BOULDER, Colo. (CBS4) – A mother in Boulder didn’t give up when her daughter was diagnosed with a terminal disease. Because of her determination, her daughter could change the future of medicine.
Julia Vitarello’s daughter, Mila Makovec, was diagnosed with a rare form of Batten Disease three years ago.
“They lose their ability to see, they lose their ability to communicate eventually. To walk, and control their muscles. They also develop seizures and ultimately it’s a fatal condition,” said Dr. Scott Demarest, an Assistant Professor of Pediatric Neurology at Children’s Hospital Colorado.
Mila was 6 years old when she was diagnosed, and at the time there was no treatment. But Mila’s mother refused to give up.
“I told myself I don’t know how I’m going to do it, but I’m going to try and raise money and I’m going to try and put the right scientists together. I’m going to do everything I can to say ‘I’ve given Mila the very best shot possible,’” Vitarello told CBS4’s Dominic Garcia.
She researched, fundraised and reached out to person after person. She finally met Dr. Timothy Yu at Boston Children’s Hospital. Remarkably, he was able to develop a treatment in less than a year.
It was named Milasen after Mila. It’s the first time the FDA has approved a drug for one person. The drug has slowed some symptoms and completely stopped others.
“The fact that this happened within a year is absolutely astounding, and that it’s just for one person. This is a medicine that’s designed in such a way that, to our knowledge, there would be no other person on the planet that would benefit from this particular drug,” said Dr. Demarest.
Mila is now being treated back at Children’s Hospital Colorado. While her symptoms have gotten better, her future is still unclear.
“I don’t know what’s going to happen with Mila. I’m reminded every single day how lucky we were that the stars aligned for Mila in so many different ways and she was allowed to become the first person to receive a drug tailored to one person,” said Vitarello.
The hope is Mila’s case opens the door for more children to receive treatment, no matter how rare their disease.
“The fact that this is possible tells us we have something to aim for and that there’s a route we can now go for to be able to make this more accessible for more people,” said Dr. Demarest.
Julia Viterello has started Mila’s Miracle Foundation. They are currently raising funds to establish a team at Children’s Hospital Colorado that can treat kids with a number of rare diseases.