CLEVELAND — The holidays are a time for reflecting on the blessings of the past while setting new goals for the future for most of us. Others will spend the holiday in the hospital, or stay as hopeful as possible they'll even make it another day.
Dan Beach doesn't like to live that way. His cystic fibrosis affects nearly every part of his life, but he's a 45-year-old father. A beloved part-time bus driver. A good husband. His extensive stays in the hospital and daily treatment could never get in the way of those most important parts of his life, though he admits it's a lot to manage.
"It's an hour or so in the morning, hour or so at night, of taking medicine, clearing the lungs. It effects the digestive system, not just the lungs. So I can put in 3,000 calories a day just like that, just to maintain my 160 pounds," Beach said while sitting in the Rainbow Babies and Children's Hospital lobby. "I do think through on a given day, 'Hey what do I have to get accomplished today? Am I going to have enough energy to get that done?'"
Cystic fibrosis is caused by a genetic mutation on the body's cells that maintain a body's water and salt flow. In a CF patient, that mutation causes thick mucus build-up, damaging the body's lungs, digestive system and sinuses. Beach has no sense of smell.
"Eighteen months ago, they put me in for a potential lung transplant. What you see now is not where I was. It was bad," Beach recalled.
This holiday season could be different, though. The FDA recently approved a three-drug combination called Trikafta that not only reaches the mutation, but fixes it, thanks to gene therapy. The approval came five months ahead of the scheduled sign-off. Word of Trikafta's effectiveness moved doctors to tears. Beach reflects on how far treatment has come.The typical life-span of cystic fibrosis patients is still only mid-40s.
"I was thinking about the younger generation, a kid who's diagnosed with cystic fibrosis at age 3, and they're told like my parents were, he'll be dead at 18, we don't have anything for him. That's what my parents were told. If he makes it to 18, it'll be a blessing."
Beach's doctor, Kimberly McBennett MD, is also the principal investigator for UH's Leroy W Matthews Cystic Fibrosis Center, that has been running trials for the drug combination. Trikafta is only approved for patients 12 and older, but McBennett estimates that's still 90% of their patient population. The mother of the first patient she told the news to cried in her exam room.
This isn't the first time gene therapy has touched Beach's life.
"A miracle baby, so to speak. Number 12 embryo, she was the last one, the rest of them didn't take. She knows her story, she knows it well. We call it the Aubrey story, from day one," Beach explained about his 8-year-old daughter.
Beach and his wife knew they couldn't have children when they got married. They put the call out to family and friends when they decided to become parents. Several embryo donors came forward, but little Aubrey was the last and only embryo to take.
Trikafta could mean more days with Beach's little miracle baby.
"You start thinking, maybe I can plan for more than 10 years. I actually might see my daughter get married. The reality of it is, in my mind I can make legitimate longer term plans that I couldn't do before," Beach said.
Beach will begin taking Trikafta in a few weeks. It's three pills per day, and continuous updates with Dr. McBennett. To learn more about Trikafta and cystic fibrosis, click here. Beach also thanks the Cystic Fibrosis Foundation for their tireless work to find a cure. To learn more about the foundation, click here.