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Brian Tseng Muscular Dystrophy Research Lab
Message from Brian Tseng
9/20/2010
Dear Families/Friends,
Hope the summer is winding down nicely for you! I write this letter to inform you that I have made an important career shift in responsibility.
I am stepping into a hybrid industry-academic position, joining up with Novartis in Cambridge, MA as a director to help steer their muscle drug development program and continue on staff at MGH/HMS with full academic appointment. With support from both hospital leadership and community, I aim to continue the Tuesday morning pediatric neuromuscular clinic at MGH. Plans are in place to hire an experienced pediatric nurse practitioner.
This is a great opportunity to help develop new medicines for a number of neuromuscular disorders that suffer unmet medical needs. This role will allow me to push from inside industry walls, build robust bridges with academic meccas like MGH and work with the FDA. Individuals with neuromuscular disorders need better medicines. It is my vision that someday better medicines will be proven through rigorous clinical trials to be both safe and highly efficacious for children with pediatric neuromuscular disorders, particularly muscular dystrophy. Much progress has been made in pre-clinical endeavors e.g. mouse muscular dystrophy but the translational gap is still daunting. Where are the new medicines for patients with pediatric neuromuscular disorders? I am both honored and apprehensive about this new challenge!
During this transition, I am committed to support the team for the mNeuromuscular Clinic, which has grown to become a fantastic multidisciplinary clinic that is now linking pediatric and adult MGH specialists. Only at an extraordinary place like MGH/Boston could this vision become a reality! I appreciate your support and flexibility in this transition phase! I will be sending out a follow-up letter describing upcoming clinic team changes and guidelines. Moreover I thank you for allowing me to be a part of your team!
Warmest Regards,
Dr. Brian
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is the most common and severe progressive muscular dystrophy to affect children. Dr. Tseng's laboratory investigates the mdx mouse, an animal homolog model of DMD, which lacks the same dystrophin protein but is able to stave off the severity of muscular dystrophy.
Although there are scores of labs working to describe the function(s) of this protein, there has been a paucity of information describing how the downstream effects of dystrophin protein absence can manifest into a severe muscular dystrophy phenotype in humans but not in the mdx mice. The long-term goal will be to elucidate the differential underpinnings in the mdx mouse skeletal muscle (from lab-bench) and exploit such knowledge to translate into better understanding and therapeutics for DMD (at the bedside).Overview: Mice without dystrophin
Dystrophin is a protein that provides strength and stability to muscles. Boys with Duchenne muscular dystrophy (DMD) do not make normal dystrophin and their muscles weaken over time. However, there are some mice (mdx) that do not have dystrophin but they do not appear crippled. Although under a microscope, some of their muscles look abnormal like boys with DMD, these mice do not lose the ability to walk. Although there are some ideas why this happens, researchers cannot explain why these mice do not lose the ability to walk. It is possible that other genes are somehow helping muscles to remain viable despite the lack of dystrophin.
Research in the Tseng lab is focused on looking for other genes in these mdx mice that helps keep their muscle function. If genes are found that keep dystrophin- deficient muscles from becoming weak, it could provide important clues for new treatments for boys with muscular dystrophy. Current techniques employed in the lab include: microarrays, bioinformatics tools, transgenic mouse technology, PCR, RNA/DNA/protein electrophoresis, immunoflourescence, confocal microscopy, magnetic resonance imaging and spectroscopy, and many others.
Philanthropy
The most challenging aspect of being both a researcher and physician is the fact that time and effort must be taken away from this important work to find funding. It is critical, yet indirectly draws away from my two primary DMD missions of medical care and research. If you know anyone or any organization looking to make tax-deductible donations, please consider designating the Tseng muscular dystrophy lab which is dedicated 100% towards muscular dystrophy research. For more information please see our Philanthropy page.
Resources
For more information about muscular dystrophy and parent support avenues, consider these websites:
Muscular Dystrophy Association (MDA) www.mdausa.org
Parent Project Muscular Dystrophy ( PPMD) http://www.parentprojectmd.org
My Favorite Client - January 4, 2009
This is an article about Gordon Sterling, one of Dr. Brian Tseng's patients
http://www.growcookeat.com/2009/01/my-favorite-client.html