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, on the advice of our scientific advisors Dr. Brian Tseng, Dr. Carlo
Rago and Dr. Robert McDonald.
We also collaborate with other non-profit organizations which have similar goals to that of
Ryan's Quest concerning current and promising advances being made in Duchenne research These organizations also have scientific advisors
who specialize in the field of DMD to determine the validity and potential of a given project.
Ryan's Quest Policy on Donated
Funds- Ryan's Quest, Inc. contributes 100% of proceeds for programs, grants and projects that support its mission to provide and appropriate
funds that benefit the community for which it serves. Funds are not to be used for institutional, administrative, overhead or indirect
costs, only direct patient care or research projects."
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
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).
(view
journal article here)
Amount Funded: $ 5,000
Project: Exon Skipping
Overview
This project is focused on helping develop new sequences and new formulations of exon skipping compounds. This
contribution was made in collaboration with Cure Duchenne. (Further announcement to be made soon).
Amount Funded: $ 100,000
Project: Biophysical Characterization
Overview
University of Minnesota-James M. Ervasti Ph.D.
This contribution will direct Dr. Ervasti in exploring the biophysical
properties of dystrophin proteins
Amount Funded: $ 37,500
©Copyright 2011 Ryan's Quest Inc., all rights reserved
THE FIGHT CONTINUES....
THANK YOU FOR THE PAST.... WORKING FOR A FUTURE!
Project: Scholarship Fund
Overview
This contribution was made through The Jett Foundation (Boston, MA) in support of their scholarship fund benefiting
young men with Duchenne muscular dystrophy
Amount Funded: $ 1,000
Project: University Of Washington
Overview
Support of Hannele Ruohola-Baker’s lab to assist in purchasing a critical piece of equipment (Strip Myograph) that will allow
the lab to perform critical functional experiments to further assess potential compounds as a therapy for muscular dystrophy.
Amount Funded: $ 5,000
Project: Duchenne Dashboard
Overview
In conjunction with other Alliance organizations the creation of the Duchenne Dashboard was designed
to optimize research funding strategies. The Alliance has a variety of interests that reflect the missions of the members
including - but not limited to - basic science, preclinical therapeutics, clinical trials, quality of care, and quality of life.
Proposals submitted through the Duchenne Dashboard enjoy several advantages: 1. transparency - the author can determine who sees the
submission, 2. thorough review - web 2.0 features allow crowd-sourcing of scientific advisors, 3. rapid response - the Alliance operates
in real-time and the author will receive feedback and funding accordingly, 4. single portal - one door provides instant access to
all participating Foundations. Submission of NIH grants with scores close to the pay line are strongly encouraged.
Amount Funded: $ 12,500
We are a member of the Duchenne Alliance and we are using the Duchenne Dashboard to optimize our funding strategies. The Alliance has a variety of interests that reflect the missions of the members including - but not limited to - basic science, preclinical therapeutics, clinical trials, quality of care, and quality of life. Proposals submitted through the Duchenne Dashboard enjoy several advantages: 1. transparency - the author can determine who sees the submission, 2. thorough review - web 2.0 features allow crowd-sourcing of scientific advisors, 3. rapid response - the Alliance operates in real-time and the author will receive feedback and funding accordingly, 4. single portal - one door provides instant access to all participating Foundations. Submission of NIH grants with scores close to the pay line are strongly encouraged.
Project: Kennedy Krieger Institute, John Hopkins School of Medicine
Amount Funded to Date: $ 25,000
Amount Committed: $76,500
Travel grant to assist Dr. Kathryn Wagner from The Kennedy Krieger Institute, John Hopkins School of Medicine for a trial
she is currently conducting to target the heart of DMD boys with an already approved FDA medication. The goal
of this grant is to help offset the financial burden to families due to the amount of necessary trips to Kennedy Krieger required
by the trial protocol. Therefore, we hope to facilitate this important trial by filling the trial to its capacity; which
leads to more reliable and valid results. We are cautiously optimistic that trials such as Dr. Wagner's (testing of
already FDA approved drugs) will lead to a promising treatment for Duchenne in the near future.
Further trial
info can be found at ClinicalTrials.gov
Project: University of Washington Department of Biochemistry
Amount Funded: $ 10,000
Overview:
This research contribution is intended to support the ongoing research of Hannele Ruohola Baker and her research team on
compounds that may help muscle regeneration and lead to a potential therapy for Duchenne muscular dystrophy.
