Desperate for a Cure

A Father Scrambles to Raise Money for a Biotech Start-Up To Try to Save His Sick Kids

JOHN CROWLEY was desperate. When his year-old daughter, Megan, was diagnosed with the rare and rapidly fatal Pompe disease, he and his wife were told there was no treatment. Months later, doctors diagnosed their new baby, Patrick, with the same genetic disease that progressively weakens muscles.

Frustrated with the slow pace of drug development, Mr. Crowley decided to take matters into his own hands. The Bristol-Myers Squibb Co. executive quit his job and took over as chief executive of a tiny biotechnology firm with a promising approach to treating Pompe. His immediate goal: Find deep-pocketed investors. But Mr. Crowley’s pedigree at a pharmaceutical company and his partner’s scientific brilliance didn’t seem enough to impress investors. He was just a dad trying to build a company to save his kids, and he was running out of time.

Wall Street Journal reporter Geeta Anand’s new book, “The Cure: How a Father Raised $100 Million— and Bucked the Medical Establishment— in a Quest to Save His Children,” chronicles Mr. Crowley’s efforts. An adaptation:

On the morning of April 7, 2000, John Crowley was in shock. He had just been snubbed in his first attempt to raise money for Novazyme Pharmaceuticals Inc., the firm founded by scientist William Canfield that he had taken the helm of just that week. “You obviously know no venture capitalist is going to invest in a company run by an inexperienced chief executive and a scientist who has never worked outside of an academic lab,” John Frick, a partner in Chisholm Private Capital, had said.

Dr. Canfield tried to reassure Mr. Crowley, saying he had another source of investment that was almost guaranteed. A small, publicly traded drug company, Neose Technologies Inc., was eager to form a partnership. Dr. Canfield scheduled a meeting for the end of the week with Neose. Before they left Oklahoma City, he opened his locked desk drawer and gave his new chief executive the company checkbook. Mr. Crowley scanned it quickly, and his eyes widened. There was only $37,000 in the bank—barely enough to make payroll for the month. Things with Neose had better work out, or this company was in deep trouble.

Accompanied by Dr. Canfield and his lawyer, Mr. Crowley flew back east for the meeting at Neose’s headquarters in a converted warehouse in Horsham, Pa., an old industrial town about an hour from Philadelphia. Stephen Roth, the chief executive, and a half dozen company scientists, regulatory, and business officials were waiting in a spare, windowless conference room. The company had awarded Dr. Canfield a research grant, recognizing the brilliance of his work. But now he was back pressing for a business deal, and company officials were trying to determine if his science was ready for prime time.

There was only $37,000 in the bank—barely enough for payroll. Things with the new investor had to work out.

Dr. Roth, also a scientist in Dr. Canfield’s area of expertise, led the questioning. At 55, he was about 10 years Dr. Canfield’s senior, far more experienced in the business world and accustomed to grilling junior scientists.

“How are you getting the PTase?” he began, using the abbreviation for phosphotransferase, the name for the first of the two processing enzymes Dr. Canfield planned to use to make his Pompe enzyme.

“We’re purifying it out of lactating bovine udders,” Dr. Canfield responded.

“Cows? Where are you getting the udders from?” Dr. Roth asked. “The stockyards,” Dr. Canfield said.

“Never in a million years can you inject cow protein into people,” Dr. Roth responded, his tone angry. “The FDA would never allow it.”

“The bovine version is only for lab work,” Dr. Canfield said between his teeth. His tone was turning defensive as the questions kept coming. He said he was working on making a different, human version of the PTase enzyme for use in the drug trials.

“And when do you believe you’re going to go into clinical trial?” Dr. Roth asked.

By now, Dr. Canfield was answering in only one or two words. “One year,” he said. He stuffed his hands in his pockets and looked down. A high-ranking scientist at the University of Oklahoma, he wasn’t used to being questioned so superciliously.

Someone from the Neose team asked Dr. Canfield to write out his manufacturing process. For the next 15 minutes, with the eyes of the Neose team trained on his back, Dr. Canfield stood at the whiteboard writing equation after equation. When he finally turned around, his choppy handwriting in black ink stretched the length of the wall.

“Oh no, you can’t do this in one year,” one of the Neose officials said, after spending a few minutes intently studying the equations.

“We can,” Dr. Canfield retorted, only to be interrupted in mid-sentence by Dr. Roth, who had risen to his feet, red-faced.

“This is bull2,” Dr. Roth shouted, stomping out of the room. A minute later, the other Neose officials followed in quick but measured order.

Dr. Canfield sat heavily into his seat between his lawyer and Mr. Crowley, his arms crossed tightly, stroking his beard like the most wronged man in the world. “Of course we don’t have all the answers—that’s why we want to partner with them,” Dr. Canfield fumed.

He and his lawyer flew back to Oklahoma, and Mr. Crowley drove the two hours home to New Jersey, feeling like a fool. He’d signed on with Dr. Canfield’s company without asking enough questions. Dr. Canfield was an excellent academic scientist, but did he have any idea how to turn his breakthrough findings into a medicine? And even if he did, how plausible was the goal of beginning clinical trials in one year? Doctors had repeatedly told Mr. Crowley that Megan and Patrick wouldn’t live past age five.

Mr. Crowley pulled up to his house to find his wife, Aileen, and the kids outside. Megan, now two and a half, and Patrick, one, sat side by side in the front yard, looking like any other neighborhood children except for the tubes connected from their necks to the microwave-oven-sized ventilators that breathed for them. Mrs. Crowley sat between them, while their eldest son, John Jr., unaffected by Pompe, rode a tricycle in the street.

“Daddy,” Megan shouted, arms outstretched. He ran over to lift her up in the air. Patrick squeaked and waved both arms.

“Daddy,” he heard little John calling from the street. He turned around to see his eldest son pulling open the passenger side door of the family van. A cloud of balloons in every color rose into the air. Aileen shrieked and ran down the street, leaping into the air in her short floralprint dress, grabbing at the balloon strings.

With a start, Mr. Crowley remembered it was his own birthday. He was 33 years old.

His wife, clutching the strings of the two balloons she had saved, was walking toward him. “Good Lord. I bought 33 balloons, and now we only have these,” she said in a fit of giggles. “Happy Birthday, John,” she sighed, kissing him

As he and his wife ate dinner that night, he tossed out an idea he hoped she would go along with. “The company needs money right now. The only money we have is in this house. I bet I can get a hundred grand out of it if we take out a second mortgage.”

“You just started and this company is already out of money?” she repeated, blinking in surprise.

“Some of the investors are taking a little longer to line up than we’d expected,” Mr. Crowley said, keeping his tone casual.

“Do what you need to do, John,” she said, as she had so often before.

Mr. Crowley arrived in Oklahoma the next week with a check for $100,000. Luckily, Dr. Canfield was having a better week. Calling down a list of local doctors and dentists, he had managed to raise another $200,000, mostly in increments of $5,000 and $10,000.

With the immediate cash-crunch averted, Mr. Crowley set about trying to build his vision of a drug company. To infuse the science and business with the sense of urgency that drove him, he invited patients and family members each week to talk to Novazyme’s employees— from the scientists to the secretaries—at what he called Lunch ‘N Learns.

Mr. Crowley scheduled these lunches when board members and consultants were expected, hoping to draw them into his world. Almost everyone came away changed. But Mr. Crowley hadn’t expected how one young woman, in particular, would affect him.

Her name was Lindsey Easton, and because she lived only an hour away, in Glenpool, a suburb of Tulsa, she was one of the first patients invited to visit. She suffered from the same non-classical infantile form of Pompe as Megan and Patrick, and she had somehow outlived the life expectancy of 5 years old for their disease type. She was, in fact, 13, but the degenerative disease had taken almost all of her muscle strength over the years.

The day Lindsey arrived, Mr. Crowley showed up at the reception area, upbeat and smiling. “Hello Lindsey, how are you?” he asked cheerfully, beaming at the girl with big green eyes and long dark hair strapped in a wheelchair that reclined at about a 30-degree angle. Where Megan and Patrick could still sit up and play, Lindsey was able to move only her eyelids, mouth and thumbs. She responded with grunting noises. Lindsey’s mother translated quickly: “How do you think I am?” Then the mother laughed, mock-glaring at Lindsey, and said to her, “Could you please hold your wise2 comments in check for one afternoon?”

Mr. Crowley chuckled, realizing that Lindsey, weak as she was, had the same feisty personality as his Megan. He led the young woman, accompanied by her parents, brothers and grandparents, to the conference room where 30 employees had gathered for lunch.

Lindsey and her mother took questions from the audience—the mother sometimes speaking for herself, at other times interpreting for her daughter.

“It’s so wonderful to see all of you working on curing this disease,” Lindsey’s mother said when the questions finally ceased. “For so long, we didn’t have hope... Each and every one of you has brought us hope.” Lindsey’s ventilator started beeping, picking up on an obstruction in air flow. Big tears raced down her motionless face.

The room grew silent, choked with emotion. Mr. Crowley, at the front of the room, looked red-faced and glassy-eyed. His voice breaking, his lips visibly trembling, he stood up to thank the visitors.

At the back of the room, Dr. Canfield held his breath, hoping Mr. Crowley could maintain his composure. Dr. Canfield had been nervous about his colleague’s plan to invite patients to speak to the staff, believing scientists should be free to experiment without being so overwrought as to be afraid to fail.

Relieved that Mr. Crowley had held himself together, Dr. Canfield retreated to his lab. He didn’t see the scene that took place moments later in Mr. Crowley’s office, down the hall from the lab. Mr. Crowley stood with his back against the door, letting tears flow freely. Hearing a doctor talk about the progression of the disease was one thing; coming face to face with it was quite another. He felt sorry for Lindsey. He felt scared for Megan and Patrick. He would work longer, harder, faster. He could not—he simply would not—let Novazyme fail.

Mr. Crowley eventually persuaded Neose Technologies to partner with him, raised $27 million for Novazyme, and later, tens of millions of dollars for another biotechnology startup, Amicus Therapeutics Inc., developing treatments for genetic diseases like Pompe. Inspired by Lindsey, he dedicated Novazyme’s manufacturing facility in her honor. Lindsey graduated as co-valedictorian of Glenpool High School, where her speech, read by her grandfather, received a standing ovation.