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.
When Jeanne and Mark Dant were told their son Ryan had Mucopolysaccharidos 1 (MPS I) at age 3, it was a double dose of the worst news parents could ever expect to hear. The bad news was their son now had a life expectancy of 10 years and, during those years, they could expect to see their son's health gradually decline while pain gradually increased. The worse news was not only was there no cure, there was no one even looking for one because their son's disease was so rare, there could be no profitability in a cure even if it could be found. Their only options: accept the inevitable or fight the indomitable. They chose the latter and devised a two-pronged battle plan.
Knowing whatever treatment or research that might save Ryan's life would require funding, they instantly set forth fundraising. Their first effprt, a bake sale, netted $342 and the Ryan Foundation was born. With the help of friends, neighbors and local businesses, bake sales soon gave way to golf tournaments, fishing tournaments and raffles. With awareness of their mission growing through local and national media attention, the foundation received $200,000.00 from an individual donor.
Enter the second part of the plan: find any research capable of leading to even a treatment for MPS I. Tenacity, persistence and tireless medical sleuthing led the Dants to the UCLA laboratory of Dr. Emil Kakkis. Dr. Kakkis had the beginnings of a treatment but his research was stalled due to a lack of funding. With the help of the Ryan Foundation, Dr. Kakkis and his small team of dedicated researchers continued their steady, gradual movement toward the treatment those suffering from MPS 1 were so desperately seeking.
By late 1997, Dr. Kakkis’ work caught the attention of newly forming BioMarin Pharmaceutical. Soon, a partnership was reached which allowed Kakkis to finish his research and get a promising new drug therapy into clinical trials. Ryan and nine other brave children from around the US, each suffering deeply from the effects of MPS I, volunteered to travel to the Harbor UCLA Medical Center in Torrance, California, just a few hundred yards away from Dr. Kakkis’ lab, to start taking weekly IV infusions of Dr. Kakkis’ new drug. The resulting new drug, Aldurazyme, almost immediately started reversing the progression of MPS I symptoms for Ryan and other trial participants.
In 2000, the FDA called for a second trial, and 50 more incredibly brave children from all over North America and Europe repeated what the original ten had gone thru in the previous two years. In February 2003, the Dants and several other families from both trials, traveled to Washington DC and spoke at the FDA Hearing to approve the use of Aldurazyme to treat MPS I. The panel voted unanimously to approve. Soon, children all over the world with MPS I began to improve with Aldurazyme therapy.
The race to beat the clock for Ryan was temporarily won, but Aldurazyme is not a cure. The damage caused to the central nervous system, heart valves, bones and ligaments in MPS I patients still needs to be addressed. That's why more science, more funding and more hope are needed.
Along the journey, the Ryan Foundation became aware of many more children facing other orphan diseases such as MPS I. Today, the Ryan Foundation continues to raise awareness and funding for orphan disease research affecting children and parents around the world. Much of the science already funded by the Ryan Foundation has now translated to other diseases like Hunter Syndrome, Morquio Syndrome, Maroteaux-Lamy Syndrome, to name just a few. The Foundation also works to help streamline the processes and legislation that slow the development and introduction of life-saving drugs targeting orphan diseases.
The Ryan Foundation has no paid employees, owns no property, and ensures that 100% of all net proceeds are forwarded to scientists working to treat and eventually cure orphan diseases.
In 1991, words such as "athlete", "graduation" and "career" were removed from Ryan Dant's vocabulary. That was the year he was diagnosed withMucopolysaccharidosis I (MPS I). Average lifespan of a child with MPS I: 10 years. Thanks to parents who refused to accept the terms of his predicament, the hard work friends, neighbors, and classmates put into the Foundation bearing his name, and a key partnership with an incredible team of scientists at UCLA’s LA BioMed, Ryan's lifespan has now extended beyond 25 years full of achievements way above average.
Like any child, Ryan wanted to be the best at whatever sport or activity he participated in. But as the disease strengthened its grip on his body, those dreams were replaced by the desire to just be a normal kid. When his joints stiffened to the point it made sports impossible, his father would often take long detours around playgrounds to spare young Ryan the pain of watching his playmates on the fields without him. By age 9, Ryan had stopped talking about normal childhood dreams like attending high school, obtaining his driver’s license, or even growing up. Ryan’s body had begun to tell him what his future would hold.
Through a drug therapy (Aldurazyme) developed thru a partnership of the Ryan Foundation by Dr. Emil Kakkis at Harbor UCLA (LA BioMed), Ryan regained the majority of motion lost to MPS I. Ryan was a participant in the first human trial of Aldurazyme in 1998 and has been undergoing weekly four hour IV infusions since his first infusion on February 13, 1998. OnceAldurazyme received FDA approval in 2003, Ryan’s parents began administering the infusions at home, saving many hours of hospital visits. Ryan is now the longest treated person on Aldurazyme in the world.
Ryan’s long desired normalcy was now in his grasp. It stayed with him all through high school, where he helped the Hebron Hawks varsity baseball and football coaches with their day-to-day tasks; actually getting in several baseball games and one football play during his senior year. Though Ryan struggled academically in High School, it wasn't until he began taking college level studies at Brookhaven Junior College in Dallas that MPS I once again threatened his dreams. The blood/brain barrier, the body’s natural systemwhich acts very effectively to protect the brain from many common bacterial infections, also prevents Aldurazyme from passing from the blood stream into the brain and spine. The reality that Ryan’s brain and spine had continued to store glycosaminoglycans (GAG’s), was now becoming more evident in short term memory issues. College or further academics would remain an impossibility without the ability to retain studied material.
Enter LA BioMed once again. Dr. Emil Kakkis had moved on to help several other Orphan Disease therapies become reality in the biotech world, but his successor at UCLA’s LA BioMed, Dr. Patricia Dickson, would prove to be just as dedicated. Dr. Dickson’s drive to continue working new therapies forLysosomal Disease became evident very quickly. The Ryan Foundation’s partnership with LA BioMed continued, and with the help of many friends and private benefactors, to date over $2,000,000 has been forwarded to help scientists at UCLA alone reveal treatments for MPS I. Dr. Dickson’s team, with the aid of the Foundation’s funding as well as other funding sources, was able to pioneer Intra-Thecal Therapy for MPS I, which introduces Aldurazymeto the brain via spinal tap.
The summer of 2011 saw a new trial for MPS I at the Harbor UCLA hospital, facilitated by Dr. Dickson and Neurologist Dr. Agnes Chen. Participants from all over the US began traveling to Harbor to begin the new brain and spine therapy. Dr. Dickson partnered the program with doctors at Oakland Children’s Hospital and the University of Minnesota, who would provide test results for the trial participants. The trial is on-going and the Ryan Foundation continues to cover all travel costs for the trial participants and parent for the multiple return trips to the campuses.
Trial protocols must be written with very specific patient parameters so outcomes may be measured consistently with each participant. Ryan was not able to qualify for the trial because of severe spinal stenosis, which may have blocked the drug from moving freely up the spinal column.
Refusing to quit, Ryan himself contacted the UT Southwestern Medical Center RN Sarah McNeil who worked with him when he returned to Dallas to receive his weekly IV infusions back in 1998. Ms. McNeil, still with UTSW Medical Center, was now working with Neuro-Oncologist Dr. Elizabeth Maher, who was quite adept at treating brain tumors with Intra-Thecal therapy techniques. Dr. Maher offered to take Ryan's case. Ryan completed three IT infusions in the spring of 2012 and is now on a protocol of quarterly maintenance IT infusions coupled with weekly IV infusions. His short term memory and academic performance are being measured at the University of Minnesota. Ryan continued taking two courses per semester throughout the UTSW trial. He completed his associates degree at Brookhaven Community College in December 2013.
Ryan spent one summer working in the visiting clubhouse of the Texas Rangers baseball club and five years working part-time for Coach June Jones and the SMU Mustangs football team as an Equipment Manager. Upon completing his Associates Degree, Ryan continues his academics at the University of Louisville. He is enrolled on scholarship as a student equipment manager with the University of Louisville football team.
Ryan is continuing to work his plan for a positive tomorrow and once again has a full vocabulary…and a full life ahead of him!
We are thankful for our Board members who choose to support and stand with us in the work that we are doing. For information about these individuals, hover over and click the photographs below.
A police officer for 30 plus years, Mark currently serves as the Assistant Chief of Police for the city of Carrollton, Texas. Jeanne has served as a Project Manager in the Information Technology field for nearly three decades at American Airlines, Electronic Data Systems (EDS) and at her current position as Project Manager in Applications Development with Santander Consumer USA in Dallas, Texas.
Mark and Jeanne founded the Ryan Foundation shortly after their only child, Ryan, was diagnosed with Mucopolysaccharidosis (MPS I). Mark has been the Volunteer Executive Director of the Ryan Foundation since its inception.
Mark and Jeanne have been friends since high school and married for 31 years. They currently reside in Carrollton, Texas.
Barney Adams is an entrepreneur, the founder of Adams Golf, inventor of the Tight Lies fairway wood, holder of numerous patents on golf products, and author of The WOW Factor. Mr. Adams has been supporting the mission of the Ryan Foundation since its inception in 1992. His personal guidance and commitment to the Foundation has brought national awareness to the mission of finding treatment for MPS and orphan disease.
The account of his first meeting with Ryan Dant’s father was read by millions in nationally renowned sports writer Dave Kindred’s two feature Golf Digest articles titled, “The Power of Charity Golf” and “How Golf and Charity Came Together to Save a Life”.
Greg is currently CEO of Buffet Partners LP, a restaurant company, and has been in the restaurant business for 25 years. He was previously President of La Madeleine Bakery and Cafe and spent 11 years with TGI Friday's in several senior level positions including Chief Financial Officer and Senior Vice-President of business development. Greg currently serves on the Finance Council for several non-profit organizations, including Mary Immaculate Catholic Church and Bishop Dunne High School.
Jeannie began her career 30 years ago in the banking industry and has spent the last 20 years raising her 4 children. In addition, she has her own business, A New Leaf, that provides home and office organization and landscape design services. Jeannie has served as a volunteer over the years at Mary Immaculate School, Jesuit College Preparatory and Ursuline Academy.
Jeannie and Greg have been married for 29 years and have 4 children, Brad, Chris, Brian, and Mary. They currently reside in Coppell, Texas.
Brian and Maggie started Consumer Credit Nationwide, a 501C3 non-profit credit counseling organization based in Davenport, Iowa 20 years ago. Brian is currently the CEO of Consumer Credit Nationwide and is a Certified Public Accountant. Maggie has started a new venture, Red Rover Children's Learning Center, where she is the Executive Director. In addition to the Ryan Foundation, Brian and Maggie volunteer for other non-profit organizations such as the Rotary Club and Gilda's Club.
Brian and Maggie have been volunteering with the Ryan Foundation since 1993. Brian and Maggie have been married for 26 years and have three adult children. They live in Bettendorf, Iowa.
James has been producing business and broadcast television for the past 33 years. For the past seven years, he has been the Production Manager of Broadcast Services at the American Airlines Center.
He has been producing “programs with a passion” for the Ryan Foundation as a volunteer since 1997. James has two children and lives in Coppell, Texas.
Danny has been an Account Manager for IBM in the DFW area for the last 15 years. He attended high school with the Dants in Louisville, Kentucky.
Chris was a flight attendant before becoming a stay-at-home mom. Chris works part time now handling billing for physician offices. Chris and Danny met at a Ryan Foundation event in 1998 when they started volunteering with the Ryan Foundation.
They have been married for 15 years. They have six children and reside in Coppell, TX.
Jerry has been a police officer for over 30 years and currently serves as Investigator Supervisor for the Denton County District Attorney in Denton, Texas. He has served the community as a volunteer for CASA (Court Appointed Special Advocate) representing abused and neglected children.
Tina has served in a business management capacity in the telecommunication industry as well as her current position of Senior Manager of Customer Service for TDIndustries in Dallas, Texas. She serves as a volunteer coordinator for Visiting Nurse Association’s Meals on Wheels program. Jerry and Tina have worked closely with the Ryan Foundation since its inception in 1992.
The Pomposelli’s reside in Little Elm, Texas, have been married for 34 years and have three children who are also Ryan Foundation volunteers.
Scott spent 7 years in the United States Air Force as a pilot. For the last 26 years he has been employed by Delta Air Lines and has been a Delta captain for the last 16 years. Susan was a flight attendant for Delta from 1985-1999. She currently volunteers at Jesuit college Prep and Mary Immaculate School where she also substitute teaches. Scott and Susan began volunteering for the Ryan Foundation in 1997.
The Truxal's have been married for 20 years and have one child, their son Sam. They currently reside in Coppell, Texas.
David and Lisa are both graduates of University of North Texas where they met. David has been employed by IBM for over 30 years and has held various Business and Real Estate Operations positions.
Lisa, after a career in small business, retired to stay home and raise their family. Now that their children are grown, Lisa works as a research analyst. Both have been actively involved in Mary Immaculate School, the Ryan Foundation, and Backyard Mansions, an organization that builds children's playhouses for charitable causes. Since 1997, when the first playhouse was built for a Ryan Foundation Auction, the Verrets have assisted various scholarship, family service, and medical research organizations to raise over $225,000 thru playhouse raffles and auctions.
David and Lisa became involved in the Ryan Foundation in 1997. The Verret’shave been married for 28 years and have four children: Jody, Will, Libby and Annie. All are graduates of Texas Tech University except Annie who will graduate there in 2015. The Verrets have been residents of Coppell, Texas since 1985.
Ed is Vice President, National Accounts - Institutional Funds Group, American Beacon Advisors. He is responsible for strategic relationship management for mutual fund products to national retirement plan platforms, national retirement and investment management consulting firms, large corporate plan sponsors, endowments, and foundations. Prior to that, he was with Mutual of America Life Insurance Company as Senior Vice President, Institutional Markets, Mutual of America Securities Corporation where he developed and expanded mutual fund product for Mutual of America. Ed is a member of various nonprofit organizations and currently serves on the National Easter Seals Board of Directors and the Ryan Foundation.
Pam has worked as Executive Assistant to the General Manager of the Dallas Stars Hockey Club for the last 13 years. Pam’s prior jobs include the Texas Scottish Rite Hospital, where she was office manager of the Child Development Division and Immaculate Heart Academy in New Jersey where she managed the front office of the all-girls Catholic High School. Pam is a member of various non-profit organizations and currently serves on the board of the Ryan Foundation.
Ed and Pam have been married for 37 years and have a daughter, son-in-law and two grandchildren.