No one had tried this before.  They needed to build a drug company, out of nothing and in no time. Pat Lacey and Meryl Witmer had to raise money from donations and summon a complex working enterprise, licensed and equipped to make the pills that were saving lives.  The only easy part was coming up with the company name: Kids Cure Pharmaceuticals.  It was already December. Dr. Giselle Shollerâs clinical trial would exhaust its small stockpile of the drug, DFMO, in just three months, the day February turned to March.  They worked feverishly and at all hours and told no one outside their circle what they were up to, even the parents of children now taking the drug. They worried the parents might panic and that their former supplier, a firm called CPP, might try to stop their new venture if it caught wind of it.  Pat, whose son Will had been saved by DFMO, worked the phones in Braintree, casting his net for donors to help raise the $2 million they needed to finance the company. It was vastly more than heâd ever tried to raise before.  Witmer, a New York hedge fund manager with connections and business experience, took the job of finding and assembling the building blocks of a drug manufacturing operation, while, in Grand Rapids, Mich., Sholler navigated the road to winning FDA permission to use the new pills they planned to manufacture. None of their other work would matter if the agency didnât sign off, and Sholler had warned everyone: Things do not move fast when youâre dealing with the government.  FDA permission would require a massively detailed application, and Shollerâs days were already booked tending to the ever-growing number of children with neuroblastoma in her trial. At night, she and her program manager, Genevieve Bergendahl, hammered away at the hundreds of pages of explanation and data. In between, Sholler squeezed in dinner with her own girls, who were now 9 and 12. When they had each turned 8, she said a silent thank you. Thatâs the age past which kids almost never develop neuroblastoma.  Days raced by quickly, but by the end of December, it felt like they were making real progress. It helped that they had started with an incredible turn of fortune.  There are only two companies in the world that manufacture the raw ingredients for DFMO. One of them was locked in an exclusive arrangement with CPP for the US market. The other was a European company, and when Meryl Witmer heard the name of the company she felt a jolt of excitement. I already have a business relationship with them, she said. And youâre not going to believe this. Iâm meeting them tomorrow.  She quickly struck a deal, then fronted the cash to get the raw ingredients on a ship and heading across the Atlantic. She also managed to find a Florida company that could turn the chemicals into a drug â and promised a quick turnaround when executives heard who the drug was for.  Pat had also had a run of luck, finding a wealthy donor moved by his story to write a check for $1 million. With that anonymous gift in hand, heâd gone to the fund-raisers at Shollerâs hospital in Grand Rapids and challenged them to raise enough to match it. On Christmas Eve, Sholler called him at home. The match had been met. The same week, Sholler and Bergendahl finished the FDA application and submitted it.  Much had been done in a very short time. It seemed possible, even, that they were slightly ahead of schedule, and as the New Year arrived they allowed themselves a faint, and unspoken, belief that they might make it in time.  The feeling didnât last long.  Ready to explode with frustration, Sholler hurried to her office at the hospital in the January morning cold. It had been three weeks since sheâd filed the application â three precious weeks â and there had been no real movement at the FDA.  The previous day sheâd finally had a quick call with agency officials, but she left the conversation feeling like they failed to appreciate the urgency of the situation.  If that wasnât enough, the ship carrying the chemicals they needed had arrived at the Port of Miami several weeks before, but customs had held up the shipment. Without the raw materials, they were nowhere.  And so Sholler arrived at her office at the hospital that morning ready to light a fire. She picked up her desk phone and punched in the number of the FDA project manager assigned to her application in Maryland.  The phone rang on the other end of the line. It kept ringing, then went to voice mail.  The next morning the FDA manager called back and listened while Sholler confided everything â the fight with their drug supplier and the need for speed.  To Shollerâs relief, the woman on the other end of the phone seemed to understand.  She laid out a long checklist of things that had to get done. If Sholler could deliver on her end, the case manager told her, she would do everything she could to clear a path at the FDA.  Later that day, Meryl Witmer called, ecstatic, with the news that customs in Miami had released the shipment, and a truck was already headed west across the Everglades, carrying the precious chemicals to the manufacturing facility on the Gulf Coast of Florida.  The logjam was broken at last, but the weeks theyâd lost had cost them dearly. The manufacturing company still had to run a batch of pills and submit them for time-consuming testing for purity and safety. Only when the drugs had passed that test would the FDA consider clearing the way for children in the trial to take them.  The manufacturer promised to work quickly, and a few days later, on Jan. 20, it sent photos to Witmer of the first batch of pills. The DFMO was then moved into laboratory control rooms to be exposed to varying levels of light and humidity. It would take weeks to see if the drug remained stable or whether mold or harmful microbes would grow. The testing had to be rigorous. It couldnât be hurried.  Three weeks passed, and they were still waiting on the test results when an e-mail arrived from the FDA. The agency said the application would be fully in order, once the safety test results were in. After that, the FDA would have 30 days to make a decision.  It was Feb. 12. Sholler and her team didnât have 30 days. They had 16.  It was time to think about a fallback.  Sholler and Bergendahl scoured the inventory of DFMO at each of the 29 hospitals now participating in the study, looking for surpluses that they could ship to hospitals where there were more patients than pills. That might eke out a little more time. Bergendahl lined up the special couriers and expensive packaging needed to safely ship DFMO from one part of the country to another.  Sholler checked in with her hospitalâs lawyers, who had continued to negotiate with CPP for more drugs. Talks had gone nowhere.  Pat talked about a last resort â going to the media. If it looked like they werenât going to make it, he would try to publicly shame CPP into supplying more. They were coming nearer to that point when Witmer finally got word that the testing in Florida was complete and that their pills had passed with flying colors. Sholler rushed the results to the FDA. It was Feb. 17.  Now it was all waiting and worrying. Eleven days on the calendar was just eight working days. It seemed impossible to believe that a sprawling, deliberate bureaucracy like the FDA could do anything in so short a time.  At home in Braintree, Pat checked in with Sholler and Witmer every day, and every day there was nothing to report.  He felt trapped. The winterâs record blizzards had dropped eight feet of snow. Roofs were collapsing. Schools were closed. The kids had been home almost every day for a month.  He worried over Will. The boy had outrun death for 10 years now, but was still recovering from the after-effects of a brain hemorrhage, possibly caused by his many harsh cancer treatments. He had regular appointments with a physical therapist. At the end of each session, Will would sit nervously while the therapist talked to Pat and Dina about his progress, then meekly ask the question: Hockey?  Not yet, was always the answer.  A mountain of fund-raising work awaited Pat at the office he was now renting for his charity, BeatNB, a few blocks from his house. Heâd gotten a good deal on it; it was above a store that sold tombstones. Some snowy mornings, the only way to get there was to strap on his boots and venture into the empty streets.  When he made the trek one day in late February, he arrived to see that plows had barricaded the door with a mountain of snow. He spent hours digging. Finally inside, he checked in with Sholler and Witmer again.  Bergendahl sat on her couch in the dark, staring at the TV, hoping it would silence her brain and let her sleep.  It was late on Friday the 27th. They had heard nothing, and with the deadline now upon them, Bergendahl had spent the day on the phone with Sholler and the clinical sites. The mood had been grim. First thing in the morning, she would start the process of dispensing their last pills, shuttling them to the hospitals that most needed them.  She checked the time. It was nearly 11. Bergendahl turned off the TV and told herself to go to bed. Before she did, she picked up her cellphone and saw something new in her inbox that made her sit up. It was an e-mail from their case manager at the FDA. Heart racing, she opened it and quickly dialed Sholler, who answered the phone already screaming.  âDid you see it?â Bergendahl yelled.  âYes!â Sholler yelled back. âWe did it!â  The following morning, the first batches of DFMO made by Kids Cure Pharmaceuticals left Florida, bound for hospitals around the United States. When the shipment arrived at Shollerâs clinic in Grand Rapids, the doctors and a small group of hospital staff gathered around to watch a young girl named Delaney Doyle receive the very first dose.  The toddler paid no attention, absorbed in a cartoon on her iPad, as her mother fed her a spoonful of applesauce containing a crushed up pill. Sholler snapped a picture of and sent it to Pat.  The family had no idea â no one outside Shollerâs tight circle knew â how close theyâd come.  Eight months later, on a damp and cloudy November morning, Sholler and a dozen others gathered in a small hotel conference room at the Sheraton Silver Spring in Maryland.  There was nervous energy in the room, like a study group cramming before the final exam. Soon, they would all get in cars for the short trip to the sprawling campus of the FDA.  Having won the battle to keep control of her trial, Sholler was now going to try to do something even more difficult. She aimed to persuade the FDA that a randomized trial of the kind CPP had insisted on was unnecessary. There was, she believed, a better way, a path that would keep kids from having to go through the horror of a relapse just to prove that DFMO works better than a placebo. She would rely on historical data and not on living children.  Sholler would have 10 minutes to make her presentation. It was almost time to leave.  Seated around a long table, she and her team rushed through the final preparations. Pat was there, as were Witmer and Bergendahl. A handful of parents had flown in to be there, too. Lisa Riniolo, a mother from Buffalo, had a tattoo of her late daughter, Melina, visible on her forearm. Kyle Matthews had flown up from Tampa. He lost his son Ezra to neuroblastoma and was preparing to leave his job to work full time for BeatNB. A half dozen oncologists who were offering the trial had also come to testify, including Shollerâs attending physician at Hasbro Childrenâs Hospital in Providence, where, all those years before when she was a medical resident, the death of her first patient, Tyler, had set her course in life.  Letâs go through it one final time, Sholler said, glancing at a clock on the wall.  She projected the first slide and started once more on her script, describing children taking DFMO and finding new life.  âThey are happy, playful, going to school. They do not require transfusions. They are growing and thriving. This is incredibly different from previous therapies.â  She raced through the presentation, speeding up as she got to the final slides and time ran short. Then she came to the end and a catch took hold in her throat.  âAt Helen DeVos Childrenâs Hospital,â she said, then stopped.  âAt Helen DeVos Childrenâs Hospital,â she began again, âwe have not had a neuroblastoma patient relapse in 3Â½ years.  âWe cannot go back to what it was like before.â  A hush swept around the room. Pat nodded his head. Lisa Riniolo bowed hers.  The presentation went well, but the FDA still wanted a lot more data. Sholler opened a new, nearly identical trial that February. If her results held, the FDA said it would take a second look at her proposal.  It was a decision she could live with because it meant that for now, every child who wanted DFMO would be able to get it.  In addition to the DFMO trial, she already had several new ideas in the works, including a huge project using genomic analysis to tailor medications for individual tumors. She had learned that curing this cancer wasnât one battle, but many. They were all long, and they were all hard.  She opened more test sites, including overseas â a hospital in Beirut, another in Marseille, France â and she spoke at conferences around the world.  But every day, new children were getting diagnosed. And always, there were children who did not make it.  Sometimes she reached a point when it felt like she couldnât handle it anymore. She wondered what it would be like to escape to a new life, a life where there were no children with cancer needing her help. But the thought never lasted for long. She thought often of Tyler, the first child she could not save, and of Will, the first one to live. They had lit a path in her mind, from what was to what might be.  On a recent trip to Kenya, where she was volunteering in oncology wards, she stopped for a second when she noticed her daughters in a field, playing soccer with some of the children from the hospital. She took a photo and posted it online. She called it âhappiness.â It was, she reminded herself, what she was working for.  One golden evening this summer, Pat walked out onto the front porch of his house in Braintree and sat on the steps next to Dina.  It had been so long since theyâd had a night like this, with no obligations. No fund-raisers, no appointments.  For 11 years, every day had been lived on the edge. It seemed hard to even remember what it had been like when they first moved into this house, the house where Pat grew up and where he and Dina had sat in the living room joyfully watching their baby son, imagining their future together.  This was not that future. But it was good. Maybe as good as it gets, Dina said.  From inside the house came the shrieks and muffled thunder of their three children playing. The screen door slapped open and Will, all 12 years and 5 feet of him, clomped onto the porch and down to the lawn and dragged a hockey net into the street. The light had grown soft, and they watched as Will raised his stick and began firing shots at the net.
Will Lacey was just a baby when doctors diagnosed a rare form of cancer and told his family there was only one end. Nobody then could imagine the journey ahead, from hospital rooms to board rooms, research labs to government offices, a furious race between hope and death.