Left Hand Valley Courier - All Local, All The Time

By Vicky Dorvee
Editorial@lhvc.com 

Busting Batten Disease

 

August 24, 2017

JULIE AFFLERBAUGH

Photo by Julie Afflerbaugh Mila’s family from left are Alek Makovec, Mila, Azlan, and Julia Vitarello.

For the average cost of a home in Boulder, rare diseases affecting hundreds of children could be curtailed or eradicated. Six-year-old Mila Makovec was diagnosed in late 2016 with one of these diseases and her family is looking to our community to raise $1M to help children in the same unfathomable situation.

Diagnosing Mila’s illness was a journey unto itself. What doctors discovered years after worsening symptoms appeared, was that Mila (pronounced Mee’-la) has Batten Disease, a fatal genetic disorder that rears its head unexpectedly between ages three to five. Everything appears normal, and suddenly a child will experience visual impairment, along with walking, cognitive, behavioral problems and seizures. The speed at which Mila’s form of the disease overtakes children is alarming, with most children succumbing to it by age ten.

Mila’s mom, Julia Vitarello, doesn’t have a background in science or fundraising. But out of necessity, she’s now well-versed in the world of rare diseases and an expert at reaching out and speaking on a cause. While Batten has degenerated her daughter’s abilities – stealing her sight, speech and motor skills, Vitarello’s job as a mother has been amplified to that of a warrior in the medical foundation world.

Raising money for a cure

Because this disease, and thousands of other rare diseases (the classification for illnesses affecting less than 200,000 people in the US), impact such small percentages of the population, the federal government doesn’t fund intensive studies to find cures. This puts the onus on private foundations, such as Mila’s Miracle Foundation (www.StopBatten.org), to produce scientific breakthroughs.

Vitarello elaborated, “Most people don’t know that funding a rare disease trial is one of the best ways of advancing science. It’s a dream space for scientists and doctors given how fast the FDA allows them to make discoveries and how many diseases they can apply the technique to.” Conducting a trial within a year, as they are, is “super-fast and usually takes years,” Vitarello said.

Our community generously helped to raise the initial $1M needed in the process of finding a cure. “This story has really moved a lot of people, because they realize this could have been their child,” Vitarello says, clearly touched by the volume of encouragement the family has received since starting Mila’s Miracle Foundation.

The first round of money raised went toward the initial and successful discovery phase of mouse models and safety studies. To proceed into clinical trials, the foundation needs another $1M by the end of September, 2017. With that funding, the drug company will produce medication, and six children with Batten Disease will undergo treatment. The treatment is one dose that is expected to correct the genetic anomaly.

Even more impressive are the far-reaching applications of this trial. “Our Batten CLN7 trial allows scientists to perfect the gene therapy technique direct to the brain. This will allow them to apply the exact same technique to hundreds of other genetic diseases, bringing each one closer to a cure,” Vitarello said.

Why the urgency of the September deadline? If they don’t raise the necessary funds, the foundation “will lose its place in line,” Vitarello explained. Scientists and drug companies will move on to their next project. Time is of the essence, for Mila and others.

This week, Mila’s Miracle Foundation received news that an anonymous sponsor is offering a $100,000 matching donation. All donations given between now and Labor Day (September 4) will be matched, doubling the impact of each dollar given.

Mila’s life today

It’s been nine months since Mila’s diagnosis and the difficulties she’s faced are accumulating. Other than being able to see light, her vision is mostly gone, her vocabulary is limited to a few words, and she isn’t able to feed herself or walk without assistance. Chelsea, Mila’s helper, is with her most days and takes her to school a few times a week. Once a week, Mila goes to hippotherapy (equine therapy) to help with her sensorimotor systems. She’s still a smiling, laughing little girl, but each day is a bit different in terms of what she can and can’t do.

Mila has a three-year-old brother, Azlan, who shows no signs of the disease. But because this is a genetic disease, he has a 25 percent chance of also having Batten Disease.

**Update: Fortunately, Mila's three-year-old brother, Azlan, does NOT have Batten Disease.

How to support and donate to Mila’s Miracle Foundation

Donate and have your money matched through Labor Day by going to: http://www.gofundme.com/savingmila or http://www.stopbatten.org

Donations may also be sent to: Mila’s Miracle Foundation, PO Box 19675, Boulder, CO 80308

Vitarello welcomes suggestions from the community for ways to raise more funds and awareness to the foundation’s purpose. Please email her at Julia@StopBatten.org

For upcoming fundraising events, please go to StopBatten.org/events.

 

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