April 2014

Land of One Thousand Hills… and Challenges Part I

Laurie Kelley April 28, 2014

A dream come true for me— Rwanda. This trip happened to coincide with the
twentieth anniversary of the start of the genocide, April 1994. I recall
vividly watching my TV each day, holding my newborn, Mary, and helplessly
comparing her blessed life with the sufferings of the refugees in Goma, and
those trapped in their villages and in the capital, Kigali. The genocide went
on for three months while the world mostly watched, indifferent, unbelieving,
and immobile. It was then and there that I resolved to do something to help those with hemophilia in other countries. Later, Save One Life was born.

Things have changed in Rwanda, which is one of the prettiest and cleanest of all the developing countries I have seen. The country has been reborn, and money is poured into healthcare. But one of the rare disorders that gets no funding or attention is hemophilia. That’s about to change.

First some facts:

Rwanda is one of the smallest African countries, located in eastern
central Africa, about the size of my home state, Massachusetts. Nearly half of
its 11 million people live in Kigali, the capital. Colonized by first the
Germans, then the Belgians after World War I, it is an independent state now
where English and Kinyarwanda are spoken. About 95% of the population is
declared Christian.

The current GDP (“income”) of Rwanda is about $15.7 billion, which ranks it at #141 in the
world. Average income is about $ 600 a year. And about 45% of the population
lives below the poverty line.

Farming impacts 80% of the population. Most crops are for internal consumption, with
only tea and coffee being exported. Thanks to the wild mountain gorilla
population, tourism is the number one industry, with farming a close second.

Life expectancy at birth is 64 years, ranking Rwanda about196th in the world.

My first impressions were all positive of this enchanting African land. I looked
at the stunningly beautiful pastoral vista: rolling hills, tiered like a cake
with green frosting, all farms and crops. Patched-worked in were squares of
other crops: banana trees, sugar cane, pineapple, sorghum. And everywhere
people walk, balancing great loads on their heads. Even the children: their
faces disappear under great loads of bamboo, until they resemble huge bails of
greenery with legs. Or small children struggle to haul water jugs back to their
homes. Infants are wrapped and tied onto their mother’s backs, asleep. Women
work hard here. Men push bicycles up hill, with a towering 50-lb sack of
potatoes or yams. The children wave at me and say “Allo!” A huge field in the
foothills is dotted with colorfully clothed Rwandans chopping and turning the
earth with hoes; backbreaking work.

For this blog I’ll write about our meetings with the hemophilia team. In Part 2 I
will cover the visits to the Genocide Museum, churches and then the Mountain
Gorillas. Because to know Rwanda, you must know more about the Genocide, and its natural wonders which attract people from around the world.

On Sunday night, April 20, Maureen Miruka and I were invited to dinner with Dr. Fabien Ntaganda and his family at his home. Dr. Fabien
is a young hematologist and the only hematologist in a country of 11 million.
He had just recently returned from training in South Africa. His daughters
Alegra and Farley were charming and we enjoyed conversing with them.

Maureen is president and founder of the Jose Memorial Haemophilia Society-Kenya. She has a son, Ethan, with hemophilia, and was compelled to found this
patient-centric organization after her other son Jose died. After knowing her
and working with her for several years, I asked her to accompany me on this
trip to create a bridge between the two countries. To me, it’s silly to have
African nations reaching out to the West constantly, and for us to help
unilaterally, when Africans can share and should share with one another.
Maureen would have much wisdom and experience to share with the patient group the next day.

On Monday April 21, we met at the Rwandan Military Hospital, where Dr. Fabien
works, to meet with many doctors of different disciplines. I gave a talk about
the need for leadership in starting a hemophilia foundation and also in
changing history—here, to create a hemophilia program that addresses problems medically and socially.

Maureen gave a presentation of her own story that led her to create the JMHS-K, and how nit is impacting lives there now.

Afterwards we toured the pediatrics ward, meeting two pediatricians who told us about their encounters with hemophilia patients. Treatment? Fresh frozen plasma. Not even cryo. The first use of factor concentrate was December 2012, when a
donation from the World Federation of Hemophilia was received! December 2012!

Maureen Miruka with Dr. Fabien and pediatricians

This fact jars remarkably in a country where The Clinton Foundation
is deeply involved, where health expenditures are 10.8% of GDP, placing Rwanda at 17th in the
world, where HIV prevalence is 3%, one of the lowest rates in Africa.

The government views healthcare as a human right. With over 400 health care facilities, 42
district hospitals, and 45,000 community health workers providing care are the
village level, Rwanda has created a system to bring health care to both its
urban and rural populations. Yet, life expectancy is only 64 years (and lower
for men).

Hemophilia most certainly contributes to this low life expectancy.

And with a
population of 11 million, there should be about 400-500 with hemophilia. Yet
only 27 have been identified. Dr. Fabien showed us the blood lab, where a one
humble machine stood. Rwanda needs a proper diagnostic facility, in order to
test those with factor VII and factor IX, those with inhibitors or von
Willebrand disease.

The pediatric wards were bright and cheery, with beautiful beds (so remarkable not to see chipped paint, stained walls, overcrowded facilities) and clean floors and walkways.

Our driver, Lucian, took Maureen and I back to the hotel after this, where we had
lunch together, and then waited for the parents/patients to show up at 2 pm for
our first meeting of the Rwanda Federation of Hemophilia. This group was formed
in February 2013, but in actuality, nothing has been implemented or become
official. The group is not registered as a proper nonprofit with the
government, which then prevents it from participating with the WFH and
receiving the resources it needs and deserves. Our mission is to get the group
together, sort out why it has not been registered, get it registered, and get
it moving, according to the tenets of my book, Success as a Hemophilia Leader.

Attendees
included: Emmanuel, Alyos, and Sylvestre, all fathers of children with
hemophilia; young people Fred and Prince, brothers, and their mother. Prince, age 15, was on
crutches and needs to have x-rays to determine if surgery is possible to fix
his patella, which was knocked loose during an accident. Dr. Fabien, in spite
of all the work he has to do, had done his part and got this community
together, bless him. We met for over an hour, talking about what needs to get
done to get this new initiative moving. We ordered soft drinks and African tea
for everyone. Maureen’s lecture on what she has accomplished with JMHS was
superb and opened their eyes to possibilities—what they as a group of patients
and parents can accomplish. As an African, she can communicate with them as I
cannot; she has that credibility that I don’t.

Right then we discussed their
constitution (It needs reviewing as it’s been a year); temporary elections were
needed today, now, to get leaders in place (done!); priorities need to be
established (blood diagnostic lab and registering the society). We accomplished
all that. Fred, only age 21, took the Minutes. We were having our first proper
meeting.

It was a good meeting, and maybe even a bit intimating, as we contemplated all
that needs to be done.

On Tuesday, Maureen headed back for Kenya, and I had a day off to read and organize notes from my hotel room. Later that day, Dr. Fabien and I met with the Health Minister, the Honorable Dr. Agnes Binagwaho, who was appointed in May 2011. The Health Ministry is located in a commercial building, and we waited about an hour until she was done with her
other guests. A pediatrician, the Minister of Health understood about hemophilia. I showed her photos on my laptops of some severe cases. And then we cut to the chase.

Incredibly, Rwanda
offers health care for all individuals to access medical services, currently
95% of the population have access to insurance (current annual insurance cost
is approximately $5). Medicine is sold at pharmacies, and patients get reimbursed
about 80% through the government. Could this be done with factor?

No, she said.
It would overwhelm the system and is too expensive. And honestly, I thought,
asking parents and patients to pay just 20% of the costs would wipe them out.
So there is no easy solution for treatment of hemophilia in Rwanda. Yet,
Barbados, a small Caribbean country, purchases factor; Honduras, one of the
poorest countries in the Western Hemisphere, purchases factor. How can we move
other countries in the same direction?

It takes a
structure fortified by the patients, families and physicians (the Rwanda
Federation of Hemophilia; it takes help from the outside world (the WFH and
entities like us and the JMHS-K); and it takes time. And over time, Rwanda will
achieve self-sufficiency.

Our meeting was pleasant and gave me a better picture of the challenges we were up against. But as I watched in later days, the women walking miles carrying heavy burdens on their heads to market, the children who were dwarfed by the huge bundle of sticks they carted about on their backs, I know that this is a country ready to work for its daily bread, its dose of factor. It has overcome much already, and will over come the new challenges too.

Laurie Kelley with the Hon. Dr. Agnes Binagwaho

To quote the country’s president, Paul Kagame, who led the revolt against the government during the civil war/ genocide, from an article he wrote in the Wall Street Journal recently, “As we pay tribute to the victims, both the living and those who have passed on, we also salute the
unbreakable Rwandan spirit, to which we owe the survival and renewal of our
country…” Think of the victims as those with hemophilia, and you can parallel
that we will succeed one day in providing hemophilia care to a nation that has
already suffered enough.

Great Book I Just Read

Shake Hands with the Devil by Roméo Dallaire

Dallaire was
the head of a UN peacekeeping mission during the 1994 civil war/genocide, in
which 800,000 Rwandans were slaughtered in three months. The book is not a
reporter’s eye-witness account of atrocities: it is a moment by moment, blow by
blow, insider leader’s view of the events leading up to the April 6, 1994
assassination of Rwanda’s president, the internal struggle to get the Arusha
Peace Accord finalized, the mobilizing of anti-governmental forces, the power
struggles between the Tutsi and Hutu tribes, the political players, and more. Dallaire
gives an exhaustively detailed account of what went on politically before
during and after the Genocide, both inside the country and globally; how the UN
on the ground reacted and the indifference of the world. It is a scathing
indictment of the bureaucratically hampered United Nations, the hesitant United
States and the self-serving Belgians. While the world watched, hundreds of
thousands of Tutsis and moderate Hutus were macheted and mutilated. How
Dallaire coped with the lack of resources, the suffering of his own troops,
constant gamesmanship of the players involved, death threats, lies from
politicians, and being surrounded by death and suffering of women and children
is nothing but heroic. He is a hero, and did his best. It is an exhausting book
to read in many ways, yet must-reading for anyone involved in charity work, war
time missions, history, the military, and huge international bodies like the
UN. So many lessons to be learned; Dallaire has done the world, history and
future citizens a vital service in providing this book. It should be a military/humanitarian
classic. God bless him. Five/five stars.

Speak Out, Create Change!

Laurie Kelley April 20, 2014

Kenyatta National HospitalIt’s
about time.

As World Hemophilia Day was celebrated in many countries, I chose to spend this year’s WHD in Kenya, a country I have been visiting since 1999. The nonprofit I founded, Save One Life, has three programs here—microgrants, scholarships and sponsorships—each touching directly the lives of many children and young men
with hemophilia.

The day was organized by the eloquent Dr. Kibet Shikuku, a hematologist at the Kenyatta National Hospital in Nairobi, and James Kago, a young man with hemophilia. Dr. Kibet welcomed about 70 family members—parents and children with hemophilia or von Willebrand disease. The day provided an overview of hemophilia for the press members present, the needs of
Kenya, and words of wisdom for moving forward from this day.

“My prayer today,” Dr. Kibet invoked, “is that we walk forward as a group, so we can advance the issues that affect us. We are one body with different endowed parts. We want to be worthy partners for better hemophilia care in Kenya.”

One main goal is to ensure better diagnosis, he added. With a population of 43 million, Kenya should have roughly 3,000- 4,000 with hemophilia. About 400 patients were identified at one point (meaning they came in at one time in their lives for treatment), but the numbers are not reliable. Only about 50 patients are regular visitors to the treatment center.

Other take aways from Dr. Kibet: “We Kenyans we have every right to be provided for by things that affect us with hemophilia. We must take charge of our own destiny. Togetherness will make us strong. Speak with one voice! We must lobby the government to support testing and availability of factor. Time to speak out and speak up about hemophilia: our community, our accomplishments, our needs.

Speak Out, Create Change was the slogan for World Hemophilia Day, the April 17 event that commemorates the birthday of World Federation of Hemophilia founder Frank Schnabel, an American who envisioned our global community working together to improve care.

Kibet thanked the WFH and Project SHARE for their support of donated factor. He also
thanked donors in US, especially those who support Eldoret project, like the
Indiana hemophilia treatment center and Novo Nordisk Haemophilia Foundation.

He also thanked the Jose Memorial Haemophilia Society and showed a photo of a man
who was in bed for four days with a severe bleed. The JMHS provided him with a donation
of factor.

He noted that there is simply not enough factor; once Kenya secures enough
regularly, then it can offer home therapy.

This is a huge point. Kenya is large, and roads can be difficult. Most patients
living in rural villages have no way to get to the treatment center in Nairobi,
the capital, or can afford transportation. I know first-hand as I have
traversed these roads quite a few times. Imagine taking a public bus, crowded,
hot, hours long, with a painful psoas bleed or worse.

The audience really responded to this idea and asked about home therapy… hoping that someday, someday speaking out… will create change.

Kenya has come a long way, and I was very excited to see at this meeting more change is afoot, all for the best, to create the kind of unity and one voice Dr. Kibet mentioned.

The informative meeting was concluded and a delicious lunch served outside on the hospital grounds. I was able to hang out with a few of the boys I’ve known for years and years: Jovan, Peter, Charles (who has a baby now!), Emmanuel, John.

Drugs Provide Cure for Hepatitis C

Laurie Kelley April 14, 2014

Even while we are basking in the hopeful glow of new long-lasting factor product approved a few weeks ago, there is even better news. A drug that can cure hepatitis C. You may know that roughly 90% of our hemophilia community was infected with hepatitis C during the 1980s, before the blood supply was tested, and before blood-clotting products were virally inactivated. It seems like another generation, but many of our guys still are fighting hep C, even though their HIV has been brought under control.This is an amazing breakthrough. Read on…

Drugs provide cure for hepatitis C, trials show Medications are hailed as a ‘triumph,’ but are pricey

BY MELISSA HEALY of the LA Times

Twenty-five years after scientists first identified the hepatitis C virus, doctors are declaring victory over an infection that afflicts more than 3 million Americans and kills more of them than HIV.

In a series of clinical trial results, a new generation of antiviral medications was able to clear the liver-ravaging virus from virtually all patients’ bloodstreams in as little as eight weeks. Even in patients with the most stubborn infections, the new drugs were capable of suppressing the virus completely at rates well over 90%.

The treatments, however, come with a steep price tag. The “sustained virologic responses” reported in the trials typically mean an infection has been permanently cleared. Only further research will establish that these cures will endure, averting an estimated 15,000 deaths a year from liver disease in the U.S. Worldwide, as many as 3% of people have the infection known as HCV.

The new medications are “a triumph of modern medical technology,” said Dr. Jeffrey Tice, a UC San Francisco physician who was not involved in any of the clinical trials. Results were published this week in the New England Journal of Medicine.

In the treatment of chronic hepatitis C, achieving such success in so short a time, with relatively mild side effects and with a single pill daily, is without precedent. Until recently, physicians relied on two wide-spectrum antiviral medications — interferon and ribavirin — that required precise dosing over periods as long as 48 weeks. Fewer than half of HCV patients are even eligible for the treatment, which comes with punishing side effects. And the regimen’s difficulties cause many to abandon it.

In the same way that antibiotics made victory over human bacterial infections possible in the 20th century, the new antiviral medications have “revolutionized” the treatment of hepatitis C, wrote Harvard Medical School liver specialists Drs. Raymond T. Chung and Thomas F. Baumert, who were not involved in the trials. The drugs — some already approved by the Food and Drug Administration, others now under consideration by the agency — offer “genuine prospects for the first comprehensive cure of a chronic viral infection in humans,” they wrote.

Among the nation’s baby boomers, the new medications are likely to avert hundreds of thousands of cases of liver cancer and dramatically reduce the number of liver transplants. Americans between the ages of 50 and 68 account for roughly 75% of HCV infections in the United States. While many contracted the virus through illicit drug use, an untold number of boomers are thought to have been infected by tainted blood transfusions before a test for the virus became available in 1992. Even as health experts welcomed the trial results, they cautioned that widespread use of the new drugs would come at a steep price. A 12-week regimen of sofosbuvir — just one of the medications in a proposed hepatitis C cocktail — costs $84,000, or about $1,000 per tablet. Treatment with simeprevir, another medication in the same class, can exceed $66,000.

At such prices, U.S. patients will have only limited access to the breakthrough drugs for some time to come, Tice said. The cost of treating even half of the Americans chronically infected with HCV would exceed the amount currently spent on all radiological imaging, said Tice, who helped assess the cost and accessibility of the new medications for the California Technology Assessment Forum. Insurers will probably limit their use to patients with advanced fibrosis or cirrhosis of the liver, a group that represents about one-third of those infected in the U.S. Those patients are at the greatest risk of developing liver cancer or liver disease severe enough to require a liver transplant, conditions that are costly and time-consuming to treat.

In a welter of trial results announced at the International Liver Congress 2014 meeting in London, medications with the chemical names ABT-450/ritonavir, ombitasvir, dasabuvir, ledipasvir and sofosbuvir were successful in bringing about sustained clearance of the hepatitis C virus in the wide range of subjects who took them. In one of the trials, a combination of ledipasvir and sofosbuvir cleared the virus in 99% of patients after 12 weeks. When the drugs were given to patients with cirrhosis for whom previous treatments had failed, 24 weeks of therapy resulted in a virtually100% success rate. The trials also demonstrated that among patients infected with the stubborn Type 1 hepatitis C genotype, success rates were above 95%. The new medications assessed in the latest trials were developed by Gilead Sciences Inc. of Foster City, Calif., and AbbVie of Chicago. The two drug companies funded the trials.

melissa.healy@latimes.com Twitter: @LATMelissaHealy
http://www.latimes.com/science/la-sci-hepatitis-c-cure-20140412,0,3083638.story#axzz2yoZmGey8

The [Infusion] Shot Heard Round the World

Laurie Kelley April 7, 2014

Unless you were watching the new streaming releases of Season 6 of Breaking Bad on Netflix (for which I might excuse you) or were abducted by aliens (for which I will not) you must have heard the news….. the first extended half-life factor has been approved for commercial sale. It’s the hemophilia B drug APROLIX. The news was announced at the HFA meeting in Tampa last Saturday, and it truly is the Shot Heard Round the World (an appropriate reference to the American Revolution). This ain’t just a new product, folks; it could shake up the industry. Physicians are watching; patients are watching; Wall Street is watching. What will the “uptake” be?

Uptake is how many patients will switch to using a new product. The hemophilia community tends to be a “wait and see” creature, perhaps due to our devastating experiences of the 1980s. When Advate was launched in 2002 (yes, drugs are launched, like ships), investors predicted a rosy 20% uptake, but initially it was more like 12%. As the risk-takers who switched early reported good results, more and more patients switched. But Aprolix is truly a different kind of drug. Not just a new generation, it is a new type of hemophilia drug: an extended half-life, meaning fewer infusions for the same results.

And Hemophilia B has not had a buffet of drugs to try like hemophilia A. There was only one recombinant (you all know what that means, right?) FIX for years, until October 2013, when Rixubis was approved. With extended half-life factor, prepare for some major shifts in our landscape. Much may depend on the price, which no one is talking about. If it’s too high, will insurance cover it?

And what about individual results? Will each patient respond well? Advate had major hiccups when there appeared to be an increase in inhibitor formation in users. Was this due to higher scrutiny of inhibitors with a new product? The initial scare seemed to be due to increased scrutiny. And these are among the most scrutinized things on the planet.

And lest we get too carried away, don’t forget there are other products in the pipeline by other companies, some extended half-life (the products, not the companies, though we hope they are long lasting too). Basically, the news about Aprolix marks the beginning of a new era in hemophilia. Maybe a Revolution, maybe the Renaissance. A Hemophilia Renaissance.

Below, an article in its entirety about the news….

U.S. FDA approves Biogen’s hemophilia B drug
Alprolix

Biogen Idec Inc has won U.S.
approval for its long-acting hemophilia B treatment Alprolix, the U.S. Food and
Drug Administration said on Friday.

Hemophilia B is a rare, inherited disorder in
which a person’s blood does not clot properly, which can lead to prolonged
bleeding and bruising. It affects about 4,000 people in the United States and
25,000 worldwide.

Alprolix is a bioengineered version of the blood
coagulation factor IX, a protein needed for normal blood clotting. Biogen is
developing the drug in partnership with Swedish Orphan Biovitrum AB. It
received approval in Canada a week ago.

Hemophilia drugs in general must be infused two
to three times a week to prevent bleeding episodes. Biogen’s drug needs to be
administered once a week or once every 10 to 14 days.

The company is also developing a long-lasting
drug, Eloctate, for Hemophilia A, a more common form of the disease, that
affects about 20,000 people in the United States. Patients with hemophilia A
lack or have reduced levels of coagulation factor VIII.

Current treatments for hemophilia B generate
about $1 billion a year, according to Biogen. The market for hemophilia A
therapies is worth about $6 billion.

Eloctate would compete with Baxter International
Inc’s drug Advate. Baxter is seeking to develop its own long-acting version but
Biogen is expected to be first to the market.

Alprolix is expected to generate sales of $286
million by 2019, according to Thomson Reuters data. Eloctate is expected to
generate sales of $1.1 billion.

(Reporting by Toni Clarke in Washington; Editing
by David Gregorio) Fri Mar 28,
2014 5:11pm EDT

For more info:

https://www.biogenidec.com/therapies_alprolix.aspx?ID=19723

http://www.alprolix.com/

United by Blood: HFA Meeting in Tampa

Laurie Kelley April 1, 2014

Laurie Kelley, Jeff Johnson, Barry Haarde

Grey skies and a chilly breeze couldn’t dampen the spirits of those attending the 20th anniversary meeting of Hemophilia Federation of America in Tampa, Florida this past week. A record number attended, estimated between 600-900, from all parts of the US. Zoraida and I arrived on Wednesday, a day early, to meet with some of our colleagues, for this is a prime meeting for business networking, fundraising and brainstorming.

Central to the meeting, like its heart beat, was the History Room. This stunning display of our past 70 years in all its pain and triumph, was a somber reminder of how far we have come in the war against bleeding, and the sacrifices of our fallen. Following a poster timeline, in which each era was clearly defined, led to the room, where dozens of resources were provided (hey, including my own stuff), a community poster board with hundreds of photos, and the Ryan White section from the AIDS Quilt. Kudos to Rich Pezzillo, Ray Datolli and Barry Haarde (and their helpers) who masterfully compiled with painstaking detail this amazing tribute to our community, to our fallen.

Selfie-time!

Symposia included a variety of topics. One on advocacy and the ACA, called Making Advocacy Personal, featured Jim Romano of PSI and Wendy Owen who answered a slew of questions on advocacy and health care policy. Another on just inhibitors, a new feature at HFA–my only concern was that is was closed only to families with inhibitors. Huh? Everyone could benefit from attending, as there will be families this year who will develop inhibitors, and there are those of us who help educate them. (Anyone want to explain that policy to me?***)

A Baxter-sponsored dinner Friday night provided talks from two young men with hemophilia who shared their stories of growing up feeling different, and who now are talking life by the horns–very inspirational!

Ray Datolli, Emily Haarde, Rich Pezzillo, Laurie Kelley

After that dinner, at 9 pm, I attended the Committee of Ten Thousand (COTT) meeting, led by the legendary Corey Dubin. We discussed the Living Memorial, a gorgeous “Vietnam Wall” style, stone memorial, to be placed in San Francisco, with the names of all who died of hemophilia/HIV inscribed on it. The artist’s rendition is spectacular, and prompted Jane Cavanaugh Smith, executive director of the Coluburn-Keenan Foundation to donate $10,000! And to pledge matching donations up to $50,000! Nathan and Sonji Wilkes, parents of Thomas, who has hemophilia and inhibitors, immediately pledged $1,000. Corey was touched and grateful, and we all look forward to learning more about the Memorial’s fundraising and financing so we can begin to help fund this, and at long last, close the wound in our community while the survivors are still with us.

The final night was a wonderful buffet dinner sponsored by Biogen Idec, complete with games for the kids and dancing. And what timing. That very day it was announced on the newswires that Alprolix, Biogen Idec’s long-lasting recombinant factor IX, was approved for sale by the US FDA!

So in addition to congratulating HFA on 20 years of service, Biogen Idec also announced to the crowd the news about this game-changing drug. And don’t forget there are many other drugs in the pipeline coming on line soon from many of the manufacturers….

It’s going to be an interesting year, folks.

Thanks to Kimberly Haugstad and her team for a fabulous meeting!*** Policy explained, April 1 from HFA: “We closed it because inhibitor families asked for it to be closed.”