It’s a harrowing tale of how one young Irish American cancer patient and his family defied extraordinary odds; 12-year-old Will Lacey of Braintree, Massachusetts, defied them in his battle to live and his father defied them again in his fight to take on the powerful pharmaceutical industry.
Will was only six months old when his parents noticed an unusual swelling. It turned out to be a tumor the size of a baseball in his chest and was a diagnosed as neuroblastoma (NB), a disease that affects roughly one in every 7,000 children and is responsible for 15% of all child cancer deaths. The survival rate once diagnosed is minimal.
Boston Globe reporter Billy Baker spent years with the Lacey family, following their unique story to keep Will alive, and learned that in the early day’s of Will’s diagnosis that the prognosis for the disease was not hopeful; the word “incurable” was used by child oncologists who had seen time and time again the aggressive cancer overpower and then extinguish the lives of their young patients.
But Will’s parents refused to let the disease claim their son’s life and the battle to find a cure would come to dominate their waking moments. In this they were joined by Dr. Giselle Sholler who had spent years researching neuroblastoma.
Desperate, they started a last ditch attempt to keep Will alive, trialing the treatment of a drug called DFMO which had been used on a neuroblastoma patient to treat a different illness but had a startlingly positive effect on her cancer too. The trial would cost hundreds of thousands of dollars, much more that Pat Lacey’s small BeatNB charity had had ever raised, but no matter.
Pat was a perennial fundraiser: for years he had sold raffle tickets, “Cure Me I’m Irish” t-shirts and thrown an annual bash on St. Patrick’s Day but now he needed to exceed all his previous fundraising records. He sold tickets like a madman, far more than in previous years, all he needed was his guests to turn up.
Mercifully, it seemed like half of Boston was there and the next day Pat sent Dr. Sholler the final $80,000 she needed to get the trial off the ground.
Will began his treatment in May, he took DFMO twice a day and despite the new treatment remained in surprisingly good form - previously any drugs he’d been prescribed had left him sick, a sign doctors had said that they were working.
A month later the family drove back to Vermont where Sholler had her practice. Will underwent a routine scan and the Laceys returned home. A few days later Sholler sent them an e-mail which stunned them: “The previously described left apical/supraclavicular mass has diminished in size.”
Will’s tumor was getting smaller. And it wasn’t just his tumor, the scans of other children enrolled in the trial showed similar reductions. It was a result that sent shock waves through the highly interconnected community of families with children suffering from neuroblastoma.
It was not a miracle drug – 14 children also on the trial had not responded to DFMO and passed away – but it was a crack of light in the fight against a disease that had long been labeled incurable.
A second trial was launched: whilst initially Dr Sholler had hoped that the drug had proved itself a cure for neuroblastoma now she thought it worked to stop children in remission from relapsing.
She moved to Grand Rapids where facilities were better and remained in regular contact with Pat, who spent his time touring the country, raising funds and speaking at specialist medical conferences.
Candid of Dr. Sholler, Will Lacey, and Patrick Lacey! #beatnb #cureme pic.twitter.com/ZVjlt1v14F
— Beat NB (OLD) (@beatnb_OLD_) March 8, 2015
The first year of the study showed cause for optimism: the first ten of patients all made it to 12 months still in remission and Dr. Scholler moved to double the size of the study to 160 children.
It looked like hope was on hand for sufferers, where once there had been none.
But Sholler’s application for the program to be expanded ran into an unexpected problem; Cancer Prevention Pharmaceuticals (CPP) had originally donated the drug to the trial in exchange for her data but the organization was in trouble and informed Scholler that, regrettably, they were in no position financially to contribute anything and instead sent her a bill for $300,000.
Pat dutifully set about raising the requisite funds but a few weeks later received a call from a fuming Sholler: CPP wanted to axe the trial’s expansion and instead move to a federally approved Food and Drug Administration (FDA) random trial.
Doing so would involve half the patients in the study being given DFMO, the other a placebo instead. Such a trial would fast-track the drug for approval nationwide but would come too late for children with the cancer in the meantime.
A bitter feud erupted between Pat, Sholler and the CPP. With stocks low on the life-saving drug and with the CPP refusing to back down it seemed like they had run out of options.
The only possible solution was to found their own drug company and manufacture the medicine themselves, a daunting task in the 106 days they had left until their supply of DFMO ran out.
Undeterred, they set about raising the eye-watering sum of $2 million they needed as covertly as possible, convinced that if CPP realized what they were up to they would be stopped before the project got off the ground.
Whilst Pat raised the cash, Sholler cared for her patients and worked her way through the tortuously detailed application required before the FDA would authorize the use of the drug from a new source.
It seemed like an impossible task but they had two big strokes of luck: first they received a donation for $1 million from an anonymous stranger – approximately half the sum needed. Secondly, only two companies in the word produced the raw materials needed to make DFMO, one dealt solely with CPP but the other was a European company who to their relief eagerly organized a shipment to Florida and the application to the FDA was submitted.
Bureaucracy swung into action and the painstaking process of gaining official approval began.
Weeks passed before an e-mail pinged into Sholler’s inbox: the drugs had been made and so long as the safety tests worked accordingly, approval for use by the FDA should follow within 30 days. That would be just after their stocks of DFMO were due to run out.
Pat suggested an eleventh-hour media campaign to shame CPP into providing stocks in the meantime.
Five days later Sholler received a further update: the drug had passed its tests. The results could now be submitted to the FDA for approval, who would then authorize distribution.
That was eight working days until their supplies of DFMO ran out. Would the FDA approve it in time?
The deadline approaching, an ominous silence reigned. It was not until 11pm the night before that Sholler received any kind of indication as to the FDA’s intentions.
The drug had been approved; tomorrow it would be dispatched to hospitals across the lands. She rang Pat, ecstatic with the news.
Today Will is 12 years old, still in remission and plays hockey. His life – for so long tumultuous – now resembles those of other boys on the cusp of teenage-hood. Now thousands of families now have new hope that a treatment for the deadliest form of childhood cancer can be made work.
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