April 17 is the birth day of Frank Schnabel, a California businessman who, over 60 years ago, founded the World Federation of Hemophilia (WFH). All across the globe on April 17, the hemophilia community celebrates unity, that we are one family united by a protein deficiency that causes prolonged bleeding and suffering. It’s called “World Hemophilia Day.” The theme of this year’s day is “Equitable Access for All.”
We are divided only in access to treatment. Up to 75% of the hemophilia global community has no access to factor. We are trying to close the “gap,” as the WFH puts it, by donating factor medicine to those in need.
Help continue to unite the world hemophilia community. Donate unused or unwanted in-date factor to Project SHARE, and the rest to me! We will find a good home for it overseas. Sponsor a child with hemophilia in poverty through Save One Life. Give back to those in need, and to honor those who have gone before us.
World Hemophilia Day, April 17, is when our community typically wears red, to signify blood, and our unity by blood. I decided years ago to wear white. Why white?
The widely used hemophilia logo features a red and white person standing together in unity. The red represents those with access to factor; the white, those without. And that’s why I wear white. To represent the people I serve most, in developing countries where little to no factor exists, or where donations of factor are not a consistent or sure thing. The logo used to feature a white feature leaning on a red figure, but this was eventually deemed disempowering. I agree!
Around the world, the hemophilia community is encouraged to speak out about hemophilia, to educate the public and to celebrate achievements on this day. Here’s an impressive video of youth in the Philippines; please watch! I visited the Philippines in 2008, when very little was being done to make substantial change. It is now happening, with even a Filipino senator getting involved with World Hemophilia Day! #PassHemophiliaBillNow
And this Facebook posting was written by a once little boy I met in Pakistan, who is grown now, and has suffered but overcome so much. I’ve been to his home; his family is poor. But he has a future and attitude is everything. Read this amazing post:
Accept Yourself
Zubair Ghafoor
This day is not to highlight your deficiencies, not to remind you about what you are deprived of. The day is to Celebrate, Celebrate we were, we are and we will together like a family. Celebrate that someone is always there to bring you all the way back towards normal life. The day is to give gratitude, To all those who put their efforts from minor to major to make our lives less-miserable, either they themselves are haemophiliac or not. To all those parents that were and are facing from social stigmas and taboos but they are still with us with more power than before. Thank to yourself that you still believe in you, becoming stronger and having consistency each day.
Last but not the least, we are all come up with our long struggle stories full of difficulties but what matter is that we are still standing all the way with positivity that God who has created us, has planned something more better than this. Imperfections makes you beautiful, unique from others. So, to All Out There, You Are Not Alone! If the glass is half empty, look at that it is half full, too. This is how life goes on… ACCEPT YOURSELF!
I was in Australia May 8-15, attending the World Federation of Hemophilia Congress. I had the pleasure of meeting a young man named Aung, from the Asian country Myanmar, formerly known as Burma. Aung had written to me months ago requesting sponsorship to the event, plane tickets that cost about $2,000. I am asked frequently for assistance and need to bank my money on people and programs I think will give a return–not to me personally, but to the cause of hemophilia. Myanmar is not a member organization of the WFH and therefore not eligible for funding to the Congresses. Kind of a Catch 22–the countries that need to be there can’t be there unless they have a hemophilia organization. These countries can’t get help forming a society, because they don’t have a society!
But one of my specialties is trying to get organizations formed in countries where none exist in the hopes that one day they will be accredited by WFH. It’s a great partnership which sometimes works! In this case, something about Aung struck me. A young man in a rather isolated country, who hasn’t walked in 7 years, in a wheelchair, willing to for the first time fly to another country, all for hemophilia and his people. That’s courage. He impressed me so much we helped fund him; please read his thoughtful and beautiful essay below and prepare to be impressed. And to realize how lucky we are in developed countries. We live in two worlds—the haves and have-nots, and the have-nots are only asking for the know-how to learn to stand on their own—literally and figuratively.
Aung with Fred Wensing of Australia
My mother’s worries perhaps foresawthe difficulties ahead. It’s not an easy task for me to deal with the airline
staff who told I am not allowed to travel on the flight because I have no
companion to look after me – because I am disabled. However I had a hard time
with my anger management in my teenage years especially on discriminations
against haemophilia and disability, I showed great demeanor in engaging the
conversation with the staff of the airline. They finally said they would make a
call the captain if he allowed me to be on the plane. It was a huge
disappointment I have ever had on the last minutes before the flight on the
airport. But this time I managed as my best to stay cool and wait for the
response, which was positive.
Just as I have come out of the
airport main building, I found Fred Wensing and his wife, Maria. It’d be funny to say
but I had never seen his photo before and had no idea how to find him. Luckily,
he found me instead. The wheelchair at least made it easy for him to spot me in
the crowd.
He and his wife took me to the hotel where I was staying during the Congress days. This is my first trip outside of my country and I have to say everything I perceived in Melbourne is total opposite of what my country has in a positive way. There’s no disabled-friendly sidewalk in Yangon, let alone the other parts of the country. There’s proper treatment for people with haemophilia except blood products which, according to my experience of catching malaria, are really risky. The main difference between Australia and Myanmar is economic conditions, no doubt. But the attitudes of the people here, especially the attitudes on the disability are what I would call really civilized. Whenever I go in Melbourne, I was never welcomed by the quizzical look saying “Why this guy in wheelchair is here?” Attitudes are not about material power or economic prosperity of a country. It’s about
education and nature, I believe. Even if so, there will be a lot of decades
before I will be seen as a person, not as a person in wheelchair.
The exciting Congress days were ahead. I was not able to sleep until 3 am on my first day of staying at Travelodge. My head was flooded by the seemingly unstoppable thoughts of what I would do and who I would meet in the Congress. It was 1:30 pm when I woke up. I
ran, of course in the motorized wheelchair, to a food shop nearby and had my brunch. The shop sold Indian food and I found for the first time that Myanmar is a cousin of India when it comes to cooking dishes. Their curries greeted my taste buds as old buddies do to each other.
It was the first day of the Congress and I planned to go to the Congress only for the opening ceremony. Fred appeared at the lobby at 5 pm. With Aboriginal music and dance, the Congress was wonderful. I met some people who have haemophilia including an elderly person, who has pseudo tumor from haemophilia, a teenager, whose legs becomes paralyzed after being injected painkillers through his spine, a severe haemophilac from Tasamania who has inhibitors and joints with bleeds. I met with some other people but the image of these three people left on my mind.
Back in my country, I always had this feeling of being alone before. I found no one who is put into wheelchair by haemophilia and related causes.
Second Day at the Congress
Second day was probably the biggest day of my life and the opening of my adventures. I went to the Congress alone in the middle of the walking crowd who were going to all the directions. I can enjoy the view of the Yarra River along the way to Melbourne Convention Center while enjoying the freedom given by the wheelchair-friendly environment. I went to the session, Management of Hemophilia with Little or No Factor. I had a conversation with one of the speakers, Dr. Daniel Hart from the U.K, on my health problems with blood products. According to him, some form of antibody might have developed in my body due to the prolonged contact with FFP or cryoprecipitate whenever I have bleeding. These antibodies make the treatment with blood products less effective and my immune system is prone to fever and cold when I have blood products as a result. It makes sense of the problems I am facing
when I inject blood products.
Appointment with a haemophilia specialist
I had an appointment with the doctor after the session. Although I am seeing haematologists often in my country, they don’t seem to be able to give me a solution to cure the bleeding episodes of my knees, which turned me into a wheelchair user when I was 14. My intention to see the doctor at the treatment room of the Congress is to consult with him about my knees. The doctor told me it would take approximately $100,000 to have knee replacement operation to be able to walk. It might be a ray of hope although I do not have any financial strength for such expensive
operation.
The doctor also recommended me to see a physiotherapist in the treatment room. I have never thought physiotherapy played a role in giving treatment to people with haemophilia before. The physiotherapist advised me to have some basic exercises in the water. The very important thing, she said, is to do the exercise slowly and to the limit my joints will not bleed. It will be a little bit difficult for me since I have to practice on my own. But I will have to try some of easy exercise. My muscles have suffered atrophy over the period of 7 years I haven’t been able to walk. Hopefully, regular exercise and diet will make the muscles and joints of my legs fit for some movement.
Wheelchair Talk
I saw the man from Tasmania again
and we headed to a restaurant for our lunch together. He is 33 years now and he is in wheelchair because of his damaged joints. Inhibitors in his body made it difficult for him to undergo a joint replacement therapy. On the way to the restaurant and at the restaurant, we were engaged in the conversation ranging from relationship and romance to our favorite music. He said he has been going out with a female friend recently but he doesn’t want it to evolve into a relationship. “I don’t want her to babysit me, you know.” I could
give him nothing but an emphatic nod. I know what it feels like to be in wheelchair and to have romance at the same time. Anyone who has been in
wheelchair for seven years will know it too.
We continued to talk about our lives and at one point he asked me if my government gives me a disability pension. It is a new word to me because our country does not have any social welfare service offering to its citizens. Luckily, I have earned money for my personal expenses from freelance translation and writing this year. I will have to thank to my parents for keeping me at the university until I graduated. I departed
him for the next sessions of the Congress after I finished my meal.
I would not forget the moments of struggles with the manual wheelchair on the way back to the Melbourne Convention Center which
was a couple of blocks away from the bar. I was going back to the Center alone and the tense feeling is running along my arms while I was pushing the wheelchair myself. I blamed myself for not noticing the steep ground towards the Convention Center. On the way up, I felt as if my arms were going to explode from the cries of muscles. But I felt the need to prove myself that I am independent and I am capable of going to where I want. Of the things that I have built, self-respect is a real ingredient of my life. I possibly cannot survive all the physical pain from haemophilia and the chronic depression without the sense of self-worth to my own existence and capability shaped by this existence.
But it was not all my effort that took me to the destination back. I was stuck at some places along the way and I would not get
back to my destination successfully without the support of the nice people among the pedestrians.
Experience Sharing
There was one interesting session waiting for me
at the Congress. There were many speakers sharing their experiences such as a
doctor, a community leader and a person with haemophilia under the main topic,
In Our Backyards: Eliminating the Gap in Care Between Developed and Developing
Worlds. As someone who comes from a country with 2 percent of the budget into
healthcare sector, I have concerns for the people with haemophilia in Myanmar.
We have to pay for all the expenses of the treatment and it is extremely
difficult even for the middle-class people like my parents. There will be much
more people in rural area who are not diagnosed with haemophilia and who passed
away because of haemophilia. Personally, I heard the sad stories of the people
with haemophilia who passed away for the inadequate treatment. After I managed
to land the mic calmly in my hands, I raised the question to the speaker on the
difficulty we faced in forming the organization from the start with no data for
haemophilia and related bleeding disorders as well as the hospital with expensive blood products and no factor products. I caught curious eyes from the audiences while I was talking about Myanmar on the mic. In the rest of that day, people came and asked me about the conditions in Myanmar. I cannot bring better treatment, better living conditions and better lives to the people with haemophilia in Myanmar myself alone. But I can spread all the information to the global community and in this way, I might be able to find somebody who is willing to work together with us to find a solution for approximately thousands of people with haemophilia in Myanmar.
Learning things with open eyes, Meeting
people with open heart
I love learning, one of the things that give you a reason to live in the world. The very first word of English I learned obviously is “Haemophilia”. When I had access to the Internet four years ago, I typed the same word in Google search box. I still want to learn more about the bleeding disorder with which I will be living until my last breath. Some of the sessions of the third day are interesting to me although I may not be able to apply the knowledge from them to me or to my friends in Yangon since they target mostly to the people in the developed world. But I acquired medical knowledge such as what happened when blood is in joints, and how to manage haemophilia with no factor products. There is hopeful
news such as the half life of the new concentrate products become longer, which means the treatment will be cheaper with less amount of concentrate used. Maybe prophylaxis treatment will be available to us in the coming decades.
Aung with Laurie Kelley
I had a fantastic meeting with the board members of the Haemophilia Foundation ACT on the third day of the Congress. Although we live in different lands and cultures, we share a common identity – haemophilia. It was never difficult to talk with the people throughout the Congress. Board members of HFACT are interested in the conditions of my country and me. I received great advices from them in running a haemophilia organization. Their organization was formed 30 years ago and they have been making progress with the care and treatment of haemophilia during
these years. But luckily, as Fred put it, we will not have to wait for 30 years
because science and technology are waiting for us.
I had a meeting with the lady from the US, Laurie Kelley, who sponsored my air tickets. Project SHARE, organized by Laurie and her team, has a positive impact on the haemophilia communities in developing countries. I had talked with her on the possibility of her visit to Myanmar. She said she might visit Yangon during her trip to South East Asia in September. I hope to be able to work with Project SHARE and Save One Life
organized by Laurie for the sake of people with haemophilia in Myanmar in the future.
Aung with Alain Weill, president of WFH
I also had a conversation with the president of WFH, Alain Weill, at the social event, Taste of Australia. He is a good listener. He promised me that he would make sure the people in Myanmar can have the treatment of factor products donated by WFH after the membership application of Myanmar Haemophilia Patient Association is accepted by WFH. My country, Myanmar, has been out of the map of the global haemophilia community
until now. I am probably the first person with haemophilia from Myanmar that has been to a WFH congress. It is very important for all the people with haemophilia in my country to have a representative in the Congress.
I also found the people from the neighboring countries of Myanmar during the Congress days from Malaysia, Indonesia and Cambodia. We also showed interest in forming a haemophilia society for ASEAN. We would be able to lobby the ASEAN governments to improve
the care and treatment of haemophilia as one voice. I will keep in touch and work with these people to form an ASEAN Haemophilia Association in a few years.
Goodbye, Melbourne
The Farewell Dinner also gave me a perfect condition to make friends with the leaders from haemophilia communities throughout the world. I talked with a president of the Vietnam Haemophilia Association –another contact from ASEAN, young people from Australia and New
Zealand, and the doctors and nurses specializing in haemophilia.
Before I came back, I took a tour around the city, Melbourne, in wheelchair. The streets were teeming with people of different sorts. The shops were full of souvenirs. There were street
musicians entertaining the pedestrians. Even the city was dancing along the tunes of the street musicians.
Having haemophilia and living in an isolated country, I always have the feeling of ‘only me’. This is the first time in my life I have seen so many people who have haemophilia at one single
place. Their happiness and motivation diffused to me. Along with other gifts
from Fred, Maria and their son, Damian, Melbourne gave me another gift – courage.
A courage to live with haemophilia and be a community leader for the people with haemophilia in Myanmar is probably the most important gift I have received from the Congress in Melbourne.
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.
Laurie with Nancy S., who has a child with VWD
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
need.
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.
Meeting FB friends: With Liz Purvis and Tater!
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
American soil.
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
treatable disorder.
With Gary Cross (L) and Dana Kuhn, of PSI
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.
Laurie Kelley with George McCoy, from North Carolina
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
matter what.
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.
Richard Minja (Tanzania), Neil Herson (President, ASD Healthcare), and Simba (Zimbabwe)
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
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