The Power of Will
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.Written by Billy Baker Illustrated by Tonia Cowan
Chapter 1 of 5
They sat in a windowless hospital meeting room around a small round table, empty but for a box of tissues: Giselle Sholler, a new doctor just a few months into an oncology fellowship, and two young boys.
The boys were the older brothers of Sholler’s first cancer patient, a 5-year-old named Tyler, who was battling a vicious childhood cancer called neuroblastoma. The disease had gone into remission, but now it was back. And when children with neuroblastoma relapsed, they died. All of them.
The brothers, ages 9 and 13, had asked to speak to her, but at first they could only cry.
Why can’t you cure Tyler? Why aren’t there any other medicines?
Sholler swallowed hard to hold back the unsteadiness coming to her voice and gave the only response there was to give, that there was no cure for what their brother had.
After leaving the boys with a social worker, Sholler walked in a hollow daze through the ward at Rhode Island’s Hasbro Children’s Hospital, looking for a place to be alone. She found an empty exam room, went inside and broke down.
Sholler hated the answer she had given because it was no answer at all. In a matter of hours or days Tyler would die. Other children would die, too, if there was no new way to treat them. There had to be an answer out there, she told herself, and she would try to find it.
One day there would be a child she could heal.
Dina Lacey looked down at her baby boy on the rug, and at her husband sitting on the couch, and couldn’t help herself.
“He inherited your fat neck,” she said to Pat, who looked at Will and laughed, partly because it was true, and partly because it was great that he had married the kind of woman who would make jokes about his fat neck.
Pat is a big guy, not imposing, but more the kind who volunteers to carry the cooler. He was never a troublemaker or a rebel, just someone who was perfectly happy to be getting along.
He’d had an ordinary suburban childhood in Braintree and then earned a degree in sociology from the University of Massachusetts Amherst, where he specialized in making jokes about majoring in sociology. He was never accused of being an overachiever, and if asked what he wanted to do with himself, he would say something like: land a decent job, get married, have kids, and pack the car for the Cape every now and then.
That’s just what he did. He got a job at a financial firm, where he met Dina, a tall, dark-haired co-worker, and won her over with an invitation to the Flag Day party he threw every year because nobody throws parties on Flag Day.
Will was born in late August of 2004, a year after they got married. They were still basking in the soft glow of a couple becoming a family when Pat’s parents offered to sell them the house in Braintree where Pat grew up. It was sweet to think about a new Lacey family starting off in that old green house on the quiet street where kids can ride bikes and play street hockey, just like he did.
It was just after Christmas and they were still getting settled in the new house when Dina made the crack about Will’s neck. He had been a big baby, 10 pounds plus, and now he was a big 5-month-old. Pat laughed as he picked Will up from the rug and began to massage the rolls on his neck.
Pat felt the lump and the sick feeling in his gut almost simultaneously.
It was about the size of a grape, in the hollow near Will’s left collarbone and when Pat squeezed it between his fingers, it felt like it was stuffed with Jell-O. He knew that nothing on a baby’s body should ever feel like that.
Dina called the pediatrician. Pat went to his computer, Will on his lap, and began to search. He figured out the lump must be in the lymph node that drained from Will’s chest, and he kept reading that it could be a sign of a malignancy. Each time Pat read that, he dismissed it. The baby in his arms was just 5 months old; surely, infants don’t get cancer.
They saw Will’s pediatrician, who sent them to a surgeon who wasn’t too concerned. Will had had a bruising birth because he was so large. He had emerged with a lump on his head. It probably had something to do with that, the surgeon said. It would work itself out. Come back in a year.
Pat heard what he wanted to hear, but Dina wasn’t sold. She thought Will had a slight droop in his left eye, and several weeks later, while visiting her family in Rhode Island, she thought his left arm looked swollen. She found a tape measure, which confirmed her suspicion. She took Will back to their pediatrician, who told them to have Will’s chest X-rayed just to be sure.
That was on a Friday. They could have had the test done that day, but Pat asked if it could wait until Monday. Let’s have a regular weekend together as a family, he told Dina. For a few more moments, let’s just not know.
On Monday, the X-ray at Boston Children’s Hospital revealed a mass the size of a baseball in their baby’s chest. They met their oncologist. They heard the name of Will’s cancer — neuroblastoma. Everything else was a blur.
Will was admitted and put in a room with a boy who had leukemia. The next morning, as Will’s crib was pushed to an elevator, they walked through the ward, passing pale children with bald heads, and when the elevator doors closed, Dina came apart. “Is that what’s going to happen?” she cried. “Is Will going to die?”
There was no answer to that question. It hovered over everything in the days and weeks that came, as they took their child and terrors home. The first treatment was to be eight weeklong rounds of chemotherapy, one round every three weeks.
The toll on all of them was brutal — the many trips to the Jimmy Fund Clinic at Dana Farber, standing by while tubes were inserted into Will’s tiny body, the hours among the clinic’s cheerful colors and sick children. At home, Will got violently ill. Pat and Dina struggled to keep his weight up. They lay awake all hours of the night, and when sleep did come, Dina often woke in a panic and stole quietly into Will’s room to listen for the sound of his breath.
Dina focused her energy on taking care of Will. Pat focused on how to beat the cancer. He spent hours on the Internet, reading everything he could. He dove deep into the world of personal blogs written by other neuroblastoma parents. Dina could not stand to look at the stuff; Pat could not look away.
He started his own blog and wrote almost daily accounts of Will’s treatment. Months turned into a year. The tumor, which had initially shrunk under chemotherapy, wasn’t responding anymore. Surgeons removed what they could and started a new round of high-dose chemotherapy — as close to lethal as they dared.
For Will’s second birthday, at the end of August, Pat and Dina brought their boy a cake decorated with M&Ms. Two weeks later, they sat with the head of pediatric cancer at the Dana-Farber Cancer Institute.
I’m sorry, she said.
There were new spots on Will’s scan. All known curative therapies had failed. They should think about how to best use the time they had left.
They drove home to Braintree in silence. Pat’s parents took Will inside the house, and Pat and Dina went for a walk around the neighborhood.
Dina, inconsolable, raged.
I am a mother, she shouted. And I am going to remain a mother. No one is going to take my child away from me.
After Tyler’s death, Sholler started the research phase of her fellowship at Brown, determined to tackle neuroblastoma. She read all the scientific literature she could find and treated more patients, but she still didn’t know where to start. Then one day at the hospital, entirely by accident, she stumbled on something.
A tainted blood transfusion had infected a relapsed neuroblastoma patient with Chagas Disease, a dangerous parasite of the tropics that, if untreated, could eventually destroy the girl’s heart. She was given a drug called nifurtimox, and, as the girl’s fever subsided, Sholler noticed a strange side effect: Her cancer began to look better. They gave her more of the drug, and her cancer went into remission.
Sholler published the results, and parents of children with relapsed neuroblastoma immediately contacted her. She won FDA permission to give nifurtimox to a half-dozen children under a “compassionate use” designation, which allows unproven therapies to be administered in a few desperate cases. But she was told that was all she could do. If she wanted to treat more patients with the drug, she would have to open a proper clinical trial.
She finished her fellowship and moved to Burlington, Vt. — not too far from where she’d grown up in Montreal — and began work at the University of Vermont Children’s Hospital. There, she was offered the chance to have both a lab and a clinical practice. The hospital also was willing to let her open a nifurtimox trial. By the end of 2006, her tiny clinic became a beacon in the childhood cancer world, the Last Chance Hotel for children with relapsed neuroblastoma.
Soon, it would become clear to Sholler that nifurtimox was not the wonder she hoped it might be. If patients got better, it proved only temporary. But the experience convinced her of something — that the key to this puzzle might lie with drugs already in use for other diseases, drugs never considered for neuroblastoma.
Pat was full of fight with nothing to swing at. He had learned everything he could about relapsed neuroblastoma except how to beat it. During Will’s 20 months of treatment, Pat had watched parents make their children sick with experimental treatments and others who accepted the inevitable.
There was no clear answer, no right or wrong way. All paths led to the same end.
Pat watched Will toddling around the house, finally free of chemo. He tried to be at peace, to savor these moments, but he could not — not when he knew the cancer would eventually take their son if they did nothing.
He scoured medical literature and talked to doctors. There was one option that Pat and Dina had been afraid to try. It was a treatment in Philadelphia that would literally make Will radioactive. It held no promise of curing him. At best, it offered him a one-in-three chance of living a little longer. At least it was something.
Pat made an appointment.
As soon as he walked into the room at Children’s Hospital of Philadelphia, Pat hated everything about it. The walls were lined with lead. Everything was covered in plastic.
But it was what you couldn’t see that made the room truly scary. The nurses clearly wanted nothing to do with the place. When they did step inside, they hustled out as quickly as possible.
They had reason. Will had just received an infusion of radioactive material so powerful it would be seven days before it was safe to be around him. Pat was supposed to go into the room only for brief periods and to keep a constant eye on the dosimeter clipped to his collar. Dina was back in Braintree because she was pregnant. It would be weeks before she could go near her son.
Pat sat in the “clean corner” of the room, directed to stay behind a lead shield that ran along the bed and blocked his view of Will.
As he talked, trying to soothe the boy, the effects of the infusion started to hit. He heard Will vomit.
To hell with the rules. Pat heaved his shoulder against the shield, so heavy it took all his weight to move. He reached Will and tried to clean and comfort him as the boy sobbed.
What had he done, Pat thought. Was he doing this for Will or for himself?
They returned to Braintree and waited. The treatment appeared to work. The tumor remained stable. There was no telling what else that much radioactivity had done to his son. The long-term side effects were potentially horrendous, though the medical expectation was that it wouldn’t matter; Will would not live long enough to find out.
One day at home, Pat received a phone call that was so perfectly absurd he laughed. It was from their trash collectors, informing him that they would no longer be serving the Laceys’ address.
At the transfer station, Geiger counters had gone crazy, and some poor guy had to dig through the pile to trace the source of the radioactivity — Will’s diapers. The truck had to be quarantined for four days.
Pulling into the driveway one evening in May, Pat heard Will shout across the yard.
The boy, pursued by their dog, Molly, ran from the porch where they had been waiting with Dina for Pat to arrive home from work.
In the two months since Philadelphia, Will had recovered from the effects of the treatment and, for all outward appearances, was a 2-year-old like any other.
Pat and Dina had been having hard conversations about which course to follow now. Parents who kept trying to save their children often crossed a line into a world of untested treatments almost certain to do nothing but cause more suffering. It was a line Pat and Dina could choose to cross or not. Pat saw a lifetime of self-doubt and anger if they made the wrong choice. He didn’t want his son to suffer while he and Dina chased false hope.
But in the end, their choice was clear: They would keep trying. Pat refused to think that the hope they were chasing was false. He called it “indefensible hope,” a phrase he picked up from a John Updike story.
“There will always lurk, around a corner in a pocket of our knowledge of the odds, an indefensible hope,” the story said, “when a density of expectation hangs in the air and plucks an event out of the future.”
A few months later, in the fall of 2007, Dr. Sholler journeyed from Burlington to Manhattan for a meeting organized by a cancer parent who was frustrated with the pace of research. Sholler and some other oncologists had been invited to speak. She already knew many of the people in the conference room, including some parents who were raising money for her work. But there was one face she didn’t recognize, a guy from Boston named Pat Lacey.
Pat had read about her research. There was some buzz about her, not all of it positive. But he took to her immediately as she gave her presentation. He could spot an impatient optimist.
When the speeches were over, Pat asked another parent to introduce him. He and Sholler spoke for a few minutes. He told her about Will, that he had relapsed, that he was incurable. Sholler stopped him.
For now, she corrected him. He is incurable for now.
Pat paused. Sholler went on.
These kids aren’t going to be incurable forever, she said. Why can’t it stop now? Someone has to be the first. Why not Will?
For months, ever since Will had finished his second trip to the lead-lined room in Philadelphia, Pat had talked to every neuroblastoma expert he could find, and every one of them had told him they were very sorry but there was nothing to be done. Now this woman was standing in front of him saying exactly what he wanted to hear a doctor say.
He knew what she was offering was dangerous.
Hope. Indefensible hope.
And yet, why not Will?
Chapter 1Will Lacey: A life in photos
Chapter 2 of 5
Darkness and the dream
Pat Lacey walked into the San Diego hotel conference room and looked for a seat among the others already at the table. He was in a great mood for the first time in months, and so, it seemed, was everyone else.
At home, they all had children battling a rare form of cancer called neuroblastoma and doctors who had told them it couldn’t be beat. But in this room there was a sense of hope. At the head of the table was the source of it, Dr. Giselle Sholler, an oncologist encouraging them to think not about death but about the possibility of a cure.
She was one of the only doctors anywhere running a drug trial for relapsed neuroblastoma. But it appeared the drug she was testing, nifurtimox, would only delay, not defeat, the disease. She needed new ideas.
It was April 2008, a few months after Pat’s first, transforming encounter with Sholler in New York. Now, the doctor was attending a cancer conference in San Diego and had suggested that everyone meet there to plan the next phase of the attack. She was already researching a few other potential drugs in her lab, but she was on the hunt for more. She wanted their input. Some of the parents came with their own leads on possible treatments. They also had to talk about money. Funding was scarce, and drug trials are expensive. And there was no time to go through the normal research grant cycle, which could take years. This handful of parents would have to raise all the money themselves.
They had been hashing out ideas for two days now and had identified some with promise. As Sholler flashed a slide on the screen to get day three going, Pat noticed one parent was missing. Neil Hutchison had filled the room the previous days with a frenetic stream of ideas. He was a storm of a man, driven by the fear they all had of losing his child. It pulled him in a thousand directions at once.
Pat had a hunch where Hutchison was — and he was right. Neil was out hunting again.
Inside the cavernous San Diego Convention Center across town, Neil Hutchison stalked through the forest of booths and exhibits. It was the annual meeting of the American Association for Cancer Research, one of the largest oncology conferences in the world. There were 10,000 presentations and twice that many doctors and researchers here. He hoped someone would have a drug, even the possibility of one, he hadn’t tried already, and he had tried just about everything.
Even in the world of desperate, try-anything cancer parents, Hutchison was legendary for his manic pursuit of something, anything, to save his 7-year-old son, Sam. He attended every major cancer conference, read obscure research publications, phoned doctors and researchers in the middle of the night. He was giving Sam a cocktail of 44 pills, most of them unproven treatments that he had picked out himself, in the hope that he could crack the riddle.
Now with a printout in his hand of all the presentations that mentioned neuroblastoma, he moved quickly from researcher to researcher, skipping pleasantries and getting right to the point: Is this something we can bring to a clinical trial quickly?
He wasn’t getting the answer he wanted, but he kept at it. By afternoon, he had exhausted himself. His head was foggy. He should eat, he thought. But not yet.
He went looking for the next exhibitor on his list and found him at the end of a long row, a trim man with a sweep of dark hair who stood by a poster about his research, recently published in the journal Nature. He’d found that a molecule derived from bacteria on bean plants could stop the growth of neuroblastoma.
The researcher started to speak, but Hutchison interrupted.
Is this something we can get to the clinic quickly, he asked.
Sorry, the researcher replied. It was still basic science. It would be years before patients might see its benefits.
Hutchison stared back. Then he lowered his head and started to sob.
The researcher, a molecular biologist at the University of Hawaii named Andre Bachmann, watched him, stunned. Bachmann had thought he was talking to a physician or a fellow scientist but now realized his mistake. This was a parent looking for a cure, a neuroblastoma parent.
Bachmann put his arm around Hutchison and led him to an open area with some tables and chairs. They sat, and Neil poured out his story — about Sam, the boy’s rapidly progressing cancer, and his quest to save him.
Bachmann listened. Again he apologized that his new research would do nothing in time for Sam.
But I might have something else, Bachmann said.
There was a drug that he’d researched years earlier and abandoned because he couldn’t find anyone willing to bring it to a clinical trial.
It was sitting on a shelf in his lab in Hawaii.
Bachmann told Hutchison the whole story, starting with his impulsive decision in 2002 to attend a neuroblastoma conference in Paris. He knew little about the disease at the time but happened to be working with neuroblastoma cells in his lab. He figured the conference would be a good way to learn more.
At the conference, Bachmann sat in on a session in which a researcher mentioned a curious trait of the disease: In the worst cases, patients’ bodies produced unusually high levels of a protein called MYCN. Bachmann knew quite a bit about MYCN — in particular, the fact that as it replicated, a gene named ODC was activated. Bachmann had an epiphany but quickly dismissed it. It was too obvious. Surely someone had already tried it.
Bachmann was thinking of a drug called DFMO, that was known to block ODC. If you blocked ODC, wouldn’t that disrupt the whole chain reaction the cancer needed to grow?
DFMO had been developed decades before as a possible cancer drug but failed to live up to its promise. It was never approved in the United States but had found a remarkable second life in Africa, treating African sleeping sickness. Transmitted by the bite of a tsetse fly, sleeping sickness was deeply feared, often leading to coma and death. DFMO’s astonishing ability to revive even far-gone patients was so startling that Africans knew it by another name.
They called it the Resurrection Drug.
DFMO clearly had not worked against other cancers, but maybe neuroblastoma was different, Bachmann thought. He hustled back to his hotel room, got on his laptop, and searched the scientific literature. He expected to find published papers, but there were none.
The idea raced through his head for the rest of the conference. He went back to Hawaii and set to work with his graduate students. He soon published research showing DFMO’s impact on neuroblastoma in mice. He wanted to take the research further, but he ran into an old problem. Because pediatric cancers are relatively rare, pharmaceutical companies have little motive to invest in research, and government funding is short. For years, Bachmann tried and failed to get anyone interested in bringing a DFMO therapy for neuroblastoma to a clinical trial.
Eventually he made the painful decision to shelve the project and move on to other work.
The research was still there and still promising, Bachmann told Hutchison. But it needed the right person to turn the idea into reality.
Hutchison abruptly stood up. He knew that person, he said, and ran off to find her.
Pat was sitting at a table on the hotel patio, watching a perfect late afternoon sun blast off Mission Bay when Hutchison raced toward him.
Where’s Dr. Sholler, Hutchison wanted to know.
Hutchison chattered excitedly, something about a drug they had to get into trial. Pat didn’t pay him much mind; Hutchison was always looking for Sholler with something they just had to get into trial.
Pat said he didn’t know where she was and watched Hutchison rush away.
He turned back to the water and the glimmering white beach and the sailboats on the bay. He hadn’t felt sun sink into his body like this in what seemed a lifetime, since the September day when a doctor told him and Dina that there was no hope left for their boy. It was so beautiful here, but he was alone and apart from those he cared about most.
He picked up his phone and called home. Dina answered. He asked about their new baby, Evelyn, and about Will.
Pat heard Dina whisper to their son.
Say “I love you, Daddy,” she said.
I love you, Daddy, Will said into the phone.
Pat told him he was in California and could see the Pacific Ocean and that he hoped that Will would get to see it someday, too.
Hutchison found Sholler and told her what he’d heard. She agreed to meet with Bachmann, and soon the three of them were sitting at the same table where Hutchison had earlier wept. She listened as Bachmann explained his research on DFMO.
His theory made immediate sense to her.
Let’s try to get this to the clinic, she said.
The next day at the hotel, she presented the idea to the parents. It was an exciting possibility, but it was still years from going into a patient. There was a lot of research left to do, and a trial could cost hundreds of thousands of dollars.
Pat Lacey spoke up and said he would try to cover it.
He’d never raised that kind of money before. He’d been tapping friends and family and community members, selling rubber bracelets and painted pumpkins, getting people to run in charity road races. He would just need to do more. He spent the plane ride home from San Diego thinking about how he would pull it off.
In June, Pat and Dina rented a house by a lake on the Cape. It was finally warm again in New England, and they needed a week to get away.
Pat did his best to be present, to stay off the Internet, to unplug. It was hard. So much of him was wrapped up in tracking the stories of the other families, watching their children like they were bellwethers of his own.
Pat knew Will’s cancer would eventually do what so many of theirs had done; it would lay quiet, seemingly doing nothing, and then one day it would burst forth and quickly spread. He put it out of his mind. Will’s cancer had so far showed no signs of that, and this was a week to forget, to enjoy a sliver of peace.
He watched Will run and splash in the lake and squeal with delight. The boy’s hair had grown in for the first time in his life, and he was getting big now, approaching age 4, a little human with a personality and opinions and a baby sister he fawned over.
When vacation was over that Saturday, they went home to Braintree and to real life. On Monday, their oncologist called.
He had reviewed a scan from the week before. He had the news Pat and Dina feared most. The cancer had spread.
There was no telling how much time they had now, or whether Will would live long enough for the cure they were chasing.
Chapter 2Will Lacey: A life in photos
Chapter 3 of 5
In a class of his own
On parade day in Braintree, the cheers followed Will Lacey as he rode through town in a red convertible, perched high in the back, his legs draped over his father’s shoulders.
Pat Lacey grinned in the brilliant summer sun while Will threw fistfuls of candy and waved. Some girls from their neighborhood wore red T-shirts printed with the words “Friends of Will” and jogged alongside, darting in and out of the crowd and stuffing fliers into people’s hands.
Will watched them and finally looked down at his father.
Why are all these people handing out pictures of me? he asked.
The question caught Pat off guard.
Sometimes, he said after a pause, we throw a big party and send the money to Dr. Giselle so she can keep giving kids medicine.
Will fell quiet, digesting the answer.
It was June 2009. A year had passed since doctors had showed Pat and Dina the scan with the livid pinprick of color indicating a new tumor, and for a year they had braced for what they knew should come next — the cancer coursing through Will’s body in a final, fatal assault.
It hadn’t happened. Not yet. Dr. Giselle Sholler had put Will on a new treatment and was about to switch him to another, hoping to keep the cancer at bay. But every day now felt like a race — for money, for new drugs, for new ways to hold off the inevitable a little while longer.
Pat and Dina tried to protect Will from their worry, acting, as far as they were able, like any other family. Dina was pregnant again. Pat volunteered as a hockey coach and drove Will and his friends to tee-ball and pizza parties. But Will was getting older and more aware, and he was asking more questions that Pat didn’t know how to answer.
Pat struggled most to contain his fear on the days when he had to take Will for a new scan at Sholler’s clinic in Vermont. The time came to go again late that month. He forced himself to talk cheerfully to Will as he drove. At the clinic, Will was sedated and loaded into the big, doughnut-shaped imaging machine.
Sholler came to the recovery room and beamed, bending her angular body to gather Will in a hug.
You’re getting so big! she said.
He looks great, she said to Pat, but she could see the tension on his face. She told him she’d get the results as fast as she could and left to find the radiologist.
In the quiet, Pat crafted bargains in his mind: Let the cancer be stable. Let Will have a shot at the next drug. Let him live long enough for his unborn sister to have memories of him.
How do souls get to heaven?
Will wanted to know. Do they take a plane? Can they come back in new babies?
They were at home. The kids had made a fort of couch cushions in the living room.
All week, Will had been asking about death. They were just the questions of a curious kid, Pat told himself, nothing more. But he found himself stumbling clumsily through his answers, evading what Will was so plainly getting at.
Will asked if Pat and Dina would die and then burst into tears, wailing and saying he didn’t want them to leave him all alone. Pat assured him his mother and father would live a long time and that Will wouldn’t be alone. Plus, people get more friends as they grow up, he said.
Satisfied, Will began to talk about the family he was going to have when he got older — four boys and three girls — and about who he was going to marry and where he was going to live and what he would do for work.
Pat listened to his son conjuring that future and felt darkness slipping over him.
Dr. Sholler put her two young daughters to bed, changed into sweats, and went to her desk in the darkened house to work. It was her routine every night. As the house settled and grew quiet, she worked late on getting new drugs to trial.
She was constantly pursuing new ideas because the likelihood that any single treatment would be a cure was low. The cancer seemed to eventually elude whatever was thrown at it. So she had to keep new options coming and hope that one of them, one day, would prove to be the key.
It was a juggling act. Money was so short that the trials lived month to month, surviving on whatever cash parents — especially Pat — could raise. And the clinic demanded so much of her emotionally. She had attended too many funerals with tiny coffins and spent too many plane rides home choking on guilt.
Twice a week, she did a rotation in the pediatric oncology clinic, seeing children with leukemia and other cancers that now had effective treatments. She needed that reminder, that even once-deadly diseases like theirs could be taken on.
Still, every neuroblastoma death crushed her. She got through by thinking of the children who were still fighting, kids like Will Lacey.
Her relationship with Pat, and her reliance on him, had grown deeper. They had long talks and made big plans about expanding the trials into a national network of hospitals. And always, there was Will to talk about: What might be coming, and what to do next. Sholler had recently put him on a new drug. If it worked, and if past experience was a guide, it might hold off his cancer for six to nine months. The boy would need something new by spring. It would take at least that long to launch a trial for the next treatment, DFMO, the drug they’d learned of in San Diego.
There was still so much to be done before she could bring it to patients. Sholler opened the DFMO file on her computer and scrolled to where she’d left off the night before.
Because he could control so little else, Pat threw himself into raising money. His BeatNB charity was shouldering an ever-increasing share of the fund-raising. Most of the parents he’d begun this journey with had lost their children, and their fund-raising had fallen away.
He rose early and stayed up late. He wrote impassioned pleas on Facebook, sold raffle tickets. He called foundations and charities and friends and friends of friends. His schedule was packed with fund-raisers. There were no easy ways to make money appear.
The cost of this trial would be high, but Sholler had encountered a stroke of luck in that regard; a small startup in Tucson, Ariz., called Cancer Prevention Pharmaceuticals was testing DFMO in colon cancer and had volunteered to donate the expensive drug in exchange for the data from her trial. But even with that considerable boost, she needed $200,000 to get the trial started. And Pat was still a very long way from that.
He had a fund-raiser coming up before St. Patrick’s Day in March. It was the third straight year he’d thrown the event, and he needed it to be bigger than ever.
A few days before the party, Sholler received word from the FDA: She was clear to open the DFMO trial. Now it was all up to Pat.
He was a ball of nervous energy as he drove to the Tirrell Room in Quincy, hours early, to make sure everything was ready. He wasn’t trying to reinvent the wheel with his “Cure me I’m Irish” event. There were Bud Lights and raffle tickets and auction items and T-shirts. It was the kind of thing people in Pat’s world have long referred to as a “time” — a party for a friend in need. They happen constantly. There are a lot of people in need. It’s hard to stand out. He had sold a lot of tickets, but he needed people to show up.
They did. It seemed like every person he had ever known was there.
The following morning, Pat mailed a check for $80,000 to Sholler, the last bit she needed to open the trial.
Just before the trial opened, two children Pat knew well died on the same day. One of them was Sam Hutchison, the son of Neil Hutchison, the man who had led them to discover DFMO in San Diego.
Pat was devastated. When Will had relapsed four years before, Pat had connected with about 30 other families fighting relapsed neuroblastoma. He thought of those 30 children as Will’s classmates, companions in the fight. Some were friends from the clinic. Others were children he followed online.
With those two deaths, they were all gone.
Will was now the last of his class.
The first batch of children enrolled in the DFMO study at the end of March, and Will Lacey was not one of them. Pat and Dina had hesitated. Will’s current drug was keeping his tumor stable, and there was no telling what would happen on DFMO — especially with the very small doses the first recipients would receive. A phase one trial is used to test a drug’s safety. Pat wasn’t worried about the side effects — children had been receiving the drug for years in Africa — but he was worried the dose might be too low to do much of anything, and reopen the door for Will’s cancer.
In May, after a month of rain and tepid snow passed, Sholler called Pat with some news. One of the patients had dropped out of the study and they had another opening in the first group. Pat and Dina took it as a sign and set aside their doubts.
They drove to Vermont a few days later, Will’s scans looked stable, and so they were cleared to drive home with a car full of cardboard cake boxes containing rows of small bottles of white powder.
When they got home, Pat poured a little bit of lemonade into one of the small bottles, shook it up, then handed it to Will, who drank it in one shot standing at the kitchen island.
How does it taste? Pat asked.
Fine, Will said, and raced off.
Life soon settled into a strangely normal pattern. Will took the DFMO powder twice a day. He wasn’t sick like he had been on all the other cancer drugs. Pat and Dina didn’t know whether to be relieved or afraid. In the past, the side effects were the sign that the drugs were working. Now, there was nothing.
A month later, in June, Sholler was in her clinic, going over test results at her computer. Will had been in a few days before. She’d looked at his scan at the time and told Pat that everything looked unchanged, and the Laceys drove home. But now she was giving the scans a closer look, and seeing something different.
She wrote it up in a report, but then decided against calling Pat. She instead attached the file to an e-mail and pushed send. He should read it himself.
Pat was at an Italian place in Braintree, with plates of chicken parm and the Sox playing on big screens. Another BeatNB fund-raiser thrown by a friend. He felt drained, but he made his way around the room, smiling and shaking hands, and then went to the bar.
He ordered a beer and stepped away to get out of the crowd for a minute and check his phone.
He saw an e-mail from Sholler with the full report. He scrolled quickly, not expecting any news, until he noticed a line that he had to read twice.
“The previously described left apical/supraclavicular mass has diminished in size.”
The tumor had shrunk. DFMO was working, or seemed to be. Would it last? Or was this to be one of those tricks cancer can play, responding to a drug before outflanking it? The only way to know was to keep going, with more children, until they had the answer.
That would prove harder than Pat Lacey or Dr. Sholler could imagine.
Chapter 3Will Lacey: A life in photos
Chapter 4 of 5
A cure and a crisis
The opulent, gold-leafed ballroom was packed. Leading pediatric oncologists had come from around the world to the conference in Grand Rapids, Mich., along with dozens of parents of desperately ill children. Officials at the federal Food and Drug Administration watched remotely on a live video feed.
All eyes were on the stage and Dr. Giselle Sholler.
On the screen behind her as she spoke that day in May 2014 was a photograph of Will Lacey holding up a brown medicine bottle as though giving a toast. It had been shot four years earlier, soon after the boy had begun taking DFMO, the drug Sholler was testing on children with neuroblastoma.
Will was 5 years old then. He was 9 now, and very much alive. On DFMO, Will’s cancer had steadily diminished until it was no longer detectable.
Sholler hesitated to call him cured. But she didn’t have another word for what had happened to Will, who no longer needed DFMO or any other cancer treatment.
Two other children enrolled in her trial had similarly incredible results. The news had electrified the global community of parents of children afflicted by this virulent form of cancer, and Will had become an icon they looked to. Many checked his father Pat’s blog regularly for updates on the boy’s condition.
But the study as a whole was not all miraculous. The other 14 children on the trial had all died.
It had been a uniquely difficult time in Sholler’s clinic. In one room, she would see Will Lacey or one of the other thriving patients. Just down the hall would be children taking the same drug, still choked by cancer. DFMO did everything for a small number of patients and nothing for many more. Then one day she asked herself if she might have been going about it wrong.
Instead of using the drug to fight relapsed neuroblastoma, maybe DFMO’s real strength was in preventing children from relapsing in the first place. In experiments in her lab, first in petri dishes and then in mice, the idea worked incredibly well. The cancer cells struggled to reproduce.
So she changed her line of attack, and in June 2012, she started a new, larger trial, for children in remission, using DFMO to try to keep them there. There would be 80 children divided into two groups — the larger one made up of those who had gone through traditional treatment and had not yet relapsed and the other of children who had relapsed once but whose cancers had been again brought into remission. Each patient would take DFMO for two years to try to prevent the cancer’s return.
Sholler had, by that time, moved away from Vermont and landed at Helen DeVos Children’s Hospital in Grand Rapids. There, with more resources to carry out her mission, she had thrown herself into building a new clinic and lab. Pat had become her emissary and proselyte, crisscrossing the country, speaking at conferences and neuroblastoma gatherings, raising money, and spreading the gospel of DFMO.
When the first 10 patients in the main group all reached the one-year mark still in remission, Sholler had moved to double the size of the study to 160 children, and Pat raised the money to make it happen. After two years, the numbers seemed even more convincing, and now, on the stage in the ballroom, she was ready to tell the world for the first time.
Slide by slide, she told the story of DFMO, arriving finally at her best slide, the one she could not wait to get to. It showed results for the 39 patients in the main group who had completed at least a full year on DFMO. Statistically, she said, a quarter of them or more should have relapsed by then.
To date, she told the crowd, we’ve had just one.
A charge went through the room. Pat Lacey could feel it from his seat in the back. It was what he had been hoping for, a jolt of enthusiasm to help him and Sholler achieve their goal of getting DFMO to every child with neuroblastoma.
In the audience, a man named Jeff Jacob was having different ideas.
Jacob was the chief executive of Cancer Prevention Pharmaceuticals, the company that supplied the DFMO for Sholler’s studies. For her first study, CPP had felt like a savior — agreeing to donate the drug in exchange for her data. But when the second study was being planned, Jacob told Sholler that the deal had to change and presented her with a $300,000 bill. The start of the trial was delayed while Pat raised the money. A child Pat knew, a young boy in Massachusetts, relapsed just before they could get it underway.
After that, Pat could never look at Jacob the same way, but the relationship had remained functional out of necessity — CPP had locked up the only supply of DFMO in the United States.
The company was facing financial constraints of its own. Its national trial to find out if DFMO prevented colon cancer polyps from re-growing was showing promise but running long and burning through money. Jacob and other senior staff had stopped taking salaries. If they could get FDA approval to treat cancer with the drug, the windfall could be huge. But they were still years away.
Now, having heard Sholler detail her remarkable results, Jacob wondered if there might be a faster way to get DFMO through the FDA.
The phone rang. Genevieve Bergendahl, Sholler’s program manager, took another call from another anxious parent. For more than a year, parents had been hearing word of DFMO’s success through the blogs and networks they followed. But now, in the days since Sholler detailed her results at the conference, they called at all hours, desperate for their children to have the drug.
Because it was experimental, there was only one way to get it — through Sholler’s clinical trial. Parents were calling months before their children could qualify for the study, desperate to book spots before it filled up.
Sholler wanted to get the drug to as many children as she could. She’d won regulatory OK to expand, and hospitals around the country were lining up to participate. For each new hospital that joined their network, Bergendahl made a mark on a map. Each new city felt like a victory.
Bergendahl had been with Sholler since the beginning in Vermont. She’d had a rule at the time against working with children with cancer. The emotional toll seemed too relentless. She had changed her mind after meeting Sholler, and since then, they had been together through every up and down. Bergendahl knew each patient in detail, down to their favorite flavor of pudding.
On the phone with the parent now, Bergendahl spoke reassuringly.
We aren’t allowed to reserve spots this far in advance, she said. But there’s nothing to worry about on this end. We’ll have DFMO for your child when she gets here.
Back in Braintree, both Lacey daughters were in school. Will was finishing fourth grade. The dry-erase calendar in the kitchen was overflowing with hockey and dance and lacrosse dates.
And there were still medical checkups. Will went to local clinics for quarterly scans. Once a year, he and Pat flew to Grand Rapids, where Sholler gave the boy a full workup. Each time, Will checked out fine.
But Pat and Dina’s worry never went away; they could never fully celebrate his cancer disappearing while living under the fear that it would return.
A few months earlier, Will had started vomiting and wouldn’t get better. Then, he complained of double vision. Pat had rushed him to the hospital, and he and Dina felt themselves in free fall when a scan showed a spot in Will’s brain.
There was an anguished wait and some complicated brain surgery. The spot turned out not to be cancer but a hemorrhage, possibly caused by the years of caustic chemotherapy and radiation. Will would recover, but there were more doctor visits and physical therapy.
Pat, meanwhile, was doing anything he could to keep Sholler’s work moving forward. He was always on his phone, always in touch with Sholler and the people who supported her.
A few weeks after the conference, Pat saw a note from Jeff Jacob appear in his inbox, addressed to him and Sholler, congratulating them on the conference and the study. He said he wanted to meet with Sholler to discuss “a framework for moving forward.”
Pat wondered: What could that mean?
A short time later, Sholler called Pat, fuming. She had talked to Jacob.
He wants us to close our trial, she said.
What Jacob told her was that CPP would continue to supply the drug for 80 patients but not Sholler’s planned expansion to 160. The sole focus now, he said, should be talking to the FDA about the fastest way to win approval of DFMO. She and Pat both knew what that would be — shutting down her current trial and launching a placebo-controlled random trial.
That was the usual course in drug approvals; half the patients receive the drug and half don’t. It was considered the only way to prove, without a doubt, that a drug works. And Sholler understood why Jacob wanted to fast-track FDA approval. It would allow doctors everywhere to prescribe it, widening the market for CPP.
But in Sholler’s mind, for her patients, such a trial was out of the question. Children would die.
Jacob had been sympathetic. “If I was a parent, I would do everything in my power to get my child treatment,” he would later say. “My position as CEO is to get a trial that the FDA would accept, which are not the trials researchers want to run or parents want to hear about.”
Sholler pushed back, but he was insistent, and it irked Sholler that Jacob suddenly seemed to be treating her like an employee of CPP, there to do the company’s bidding. Her group, after all, had created, written, administered, and paid for the entire trial; all he’d done was sell her the drug.
She thought he was wrong that only a conventional random trial would persuade the FDA. She believed she had an idea the agency might accept: Instead of giving a placebo to desperately ill children, she would create a control group using published data on patients whose treatment had been identical, except for DFMO.
Getting the drug to as many patients as possible remained her only goal. She wouldn’t let CPP reverse course now.
She dug in for a fight, but still she worried. CPP was the only source of DFMO in the country. If their relationship foundered there was nowhere else to get it.
In early June, Jacob asked Sholler to join in a conference call with prospective CPP investors. She refused. Hospital lawyers preferred it that way, but she also relished the opportunity to send a signal: She did not work for CPP.
Jacob was frustrated. In e-mails and conference calls through the summer, he and Sholler argued over how to proceed. Positions hardened; neither would budge. By late July, with negotiations going nowhere, CPP informed Sholler that it was working with another prominent pediatric oncologist for future trials.
Sholler felt cornered. Pat was in a panic. They would hit 80 patients in a few months. After that, there would be nowhere for children to get DFMO. Any trial that CPP tried to launch would take years to get underway.
She had no answer because she thought there was no answer.
Then one day, she received a phone call out of the blue from an old friend who had a wild idea.
Meryl Witmer had called just to check in.
She was a longtime supporter of Sholler’s. Her son had managed to beat a related form of cancer, but the experience had left her frustrated by the pace of pediatric research. She was the one who had, years earlier, arranged the gathering in New York where Sholler met Pat Lacey for the first time. She also happened to be a powerful businesswoman who ran a hedge fund with her husband and was one of two women on the board of Berkshire Hathaway.
It had been a year or so since they’d last talked, and Sholler told her everything. Witmer listened, then asked a question: If DFMO is out of patent, can’t you just start your own company to make the pills?
Outlandish as the idea seemed, it was one that had occurred to Pat before. But he and Sholler had dismissed it as too big an undertaking and one that couldn’t possibly be completed in time.
Sholler dismissed the idea again, but over the next few days the fight with CPP only escalated. She now saw no other way. She e-mailed Witmer: You need to do this now!
That fall, the DFMO shipments from CPP to Sholler’s clinic stopped arriving. Her supplies were running low, and as her hospital’s lawyers argued with CPP, a new worry crept in: Was CPP going to stop supplying DFMO even for the 80 patients they agreed on? Pat pounded out a livid e-mail, telling the company they barely had enough DFMO to get through the next two months. “How is this possible?” he wrote. “We are buying the drug. . . . You are quite literally withholding lifesaving medicine.”
He didn’t get an answer. But a short time later he did get word that someone from CPP would be in Boston and wanted to meet with him.
Pat looked at his phone, checked the time, and drummed his fingers on the sleek modern table. It was a crisp day in October. He’d left his office in Boston’s Seaport a few minutes earlier to walk a few blocks to District Hall, a new public “innovation” space.
Pat had never heard of the man who was coming to meet him. Dan Donovan was his name, CPP’s chief business officer.
The meeting felt to Pat like an attempt to take the dispute out of Sholler’s hands, to get him alone and see if he could be recruited to help convince Sholler to come to terms. Pat would keep his guard up.
A middle-aged man in a suit and a sideways smile appeared before him.
They shook hands and Donovan sat, all friendliness and small talk, like they were co-conspirators, Pat thought, the only level heads in a house of crazies. They were going to figure this thing out. Pat listened and nodded at the right times, smiled, until finally he tried to pin Donovan down. He wanted him to commit to at least sending the DFMO they needed for the original 80 patients.
Donovan leveled his gaze. If they wanted that DFMO, Pat recalled him saying, Sholler had to go.
A few days later, CPP sent one last small shipment of the drug — they called it a good-faith gesture. The company made clear in an e-mail that would be all until the drug was proved safe and effective. And they wanted another oncologist “designated as a replacement for Sholler.”
Sholler wasn’t going anywhere. She asked Bergendahl to run an inventory of the DFMO they had remaining at the various test sites. She wanted to know how much they had left and how long it would last, factoring in new children. She had no plans to refuse anyone.
Bergendahl called her with the answer: March 1.
A shiver of dread ran through Sholler. She hung up and called Pat to tell him the news. The secret plan was now the only plan, and the odds against them were sobering: Meryl Witmer and Pat Lacey would need to start a drug company, manufacture pills from raw chemicals, and get regulatory approval to give it to children. It could easily take years. They had 106 days.
Chapter 4Will Lacey: A life in photos
Chapter 5 of 5
Their indefensible hope
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 Sholler’s 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 he’d 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 didn’t sign off, and Sholler had warned everyone: Things do not move fast when you’re dealing with the government.
FDA permission would require a massively detailed application, and Sholler’s 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. That’s 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 you’re not going to believe this. I’m 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 – 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, he’d gone to the fund-raisers at Sholler’s 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 didn’t 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 she’d filed the application — three precious weeks — and there had been no real movement at the FDA.
The previous day she’d 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 wasn’t 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 — the fight with their drug supplier and the need for speed.
To Sholler’s 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 they’d 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 couldn’t 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 didn’t 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 hospital’s lawyers, who had continued to negotiate with CPP for more drugs. Talks had gone nowhere.
Pat talked about a last resort — going to the media. If it looked like they weren’t 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 winter’s 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. He’d 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.
“Did you see it?” Bergendahl yelled.
“Yes!” Sholler yelled back. “We did it!”
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 Sholler’s 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 — no one outside Sholler’s tight circle knew — how close they’d 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 Sholler’s attending physician at Hasbro Children’s 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.
Let’s 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.
“They are happy, playful, going to school. They do not require transfusions. They are growing and thriving. This is incredibly different from previous therapies.”
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.
“At Helen DeVos Children’s Hospital,” she said, then stopped.
“At Helen DeVos Children’s Hospital,” she began again, “we have not had a neuroblastoma patient relapse in 3½ years.
“We cannot go back to what it was like before.”
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 wasn’t one battle, but many. They were all long, and they were all hard.
She opened more test sites, including overseas — a hospital in Beirut, another in Marseille, France — 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 couldn’t 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 “happiness.” 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 they’d 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.
Chapter 5Will Lacey: A life in photos