Have you ever wondered if faith and science can coexist? I have. My six-year-old daughter lives with cystic fibrosis, and from the moment I learned of her diagnosis, I have believed whole-heartedly in the power of both faith and science to shape my daughter’s future and my own outlook on her life.

Every evening, I hold my daughter’s hand and pray with her as she lies in her bed. Our final verse is always the same. “Please watch over my baby. May she be healthy, happy, safe, and strong. I am so proud of her, and I am so glad that You gave her to me.”

When something particularly nerve-wracking is happening, I give my daughter a tiny porcelain angel to clutch. 

She holds it during blood draws at the hospital and during every day occurrences that bring her worry. Yesterday, she held it in her palm as she walked into school by herself. Her big brother wasn’t there to take her to her classroom, and she was nervous that she couldn’t navigate the school’s maze of hallways herself. She could have walked those halls in her sleep, of course. But I like to think the angel gave her a little extra confidence.

I routinely am reminded of what my daughter’s pulmonologist told us at our very first clinic visit. “Your daughter is going to live a long and full life. It’s my job as her doctor – and yours as her parents – to help make that happen.” From that day on, I put all of my trust in this amazing woman. She led the creation of evidence-based approaches to CF care in infants and preschoolers. She was the principal investigator for a number of clinical trials. She was a mother herself and knew from that first appointment how to speak to me as a partner, a fighter, and a protector. She encouraged questions big and small. She gently reminded me to not “put my daughter in a tower” and gave me the subtle but effective eyebrow raise when I got wound up over something relatively benign. She also took an aggressive approach to treating illness and helped me understand how every cough would be treated; what pathway of drugs we’d try at any given point in time; etc. Over time, she gave me confidence that no matter what came our way, she would be there to see us through.

When she moved across the country to be closer to her family, our confidence didn’t waver. She had trained her protégé, her whip-smart and compassionate former Fellow, to treat CF in the exact same way. That’s the beauty of medicine. 

One person alone doesn’t have to bear the load. Others can be taught and trained to follow the same clinical pathways and to give families the confidence that they so desperately need.

Every evening, my daughter and I thank God not only for our doctors, but for the medications that help us stay healthy. I am acutely aware that without scientists’ tireless devotion to improving the management of cystic fibrosis, our lives would be very different. We wouldn’t have a rich selection of antibiotics to treat pulmonary exacerbations. We wouldn’t have the medical devices to perform respiratory therapy on a daily basis. And we wouldn’t have the groundbreaking medications that help address the underlying cause of cystic fibrosis.

Earlier this week, the U.S. FDA approved Trikafta, the first triple combination therapy available to treat people with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older with CF who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population.

When my daughter was diagnosed in 2013, only one therapy of this kind existed. It was available to 4% of the CF population, which did not include my daughter. Six years later, we have a medication that can treat roughly 90% of the population, including my baby. 

Granted, we have to wait until it’s approved for ages 6-11. We need to make sure that insurance plans grant access so that families can actually afford it. And we need to make sure that researchers are continuing to focus on the remaining 10% of the CF population who deserves to benefit from a medication that can change their lives, too.

Because this medication will be lifechanging, indeed. Clinical trials demonstrated improvements in lung function by an average of 13 percentage points, along with improvements in sweat chloride, quantity of pulmonary exacerbations, and body mass index. 13 percentage points might not seem like much to some, but imagine a lung function curve that is trending downward, and all of a sudden, it changes course and ticks upward by 13 points. That is huge. That is powerful. That is the miracle so many of us have been praying for.

And that is why I can believe so fiercely in both our pulmonogist’s words and in the plausibility of my prayers. With a drug like this, my daughter is going to live a long and full life. My daughter can be healthy, happy, safe, and strong.

It’s just like the Serenity prayer says:

God grant me the Serenity to accept the things I cannot change, the Courage to change the things I can, and the Wisdom to know the difference.

To all those who had the Courage to change the things you could, I salute you. To the scientists at Vertex Pharmaceuticals. Your commitment to pushing the Trikafta research forward is changing lives. To the visionaries at the Cystic Fibrosis Foundation. Your novel approach to venture philanthropy has made this possible. 

To the generous friends, family members, and strangers who gave from their pockets to help fund scientific breakthroughs and one day, a cure for CF. A little bit can go a long way. To the doctors who chose to devote their lives to treat this disease. You are our rocks. And to the warriors who have tirelessly brought awareness and advocacy to the cause. Brian Callanan, Barbara Harrison, Erin Evans, Rod Spadinger, Andy Lipman, Jerry Cahill, Gunnar Esiason, Margarete Cassalina, Emily Schaller, Emily Kramer-Golinkoff, Laura Bonnell, Tim Wotton, and so many others. You inspire hope and courage every day. 

I have faith in God.

I have faith in our physicians and scientists.

I have faith in this community.

And I will give prayers, money, time, blood (for clinical trials), and anything else that is needed to help ensure that everyone with cystic fibrosis can live a long and full life.

Amen!

Help Others Live STRONGER and LONGER- 

   

   

 

 

 

Laura Spiegel is the mother of two wonderful kiddos, one who has CF.  She is a member of the CFLF Board of Directors. Laura is also the founder of Paint Her in Color, a new web site that offers emotional support to parents of children with special health care needs. She can be contacted at laura@paintherincolor.com, on Facebook at facebook.com/paintherincolor, on Twitter @her_color, or at www.paintherincolor.com

 

 

 

 

***Views expressed in the CFLF Blog are those of the bloggers themselves and not necessarily of the Cystic Fibrosis Lifestyle Foundation*** 

***Please speak with your physician before making any changes to your CF management***