The funds received through Ryan's Quest are directed, in full, to Duchenne muscular dystrophy research. Ryan's Quest bases all decisions
regarding the awarding of funds to research projects, by speaking with lead doctors, researchers and scientists concerning current
and promising advances being made in Duchenne research. We also collaborate with other non-profit organizations which have similar
goals to that of Ryan's Quest. These organizations also have scientific advisors who specialize in the field of DMD to determine the
validity and potential of a given project.
We are also honored to have our very own scientific advisors, Dr. Brian Tseng, Dr. Carlo
Rago and Dr. Robert McDonald
Dr. Brian Tseng
As an assistant professor, Dr. Tseng joined the faculty at Harvard Medical School/
MGH in the Department of Neurology, Division of Child Neurology. With an MD/PhD background, he is a physician-scientist working to
find better treatments and better care for Duchenne muscular dystrophy (DMD). Dr. Tseng is also a board-certified pediatric neurologist
with neuromuscular interest.Dr. Tseng received his medical and doctoral degrees from the University of Texas, Houston. He completed
his Pediatric Residency at the University of Colorado Denver School of Medicine and his Fellowship in Child Neurology at the University
of California San Francisco.
Carlo Rago Ph.D
Dr. Rago has authored nearly 30 papers in high-profile journals including Science,
Nature, and PNAS. He received his BS in Biology from Rider University where he honed his passion for research while working
in the immunology lab of Dr. James Riggs. As a graduate student in the Cellular and Molecular Medicine program at the Johns
Hopkins School of Medicine, his work focused on cancer genetics and therapeutics. He is currently a post-doctoral fellow in the
Vogelstein lab. Many of Dr. Rago's experiences in cancer research share extensive overlap with work in the field of DMD, including
small molecule drug screens, use of AAV, and development of biologic and antisense therapeutics.
Dr. Robert McDonald
Dr.
McDonald currently operates his own private practice in Jefferson City, Missouri. He received his medical degrees from the University
of Missouri-Columbia and postgraduate training in Neurosurgery at Temple University. Dr. McDonald has a wide range of research
experience to offer in the area of microbiology, cell biology, and molecular genetics.
In addition to his medical
background, Dr. McDonald is also a parent of a young boy with Duchenne muscular dystrophy.
How we choose the projects which we fund..........
Project: Compound Quest
Overview
Compound Quest is a research initiative being carried out by Dr. Herman Vandenburgh and his team at Myomics, Inc. in
Providence, Rhode Island. Compound Quest utilizes a unique technology that allows for the testing and measurement of the effect
of pharmaceuticals and nutritional supplements that can benefit boys with DMD and move them quickly into a human clinical trial.
This
contribution was made through the Jett Foundation of Pembroke, MA who has partnered with neuromuscular specialist,
Dr. Brian Tseng of Mass General Hospital for Children. This project was funded in collaboration with DMD Foundations which
allowed for matching grants to double the initial investment.
Project: CombinatoRx
Overview
CombinatoRx is a unique pharmaceutical company based in Cambridge, MA. The company focuses on developing new medicines
from combinations of existing FDA-approved drugs that can work together to achieve a desired effect. CombinatoRx has a team of six
scientists devoted to developing a treatment for DMD. They are looking at millions of combinations of drugs that have been approved
for other uses to see if any of the compounds can work in tandem to slow or stop the relentless progression of DMD. This research
is funded by several DMD foundations, including:
• Charley's Fund (South Egremont, MA)
• The Nash Avery Foundation (Minneapolis, MN)
• The GM Trust (London, UK
Project: Biglycan
Overview
Brown University -- Justin Fallon, PhD
Dr. Fallon has developed a drug called Biglycan, which stimulates the cells affected
by DMD, forcing them to continue producing utrophin which is an important protein people normally lose around the age of four or five.
Utrophin is a compensatory protein that can act as a substitute for dystrophin, the missing protein in DMD boys. Dr. Fallon has discovered
that biglycan can upregulate utrophin expression in a muscular dystrophy mouse model. He has observed other beneficial effects of
biglycan, including reduction of muscle fiber cell death. Dr. Fallon is now scaling up the production of biglycan in preparation for
preclinical safety studies.
Project: Project Catalyst
Overview
This contribution was funded through PPMD's Project Catalyst. Project Catalyst is a first of its kind research initiative
designed to develop oral medications that may delay muscle degeneration in boys with DMD.
Raising Awareness
Finding a Cure
For this Generation of Boys with DMD
©Copyright 2009-2010 Ryan's Quest Inc., all rights reserved
Project: Stem Cell Therapy
Overview
University of Milan--Dr. Giulio Cossu
A promising stem cell approach that uses mesoangioblasts, adult stem cells precursor
cells derived from blood vessels of muscle biopsies. These cells would come from either a matched healthy donor, or from the
DMD patient. The harvested stem cells are amplified and treated outside the donor's body, then re-introduced into the patients
body.
This contribution was made through Cure Duchenne of Corona Del Mar, CA, who is currently funding Dr.
Giulio Cossu at the University of Milan.
Amount Funded: $15,000
Amount Funded: $15,000
Amount Funded: $50,000
Amount Funded: $50,000
Amount Funded: $15,000
Project: Compound Quest
Overview
Contribution towards helping Compound Quest start developing their own human DMD muscle cell lines. This will yield
a more effective and accurate result of the tested compounds to treat DMD. This was designated to Dr.Brian Tseng's lab at Mass General
Hospital in Boston, MA through the Jett Foundation.
Amount Funded: $ 5,000
Project: Project Catalyst
Overview
University of Pennsylvania-Lee Sweeney Ph.D.
Continued testing of project catalyst lead compounds (utrophin, myostatin,
and IGF-1) at the University of Pennsylvania.
Amount Funded: $ 25,000
Project: Osteopontin
Overview
UCLA-Melissa Spencer Ph.D.
Potential of Osteopontin as a promising therapeutic target to help reduce inflammation
and fibrosis in Duchenne MD. This contribution was made through PPMD's End Duchenne Grant Program.
Amount Funded: $ 25,000
Project: Clinical Trial Research Personnel
Overview
This contribution was made to provide support with DMD clinical trial research personnel at MassGeneral Hospital for
Children in Boston, MA. This contribution was generously matched resulting in $20,000!!
Amount Funded: $ 10,000
Project: Clinical Outcome Measures Initiative
Overview
An ongoing priority with respect to DMD research is the need to establish an international consensus in developing
age appropriate clinical and biochemical outcome measures. The intent of this project is to provide a data driven, international recommendation
to define the most appropriate clinical outcome measure or measures, as a function of age, to use in clinical trials in DMD and to
define the most appropriate biochemical outcome measure (dystrophin quantification), to use in clinical trials in DMD.
This contribution
was made through Treat-NMD (United Kingdom) and the Wellstone Muscular Dystrophy Research Center (Washington, DC).
Amount Funded: $ 5,000
Funding
THE FIGHT CONTINUES....
THANK YOU FOR THE PAST.... WORKING FOR A FUTURE!
