What can you do if someone you love is diagnosed with a disease that is so rare that drug companies won't develop a treatment because it would never turn a profit?
You might try doing what father Mark Dant did for his son, Ryan.
As Correspondent Vicki Mabrey first reported two years ago, Ryan Dant was one of only 40 babies in the U.S. born each year with a fatal, genetic condition called Mucopolysaccharidosis, or MPS 1.
Ryan's parents, Mark and Jeanne Dant, were told they could do little more than wait for their boy to die. But that was a fate the Dants were unwilling to accept. So Mark Dant went to work.
Ryan was born in 1988. From what his parents could see, he was a healthy, active toddler. Mark was on the police force and Jeanne worked in the airline industry. Together, they were building a quiet, peaceful life in the suburbs of Dallas.
In 1991, when Ryan turned 3, a physician noticed that his liver was abnormally large. The doctor told the Dants that Ryan was sick and would probably die before the age of 10.
He had Mucopolysaccharidosis-1, which means he was missing an enzyme that the body needs to break down certain waste materials. Over time, buildup of the waste material damages organs like the heart, stiffens the joints, affects breathing and stunts growth. In some cases, it can lead to severe brain damage and mental retardation.
There wasn't much research on the disease, because so few people got it. So the Dants decided to look for a cure themselves. They started the Ryan Foundation to help fund a cure. It became a community project with all their neighbors joining the fight. Their first fundraiser was a bake sale. They netted $342.
"It was a beginning, a first step," says Mark Dant.
Meanwhile, Ryan's health continued to decline. His headaches became so excruciating, he would vomit and pass out. His fingers started to curl. At 7, Ryan’s hands were too stiff to play his favorite sport, baseball. To keep him in the game, his father attached velcro to his bat. Eventually, even that didn’t work, and Ryan had to quit the team.
Mark still worked full time as both a cop and as Ryan’s dad. Every day after walking the beat, he changed his uniform to knock on doors, shake hands and raise money to give to someone -- anyone -- working on a cure.
Over three years, the Ryan Foundation graduated from bake sales to $100,000 golf tournaments with Ryan as their poster child.
“Every time I walked into a corporation or a company or a store, I would show them a picture of Ryan and I would say, 'I’m here not as a police officer, I’m here as a dad. And here is my cause.' And I’d show them a picture," says Mark. "If I went to 100 places, 99 would still say no, but one would say yes. That was a big day.”
His biggest day came in 1995, when Mark met a young scientist at UCLA named Emil Kakkis. Kakkis was one of the few researchers in the world studying MPS1. Kakkis was about to stop research because of a lack of money, so this meeting couldn't have come at a better time.
"It was very frustrating to feel that you had something that would be beneficial for a disease that wasn't currently treated, but couldn't do anything about it," says Kakkis.
Kakkis had a sense that this complicated disease had a fairly simple solution: Create a synthetic version of the enzyme that’s missing in kids who have MPS1.
The science was solid. But to take his synthetic enzyme from the lab to the bedside, Kakkis needed money. And Mark Dant knew that time was running out for his 7-year-old son, who found even the simple act of breathing unbelievably difficult. Over the next few years, the Ryan Foundation raised a million dollars and gave it all to Kakkis.
Within three years, Kakkis had attracted additional funding from a small biotech firm and had gotten FDA approval for a clinical trial of his synthetic enzyme treatment. Ten kids were chosen for the trial at Harbor/UCLA hospital in California. Ryan was one of them.
In February 1998, Ryan's neighbors gathered at Dallas/Fort Worth airport for a big sendoff. The Dants finally had something to hope for.
At the hospital, Ryan was tired of needles and doctors and feeling weaker and weaker every day. "When is this going to end?" he said. "I just want to go home because I don't like it here and I don't like to do all these tests."
Two months before Ryan’s 10th birthday, Kakkis and the Dants administered the first dose of Ryan’s enzyme treatment. Week by week, Ryan’s life improved.
Now at age 13, Ryan is back playing baseball, something his parents thought they may never see their son do again.
“It feels like I can do everything that I ever wanted to, like ride my bike every day or play baseball, basketball, you name it, I can do it,” he says.
All 10 kids showed improvement, and the results were so impressive they were published in The New England Journal of Medicine.
Three years after that first trial, Ryan still receives the enzyme treatment once a week at a Dallas hospital. And with him is Spencer Holland, whose own family is facing a similar crisis to the Dants, only three times worse.
Amy and Steve Holland have three kids, and all three suffer from MPS1. When the clinical trial started, the Hollands were told only one of their children could get the drug. They were asked to choose which one.
"How could any parent make that decision for their child?" asks Amy Holland. They left the decision to the doctors, who chose the oldest, Spencer.
Like Ryan, he has improved significantly. But for the Hollands, it's been a mixed blessing.
"We saw Spencer getting better and better and stronger and more like a normal, healthy child," says Amy. "Meanwhile, our little girls were getting sicker and sicker and weakening."
When 60 Minutes II met with the Hollands two years ago, the FDA had not yet approved the drug, and was requiring a second clinical trial with a larger sample -- one with 42 kids worldwide.
When the Hollands heard about the second clinical trial, they submitted both of their daughters names. Both of their girls were chosen, and just in time. Lanie and Maddie Holland had been deteriorating quickly. Lanie had to have brain surgery, and Maddie was losing her ability to walk.
Although the Dants and the Hollands consider this treatment nothing less than a miracle, it's a miracle with limits.
It hasn't reversed all of the damage in Ryan's hands. And doctors suspect the enzyme is not reaching the brain, which means it probably will not stop the mental deterioration typical of MPS1 victims.
"If you asked Ryan what he was going to be when he grows up, he used to always say a baseball player," says Mark Dant. "For a while, he stopped saying that. Now he says that again. He still believes he's going to be a baseball player. "
In April, the FDA finally approved the drug that saved Ryan's life. It will now be available to the hundreds of other children in the U.S. with MPS1.
The bad news: It will cost the families, or their insurance companies, more than $300,000 a year for the rest of their lives.