Sunday, May 30, 2010
I’ve been to a lot of places recently, and one of the most interesting ones was the GTC Biotherapeutics farm in Charlton, Massachusetts, on Friday, May 21, to see transgenic goats.
I wrote about transgenic animals in the February issue of PEN, when I discussed hemophilia products that are being developed now. Factor produced through transgenic animals may one day become a source of factor concentrate. And if production proceeds as hoped, transgenic animals may represent a high volume and thus low-cost way to produce factor and other proteins.
What are transgenic animals? These are animals that have been genetically modified to express human therapeutic proteins in their milk. In other words, human factor can be collected by milking these animals. The source material is raw milk. And once the proteins are collected and purified, they can be used to treat diseases and genetic or metabolic disorders in humans.
Now, the goats located on the 167 acres that bridge the towns of Charlton and Spencer, Massachusetts, are not being raised to create factor, although factor IX is being produced by transgenic pigs in Virginia, and factor VII is being made by transgenic rabbits in France. The goats we visited express the protein used in ATryn®, a recombinant form of human antithrombin. Atryn® is the first transgenically produced therapeutic protein to receive FDA approval and the first recombinant antithrombin approved in the U.S. So as wild as this all sounds, GTC already has an FDA-approved, transgenically produced therapeutic in the marketplace. And GTC is hoping to initiate a clinical trial for their factor VIIa product later this year.
My husband Kevin and I requested a tour, and we were enthusiastically greeted by Ashley Lawton, Vice President, Business Development. Before we entered the building, we had to dip our shoes into a disinfectant solution, to kill any microbes. Of course, I was wearing sandals!
Safety was stressed at every part of the two-hour tour.
Ashley told us that the goats originated from New Zealand, because this is one of the few countries free of scrapie, a fatal and degenerative disease in sheep and goats. Why use goats and not cows? Goats produce more milk and their milk has a high protein content; they also have a four-month gestation period. And they are relatively easy to breed and maintain.
GTC is a pioneer in the field of transgenic technology, and because it involves making human therapeutic proteins, regulatory agencies want extensive documentation. Everything done to the animals, from having their hoofs cleaned to operations, is documented fully.
Dr. Bill Gavin, General Manager of Operations and chief veterinarian, joined our tour and told us, “Because we were a new technology… we were held to an even higher level of safety.”
We toured the labs where the genes are spliced into the embryos. We traveled to the barns where the goats are penned. Somehow I expected sterile, see-through cubes with goats hooked up to machines. Instead, we saw adorable goats comfy in stalls, much like you’d see on any farm. The “kids” were cuddled together. Young adults were able to mingle and hang together; they even had toys to play with like bouncing balls. Goats love to climb, and these had cubes to scale.
Goats are fascinating. They are curious, sociable and always hungry! They’ll nibble on anything and eat just about anything. I couldn’t go too near them but got close enough to see these are probably the best cared for goats on earth.
I’ve been invited to view the pigs in Virginia that are being researched currently to produce factor IX; after this trip I think I need to go check it out.
Actually seeing the goats and the technology made this seem more than just a pipedream. Perhaps the biggest revelation is that we would not “drink” the treatment, which I actually believed! Once the protein is purified from the milk, it will be freeze-dried and prepared as an injectible, biological product, very much like the factor concentrates available on the market today.
I know there are those in our community who might think this is unfeasible. But for me, this is a ray of hope. Transgenic animals might be particularly attractive as a way to treat hemophilia in developing countries, where there is great need for a low cost product, and plenty of it. Do you know which countries are the top five in population?
In order: China, India, US, Indonesia, Brazil. Four of the five most populated countries on earth are developing countries. So it stands to reason that a huge population of people with hemophilia are underserved and in need. This wonderful tour on a warm spring day left me hopeful that plentiful help for them might someday come from this fascinating new technology from humble farm animals.
Interesting Book I Just Read
The Seven Powers of Questions
By Dorothy Leeds
This book asks questions, actually: why do we talk so much and not listen? How can we better focus and learn? What are effective ways to ask questions? Leeds covers a lot of ground about how we communicate, mistakes we make in trying to form relationships (whether business or personal), and how to be a better listener. Asking questions makes us better listeners, helps us to focus, diffuse defensiveness in others, calms situations. She notes the things that make us poor listeners: having preconceived ideas, jumping to conclusions, being emotionally charged. Questions help break down those barriers, and make us more open to listen, and then to think. This book got off to a very weak start (I think it was opening with a not-so-good quote from the 1960s Star Trek TV show [and I consider myself a fan]) with a plethora of cliché quotations from oft-referenced works (Ask a better question to get a better answer: how many times have I heard that?) and also had a weak ending, by evolving into a “Where is your entire life going and how will you get motivated to be an empowered person and make your dreams come true…” But overall there is material that is vital to know and this book will be welcome to read if you are new to self-help books or any book on communication. Two stars.
Sunday, May 23, 2010
Last Tuesday I dusted off my salwar kameez outfit from Pakistan, hopped on a plane with executive director Martha Hopewell of Save One Life, our child sponsorship nonprofit, and went to New York City for a wonderful fundraiser called "Bollywood Chic," hosted in the art studio of Hunt Slonem.
The studio takes your breath away: cavernous at 15,000 sf, and filled from ceiling to floor with framed art, statues, and even a museum quality collection of butterflies that was stunning. In the center of the studio: bird cages filled with colorful macaws and doves. Hunt's specialty is painting birds; you can see his famed parrots on a new Tiffany set of serving ware. The birds are raucous when not being fawned over; they demand attention by screeching. This added an exotic flavor to the evening!
How did we snag the goodwill of an artist of Hunt's calibre? Through board member Tara Reddi, who is vice president of the Marlborough Gallery in New York, and has a nephew with hemophilia in India.
Guests arrived around 6:30 and stayed till 9, sampling some wonderful Indian-style food, and wine at our bar, and listening to Bollywood soundtrack. Guests could wander freely throughout the studio, which is subdivided into different rooms, each with a theme. The Lincoln room is filled with Hunt's portraits of Lincoln in many colors. Another room is filled top to bottom with framed paintings of rabbits. Another room is filled with Greek or Italian statues. Another room contained a massive round table and filled with candles, looking like an invitation to a seance! Guests were really intrigued by the decor!
We were pleased to have with us Dr. Ranjan Kulkarni from the Nasik Chapter of the Indian Federation of Hemophilia. She is our program partner there, and has enrolled many children with hemophilia in Save One Life. She just happened to be visiting in New Jersey, which worked out perfectly! The fundraiser gave her a chance to meet two other doctors and sponsors: Dr. Lucy and Dr. Shipra, both attired in lovely Indian dresses!
Save One Life now sponsors over 550 children in nine countries, giving them funding that they would normally never receive. Funding is used for transportation to clinic, to buy medicine, and to pay school fees. The nonprofits that administer the program--our program partners--check in regularly with the children, to ensure they are doing well. So more than just a charity, we are guaranteeing these kids get seen, are known and are helped.
Our deepest thanks to Hunt Slonem for generously allowing us the privilege and pleasure to spend an evening at his studio. Thanks to Tara Reddi for organizing this marvelous event, which required a lot of hard work. Thanks to all the guests who showed up despite the dreary weather, many of whom were friends and patrons of Tara's and Hunt, and who supported the fundraiser. And thanks to my friends at NHF, whom I was so happy to see present!
Many thanks to two companies who helped underwrite the event: CSL Behring and Inspriration Biopharmaceuticals. All proceeds of the event will go towards our India program, where the bulk of our beneficiaries are located. I'll be traveling to India in September to check on our program and will be sure to keep everyone posted. Namaste!
For more information or to sponsor a child in need: www.SaveOneLife.net
Also see: www.cslbehring.com, www.inspirationbio.com, www.marlboroughgallery.com
Sunday, May 16, 2010
I'm very happy to report that the Inhibitor Summits are back this summer. Two Summits are scehduled, one in Houston and one here in Boston.
August 5-8, 2010 -- Houston, TX
August 19-22, 2010 -- Boston, MA
Come join other people with hemophilia A or B with inhibitors and their families for a weekend of education designed to improve your overall health and quality of life.
REGISTER NOW! http://www.nhfinhibitorsummits.org/
From the NHF announcement: Having an inhibitor can seem overwhelming at times that even day-to-day life can be a challenge. This can affect your well-being in ways that only other people with this condition can understand. The annual Inhibitor Education Summits connect people with inhibitors, their caregivers and members of their support network with expert healthcare professionals. They also connect you with others who have been where you are--people who can share their experiences or suggest a coping skill.
New this year:
Multiple educational tracks
Exciting Youth Camp activities planned for kids ages 4-12
Coping mechanisms for improving the psychological well-being of patients and their caregivers
To register or for more information, visit the NHF Inhibitor Summits Web site, call 877-560-5833, or e-mail email@example.com. Travel and lodging assistance is available for eligible patients and their caregiver(s).
Atención: los representantes hablan español.
This educational program series is provided by the National Hemophilia Foundation and supported by an educational grant from Novo Nordisk.
Hope to see you there! Register today!
Great Book I Just Read
Tears in the Darkness
Michael and Elizabeth Norman
This book, about the horrific Bataan Death March in the Philippines in World War II surprised me. It's told mostly through the eyes of ordinary soldiers, one in particular, Ben Steele, a 20-something year old, budding artist from Montana. The book chronicles the war-fever (Steele's own mother encourages him to sign up for military service), the surrender of 76,000 Americans and Filipinos to the Japanese in the Bataan peninsula, and the infamous death march that led to the starvation, dehydration, beatings and executions of so many. Japanese military brutality is on display with a candidness that is chilling. The suffering Ben Steele endures, along with his fellow prisoners, is almost not to be believed. Ben eventually becomes a POW, and becasue he is sick, is put in sick bay, where he bides his time be sketching what he sees. His illustrations are in the book, though it's not always clear what the pictures are of. This is a sad tale of Japanese brutality and total American military abandonment of its own soldiers in their hour of need. How anyone survived the march is beyond belief. Eventually of course, Douglas McArthur ("I shall return") does return and the Americans capture the peninsula. Interestingly, the Japanese side is told thorugh the eyes of commander Homma, who acts unaware of the rtagedy that unfolded under his watch. It's almost sad to see what happens to him and his family. A must read for WWII fans. Four stars.
Sunday, May 09, 2010
Here in America it's Mother's Day, and I've been enjoying the good wishes that have been emailed to me from friends in hemophilia around the globe. I received a great gift: my luggage! It went missing for three days following my return from Africa a few days ago. I'm back, it's back and the little parasite I picked up is mostly gone.
(Photo: Mother and cub, Kenya, shot on safari)
My trip to Kenya was wonderful, full of rugged rides into the countryside to visit the mothers and sons, families with hemophilia. In Kenya, as in most developing countries I have studied, it's often the mothers who are the primary caretakers of those with hemophilia. Actually, it's true of any country I have studied. For whatever reason, culture, biology or psychology, mothers are deeply connected to their sons with hemophilia. They also become the primary advocates, and often the hemophilia leaders in their communities, and sadly, the self-promoted whipping post for when things go wrong.
I thought about our roles as mothers while in Morocco. I landed there a week ago, around midnight, and promptly started feeling the grinding pain of the parasite. Lovely. I missed most of Morocco, though I was there for three days. At least one thing was very memorable: a visit from a young man with hemophilia, Mohammad.
He traveled four hours by train to see me, and to pick up a donation of factor I had for him. We sat and chatted for over an hour, and shared green tea, which was delicious and strong. Mohammad is soft-spoken, respectful, and highly intelligent. He speaks three languages fluently, and studied physics but had to drop out of the PhD program when he missed two months of school due to a psoas bleed. Actually, he only mentioned “a bleed,” and when I think of what bleed could possibly keep you out of school that long, I knew it was a psoas bleed. A knee bleed you can hobble around on crutches; an elbow bleed, you can still walk. Shoulder bleeds are terribly painful, but again, you can walk. A psoas bleed means you cannot move. So he laid in bed for two months, trying not to move, with no factor. His professor thought he was lazy or lying, and no longer allowed him in the program.
Mohammad is the first in his family with hemophilia. He was diagnosed at age 2. His father was a miner, now retired. His mother never worked. At age 12 he had his first factor infusion… and then nothing until Project SHARE sent him his second infusion one month ago.
Mohammad was having an elbow bleed as we spoke. I thought maybe he should take a dose of the 14,000 IUs I brought him, but no; he would tough it out. “This will last me about a year,” he said smiling, holding the bag of factor.
Such simple gratitude, for an amount of factor that would last us two months, and the average adult US patient on prophy with hemophilia a week.
I thought of his mother today. What it must be like to see your only son suffer so much, giving up his academic dreams, knowing how intelligent he is, being able to do nothing to control his pain. So many young men with hemophilia are stoic, because for them the pain of seeing their empathic mothers suffer is greater than the pain of the bleed. They learn to mask it. Indeed, Mohammad was limping but hid it well, from many years of practice. I wondered if his mother was as stoic? She has done a marvelous job, because despite the odds, he has survived, without complaint, without bitterness, and with so much respect for his parents.
This Mother's Day we can think of all those mothers worldwide who suffer so much when their child has no medicine, like those in most countries of Africa. We can honor them today as heroes, empathic fountains of love and strength for their children, and soldiers in the war against poverty and pain when there is no factor.
Great Book I Just Read
The Africans by David Lamb
This in-depth and comprehensive book about the Dark Continent was banned in several African countries when it was published, for its no-holds-barred look at the culture, politics, history and development of Africa. Lamb spent four years traversing the continent, speaking with people on the streets as well as presidents. Lamb successfully weaves fascinating stories in with geopolitics and economics: this is no travelogue. It's a serious piece of journalism that mostly leaves his own story out, and focuses on the subject. Skillfully told, you will admire the resilience of the African people, who have survived the Arab slave trade, the invasion of colonialists, the dividing up of their continent by Europeans, their own tribalism which eats at them like a cancer, idelogical battles about Marxism vs democarcy, and lastly, their own corrupt leaders. You admire the Africans, and pity what they have endured, and come away fascinated with the complexities of this amazingly beautiful land and its beautiful people. Published in the 1980s, it does stand the test of time, but you'll need to read more about Africa to get caught up, as much has happened in the 20 years this was written--both positive and negative. Four stars.
Sunday, May 02, 2010
(Photo: Road to Murang'a)
Tuesday April 27
Today was a field trip, outside of the bustling, clogged streets of Nairobi. We headed to Murang’a, a town about 90 minutes away, off on the highways pitted with deep and numerous potholes. The shoulders of the highways are crumbling and soft so in dodging the potholes, we have to be careful not to veer too close to the shoulders or we will need up in a ditch.
You may think Africa is hot (the very name means “Away from the cold”—A frika), but Kenya is quite pleasant, with cool breezes, mostly dry air, and even chilly in the evening. No wonder so any settlers came here and stayed. The climate, the rich red soil, the friendly people make Kenya a country where people dream of living.
Maureen Miruka, mother of two-year-old Ethan and founder of the Jose Memorial Haemophilia Society, was our fearless driver. With us also was Paul, the 24-year-old administrator of the Society. How nice that Maureen hired him—he has hemophilia and many of the young men with hemophilia cannot hold down jobs. Remember that there is no factor in Kenya unless it is donated, and the donations are only enough for a few.
(Photos: Dr. Ngyo gets first donation of factor; Waiting room)
Our first stop was the Murang’a District Hospital, where we met the gracious Senior Nursing Officer Danny Mengai. Joining us later was Dr. Nguyo. Maureen had met them previously, explained about hemophilia and this time brought with her their very first ever donation of factor! This is factor that my organization Project SHARE had donated to her organization. This was the kind of teamwork and outreach I like to see. Imagine: Murang’a has a higher than normal concentration of people with hemophilia and there has never till now been any factor in the hospital. And you can bet that fresh frozen plasma is hard to come by as donating blood is not a tradition in Kenya, or in any African country.
I learned today that a big teaching opportunity for the JMHS would come in December: this is when the vast majority of circumcisions are done. Why? I asked. For religious purposes? No, Maureen replied: school holidays. Boys are circumcised around age 12 and this is when we can get referrals for bleeding disorders to the Society. Dr. Nguyo assured us he would check with Maureen at that time, and also refer anyone suspected of a bleeding disorder.
Back in the Toyota and then on to visit patients. This was our very first official field visit—sorry, the second. We must give Paul credit. He had visited previously by himself to do the groundwork, and take down patient information for Save One Life. And he did an excellent job! Our goal is to start enrolling patients from Kenya, and ask anyone reading this blog to consider sponsoring a child form Kenya. The needs are terribly great and it won’t take much to change their lives.
Take Peter for example. Just down the street from the hospital, on a red dirt road, in a small apartment lives Peter, age 19, with hemophilia. He is thin and speaks softly, like most Kenyan boys. He was so curious about his condition, and wants to be a doctor when he grows up, “So I can help other people like me. Because I know how they suffer.”
That’s an understatement. I am not sure we can understand how much these children suffer, with no relief, night after long and desperate night. (Photos: Peter; his kitchen; Me with family)
Let’s imagine what it is like for Peter when he has a bleed. There’s no money to get to the hospital. He has no crutches, of course, no wheelchair. He remains housebound. He tries to hide it from his distraught mother. She is single, and caring for Peter, and his brother, who has emotional problems as well as hemophilia, and also has adopted her two nieces, abandoned by her sister. The mother makes only about $20 US a month washing clothes and housekeeping for people. Peter’s bleed worsens, as does his pain. At night they all sleep in one room (the only other room they have, which also functions as a “kitchen,” because it has a charcoal pot in it) probably a few of them crammed into one bed. If anyone moves, this sends shooting, excruciating pain from the bleeding joint. Peter can’t get up and watch TV as a distraction, or Facebook, or do Wii, or pop a painkiller, or grab ice from the freezer or treat himself to a Coke. They don’t have a refrigerator, and own nothing but the basics—a table, chairs, couch, a bed or two, a boom box. If he gets up he’ll wake everyone. And in Africa, kids don’t whine and demand and complain. They suck it up with dignity. He lies there all night, trying to control the pain. The last thing he wants is for his mother to find out, which will add to her worry and suffering. He learns to be stoic and fight the pain, because there is simply no other choice. This bleed goes on day after day and night after night. It finally subsides… until next week, when this scenario happens all over again.
This is his life, and he cannot imagine any other.
We record his family history, ask him about his schooling, and his plans. We learn that his brother is in a mental ward. The entire family was terrorized last year during the post election violence. Thugs entered their home, set it ablaze, and chased them out into the streets. This is a nice, Christian, impoverished family, with two young men with hemophilia; they deserve nothing of what has happened to them. The younger brother, already fragile, is suffering from post-traumatic stress disorder. We make plans to go visit him. He’s ready to come home, but the family has incurred $350 in fees and like most developing country hospitals, the patient is not released until the bill is paid. You hear that right. Peter’s brother could be there indefinitely. We’ll see about that.
Who wouldn’t want to sponsor Peter? What a lovely young man; so sensitive and sweet, so gracious and kind. If he were placed in America, there would probably be nothing he couldn’t accomplish with his winning perspective.
The road calls, and we soon leave, after first handing out some presents to the family. On to the next patient: Peter’s uncle, also named Peter. He is older, with a family: wife and two children. He lives, well, basically, in a jungle. We park by the side of the road, surrounded by banana plants, overgrowth, trees and vines. Someone actually lives in that thicket of vegetation. We pull over under a big tree and a crowd of children gather, from the other side of the street. They giggle and whisper, “Mzungu!” and cover their mouths. What does that mean, I ask Maureen. Maureen smiles and says, “White.” Not many people like me make it out here, I guess.
I dissolve our differences by hauling out my stash of Tootsie Pops. Never, ever travel in developing countries without them. They withstand the heat and luggage manhandling, and are beloved by everyone. I hand out one to each bystander and immediately have fans!
We should have brought machetes because to get to Peter the uncle, we have to step over rocks and push back the big banana leaves. A short walk down the twisty sort-of trail and we arrive at their plot of land. Such poverty.
A split-log home, tin roof, muddy ground. The floor inside the house is missing; it’s just mud. There is no electricity. There are indicators of various levels of poverty and this is one key: do they have electricity? Believe it or not, you can do without plumbing. An outhouse will do and most people in the villages have them. But when you don’t have electricity…. You might as well be living in the 1800s. No, even earlier, in this case.
This family owns nothing, nothing. No vehicle, hardly any furniture. A homemade chicken coop housed a few chickens that had no food or water and the heat was growing. I felt for them, sadly. They cackled unrelentingly. A dog was penned in the back, also in a homemade kennel, with hardly any ventilation, whining to be released. The children padded around barefoot, the wife was a bit cautious, not too friendly. (Photos: the outhouse; Peter with family)
I gave the children the customary Tootsie Pops, which they eagerly accepted, and a superball and a toy kitten that shook when you pulled his tail. They were frightened by the toy at first. Then they thought it hysterical, and they made it shake over and over. I had rescued some toys from the CVS store which operates right beneath my office. Apparently, they ditch whatever they don’t sell every month: perfectly good toys, baby items and medical items. I retrieved toys for kids, baby strollers, thermometers, heat wraps, you name it. Yeah, I am a Dumpster Diver and proud of it. Everything is neatly wrapped in clear plastic bags and in perfect, new condition. And these kids had a ball with the toys.
After our interviews with Peter, we headed for the next house. Also perched in a jungle-terrain, on a hill, Charles’s home at least has electricity. And the animals look well cared for. We were greeted by the father, Sampson, an elegant elder man, his wife, who eyed us warily, and later told us this was the first time she ever met anyone else who had a child with hemophilia (! She must be about 60), Charles and his new wife, Hannah.
Charles approached us on a crutch, with obvious crippled joints. But what a nice young man: age 24, speaking fluent English (Kiswahili is the national language but most educated people—meaning grammar school and high school—can speak English as Kenya was once an English colony). Charles shared so much with us: his hopes to return to college and study electrical engineering, because he has a knack to fix anything, and he longs to contribute to his family. His parents are elderly. How much longer can they work and support him? It’s such a source of shame for an African man to be supported by his own parents. Hannah was sweet and gave me a little tour of their farm. The animals were all housed in self-made, split-log cages, but really well done and humane. A cow, goats, chickens and a dog. As clean as can be for living in jungle-like surroundings. They don’t have a refrigerator and Charles could really use one for ice for his joints. He lives so far from any hospital and they of course don’t own any kind of transportation! Not even a horse.
We were so impressed with the family. So good, high hopes, willing to work hard. Charles needed $350 to get his school fees paid for so he can return and get his degree. He just doesn’t have that kind of money. People like him, Peter and all the others we met live day to day. It’s truly survival. And when thugs burn your house down, or the rains come and wash away your farm or crops, you are really threatened with starvation and just pure survival.
We just cannot imagine their daily lives. I have a hard time, and I have seen it in front of me. How do they do it?
And yet I never hear a complaint, or curse. The only think I hear are blessings: May God bless us for our work, may we have a safe journey, thank you for helping us. Now maybe you can see why I return over and over. Despite their poverty, they have a richness many of us in developed societies lack.
We parted on such happy terms, and we all chattered on the way back about what a diamond in the rough Charles was! Peter too, in a different way.
We visited two more families, Stanley, a grown man, and Derrick, a one year old and actually Paul’s nephew. By then it was really growing late and I was out of Tootsie Pops and just about anything else in my purse that I had given away.
Our last stop of the day was at a home of a better off family. They have a proper concrete home, electricity, and the father is employed as a teacher. The son with hemophilia neatly dressed with shoes. We had a nice courtesy visit, and afterwards, to show their appreciation, the mother presented Maureen with a magnificent rooster!
They popped the rooster into a plastic bag, tied the top and carried it out to the car, its crested head popping out. When they opened the “boot” (trunk), the animal lover in me sprung out. We just can’t put a rooster all trussed up in a plastic bag into a hot trunk! It was 90 degrees and we had a two-hour journey back on horribly bumpy roads! The poor thing…
So I rode with the rooster in my lap, all the way back to Nairobi for two hours at night, petting it as if it were a cat. The doorman at the Holiday Inn was pretty surprised when he opened my door to let me out!
I’d call it a successful day all around for Save One Life and Save One Rooster.
(Please consider sponsoring one of the families mentioned above! Just $20 a month. See http://www.SaveOneLife.net)
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