Research
Liam’s Lighthouse Foundation is ecstatic to announce the funding of its first research grant to Cincinnati Children’s Hospital Medical Center in the amount of $100,000 over the next two to three years for the research of gene therapies for Hemophagocytic Lymphohistiocytosis (HLH).
Background: Since 1999, researchers have known that mutations in a gene called PRF1 are one of the causes of HLH. This gene encodes a protein called perforin. In healthy people, perforin is used by immune cells to kill malfunctioning cells and keep them from damaging the body. Many patients with HLH have mutations in the PRF1 gene or in related genes that makes their immune systems less effective at destroying abnormal cells. In theory, HLH caused by perforin deficiency could be cured if patients could be given a working copy of the PRF1 gene. Cincinnati Children’s researchers are studying whether a modified virus can be used to transport a new copy of this gene into the cells of patients with perforin mutations. So far, this approach has shown promise in the laboratory, and groundbreaking work is now underway in laboratory mice with abnormal perforin genes. When this approach is developed for humans, it will be applied first to patients lacking readily available matched donors for transplantation. Cincinnati Children’s is a world leader in the development of potential gene therapies for immune deficiencies, cancers, and blood disorders. If they can achieve success with this study, imagine the implication for other gene mutations as well. Perforin is one of the most common gene mutations found to cause HLH. Liam’s Lighthouse Foundation will be updated annually on the progress and outcomes of this study. Liam’s Lighthouse Foundation will be traveling to Cincinnati Children’s Hospital and presenting Dr. Michael Jordan and Dr. Lisa Filipovich as well as other research team members with this donation on August 2, 2012. It’s extremely important to acknowledge that this could not have happened without the help of the entire histio community and LLF supporters who worked so hard to help us get to this point. For those who held fundraisers or a special event in their child’s name and donated the proceeds to LLF, those who ran marathons and raised money selflessly, for our sponsors, our amazing volunteers, for all who help with our annual memorial golf tournament, for those who donate through their employers, for every histio scarf purchased, for every piece of awareness jewelry bought, and for all who attend the NYC awareness event with us, THANK YOU FROM THE BOTTOM OF OUR HEARTS! We would also like to THANK Caleb’s Crusader’s Foundation, The Abigail Buckner Foundation, and Team Taylor and The Ryan Family for their generous donations and hard work to help fund this study. We are also thrilled to announce Dr. Lisa Filipovich has joined Liam’s Lighthouse Foundation as our primary scientific research advisor! Welcome to the team, Dr. Filipovich! Dr. Ken McClain of Texas Children’s Hospital and The Histio Cure Foundation have done such wonderful things to help fund research and increase awareness of Histiocytosis. We are looking forward to working with them in the near future to help fund specific research of Langerhan’s Cell Histiocytosis (LCH). Ryunosuke Satoro said: "Individually, we are one drop. Together, we are an ocean." Together, we will continue to fund research for a cure and make a difference in the world of Histiocytosis. On behalf of all of us at Liam's Lighthouse Foundation, thank you for believing in us and supporting us over the past 2 years. This is just the start of something much, much, bigger than us all. 
Sincerely,
Michelle Schulze Founder/President |
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RESEARCH UPDATE: 5.1.13
Dr. Michael Jordan, one of the primary HLH researchers at Cincinnati Children's, developed the perforin-deficient animal model that is helping advance the study of better treatmens for HLH. It was a pleasure meeting him last year when we made our reseach donation. I was so thrilled to receive a copy of Research Horizons, Cincinnati Children's Research Foundation's publication to showcase the researchers and their work! Please take a moment to see what's going on with HLH research and to see what the experts are saying. "For patients that have no perforin function, there is no way to fix it apart from a bone marrow transplant, which has significant risks. If this is successful, we could fix the perforin deficiency with gene therapy, replacing it in their own cells." - Michael Jordan, M.D.
