Cure Duchenne to Host Inaugural Duchenne Muscular Dystrophy Summit
March 8, 2010 by CureDuchenne
Filed under Featured
On March 19, 2010, CureDuchenne’s Scientific Advisors from around the country will come together in an unprecedented meeting to find a cure for Duchenne Muscular Dystrophy (DMD).
The group of ten includes world-class scientists coming together for the chance to sit with each other and share new strategies around finding a cure for DMD.
“We know that the path to a cure starts with collaboration,” said CureDuchenne co-founder Debra Miller. “By bringing together ten of the world’s most forward-thinking scientists, researchers and drug development experts in the field of DMD research, we hope to be able to best identify the strategy necessary to move treatment options into the human clinical trial stage. We’re racing the clock to save our boys, and if there ever was a group capable of finding a cure, it’s this one.”
10 Leading DMD Scientists from around the country together in one place to share with Parents and Advocates.
The inaugural DMD Summit will include these muscle disease and drug development experts:
- Barry Byrne, MD, PhD, University of Florida
- Jeffrey Chamberlain, PhD, University of Washington
- Kevin Campbell, PhD, University of Iowa
- Eric Hoffman, PhD, George Washington University School of Medicine
- Edward Kaye, MD, Genzyme
- Douglas Macdonald, PhD, CHDI Foundation, Inc.
- Carrie Miceli, PhD, UCLA
- Stanley Nelson, MD, UCLA
- Pier Lorenzo Puri, MD, PhD, The Burnham Institute
- Brian Tseng, MD, PhD, Harvard University and Massachusetts General Hospital
This group of scientists serves as our scientific advisory board and works with the org
anization to determine what projects around the world are closest to the human clinical trial phase and the most viable for funding. To date, CureDuchenne has provided millions of dollars in funding for DMD research around the world and has been integral in bringing 3 treatment options to the human clinical trial phase.
The DMD Summit will take place over the course of three days and will include closed session discussions, research project evaluation, and a breakfast event where each participant will report on the findings from the weekend.
The breakfast will be attended by DMD parents, advocates and supporters.
We want everyone who is interested in finding a cure to DMD to attend the Saturday morning breakfast where you can talk personally with these leading researchers. This is a rare chance to get to learn from the top minds all in one place!
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PTC Therapeutics and Genzyme Corporation Announce Preliminary Results from the Phase 2b Clinical Trial
March 3, 2010 by CureDuchenne
Filed under Research Articles
Please take some time to read this important announcement:
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Dr. Barry Byrne, one of Cure Duchenne’s scientific advisors, talks about his role in the movie, “Extraordinary Measures”
February 26, 2010 by CureDuchenne
Filed under In The News
CureDuchenne is very honored to have Dr. Barry Byrne as one of our scientific advisors. Dr. Byrne’s dedication goes beyond science and medicine; he has a true heart for his pediatric patients and is working hard to make a future for all of them. Dr. Byrne was the first person to register for the “2009 Climb to CureDuchenne” and we are looking forward to having him on our team in the “2010 Climb to CureDuchenne: Pick Your Peak.”
Q. Tell us a bit about how you came to be involved with the Crowley family, whose story was recently made into a major motion picture “Extraordinary Measures”?
A. Our group began a scientific collaboration with a laboratory in Oklahoma working on biochemical modifications of the enzyme planned for use in Pompe disease. We conducted the early animal studies with this group and I had the opportunity to serve as an advisor in developing the clinical program which grew out of this collaboration. John became a business partner with the Bill Canfield and we have worked together since that time.
Q. What was it like seeing your work translated into a book and then a movie?
A. The remarkable thing about scientific discovery is that small incremental advances are made by a series of individuals and this contributes to the overall story. Occasionally, a big leap in understanding moves the story along in a more substantial way, but mostly any large problem is solved with a series of small discoveries. So our contributions are embedded in a quilt of contributions from many individuals and groups. It is great to be part of that effort and recognize the moments where we helped move the story along.
Q. Harrison Ford’s character, Dr. Robert Stonehill, is based around a group of scientists, is that right? How close to the truth does the movie come?
A. A number of people contributed to the scientific side of this story as it is described in the book “The Cure” by Geeta Anand. Dr. William Canfield and myself were the two physicians working with John Crowely in the time frame of the book and subsequent screen plan. John had meet with many doctors in his quest to understand what options he had for Megan and Patrick and Bill Canfield and I had started a scientific collaboration before Canfield and Crowley became business partners. There are some parts of the movie where the time line is compressed to make a logical sequence of events but I thought the essential elements of the book were captured faithfully in the screen play.
Q. The movie highlights the red tape involved in bringing new treatments for rare diseases through to clinical trials, what has your experience been with that?
A. This is one aspect where I think the movie helps inform the public on this important issue which a key point of getting bench work back to the bedside. In this particular case the work moved very quickly in part because of the competition which was alluded to in the movie. Four different products were underdevelopment in the Netherlands, Duke University, Genzyme and the Canfield/Crowley company called Novazyme. We did the comparative experiments to evaluate these products side by side. At the point of moving one these products into the clinic, Genzyme had to make the decision which enzyme was to be used. Based on a series of considerations, the management team chose the Genzyme cell line to manufacture Myozyme. The process was costly and this is often a huge barrier for rare disease treatments. Of course the time involved is glacially slow when faced with children who are affected by progression of the disease.
Q. You’ve been a great supporter of CureDuchenne, even joining us on our first annual Climb to CureDuchenne up Mt. Rainier, how has your work in cardiology lead you to working on rare childhood diseases like Pompe and DMD?
A. In my medical training I was initially interested in becoming a geneticist and then found that pediatric cardiology was also a fascinating area where there were many interventions available to help children with structural heart disease. Over the past 15 years, we have learned so much about the fundamental genetic causes of how the heart functions, I believe we are entering an era of developing ways to treat the functional problems of the heart with genetic therapies. I feel very fortunate to have found a way to merge these two interests.
Q. It seems this film has shed an important and necessary light on rare childhood diseases, like DMD. What do you see as the future for DMD research? What projects are you currently on?
A. There are many parallels to the efforts of the large group of scientists working on Duchenne muscular dsytrophy (DMD) as there were in the area covered in the film. As these diseases represent two important forms of muscular dystrophy, I hope the scientific efforts are complementary in a way that moves the field along even faster.
We are focused on developing the gene therapy methods which will enable systemic treatment for both DMD and Pompe disease. Again, this type of work takes a team and I am very glad that our team is moving quickly toward this goal.
Q. You were one of the climbers last year on the Climb to CureDuchenne at Mt. Rainier. Why did you sign up for that and what was the experience like?
A. The challenge to climb Mt. Rainier was one way to show my commitment to helping advance research in this area. The summer before Rainier, my son and I had a great experience climbing at the Philmont scout ranch in New Mexico. It struck me how this sort of experience should be shared by all parents, especially for boys with DMD. If we are successful in finding a really durable cure, I would consider my career a success if this feat were possible for a young man with DMD.
Q. The 2010 Climb to CureDuchenne: Pick Your Peak has just been announced. Will you rally again to be part of a climbing team?
A. One of the unexpected benefits of participating in the 2009 Climb is that I am lucky to have 11 new close friends that formed the original team. Our team in Florida is working on way to continue the tradition and I hope to contribute to this effort as long as I can. As Bill Procko said, climbing is like the work we do in the lab, putting one foot in front of the other until we reach the goal. The 2010 Climb is brings us another step closer to a cure for DMD. I’m IN.
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