Her name is Ellie.  She is a sweet and outgoing eight-year old girl who serves as nature’s own anti-depressant to family and friends, as well as to the many doctors, nurses and researchers who’ve come to know her at the Kennedy Krieger Institute in Baltimore, Maryland.  What began as noticeable balance issues and leg spasms when she walked at age three was later diagnosed as Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and Lactate Elevation (LBSL), a very rare and progressive genetic disorder that affects the brain and spinal cord; most often developing in children, affecting their ability to walk, then even to stand and coordinate fine and gross motor skills.    

LBSL falls within the also rare Mitochondrial Disease family.  But Ellie is not alone – through her website, which describes the disease and Ellie’s journey (A Cure for Ellie) she has attracted interest from families in Italy, New Zealand, Canada, Russia and across the United States who are also searching for a cure for their children.

While some may see Ellie’s future as bleak, her family and friends are full of hope. Currently, Ellie is given an antioxidant mix of medicines – but there are no definitive long-term therapies.  Dr. Ali Fatemi is launching a research project at Kennedy Krieger Institute which will provide a roadmap towards nanotherapy for LBSL.  The outcomes of the research will become a very powerful tool for testing new effective medications.  


There are four milestones within the project which require funding. The research within each of the milestones will take place over the next several years. Year one of "Milestone 1" required $60,000, which was met in October 2015 and allowed the project to begin. The second phase of "Milestone 1" requires $231,000 and funds raised to date have allowed our researchers to continue with increasingly important studies and testing over the past 18 months to position us for "Milestone 2", which will require just over $1,000,000. We are excited and encouraged by the progress that has been made and know this will be a "marathon" effort - not a sprint. With your help, together we can save Ellie and the children like her across the globe - to help them walk and keep them strong for years to come.