Fighting cystic fibrosis
New medicines, fundraising give Upper Arlington family hope
Medical developments and fundraising efforts like the one an Upper Arlington family started 13 years ago now provide considerable hope to some fighting cystic fibrosis.
Six months after Tom and Marie Hennessey's 8-year-old son, Matthew, and 3-year-old daughter, Kate, both were diagnosed with cystic fibrosis in May 2000, a friend organized a golf tournament to raise money for disease research.
From that "amazing gesture of kindness," as Mrs. Hennessey called it, the annual Hennessey Family Fore-Love Classic was born.
Since its beginning, the event has raised roughly $500,000 to help find treatments for cystic fibrosis, an inherited chronic disease that affects the lungs and digestive system. There is no cure.
On Sept. 9, the Hennesseys will host the 13th installment of their golf outing and dinner at Brookside Country Club, 2770 W. Dublin-Granville Road, Columbus.
Supported by the organizations such as Liberty Mutual and the Columbus law office of Dinsmore & Shohl LLP, as well as the typical mass of 100 golfers and approximately 200 who attend the dinner, the event generates approximately $65,000 each year.
"Since there are only 30,000 people in this country with cystic fibrosis, we do not represent much of a profit center to big pharmaceutical companies," Mrs. Hennessey said. "We could not have made the kind of progress that has been made in cystic fibrosis without the kindness of the thousands of people across the country who are willing to financially support the search for a cure, among them our friends here in Columbus."
Registration for the golf outing starts at noon Sept. 9 at Brookside. Additional information is available at centralohio.cff.org/golf.
In addition to the emotional and financial support the Fore-Love Classic has provided to the Hennesseys, they and others fighting cystic fibrosis -- about 70,000 worldwide, according to the Cystic Fibrosis Foundation -- have been buoyed by recent medical developments.
"For us in the cystic fibrosis community, we have what we talk about as a CF pipeline," said Dr. Melissa Holtzlander, a physician in the Nationwide Children's Hospital Pulmonary Medicine section. "That is a pipeline of all the drugs we use or that are coming out."
People who suffer from cystic fibrosis have a defective gene; its protein product causes the body to produce unusually thick, sticky mucus.
As a result, their lungs clog, making them susceptible to life-threatening lung infections. The condition also leads to obstructions of the pancreas and stops natural enzymes from helping the body break down and absorb food.
According to the CFF, most people diagnosed with the disease in the 1950s lived no more than a few years. Now, its website states, many "can now expect to live into their 30s, 40s and beyond."
"The goal of these new medications is to actually correct the defect at the cellular level," Holtzlander said.
Two such medications that excite the Hennesseys are Kalydeco, approved by the U.S. Food and Drug Administration in 2012, and VX-809, a drug currently still under clinical trial.
Matthew Hennessey, now 21 and entering his senior year at Ohio State University, is taking part in a six-month trial by Vertex Pharmaceuticals Inc. which is studying its cystic fibrosis drug Kalydeco in combination with VX-809 and their effects on people with the most common mutation of cystic fibrosis, Delta F508.
Both drugs are designed to treat the underlying cause of cystic fibrosis -- a faulty gene and its protein product.
Matthew said fighting cystic fibrosis is a "daily battle" that requires mental fortitude due to living in the shadow of early death, discipline to maintain a daily regimen of 15 to 20 pills and chest therapy to help clear mucus from the lungs.
While at Upper Arlington High School, Matthew said he played baseball and tried to live a relatively "normal" life as a teenager, but he also suffered five lung collapses before graduating in 2010.
Since the introduction of Kalydeco and his involvement in the clinical trial, however, he said he's felt consistently better.
"It's been very, very positive," he said. "Instead of having good days and bad days, good weeks and bad weeks, good months and bad months, it's just providing a higher baseline of feeling well.
"I want to stress how healthy I feel. I do everything I want to do."
Mrs. Hennessey said she's energized by the CF pipeline and its prospects for helping Matthew and Kate -- now 16 -- and others with cystic fibrosis improve and extend their lives.
"It is incredibly exciting to be part of the search for a cure for cystic fibrosis at this time," Mrs. Hennessey said. "Kalydeco and ... drugs currently in clinical trials represent an unprecedented advance in this disease as they correct the defect at the cellular level.
"That is the next-best thing to gene therapy from a patient perspective, as the disease cannot continue to destroy lung tissue and negatively affect other parts of the body. We believe we are winning the battle against this disease."