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For head of state’s biotech trade group, drug progress is personal

Bobby Coughlin, 16, who has cystic fibrosis, with his father Robert, president of the Mass. Biotechnology Council.
Bobby Coughlin, 16, who has cystic fibrosis, with his father Robert, president of the Mass. Biotechnology Council.(Jonathan Wiggs/Globe Staff)

DEDHAM — For 11 years, Robert K. Coughlin has been the public face of the state’s booming biotech industry, trumpeting its breakthrough drugs and defending their often-dizzying price tags.

As president of the Massachusetts Biotechnology Council trade group, he lobbies for firms that have marketed life-saving medicines for devastating blood cancers, a rare inherited muscle-wasting disorder, and many other terrible diseases.

But there’s no approved drug to slow the progression of his 16-year-old son’s deadly illness. Bobby Coughlin, a Dedham High School junior and the youngest of three, was born with cystic fibrosis, a rare genetic illness that attacks the lungs and other organs. Boston-based Vertex Pharmaceuticals has introduced three drugs since 2012 that treat the underlying cause of the disorder in some patients, but none of them targets his particular mutation.

That could soon change. One of two new experimental medicines from Vertex is likely to be approved in about a year, based on encouraging late-stage clinical results. If that happens, it’s expected to benefit patients with Bobby’s mutation and help the company reach a goal of making cystic fibrosis treatable for 90 percent of patients.

Coughlin, 49, knows better than most that drug development takes years, but that hasn’t made the wait any easier. His son, a cheerful, slender teenager who likes to shoot baskets in his driveway, fish for striped bass, and play video games, looks fine. But he has advanced liver disease as a result of cystic fibrosis, his dad said, and may eventually need a liver transplant.

“As far as I’m concerned, it’s crunch time,” said Coughlin, a former state representative from Dedham who served in governor Deval Patrick’s administration for seven months before joining MassBio in 2007. “If that drug can be approved within the next year or so, that’s great news.”

Coughlin often publicly mentions Bobby’s ordeal to underscore the high stakes of drug development. Indeed, Coughlin’s experience with cystic fibrosis contributed to MassBio’s decision to hire him, according to Mark Leuchtenberger, a drug industry veteran who chaired the group’s board of directors at the time.

“Anyone who is touched by these terrible diseases has an understanding that others just can’t feel or understand,” said Leuchtenberger, interim chief executive of Brooklyn ImmunoTherapeutics of New York, and still a MassBio board member. “That’s a source of his energy.”

Bobby Coughlin takes a range of medications for his cystic fibrosis.

Jonathan Wiggs/Globe Staff

12/08/2018 Dedham Ma- Bobby Coughlin (cq) is a 16 years old who has Cystic Fibrosis. His family is hoping for a new drug to come to the market that would be beneficial to him. He is photographed ,with some of his current medications. Jonathan Wiggs /Globe Staff Reporter:Topic:

Coughlin, of course, brought other assets to the job. His political experience has helped him lobby for biopharma firms that have received millions in state tax incentives, and to fight government efforts to limit drug prices. MassBio represents 1,200 companies and gives Coughlin a generous compensation package that in 2017 totaled $774,114, according to a tax filing for the nonprofit.

He’s also a good schmoozer, by pedigree and practice. The youngest of six sons of the late Paul Coughlin, a Dedham selectman, Robert Coughlin was elected to Dedham’s School Committee at 20 and then served on the town’s Board of Selectmen before his 2002 election to the Legislature.

Coughlin’s schmoozing landed him in trouble right after he joined MassBio. The state Ethics Commission fined him $10,000 in 2008 after it concluded he secretly sought the MassBio job while negotiating tax breaks for the biotech industry as a key aide to Patrick.

As Coughlin recently discussed his son’s cystic fibrosis with a reporter at a hotel in Boston’s Seaport District, however, his affability gave way to sadness. His voice quavered with emotion. He brushed away tears at times, but said he’s more optimistic than ever that “we’re going to be able to beat this.”

Cystic fibrosis afflicts about 30,000 Americans. It’s caused by a defective gene that results in a thick, sticky buildup of mucus in the lungs, pancreas, and other organs. People with the disease have inherited two copies of the defective gene, one from each parent. They have to take a slew of drugs to improve their breathing and ability to process foods. The average life expectancy is about 37, according to the National Institutes of Health.

Bobby takes 50 to 100 pills daily to aid digestion, ward off infections, and treat his organs. He spends at least 45 minutes a day inhaling medicated mists through a nebulizer. For about 20 minutes a day he also uses a contraption that resembles a defibrillator and emits sound waves to loosen mucus in his lungs.

Between the regimens, homework, and other activities — he plays basketball with friends but no longer plays ice hockey because he can’t risk a blow to his liver — Bobby only gets about five or six hours of sleep a night.

“The hardest part about having CF is probably just maintaining all the treatments when pretty much all of my friends, none of them have to really do any of that,” said Bobby, who hopes to go to college in the Boston area.

He has to be particularly careful to avoid germs. His mother, Christine Coughlin, said, “I can pick out people coughing when I enter a room, I’m so sensitive to it.”

In an odd coincidence, Robert Coughlin got involved in the fight against cystic fibrosis long before Bobby was born — even before he was married.

Since 1995, he has served every year as honorary chairman of the nonprofit Cystic Fibrosis Foundation’s Great Strides fund-raising walk in Dedham. Coughlin said he began volunteering as a selectman because a sister-in-law and a friend from Dedham High School have family members with the disorder.

But his immediate family wasn’t touched by the disease until December 2001. Christine was pregnant with Bobby when a prenatal screening test — one that had just become routine — revealed that she had one copy of the defective gene, making her a carrier of the disease. Coughlin got tested and discovered he did, too.

On New Year’s Eve in 2001, the Coughlins met with a genetic counselor at Boston University Medical Center. He told them that their baby had inherited both defective copies and would be born with the disease.

The Coughlins were staggered. They had two healthy children, Mary Kate and Paul. (Mary Kate, the couple later learned, is a carrier, but Paul isn’t.) The Coughlins, who are Catholic, would not consider terminating the pregnancy.

Coughlin vividly recalls the reaction of friends with whom he volunteered at the foundation when he told them his son would be born with cystic fibrosis: “People were like, ‘Oh, my God, so that’s why you were doing all this.’ I said, ‘No, we didn’t even know.’ ”

Bobby was born in May 2002 as Coughlin ran — successfully — for the first of three terms he won in the House.

Coughlin has drawn strength from others affected by the illness, including Joseph J. O’Donnell, a Boston corporate tycoon whose son, Joey, died of cystic fibrosis in 1986 at the age of 12. O’Donnell has helped to raise hundreds of millions of dollars for the foundation, much of which has gone to Vertex for research.

“We’re going to raise whatever money it takes to buy a cure for your kid,” Coughlin remembers him promising in a phone call. “Your kid isn’t going to die from this disease like my kid did.”

The fight against cystic fibrosis has made great progress since Joey’s death. In 1989, Dr. Francis S. Collins, now the director of the National Institutes of Health, codiscovered the gene that causes the disease.

“We thought it was all over,” O’Donnell recalled. But that breakthrough also revealed that developing a drug to treat the underlying cause of the disorder would be “5,000 times more complicated than we thought,” O’Donnell said. Scientists have identified over 1,700 different mutations.

In 2012, the Food and Drug Administration approved the first such drug, Kalydeco, made by Vertex. The FDA has since approved two more Vertex medicines, Orkambi and Symdeko. Today, more than half of the 30,000 people with cystic fibrosis are eligible for at least one of the drugs, according to Vertex.

Then came news in November about a potential treatment that could finally help Bobby. Vertex reported encouraging data from late-stage trials of a new medicine that combines three compounds.

The combination improved lung function up to 14 percent, which may not sound huge, but is “a bigger magnitude of clinical benefit for any drug we’ve ever seen in cystic fibrosis,” according to Brian Skorney, an analyst for Robert W. Baird & Co. in New York.

Data from a second Vertex triple-combination drug in late-stage trials are due early this year.

Both medicines are expected to treat patients with Bobby Coughlin’s mutation. Vertex plans to seek FDA approval for the one that yields the best results by the middle of this year.

“This marks an important milestone in our journey to develop medicines to treat the underlying cause of CF in all patients with this devastating disease,” said Kym White, a Vertex spokeswoman.

While the Vertex drugs have already transformed some patients’ lives, their price tags have drawn fire. Kalydeco has a list price of more than $311,000 a year, Orkambi $272,000, and Symdeko $292,000.

Paul M. Quinton, a professor of pediatrics at the University of California San Diego School of Medicine, has cystic fibrosis himself, has spent his career researching the disease, and takes Symdeko. (At 74, he says he is “an outlier” for having survived so long.) He said the medicine has dramatically improved his health, but the cost is “horrible.”

Although Medicare and his wife’s health insurer pick up most of the expense, he said, that’s passed on to taxpayers and insurance customers. He pins much of the blame on the salaries of Vertex executives.

“When the executive officers are all in the multimillion-dollar range, of course you’re going to have to charge more for the drug,” Quinton said.

In 2015, Vertex shareholders, in a nonbinding vote, slammed company CEO Jeffrey Leiden for his pay. Leiden in 2014 received a compensation package of $36.6 million, the highest in the state for the head of a public company. His compensation last year fell to $17.2 million.

But Coughlin, who speaks often to Leiden, rejects the criticism. He said drug development is expensive and the company reinvests the money it makes in research. Efforts to limit what Vertex charges for medicines — such as New York’s recent request for a 70 percent discount on Orkambi for Medicaid patients — would be “catastrophic for patients,” he said.

“If the early-stage Vertex drugs weren’t reimbursed, there’d be no next generation,” Coughlin said. “We wouldn’t even be having this conversation today if there was no reimbursement or coverage for Kalydeco, for Orkambi. The drugs that we pay for today, that revenue is investing in the drugs of tomorrow.”

Jonathan Saltzman can be reached at jsaltzman@
globe.com
.