Dry cleaner's fight against sons' rare disease could lead to other life-saving treatments

July 21, 2008

Phil Milto sat across the table from a wealthy local businessman in disbelief. He'd asked the man to help fund medical research to find a cure for the rare disease that was killing his eldest son, Nathan--only to be told that not enough people were affected to merit a contribution.

Milto, an Indianapolis dry cleaner, thanked the man for his time and moved on.

Luckily for countless people with diseases ranging from Parkinson's to Alzheimer's, he didn't let the rejection derail his efforts.

Years later, the Milto-led Nathan's Battle Foundation has evolved into what he calls a not-for-profit biotech company that has raised money and guided research that resulted in a promising treatment for Batten disease. Now, some of the gene therapy techniques researchers developed are being applied to other disorders.

"The message I kept trying to tell people all along was that we hoped to develop a treatment that could be used for a broad spectrum of diseases," Milto said. "Now we see this could have a worldwide impact."

One of the nation's leading medical journals recently published the results of the successful foundation-funded treatment trial, and the U.S. Food and Drug Administration has approved a second round of testing. Milto, 40, is hopeful that within five to 10 years, the treatment will be available to everyone diagnosed with Batten disease--about 500 to 1,000 people worldwide.

Batten disease is a neurological disorder that robs children of brain function as it progresses, eventually leading to loss of sight, speech, hearing and motor skills. The treatment developed with funding from Milto's foundation involves brain surgery to inject a gene that will create the enzyme patients lack.

A group in France also is using the Batten treatment platform to develop therapies for other life-threatening infantile and childhood disorders. And a Parkinson's disease trial in the United States used the same gene therapy delivery methods and techniques.

Credit Milto's relentless drive for a cure--and his use of business skills rarely seen in the medical and research fields. Milto, a partner in locally based Milto Cleaners, has raised $5.5 million since the foundation was founded in 1999. He also has guided the research by brokering deals with the likes of Cornell University, Rutgers University and Boston-based biotech firm Genzyme Corp.

"For someone with no medical, research or pharmaceutical background to navigate this path is almost unbelievable," said Craig Svensson, dean of Purdue University's College of Pharmacy, Nursing and Health Sciences. "Getting this far through the FDA, especially with an invasive procedure like this one, is extremely complex, not to mention expensive. It's a Herculean effort."

Giovanna Spinella, a consultant for the office of rare diseases at the Bethesda, Md.-based National Institutes of Health, said she has never before seen such a "single-handed effort."

"It's scary to think where we'd be without Phil and Nathan's Battle Foundation," said Susan Balog, a Pittsburgh mother of a child afflicted with Batten disease. "Without the Miltos' efforts, we'd have nothing."

Business, meet science

Perhaps one of Milto's most challenging tasks was convincing people that donations were not about merely helping his family.

"This wasn't a sick kid story where you gave money to a cause with no understanding of the return," said Milto, a 1990 Indiana University business school graduate. "Every donor or investor in our foundation has received a return on their investment with the delivery of therapies to children. And we think that return could become much broader."

Before Milto's efforts began, the usual life span for Batten disease patients was 12 years. Now there is hope for more. Though his health is still tenuous, Nathan recently turned 14. P.J., another of Milto's sons who has been diagnosed with the disease, is 11.

Milto and his wife, Tricia, also have two healthy sons, Nicholas, 12, and Joey, 4.

Normally, scientists who want to research a disease ask foundations and governmental agencies for money. Milto's foundation, however, raised the money first, then pleaded with researchers to help. Milto also brought together biotech companies and researchers interested in finding broader applications for the work.

"Most scientists have no idea how to move basic research from the bench to the clinic, so many discoveries are left in the lab," he said.

Nathan was the second child treated with the new gene therapy transfer at Cornell in June 2004. P.J. underwent the brain surgery needed for the treatment one month later.

Nine of the 10 patients that participated in the first trial are still alive, although the damage caused by the neurological disease cannot be reversed. If the treatment is applied before the disease progresses, researchers hope to stop it before the damage occurs.

This summer, highly respected trade publication Gene Therapy Medical Journal published the results of the first clinical trials, reporting that the treatment significantly slowed or stopped the disease.

When asked about the progress, Milto takes a deep breath and exhales slowly.

"It's been a long journey," he said. "We had a lot of supporters along the way."

He hasn't forgotten the naysayers he's met along the way, either.

"We want to thank everyone who has been there for us and thank those who have told us that we couldn't achieve our goals, which continued to drive us to be successful," Milto said.

Milto isn't one to get over-emotional, at least not with strangers or newspaper reporters. But the toll of the journey is obvious in his tone. He knows there are more miles to travel to realize the full benefit of this sojourn. He sounds both like someone ready to climb the next mountain and someone ready to pass the torch.

He realizes, too, that time with two of his sons--despite the treatment--still may be short.

"Nathan is still a very sick boy," Milto said.

More money needed

Nathan's Battle Foundation still owes Cornell $176,878 for the first round of the trials, and another $2.1 million will be needed for the second round.

With research funded by NBF, Cornell officials think they've found an even better way to distribute treatment more thoroughly through the brain. Milto is hopeful this round of trials could lead to the treatment of younger Batten children, who could go on to lead "normal" lives.

"Hopefully, this is the beginning of the end of this disease," Milto said.

Milto's fund-raising efforts continue next month, with the Eighth Annual Nathan's Battle Foundation Invitational golf outing Aug. 28 at Dye's Walk Country Club in Greenwood.

"Phil and his foundation's efforts have been non-stop for a decade," said Balog, the Pittsburgh mother.

Milto insists he's not looking for recognition for himself, just the cause.

"The only title I ever wanted was 'Dad,'" he said.

It seems like a very long time ago that Milto and his supporters from the city's south side held their first fund-raiser in August 1999. The golf outing raised $14,000.

In 1997, 3-year-old Nathan suddenly started to lose his balance and had vision problems. In 1999, he was diagnosed with Batten disease.

"Our doctors told us to go home and enjoy the time we had with him," Milto said.

He refused to heed their advice.

Instead, he wrote out a business plan, the initial copy on the back of a napkin. Then he refined it and put it to work.

"For my wife and I, our kids are the most important things to us," Milto said. "Our job is to try to make their lives better, in ways big or small, whether it's tucking them in safely at night or developing a new drug. That's what we've dedicated our lives to."

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