Where there is a Tomorrow
While no one can be certain of what tomorrow brings, most of us have dreams and plans for the future – especially for our children. That was certainly true for me with my daughter – so I was especially touched by the story told by Mark Dant at the LA BioMed Annual Gala on December 6, 2012.
Mark and his 3-year old son, Ryan, loved playing baseball, and Mark had big dreams for Ryan’s future. He seemed a normal, healthy child, so Mark and wife Jeanne were stunned, following a routine check-up, to learn that Ryan suffers from a rare genetic disease, mucopolysaccharidosis or MPS-I. Ryan’s body lacks a crucial enzyme that breaks down large molecules in his cells, an insufficiency that invariably results in stiffened joints, damaged organs, and since there was no available treatment or cure, an end of life around age 10.
Unwilling to accept this prognosis and wanting to give their son the gift of a tomorrow, Mark and Jeanne Dant launched a mission to reverse this situation by forming the Ryan Foundation in 1992. Their first fundraiser, a bake sale, raised $342, but as they and their network of grassroots volunteers approached corporations and held golf tournaments, they raised hundreds of thousands of dollars.
Enter LA BioMed! About the same time Ryan was diagnosed, Dr. Emil Kakkis at LA BioMed was conducting research to develop an enzyme-replacement therapy for MPS-I patients. His breakthrough research was in need of additional funding to produce sufficient levels of Ryan’s missing enzyme to test in clinical trials. Through the support of the Dants’ fundraising efforts, Dr. Kakkis’ team produced the required quantities, and Ryan himself participated in the first clinical trial conducted at Harbor-UCLA Medical Center just before his 10th birthday. His condition steadily improved in the following weeks and months and Ryan’s symptoms lessened and even disappeared in the subsequent years. He is now 24, attends SMU, and—indulging his love for sports—is a member of the football team’s equipment staff.
Currently under the leadership of Dr. Patricia Dickson, the MPS Research Lab at LA BioMed continues its research using similar approaches on other forms of MPS, for which there are as yet no treatments. Dr. Dickson’s studies have become the template for clinical trials for MPS II and IIIA, as well as planned studies for other disorders caused by missing or defective enzymes. Through this and other research, LA BioMed is changing lives around the world, and bringing with it the gift of tomorrow.
Rick Learned is Treasurer of the LA BioMed Board of Directors and a member of the Friends of LA BioMed.
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