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When Good Science and Good Medicine Aren't the Same Thing

Usually, efforts to find a new treatment or cure for a disease go something like this: scientists spend years figuring out something about the basic biology of the disease, and then spend more years finding ways to target a part of that process. When they hit on something that works in the laboratory, they (or, often, a pharmaceutical company) spend even more years - and a lot more money - doing the tests necessary to prove a drug is safe and effective for use in human patients.

When your two year old son has an incurable form of cancer, you don't have that kind of time. That's the situation Pat Lacey found himself in ten years ago. 

Neuroblastoma is a cancer that primarily affects young children. It’s the most common form of cancer in infants. For low- to moderate- risk patients, the survival rate is greater than 90%. But for those whose neuroblastoma recurs, it is almost always fatal. At least, it used to be.

Will Lacey was diagnosed with neuroblastoma when he was six months old. By the time he was two, doctors had exhausted their repertoire of treatments, and the cancer was coming back. Today, Will is a healthy twelve year old, quite possibly the first person to have survived relapsed neuroblastoma.

Will's father, Pat, has had a lot to do with that. He is the one who found the tumor, and the one who took Will to Philadelphia for what Lacey describes as a "barbaric" radiation treatment that made the toddler so radioactive he had to be isolated in a lead-lined room for days.

"That was the best option available. That was what we had to do," said Lacey. "So we went ahead and did it."

Then, the Lacey's began a search for something better. 

"We began a search of neuroblastoma expert in the world," recalled Lacey. "We visited people across the country, I spoke on phone calls, I read papers. And, in that journey, the idea was to find someone - anyone - who had an idea. Someone who had something we could fund, something we could raise money for, and something we could get behind."

That's how Pat Lacey found Dr. Giselle Sholler. Sholler shared his vision that neuroblastoma didn't have to be incurable, just because it always had been. Lacey recalls another parent who called Sholler's clinic "the last-chance hotel."

"When you look at the situation in the medical culture of the society, no one is ultimately tasked with the responsibility of saving these kids' lives," said Lacey. "Everyone shares that goal. Everyone wants that to happen. But who is ultimately responsible for that? Sadly, what we discovered is no one. Sadly, what we discovered is, in order to get cures to kids who desperately needed them today, that something that we needed to do. We needed to lead."

Over the past ten years, Lacey and Sholler have repeatedly found ways to shorten the process of identifying and testing potentially life-saving treatments for neuroblastoma. Often, those are drugs that have already gone through safety testing and are approved for other purposes, but may work for neuroblastoma. The most notable example is a drug called DFMO, which Lacey credits with saving his own child and hundreds of others.

Lacey founded the non-profit Beat Neuroblastoma, and has raised an estimated five million dollars to support Sholler's work. He even helped create a new drug company to produce DFMO when the only other supplier refused to sell the drug to Sholler for her research.

Today, Sholler and Lacey are trying to convince the FDA that they don’t need to do traditional clinical trials to prove DFMO works. In fact, they say, it would be unethical to do so. That's because a randomized, controlled study would mean withholding DFMO from half the study participants.

"Perfect science would be a controlled study. Perfect science would be having one hundred kids enrolled in drug, and one hundred kids not, and then counting up the pile of dead kids," he said. "That's how you dictate which one works, in a perfect world. That's great science. It's terrible medicine."

Instead, Sholler is making the case that she should be allowed to use past patients who did not receive DFMO as virtual controls, rather than knowingly sentencing children to death in the name of science. It's an argument that Ebola researchers have also used.

"It does seem to be a growing question that people are asking," said Billy Baker, who has written this story in a five-part series for the Boston Globe, called The Power of Will. "People like Dr. Scholler are forcing that question to come up."

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