Since my son was diagnosed twenty years ago, there have been so many treatment type wolf warnings that it's hard to not be sketical when people talk about treatments and cures. Parents want to see work with results that are going towards some identifiable end. We get reports and announcements and articles but where's the timeline? We fought for and got big dollars to ramp up results not pay someone's salary. Who will deliver these results?
Put another way, are the scientists whose muscular dystrophy research we count on to save our children's lives, worthy of our trust and hope?
Here in DC, they are and it starts at the top.
I've worked in the Hoffman Lab in Washington DC for almost seven years. The lab has grown to close to 100 researchers. I've had a chance to observe the comings and goings of many scientists and gotten to know them in ways not normally available to parents. Would I say they are worthy of my trust and hope? What is different about people here that gives them an advantage I look at what they do with their lives - and what is important to them.
Even before Eric Hoffman moved his lab from Pittsburgh to Washington, DC, he offered the researchers who work for him a paid week off to volunteer at MDA Summer Camp. He's even worked as a counselor himself. As we parents know, when you live, work, play every breath from the care givers' point of view, then muscular dystrophy becomes more than test tubes and proteins. It's the difference between reading about a sunset and seeing one.
This year, as in years past, I go to camp with a team of massage therapists to give the counselors and kids there a massage. So many of the counselors there were from work that I might as well been at the hospital! At least one-third of the volunteers and staff either work in the lab now or had worked there and since moved on. Many of them raved about camp as a life-changing experience. And they acknowledged Eric for giving them this opportunity.
It would be remarkable if this were the one instance of giving back to the community but there are other, individual instances, like bringing a scientist from India to the States for training so she could run a diagnostic lab back home or the art work he bought featuring people with muscular dystrophy for the corridor outside our lab or offering a room to visiting scientists and even me, when my son had an operation two years, giving me as much time as I needed for his recovery.
But Eric is only the beginning. I worked in different places over the years and have never been so aware of the generosity of others as I am here.
Paola Canelos, a clinical coordinator from our lab and Tina Duong, our physical therapist, just got back from a week in Ecuador where there was no such thing as a muscular dystorphy clinic. Even physical therapy which costs $3 a session is out of the question for some families particularly from the outlying areas.
Paola and Tina are in the process of setting up a muscular dystrophy clinic. Streets are not accessible, equipment that we take for granted, like wheelchairs are a luxury item and the only time children with muscular dystrophy see a doctor is for a diagnosis. A hastily put together session to teach physical therapists stretching techniques that they can in turn teach parents to lessen contractures. Instead of the twenty or so therapists they expected, they were greeted by 60 therapists wanting to learn stretching techniques and how to train parents to lessen contractures.
Lakshmi, who came from India last year in order to be trained in lab techniques, listened to the experiences from MDA summer camp related by people in the lab. She came back this year for more lab training and she timed her visit so that she could spend a week at summer camp herself. In India, children with muscular dystophy don't go to school and are excluded by virtue of their immobility from having a normal social life. Lakshmi went back to India in July and last weekend held the first MDA India camp; a 'fun weekend'. Next year she plans to devote a whole week, with a volunteer for each child.
Rob Freishtat, one of our Center docs spent a week in Egypt performing cleft palate surgeries. He performed one surgery after the next
Naomi Bartley, another clinical coordinator (she used to work with Petey!) will host the first ever 'Naomi's Hope for a Cure' a fundraiser for childhood cancer research. She came up with the idea of auctioning off Hope Chests, painted by different renowned artists such as Alan Bateman, Senator Diane Fienstein, and Val Bradley . The Gala will be held on November 16th at National Building Museum, Washington DC. Naomi is a childhood cancer survivor and Eric's step-daughter.
Those are the big Wow items. Just as impressive are the always needed and given helping hands: the fundraiser happy hours to help pay for families coming to Children's National Medical Center for clinical trials; the money donated, the weekends and evenings spent at work nursing some experiment or making sense out of data. We even have someone who found stray cats at the hospital parking lot and paid for them to be neutered and spayed!
The giving goes on and on and on.
We are pushing for a treatment. The Center hopes to start morpholino clinical trials in the next year that will lead to a treatment.
Over the years I have developed a healthy sketicism when it comes to muscular dystrophy treatments and cures. Right now, I reserve judgement They proved themselves, in a word, worthy.
Interested In receiving a FREE copy? Email Tina Duong at tduong@cnmcresearch.org
October 6, 2007
October 4, 2007
A Four Star Night: Dining Away Duchenne
I can’t decide if James Wood looks more like his mom, Dana, or his dad, Joel. But, 
he’s a scamp and a cutie. He gets that gleeful smile thing going, especially when he thinks he’s getting away with something. James is the impetus behind ‘Dining Away Duchenne’.
Seven years ago Joel and Dana held the first ‘Dining Away Duchenne’. James was three, just getting the hang of independence; exploring the world, pushing limits – the kinds of unremarkable things that little boys do when they are three. But with a difference; as his parents had just found out, James has duchenne muscular dystrophy.
When I first met Joel and Dana, they were at a muscular dystrophy support group meeting. My husband, Peter, and I were there with our son, Petey, who was se
venteen at the time. Petey has duchenne’s too and was preparing to move away to college in the fall.
And because duchenne’s progression has been the same since time immemorial, older kids with duchenne, with wheelchairs and breathing tubes, are a fearful reminder to new parents of what is in store for their kids.
Joel told Petey that meeting him gave him hope, for James’ future. As a parent of a child newly diagnosed with duchenne, his was a courageous and healthy response.
The Woods began mobilizing resources and people, the likes of which I had never seen before. The next thing I know, they were throwing this shindig, a fundraiser for research into a treatment for duchenne. And they put it together in a matter of months.
It was the first ‘Dining Away Duchenne’ and they made $620,000 that year, a remarkable
feat. Thus began their fundraising efforts and what became the “Foundation to Eradicate Duchenne”, their non-profit group dedicated to raising money for duchenne research.
Dining Away Duchenne is a four star ‘taste of DC’ and the chefs who prepared the food samplings are stars in their own right. This spectacular event was held in downtown Washington, DC. The night was beautiful and the guests milled around on a veranda in the shadow of the Capital Dome. I felt like I was in a movie or something.
At that fir
st event, I met Senator Paul Wellstone, who, along with Representative Richard Wicker, took a leadership position in support of the MD Care Act – an act of congress that catapulted duchenne research to a funding level where life-changing research became possible.
That was seven years ago, and through the years some things are different but the main thrust is the same: We need funding and research, funding and research, funding and research; to create a treatment for the Jameses and the Peteys of the world.
This year’s event, honored Senator Susan Collins, one of the principal authors of the MD Care Act. Dining Away Duchenne was held on September 18, at Johnny’s Half Shell, a four star restaurant a stone’s throw from the Capitol. The food was excellent, as usual, with samplings from Johnny’s, Tosca, Oyam
el, Café Atlantico, Bistro Bis, and Colorado Kitchen, among others.
This was the finest event – ever, bringing in $630,000!!
It’s been seven years since James was diagnosed and almost as long since Joel and Dana began the Foundation to Eradicate Duchenne. Research is progressing with stop-codon advancement and morpholino translational research moving forward to human trials. Sadly, some of our good friends, including Josef Gapco, who sang at a previous ‘Dining Away Duchenne’, have died fighting this disease.
The Wood Family is joined in this endeavor by other parent’s fighting for the lives of their boys, including good friends Dave and Monica Heil, whose son, Alex, was also diagnosed with duchenne.
Research is further along than I thought possible, but not far enough yet. As Joel said, we have a treatment “we know will work. Only time, money and regulatory hurdles stand in our way.”
James is 10 now, an age when Petey was transitioning into a wheelchair. James moves independently but his parents can tell he’s weaker. How much time does he have before he’s in a wheelchair? Will research provide the answers necessary to keep him healthy and strong?
“Only time, money and regulatory hurdles stand in our way.”
GenMed Lab Mom
Lab Word on the Blog (http://www.pbs.org/wgbh/nova/genome/expl_03_stop.html)
Stop codon: A codon is a group of three bases - A, T, C, or G - and codes for a single amino acid, the building blocks of proteins. A stop codon tells the cell's machinery that it has reached the end of the protein and should stop translating the code. Stop codons come in three different forms - TGA, TAG, and TAA.
Quote:
"The winter has been long. This is the first dawn when anyone could bear to speak of spring." from "A Sense of the Morrning: Nature Through New Eyes" by John Brendan Hope.
he’s a scamp and a cutie. He gets that gleeful smile thing going, especially when he thinks he’s getting away with something. James is the impetus behind ‘Dining Away Duchenne’.Seven years ago Joel and Dana held the first ‘Dining Away Duchenne’. James was three, just getting the hang of independence; exploring the world, pushing limits – the kinds of unremarkable things that little boys do when they are three. But with a difference; as his parents had just found out, James has duchenne muscular dystrophy.
When I first met Joel and Dana, they were at a muscular dystrophy support group meeting. My husband, Peter, and I were there with our son, Petey, who was se
venteen at the time. Petey has duchenne’s too and was preparing to move away to college in the fall.And because duchenne’s progression has been the same since time immemorial, older kids with duchenne, with wheelchairs and breathing tubes, are a fearful reminder to new parents of what is in store for their kids.
Joel told Petey that meeting him gave him hope, for James’ future. As a parent of a child newly diagnosed with duchenne, his was a courageous and healthy response.
The Woods began mobilizing resources and people, the likes of which I had never seen before. The next thing I know, they were throwing this shindig, a fundraiser for research into a treatment for duchenne. And they put it together in a matter of months.
It was the first ‘Dining Away Duchenne’ and they made $620,000 that year, a remarkable
feat. Thus began their fundraising efforts and what became the “Foundation to Eradicate Duchenne”, their non-profit group dedicated to raising money for duchenne research.Dining Away Duchenne is a four star ‘taste of DC’ and the chefs who prepared the food samplings are stars in their own right. This spectacular event was held in downtown Washington, DC. The night was beautiful and the guests milled around on a veranda in the shadow of the Capital Dome. I felt like I was in a movie or something.
At that fir
st event, I met Senator Paul Wellstone, who, along with Representative Richard Wicker, took a leadership position in support of the MD Care Act – an act of congress that catapulted duchenne research to a funding level where life-changing research became possible.That was seven years ago, and through the years some things are different but the main thrust is the same: We need funding and research, funding and research, funding and research; to create a treatment for the Jameses and the Peteys of the world.
This year’s event, honored Senator Susan Collins, one of the principal authors of the MD Care Act. Dining Away Duchenne was held on September 18, at Johnny’s Half Shell, a four star restaurant a stone’s throw from the Capitol. The food was excellent, as usual, with samplings from Johnny’s, Tosca, Oyam
el, Café Atlantico, Bistro Bis, and Colorado Kitchen, among others.This was the finest event – ever, bringing in $630,000!!
It’s been seven years since James was diagnosed and almost as long since Joel and Dana began the Foundation to Eradicate Duchenne. Research is progressing with stop-codon advancement and morpholino translational research moving forward to human trials. Sadly, some of our good friends, including Josef Gapco, who sang at a previous ‘Dining Away Duchenne’, have died fighting this disease.
The Wood Family is joined in this endeavor by other parent’s fighting for the lives of their boys, including good friends Dave and Monica Heil, whose son, Alex, was also diagnosed with duchenne.
Research is further along than I thought possible, but not far enough yet. As Joel said, we have a treatment “we know will work. Only time, money and regulatory hurdles stand in our way.”
James is 10 now, an age when Petey was transitioning into a wheelchair. James moves independently but his parents can tell he’s weaker. How much time does he have before he’s in a wheelchair? Will research provide the answers necessary to keep him healthy and strong?
“Only time, money and regulatory hurdles stand in our way.”
GenMed Lab Mom
Lab Word on the Blog (http://www.pbs.org/wgbh/nova/genome/expl_03_stop.html)
Stop codon: A codon is a group of three bases - A, T, C, or G - and codes for a single amino acid, the building blocks of proteins. A stop codon tells the cell's machinery that it has reached the end of the protein and should stop translating the code. Stop codons come in three different forms - TGA, TAG, and TAA.
Quote:
"The winter has been long. This is the first dawn when anyone could bear to speak of spring." from "A Sense of the Morrning: Nature Through New Eyes" by John Brendan Hope.
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