(Bloomberg) -- On a rainy morning in February 2013 at SeattleChildren’s Hospital, doctors and nurses were rushing in and out ofthe tiny room of a 5-year-old kidney cancer patient, Stellablue, who wasgetting a second round of aggressive chemotherapy following arelapse.

Taking a break from the hectic atmosphere, her father, AndyWoods, headed to the hospital computer library with his otherdaughter, a toddler, on his shoulder.

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The first article he read that day described recent experimentsin mice suggesting that a drug called IMGN901 could kill cells fromWilms tumor, Stellablue’s rare childhood disease. Woods, a bathroomand kitchen tile contractor from Bozeman, Montana, was becomingproficient in understanding medical papers. If Stellablue’streatment failed again, he knew this could offer a potentialoption.

“I said, ‘That’s just what I’m looking for,’’’ Woods recalled,reciting the paper’s title from memory, like a mantra. “‘Theisolation and characterization of renal cancer initiating cellsfrom human Wilms tumor xenografts unveils new therapeutic targets.’It was amazing.”

Like thousands of parents before him, Woods soon discovered thatdrugmakers avoid testing experimental drugs like IMGN901 onchildren, even on kids who didn’t respond to last-resorttreatments.

He had stepped into the blunt reality of research on rarepediatric diseases. Pharmaceutical companies have developed few newchildren-focused drugs because the market is small and testing isfraught with dire risks for young patients who can’t giveconsent.

For Woods, it was the beginning of a yearlong journey to get apediatric clinical trial of IMGN901 under way.

He was up against a system that pits desperate parents againstcancer-drug makers. The U.S. has taken an incentive approach,offering rewards worth tens of millions of dollars to encourageresearch. Families and advocates say it’s not enough, and are usingsocial media to get organized and pushing for legislation inWashington. In May, they got support from a group of 26 senators,who wrote to the U.S. National Institutes of Health, urging it tomake sure children are included in studies when possible.

“Too often, children get left behind as treatments are developedfor adults,” Mississippi Republican Roger Wicker and Rhode IslandDemocrat Jack Reed said in the letter. “It is critical that wecontinue to make progress on this front.”

IMGN901 roots

IMGN901 traces its roots to the 1980s, when ImmunoGen Inc.started developing a drug targeting a molecule called CD56.Following setbacks in multiple trials, the drugmaker most recentlyfocused its research program on adult lung cancer, the world’s mostcommon and deadly tumor.

CD56 is found on the surface of almost all cells in Wilms tumorand some other pediatric cancers in nerves and muscles. Children’scancer experts found IMGN901 promising as early as 2009, accordingto Malcolm Smith, associate branch chief of pediatric oncology atthe National Cancer Institute. ImmunoGen made the drug availablefor testing in mice and models but when researchers showed interestin testing it in children in 2010, the company indicated thatit wanted to wait until its own lung cancer trial was complete,Smith said. ImmunoGen, in an e-mailed statement, said it was unableto verify 2010 communications about a possible pediatric trial.

Most drugs aren’t studied in children -- sometimes for goodreasons, as trials can harm patients. Still, pediatric doctors needinformation on dosage and side effects specific to their youngpatients.

‘I beg you’

When companies do agree to pediatric trials, they typically waituntil after mid-stage studies on adults are done, said PeterAdamson, chairman of the government-supported COG, which organizesstudies in a network of about 200 hospitals. It’s not soon enoughfor researchers and parents like Jennifer Seigel.

“As a mother, it is horrific watching your child suffer waitingfor a successful treatment,” Seigel, a nurse practitioner, wrote ina 2013 letter to ImmunoGen. “I beg of you to consider pushing thisdrug into a pediatric clinical trial.” Her son died in January2014, 3 1/2 years after being diagnosed with Wilms tumor.

Oncology drugs sell about $100 billion a year globally. The vastmajority of the 1.7 million cancers diagnosed in the U.S. annually,about 77 percent, are in people at least 55 years old. Children andteens represent about 1 percent, and the chance that a child willdevelop cancer before 20 is about 0.3 percent.

From a business perspective, it’s an unprofitable market formost drugmakers.

“They’re not able to make any money in tiny patient populations,so they’re not going to invest in the drug,” Woods said.

Congress has stepped in to encourage children-focused cancerresearch with the Creating Hope Act of 2011, which some lawmakersare currently pushing to extend to 2027. The legislation rewardscompanies that get a drug approved first for use in children with avaluable voucher for fast review of any other drug: It’sessentially the right to get a more lucrative drug to the marketmonths earlier than through the usual approval track. Six vouchershave so far been awarded, and four of them were resold -- the mostrecent for $350 million.

Stellablue’s cancer

When Stellablue was diagnosed at 4, Woods dropped six tile jobs,and the family packed up their home facing Montana’s ChestnutMountain and drove 700 miles to Seattle Children’s Hospital.

Woods and his wife, Andrea, had just had their second daughter,Soren. To be close to the hospital, the Woods bought a used 25-footcamper for about $4,000. They lived for months in the parking lot.“We’re a close family,” Woods said. “Living on top of each otherisn’t the end of the world.”

During the nine months of Stellablue’s first treatment, Woodsspent most of his days reading about cancer.

“I had to become an oncologist,” he said.

When Stellablue relapsed four months later, her chances forsurvival plunged to 30 percent from 92 percent. She started a moreaggressive program. If it didn’t work, she would effectively be outof options.

Woods’ petition

Woods reached out to ImmunoGen by e-mail, pleading with thecompany to start testing IMGN901 in Wilms tumors.

“Right now, I feel like we are waiting around,” Woods wrote inSeptember 2013. “Waiting for you (ImmunoGen) to take the initiativeand move forward with this research on your drug and try it inWilms tumor.”

In November 2013, ImmunoGen announced that its adult lung cancertrial had failed and it would stop making and experimenting withthe product. Woods contacted leading experts including Smith, theNational Cancer Institute pediatric oncologist, and rallied otherWilms tumor parents. Woods also made a surprise visit toImmunoGen’s booth at a cancer conference in Chicago. A few dayslater, on June 4, 2014, he started a petition on change.org, sayingthat the Children’s Oncology Group, or COG, was ready to testIMGN901, and only one thing stood in the way:

“The drug company which owns this drug (ImmunoGen) has beenhesitant to provide a supply of it to the COG to run this trial,”the petition read. “They have sent mixed messages about whetherthey will provide the drug or not.”

By June 18, about 1,000 people had signed: “Great job everyone!”Woods wrote. Five days later, “a petition you signed wasvictorious!” he wrote, after learning the company would supply thedrug to the trial.

Limited supplies

Supplies of the drug are limited, and ImmunoGen said it wantedto ensure they were adequate to last for a full trial before makingthe drug available to the COG for testing on children. ImmunoGensaid it had agreed to give away the drug for free before Woods’campaign began, but was also making sure the group had thenecessary funding to run the study.

“We had put considerable scientific, financial, and emotionalcapital into it and were happy to provide supplies once the COG hadan FDA-compliant trial in place,” ImmunoGen said in an e-mailedstatement. “The petition had no impact on our decision.”

The drugmaker said it’s committed to making a difference forcancer patients, and is also providing IMGN901 to the University ofTexas MD Anderson Cancer Center, for a separate study on adultblood cancers.

‘Premature success story’

Funded by NCI, the pediatric trial began in 2015. It has nowabout 20 children with relapsed cancers, including Wilmstumor, but is enrolling at a brisk pace with the aim to reach 150,according to NCI’s Smith.

“This is a premature success story because we don’t know whetherthe drug is going to work,” said James Geller, the pediatric cancerdoctor at Cincinnati Children’s Hospital Medical Center who runsthe trial. “The biggest success will be if it impacts the health ofchildren and their families.”

For now, Stellablue doesn’t need the experimental drug herfather worked so hard to get into trials, and her family hopes shenever will. Three years after undergoing the aggressivechemotherapy program that sent her father looking for otheroptions, she remains cancer-free. Although she still suffers fromside effects of the treatment, including pain and breathingdifficulty, she’s the strongest she’s been in years. She wentskiing this winter and recently ran a half-mile without stopping towalk.

Back at work in Bozeman, installing tile, Woods is aiming for anew career at 40: He’s taking a temporary position at a cancerlaboratory near Portland, Oregon, where he’ll be looking athard-to-treat cases of Wilms tumor. If the situation works out,he’d like to work in cancer research full time.

“No matter how it goes, lots of good things will come out ofit,” he said. “Cancer is never far from my mind.”

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