mila’s miracle foundation
Mila's Miracle Foundation to Stop Batten was formed in December 2016 upon learning our daughter Mila had Batten Disease: a fatal, devastating genetic disease. We now realize that Mila’s life has a greater purpose than we had ever imagined when she was born. She isn’t just our sunshine. She is a little ray of light, a little spark, for everyone. Our hope is that her spark will ignite others, and one day soon there will be a bright light where there was once just dark.
Batten Disease is a rare neurodegenerative disease with no cure... yet. We are racing against time to change that.
WHAT IS BATTEN DISEASE?
Batten Disease is a rare neurodegenerative disease causing seizures, vision loss, cognitive impairment and early death. There is no cure... yet. Parents are forced to make excruciatingly painful decisions as the disease takes its course. Eventually, these families are left to face life without their child.
HOW COMMON IS BATTEN DISEASE?
Every year, hundreds of children are born with some variant of Batten Disease, all of which are fatal and have no cure. Worldwide, roughly 14,000 children are afflicted with this disease. Siblings also have a 25% chance of developing the disease, which makes the diagnosis all the more devastating for families as they wonder the fate of their other children.
THE SCIENCE BEHIND BATTEN
In Batten Disease, a simple gene mutation prevents cells from breaking down fats and proteins, leading to a build-up in the cells' trashcans, or lysosomes. This results in cell death and the body’s inability to function normally. The condition is autosomal recessive, meaning both parents must carry the same abnormal gene in order for their children to develop the disease. There are fourteen known variants of Batten - CLN1, CLN2, and so on - and each is unique enough to require its own research and trials. We are focused on Mila’s variant, CLN7. While great strides have been made over the past few years, there is still much to be understood about Batten as a whole.
IS THERE A TREATMENT?
There is no cure for Batten Disease... yet. However, we are working hard to start and fund new treatments that could help children with Batten and other devastating neurological disorders. Our work is for Mila and for the millions of other children like her that deserve a chance.
Our mission is to find and fund paths to a cure for Batten Disease and other devastating neurological conditions. Families in this community have been pivotal in raising the necessary funds and awareness. Our hope is that together with our incredible scientists and partner organizations, our work will give Mila and many others like her a second chance at life.
Here’s how we plan to do it…
Our foundation started an exciting new Antisense Oligonucleotide (ASO) treatment which Mila is currently undergoing. Pioneered by the incredible scientist and neurologist at Boston Children’s Hospital, Dr. Timothy Yu, this first-ever fully customized treatment targets Mila’s disease-causing mutation. See the animation below to learn more on the drug, affectionately named Milasen. We are now working to turn Mila's treatment into a brand new path to treat children across hundreds of devastating genetic diseases.
Together with renowned gene therapist Dr. Steven Gray, our foundation began the work on a new gene therapy direct to the brain for children with Mila’s type of Batten Disease (CLN7). This promising approach, which is still in the pipeline, would replace the broken gene with the corrected one in a one-time shot into the spinal fluid. Our trial is being developed by the University of Texas Southwestern in partnership with Aashi’s Hope Foundation and the Batten Hope foundation.
In addition to funding therapies, our foundation is dedicated to better understanding the basic science behind Batten Disease. Currently, we support scientists trying to crack the CLN7 gene by hosting an annual Batten CLN7 symposium with researchers and physicians from around the world. We also provide grant funding to labs working on biomarker discovery and data collection, with the hope of contributing to clinical and research-based advances in the diagnosis and treatment of Batten as a whole.
2019 Funding Opportunities
2019 Batten Symposium
mILASEN - hOW IT WORKS
WE NEED YOUR HELP NOW
Mila has lost her vision and much of her ability to walk and talk. But she is a fighter. We need your help now to give children like Mila a second chance at life.
Mila's Miracle Foundation, Inc. is a 501(c)(3) nonprofit organization. All donations are tax-deductible.