Spencer, Madison and Lanie Holland have a genetic disorder called MUCOPOLYSACHARIDOSIS, or MPS. They lack an enzyme needed to break down a protein crucial to normal growth. As a result the protein builds up in joints and organs, generally causing death by the age of ten.
"I thought about Maddie and Lanie walking down the aisle, and about Spencer going to the prom and driving a car, " says Mom Amy Holland. "It was as if all of it had been taken away from us."
Little did the Holland's know...a more agonizing scenario was still to come: In 1997 Spencer was one of ten children enrolled in a clinical trial of an experimental treatment in which the missing enzyme is replaced. At the time he was going downhill fast.
But within weeks: "I could just not believe the difference in my child and that was the day that I realized it was working," says Amy.
The Hollands expected results from the trial would win the drug swift FDA approval, but that didn't happen. "Looking at a drug in ten patients establishes safety in those ten patients," says Dr. Marlene Haffner of the US Food and Drug Administration (FDA). "We still don't know enough about a product."
Now the FDA wants a second trial. That means Madison and Lanie have to wait and become weaken while their brother Spencer who still gets the drug, thrives.
"We feel like absent doing something radical like stealing the enzyme or something," says Dad, Steve Holland. "I don't know what else I can do."
"The longer they take to make up their minds on this drug, the more my child suffers," says Amy.
"I hope your really know how hard we try to get these drugs out to patients," says Dr. Lewis Waber at University of Texas Southwestern Medical Center. "But the last thing you want is to find out that a drug is hurting people."
"What needs to be fixed here is to give these children a shot at survival," says Waber. "I think the FDA needs to loosen up their requirements for these very rare diseases."
The Hollands are now waiting, hoping to get both girls into the next clinical trial which has been delayed. "We cherish every single task and every single accomplishment that our children have," says Amy Holland.
For this family, not a day, not a minute can be taken for granted.
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