funding
science.
finding
hope.
articles &
videos
CBS
60 minutes:
our story.
we need your help now.

We need your help now.

Dickson_w_samantha.jpg

Children with MPS I around the world are suffering from cognitive impairment and memory loss because of MPS. Dr.'s Agnes Chen and Patricia Dickson at Harbor UCLA's LA BioMedical Research Center in Torrance, California have initiated a study to help treat those who suffer with these horrible signs of MPS. Dr's Chen and DIckson's trial involves placing a small amount of Aldurazyme, the drug the Ryan Foundation provided invaluable funding to help develop (see 60 Minutes link), into the spinal cord where it is then taken to the brain by the patient's cerebral spinal fluid. Sixteen trial participants are being identified from across the US and Canada. Each participant will need to travel to Torrance, California and/or Oakland Children's Hospital in Oakland, California to receive testing and treatment once before their trial begins, once a month for four months, and then once every quarter until the trial concludes. In addition, each participant will need to travel to the University of Minnesota in Minneapolis, Minnesota prior to the trial and twice yearly once the trial begins where Dr. Elsa Shapiro and her team of experts will test and perform detailed MRI's on the participants so that data may be gathered on how the brain is responding to the treatment.

All of this travel is quite expensive. The Ryan Foundation and our partners at UCLA, Oakland Children's, and the University of Minnesota have estimated that $10,000 per month is needed to pay for airfare, hotel, food, and ground transportation for each of the participants and one parent or guardian. That's $10,000 per month for five years. These children deserve the chance to live a healthy life, but they can not achieve their goals without YOUR help. The Ryan Foundation has accepted the challenge to provide all of the necessary funds for these brave and heroic children and adults to travel to receive treatment - the treatment that may provide them the gift they dream of each day... ...the gift of tomorrow.

Every dollar donated to the Ryan Foundation in 2014 will be used to fund the travel for this ground breaking trial. Won't you please partner with others around the world and help these children find their tomorrow's at LA Biomedical at UCLA, Oakland Children's Hospital, and the University of Minnesota? 

You may make donations by using our PayPal account or by contacting us for other payment options. Thank you for supporting our mission of funding science and finding hope for the lives of children in need. 

success stories.

Funding Science: Current Projects

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. Patricia Dickson, P.I, Dr. Agnes Chen, M.D.

Purpose

“A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I”

Results/Updates

This is a 24-month study of the use of laronidase administered into the spinal fluid to treat cognitive decline in mucopolysaccharidosis I (MPS I).

More Information can be found at:
http://clinicaltrials.gov/show/NCT00852358
http://mpsresearchlab.labiomed.org/joomla2/index.php/our-research/14-funding/18-ryan-foundation

Location

University of Minnesota

Investigators

Dr. Chet Whitley P.I., Dr. Elsa Shapiro P.I.

Purpose

To identify abnormalities in brain structure and function over time in treated and untreated MPS patients.

Results/Updates

To identify abnormalities in brain structure and function over time in treated and untreated MPS patients. To identify distinct neuroimaging and neuropsychological patterns for each MPS disorder.  To develop quantitative measurements of change.   To examine the degree to which risk factors influence brain and Quality of Life outcomes.

For more information:
https://events-support.com/Documents/Shapiro.pdf
http://www.mps2012.eu/upload/siteimages/MPS2012/S04.3.pdf

View More

Location

Icahn School of Medicine at Mount Sinai, New York

Investigators

Calogera M. Simonaro, MPh, PhD, Associate Professor of Genetics and Genomic Sciences
Edward H. Schuchman, MPh, PhD, Professor of Genetics and Genomic Sciences

Purpose

To conduct clinical studies of pentosan polysulfate (PPS) in patients with mucopolysaccharidoses (MPS).

Results/Updates

Dr. Simonaro and Dr. Schuchman have demonstrated in MPS animal models that PPS has the effect of reducing the chronic inflammation associated with substrate accumulation, resulting in improved motility and a slowing of bone/cartilage disease.

http://www.mpssociety.org/wp-content/uploads/2013/05/bene-pharnaChem-GmbH-5.15.13.pdf

Funding Science: Past Projects

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. Patricia Dickson P.I.

Purpose

“Early Versus Late Treatment of Spinal Cord Compression With Long-Term Intrathecal Enzyme Replacement Therapy in Canine Mucopolysaccharidosis Type I.”

Results/Updates

http://www.ncbi.nlm.nih.gov/pubmed/20655780

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center and Iowa State University

Investigators

Dr. N. Matthew Ellinwood P.I. Iowa State University. Dr. Patricia Dickson P.I. Dr. Agnes Chen P.I. Harbor-UCLA LA Biomedical

Purpose

“Replacing the Enzyme α-L-Iduronidase at Birth Ameliorates Symptoms in the Brain and Periphery of Dogs with Mucopolysaccharidosis Type I”

Results/Updates

http://www.ag.iastate.edu/news/releases/903/
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3075726/
http://harborpeds.org/news/pattidicksonagneschenMPSpaper2010
http://stm.sciencemag.org/content/2/60/60ra89.abstract

View More

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. Patricia Dickson P.I.

Purpose

“Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs”

Results/Updates

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2889197/

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. Patricia Dickson P.I.

Purpose

“Immune Tolerance Improves the Efficacy of Enzyme Replacement Therapy in Canine Mucopolysaccharidosis I”

Results/Updates

http://www.jci.org/articles/view/34676/files/pdf

Location

Iowa State University, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. N. Matthew Ellinwood P.I. Iowa State University. Dr. Patricia Dickson P.I. Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Purpose

"Gene Expression Alterations in Cortical Neurons and Response to Intrathecal and Intravenous Enzyme Replacement Therapy In the Mucopolysaccharidosis I Dog”

Results/Updates

http://mps2010.eproceedings.com.au/presentations/Friday/HallG/1400%20Dickson.pdf

Location

Washington University School of Medicine St. Louis, Missouri

Investigators

Dr. Katherine P. Ponder M.D.

Purpose

“Challenges in Treating Musculoskeletal Disease in Mucopolysaccharidosis”

Results/Updates

http://www.mps2012.eu/upload/siteimages/MPS2012/S01.2.pdf

Location

University of Pennsylvania

Investigators

Dr. Mark Haskins, VMD, PhD

Purpose

“Gene Therapy for Lysosomal Storage Diseases (LSDs) in Large Animal Models”

Results/Updates

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3340575/

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Patricia Dickson, P.I.

Purpose

“Inadvertent Propagation of Factor VII Deficiency in a Canine Mucopolysaccharidosis Type I Research Breeding Colony”

Results/Updates

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2779214/

Location

Harbor UCLA Research and Education Institute

Investigators

Dr. Emil Kakkis M.D., Ph.D

Purpose

Enzyme Replacement Therapy for Mucoploysaccharidosis (MPS Type I)

Results/Updates

"Enzyme Replacement Therapy for Mucoploysaccharidosis (MPS Type I)" - PDF

Funding Recipients

The Ryan Foundation provides funding to a wide range of research institutions and facilities in the pursuit of treatments for rare disease. These include:
UCLA
UNIVERSITY OF MINNESOTA
IOWA STATE UNIVERSITY
UNIVERSITY OF PENNSYLVANIA
WASHINGTON UNIVERSITY / ST. LOUIS
UNIVERSITY OF UTAH