Chip Chick
At the time, there were no cures or treatments for Batten disease, but Julia would never give up. She started “Mila’s Miracle Foundation” to raise money to develop gene therapy for her daughter.
One year later, Boston Children’s Hospital scientist Timothy Yu saw a Facebook post talking about Mila’s condition, and he was intrigued.
Through family genome testing, Yu was able to determine her affected gene. At that time, a new drug targeting a different neurodegenerative disease had just been released.
This new drug could target the malfunctioning gene and “override” its function. So Yu thought they might be able to tailor the new drug to target Mila’s CLN7 gene, stopping it from malfunctioning.
Yu dedicated his time and massive resources, coordinating the development and production of the therapy, now called “milasen.”
He jumped through hoops and miraculously helped push milasen through the FDA’s stringent emergency use protocol. Because of Yu’s dedication, expertise, and great care, Mila received the new drug.
Although therapy was finally available, Mila’s condition took a turn for the worse in the last two months, waiting for milasen.
According to https://cen.acs.org, “She could barely walk without assistance and had become reliant on a feeding tube for food and water. Her already limited vocabulary disappeared. Worst of all, she began having seizures that took all of Vitarello’s strength to control.”
Although Mila showed improvement in the number of daily seizures she had (previously 30 per day, decreased to a few) and began eating meals by mouth, she had already lost too much to sustain life. If only they had been able to get help sooner.
She succumbed to Batten disease and sadly died on February 11th, 2021. On February 12th, Mila’s mother wrote, “yesterday, Mila’s spirit left her body. A day so trying that it will forever change me….” Mila did not die in vain.
Julia is spreading hope to other children and adults dealing with rare genetic diseases and promoting change by telling Mila’s story and raising money for continued research and development of targeted replacement gene therapies.
