Leigh’s Disease is a rare neurological disorder affecting young children. Most children suffering from Leigh’s die before age three. Logan, a courageous eight-year-old little boy, has recently been diagnosed with this tragic disease. No one knows why he has not previously displayed symptoms, but since his diagnosis, the symptoms have rapidly progressed. Within the span of a few months, Logan has gone from a thriving taekwondo student, to completely wheelchair dependent. His speech has slowed dramatically. He no longer can attend school, requiring 24 hour care. His muscle tone and motor skills are failing quickly. Logan will soon lose the ability to see, to talk, and eventually breathe.
Very little research has been completed on Leigh’s disease, and even fewer drug companies have proposed any sort of treatment for mitochondrial diseases such as this. To learn Edison Pharmaceuticals has developed a treatment, EPI-743, which has already shown improvement in children in previous clinical trials, was a great blessing, and the hope Logan and his family needed. Unfortunately, shortly after learning of this treatment, the gleaming beam was extinguished due to irrelevant regulations.
To pass FDA approval, the drug manufacturer must propose criteria for their studies. One of the criterions in Edison Pharmaceutical’s application for EPI-743 treatment for Leigh’s disease requires children to be genetically confirmed to have the disease, which excludes Logan.
Logan has had confirmation through MRI. His symptoms follow those expected in genetically confirmed Leigh’s disease patients. He has undergone genetic testing and, after seven weeks of waiting, was 50% confirmed to have Leigh’s disease by a mutation on a single allele. Further genetic testing was ordered, but Logan does not have time to wait for the results. The clinical trial for EPI-743 has already begun recruiting patients. The FDA will not allow him into the study without complete genetic confirmation.
Logan is dying. It is obvious. And there is nothing to do but watch.
This petition is designed to change the criteria proposed by Edison Pharmaceuticals and approved by the FDA so patients like Logan can benefit from EPI-743 treatment. Children with such mitochondrial diseases do not have the luxury of time. Time for genetic testing. Time for placebo treatment once accepted into the study. Time for drug approval. This is a terminal disease with rapid symptom progression! Parents should be allowed the option to waive liability for the drug companies and federal government if their only other option is to watch their children deteriorate. Please help Logan celebrate his 9th birthday by signing this petition.