Funding

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 and Dr. Carlo Rago

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.

Dr. Carlo Rago

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.

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

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: $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