hemophilia continent, and to reduce isolation.
Topics of the two day conference included: clinical management of hemophilia, outreach to community members, genetics, hemophilia in the Caribbean, WFH programs, comprehensive care, the role of nursing, physiotherapy, and patient involvement.
Who attended? Erica Worrell from Barbados, mother of a child with hemophilia, who just started a new society there in April and already held a successful walk to raise awareness; Brian and Ritchie Bardalez of Belize, young men who I have known since they were children, as I visited
Belize twice back in the early 2000s (they are now taking a leadership role);
Issa from Trinidad and Tobago, a patient and fiery orator, all my colleagues
and friends from Jamaica (Denton, Alex, Kirt, Berverly, Kerry-Ann, Milton,
Tamaicka, Sharon, Gricell, and the medical staff of UWI), Laurence Bakhsh, a brave young man from Guyana who we have helped for years with factor donations—first time meeting him!
The challenges are vast: Erica shared how there is no national registry of patients in Barbados, and how difficult it is to get factor. While the country buys some—a miracle in itself—you must get a prescription (wait time long), then go to the pharmacy (wait time even longer). You must pay for all ancillaries, right down to butterfly needles, which often are not even available! And tourniquets? Forget it. (We promised to ship her some ASAP.) Ritchie pointed out that Belize has closer ties with the Caribbean than with Central America, where he is located. There are only 16 known patients in Belize, and the terrain is the opposite of flat Barbados; it is mountainous with poor roads. A plane—“puddle jumper”—is the safest way to travel and distribute factor. 56% of the patients do home therapy, which spares them from expensive travel to clinics. There’s no hematologist—anywhere.
There’s also no hematologist in St. Lucia, reported Dr. Jackie Bird, a 58-year-old dynamo who seems to have single-handedly taken on care for all people with hemophilia on this volcanic island. Poverty is 28%, and there are only 6 known patients—all with factor IX deficiency! Factor is not available and never has been.
That made me sit up. Could we be the first to ship factor IX concentrate to St. Lucia? Jackie’s knees buckled, as she made a prayer sign with her hands, smiled and shouted, “Yes, thank you!” The room exploded in applause. This is the magic of these meetings: putting people together, those with and without, sharing honestly, and finding solutions. Yeah for us!
Laurence, a 33-year-old from Guyana, just founded a hemophilia society in 2015 and is struggling. After all, he has limited mobility, limited funding (he cannot work), and there are only 5 known people with hemophilia, including his brother and cousin. Guyana’s population of 755,000 means there are about 75 people with hemophilia. He has his work cut out for him. But with his natural charm and humility, he quickly became a favorite of all, and with his new network of colleagues, is bound to make improvements soon. “Strive to stay alive” is the organization’s tagline.
The Bahamas was represented by Florence Roker, mother of 21-year-old Chavez, who I met years ago. She has now stepped forward
to grab the reins of the floundering national organization. She broke into tears describing how Chavez has suffered: “You physically feel their pain,” she sobbed. Now, she said, regaining her composure, there are other children who suffer and who need our help. You have to find the silver lining behind the clouds. “This is a passion for me,” she concluded. “I’m motivated to get the organization to where it should be.”
An empowering and passionate speech was given by Issa, chair of the Society for Inherited and Severe Blood Disorders Trinidad and Tobago, an association for those with hemophilia, sickle cell and thalassemia. This is a brilliant strategy, to link up the three blood disorders to gain strength and have a stronger voice. And oh boy, does Issa have a strong voice! Motivating us and making us laugh, he stirred the audience emotionally with obvious leadership skills. T&T has been a WFH national member organization for 25 years, making it the oldest organization for hemophilia in the Caribbean. Issa declared that in the 1980s, West Indies cricket dominated the world. “Dominated!” he shouted. “Dominated! It happened when we united as one! We conquered the world!’ The audience laughed and applauded. Comparing this to diseases and disorders, T&T united as well. But unlike the West Indies cricket team, which had its glory days in the 1980s, “we are still a force to be reckoned with!” Issa declared.
But the most quoted person these two days was Jamaican hemophilia patient Tevon Brown, who stressed how isolated he felt growing up, as if he were the only person with hemophilia. He suffered terribly with pain, which felt like an “electric lead to my heart.” Strong role models and good teachers helped him. And he stressed now that we must unite and bring those who are isolated together, including nations. “We must have stick-to-it-iveness!”
But the greatest joy for me was seeing 22-year-old Kishroy Forde, who I’ve known since he was six, attend. Kishroy lives on Mayreau Island, remote in the Caribbean Sea, part of St. Vincent and the Grenadines, accessible only by boat, with no health care on the island of 300 people. It’s surreal to visit, which I’ve done twice. We provide Kishroy with factor when he needs it, and he knows how to self-infuse. We’ve also helped him attend a tech school, and with the help of his sponsor, purchase a new fishing boat for his father. They live in a rustic community; Mayreau has no towns and you can circumambulate the island in less than an hour. And yet, it is a tropical haven.
This was the first time Kishroy has met others with hemophilia and taken a plane. It was amazing to see him conversing with other guys his age who have hemophilia. He even got up to share his story!
I must thank the World Federation of Hemophilia for organizing this conference, funding it, and inviting me, which allowed me to invite so many of the people I’ve met over the past 20 years. Kudos to Luisa Durante, regional manager of the WFH for Latin America, who is as hard working as she is fun as she is beloved by the people she serves. She and her team (Salome and Felipe) provided a magical two days. The WFH does great work, and we are
proud to support them and volunteer when possible. It takes stick-to-it-iveness to make long term changes in hemophilia care, and this is how it happens. Unity, hard work, passion.
I attended the annual National Hemophilia Foundation meeting from November 7-11,
as I have done since 1992 faithfully. As usual it was filled with interesting
sessions on treatment of hemophilia, attractive displays of company booths and
wonderful reunions with many friends and colleagues. But something new: in his
opening speech, NHF CEO Val Bias, a man with hemophilia and one of the foremost
advocates in helping to get the Ricky Ray Law passed, spoke about uniting our
communities globally. This is the first time I believe that NHF has shown a
targeted interest in helping the world’s 75% with hemophilia who have little or
no access to care.
This was exciting to me as I have been working to help the underprivileged with hemophilia since 1996. First with a leadership training program called
L.I.G.H.T., then a factor donation program called Project SHARE, still ongoing,
and my nonprofit Save One Life, which sponsors children overseas. I was
thrilled that NHF would bring its resources and brainpower to help those in
Why not before now? Timing, focus. We have been through many phases: the contamination of the blood supply and subsequent infection of thousands occupied our time and focus for years. Then came product shortages. The inhibitors. Even NHF had its own problems internally and struggled to assess leadership (CEOs came and went) and structure. That seems behind us now. And with Val’s leadership and vision, NHF turned its powerful eye overseas, especially to Africa.
Val Bias and Neil Frick visited Nigeria in September, marking the first time that someone from NHF has been to Africa! I’ve been traveling there since 1999, when I first visited Zimbabwe and Kenya, and am returning on November 30. Finally, finally America is showing its leadership and true giving nature in joining the international community and participating in partnerships that will help
hemophilia nonprofits and its members in developing countries.
Val has bigger plans: the 2016 NHF meeting will be held in Miami, and Miami was
also selected for the 2016 World Federation of Hemophilia biennial meeting.
This means that attendees to either meeting can also extend their visit and
join the other meeting. WFH gets about 4,000 visitors and NHF gets about 2,500.
This would potentially be the biggest meeting on hemophilia in history, and on
I really applaud NHF becoming involved globally. The world needs it and it’s good
for us. I’ve always felt that we have more than enough to share, if we can just
find ways to do it. With NHF’s power, hemophilia care globally can progress at
a faster pace to reach more in need, an estimated 300,000 who suffer from this thoroughly
And looking at all the photos from NHF here, I realize that almost evey person pictured either sponsors a child with hemophilia or VWD in a developing country through Save One Life or has contributed in some way to Save One Life or Project SHARE. From summiting Kilimanjaro to raise funds, to making handicrafts that support a child, to sponsoring one directly, Americans have already been uniting globally—and now can take pride that their national organization will as well.With Reid Coleman of NC
“Are you a madman or a guinea pig?” asked a man from France at the final symposium of the World Federation of Hemophilia Congress in Paris, France last Thursday. He addressed his question before the 500 or more attendees in the huge amphitheater at the Palais de Congres to our own George McCoy, from North Carolina, one of the fascinating speakers on the panel debating and comparing long-acting factor to gene therapy.
Great question, particularly as George had just revealed that he was the very first human ever to be injected with recombinant factor VIII, back in March 1987. I was riveted to George and his reply. The whole audience was. But more on that in a bit!
The session opened with Dr. Paul Giangrande, director of Oxford Haemophilia Centre in England and world-renowned hematologist, purposely supporting continued recombinant therapy, and listing the many pros of our current treatment. Paul reminded us of the Hippocratic oath Primum non nocere: First, do no harm, and cited the long and many years of improved quality of life and quantity of life on these therapies.
(Dr. Giangrande also prefaced his presentation with an outrageously humorous skit on how he gets roped into presenting so many talks at symposia, all seemingly taking contrary positions! He feels it is his duty to provide food for thought on the pros and cons of all treatment; here, he was asked directly by WFH to make a case for recombinant therapy)
Long acting factor, Dr. Giangrande said, which many manufacturers are working on, is based on recombinant technology, a stable and known technology, which is considered safe. It provides breadth of product for factor VIII, IX, and inhibitor patients. WFH president Mark Skinner even said earlier at the Congress that treatment should aim for a trough level of 15%—which recombinants can provide. Giangrande added that we know the manufacturers already so we know in essence from where the product comes and what we are getting.
Giangrande then cited some of the downside of what we know and can expect from current gene therapy, still in clinical trials: more hospitalvisits and blood tests. He asked if patients truly were giving informed consent. Most parents don’t understand what the studies involve. And he questioned the
ethics of using patients in developing countries in these clinical trials—there are medical risks, and the patients could be desperate to have product no
Will gene therapy really lower costs? Currently Dr. Kathy High from Children’s Hospital of Philadelphia is investigating a factor IX gene therapy treatment. If successful, Giangrande reminded us, it will be commercialized, just as all the past research in hemophilia has been.
Next came Dr. Kathy High herself, who reminded the audience right away that the ultimate goal is to cure hemophilia. We aren’t used to thinking of cures for genetic diseases. Some have been radical, such as bone marrow transplants or liver transplants. But we have alternatives now. In a recent published gene therapy article, research in the UK showed that a single infusion led to more than 18 months expression of factor IX at
about 5% levels. This was repeated in two other patients. Unfortunately, eight weeks later one patient required steroids. While it looks like a virus vector may trigger an immune response, Dr. High felt that the risk of a short course of steroids outweighed the risk of bleeds. Other risks noted included the viral vector appearing in semen; and one mouse model showed an increase in carcinoma (but he was an old mouse with lysosomal storage disorder! The things you learn at these symposia).
How long will this gene therapy last in the body? We don’t know. In dogs, over ten years. And it only requires a simple infusion, with no organ transplant.
Dr. High posed the question on everyone’s mind: when will gene therapy happen for hemophilia A patients? High believes it will start in 3-5 years. Some studies are already underway, such as one with AV canine factor VIII, with Dr. David Lillicrap of Canada. High gave a positive and detailed summary of currently gene therapy—very exciting presentation!
So back to George McCoy, who then spoke, eloquently, sincerely and humbly. He described his childhood, and how he was diagnosed at age two. One of four boys, his eldest brother Richard had hemophilia for years and no one knew. Sadly, he died. George’s speech was moving and thoughtful, especially when he cited WFH founder Frank Schnabel, who described patients with hemophilia as “tortured pieces of human geography
confined to a wheelchair.”
Dr. Gil White contacted him about being the first rFVIII patient ever in March 1987, and hence the question: is he a madman or a guinea pig? George simply replied that he would do it all again if he had to—he would volunteer for gene therapy if—or when—the time comes. And he stressed that we need volunteers for gene therapy now.
He closed with the beautiful thought: “What brings us here is the will to live, the
will to prosper, the will to learn. We all do need each other.” And while all
the speakers weighed in on whether they would elect gene therapy over
long-acting factor for their hypothetical grandchildren with hemophilia, were
they to have one, it was Dr. High who made a memorable statement: we need access to all therapies, both gene therapy and long-acting, for individual patients with
individual biological make-ups, for those in developing countries— access to all products and treatments for us all. George was right: we all do need each other, and this past week at WFH reminded us all that 300,000 have little or no access to hemophilia treatment. The burden—to me the joy— is on us to help them all.
CEO of Biogen, George Scangos, Laurie,
Sr. VP Medical Affairs, Biogen, Glenn Pierce
I’m in a soggy Paris (not complaining though!), attending the wonderful 50th anniversary of the World Federation of Hemophilia, taking place during its biannual Congress. The WFH, founded by American Frank Schnabel, is the premiere hemophilia nonprofit in the world, dedicated to improving the lives of those with hemophilia around the world, uniting those with hemophilia globally, and advancing access to care in developing countries. Today was the opening day, and a staggering, record-breaking 5,300 are in attendance from 130 countries! It’s a great opportunity for me to meet with our many country partners, who work with us to get factor to patients in developing countries.
I’ve been attending WFH meetings since 1996, and attend every two years. It’s a great chance to learn more about WFH efforts, to meet up with those we serve, and to make new acquaintances. Right away I bumped into Dr. Tatiana Andreeva, a pediatric hematologist from St. Petersburg whom I’ve known since 1998. She even visited my home once!
The incomparable Jill Smith, RN from Australia—who traveled with me once to Zimbabwe for a week to do an assessment of hemophilia care there (and shared a tent with me on safari!)—and incredible Anne Gillham, RN from South Africa, who has been a vital source of training and support for so many countries in Africa besides her own.
The opening ceremony was excellent. Mark Skinner, past president of NHF and who has been president of WFH for 8 years now, gave an insightful talk about the accomplishments of WFH in helping advance care. But never have I seen the WFH so intensely focused on developing countries. This made my heart sing, for it’s been my mission and desire since 1996, that we do not forget those at the bottom of the wait list, the ones whose government does not provide funding for treatment. A very touching and powerful movie was shown about how WFH has worked in Senegal to improve care, and I was pleasantly surprised to see that producer Marilyn Ness had created it! It was beautiful, so well done. I’ve been to Africa many times and have traveled those same dusty roads, been in the same hospitals with paint-peeled iron beds, no AC, suffering patients. Marilyn captured the feel of hemophilia care in Africa well.
The WFH is now making a serious push to “Close the Gap” with a new campaign, designed to raise $5 million to help create programs to reach the impoverished countries where care is lacking. We still have four more days of the Congress, and already the tone has been set: we must act now to help save the lives of those without treatment. It’s what we’ve been focusing on at Save One life, my nonprofit, and I applaud WFH for putting the spotlight on developing countries as it moves forward, and asking everyone to join in.
After the ceremony, we all moved into the exhibit area and visited the various booths by the pharmaceutical companies. There we met even more colleagues and friends. I was thrilled to meet up with my friends from Africa, especially Megan Adediran, president of the Nigerian Haemophilia Foundation, who just published a new book about her experiences with hemophilia, called A Light at the End of the Tunnel.
The World Federation of Hemophilia Congress was a wonderful time, and very informative. Representatives from all over the world came to Buenos Aires, Argentina, all last week, to share news, present studies and network.
One of the most talked about subjects was not gene therapy, but longer acting factor. As we mentioned in the February issue of PEN (https://www.kelleycom.com/newsletter.html) it looks like extended half-life factor will be next on the horizon for new products. Of all the companies presenting, Biogen Idec perhaps stole the show. The company reported positive results from a Phase I/IIa safety and pharmacokinetic study of its recombinant factor IX protein, called “rFIXFc,” in hemophilia B patients. The primary objective of the study was to assess safety, and the product was well tolerated (albeit in a single-dose) with no signs of inhibitor development or antibodies.
Chief medical officer of hemophilia products at Biogen Idec, is none other than Glenn Pierce, twice president of the National Hemophilia Foundation. As president of NHF a long time ago, Glenn pushed hard for gene therapy and was hopeful that it would be found in ten years, and then dampened that hope to in our lifetime. For now, it seems we may have found the next best thing: long acting factor.
The Congress gave me a chance to meet with a variety of colleagues from around the world, including those from Pakistan, Honduras, Tanzania, Ghana, India, Belize and New Zealand, just to name a few! Nice break for me: instead of traveling to meet them all in their countries over several years, I was able to meet with them all in one week!
Great Book I Just Read The Man Who Tried to Save the World by Scott Anderson
This book gets better each time I read it. The enthralling story of larger-than-life Texan Fred Cuny, the “Master of Disaster,” who disappeared in Chechnya in 1995. Anderson actually travels there during wartime (dubbed one of the scariest places on earth) to learn what happened to Cuny. Cuny was a visionary, who turned the international disaster relief world on its head with his revolutionary–and effective–ways of approaching disaster relief. Largely self-taught, his compassion, passion and “bedrock practicality” led him to devise better and cheaper ways to save lives–not always a welcome thing to the comfy and bloated big international nonprofits. He was drawn to danger and this book reads like a spy thriller and a fascinating biography of a fascinating American. He is a true American hero. Harrison Ford bought the rights to the movie in 2002: come on Ford, where is the movie? Three stars.