Caleb Nolan is Receiving Life-saving Therapy for Rare Form of Cystic Fibrosis; Bennet Legislation Key to Expanding Therapy to Treat Most Common Form of CF
Broomfield second-grader Caleb Nolan today shared his story with U.S. Senator Michael Bennet about what it’s like to live with Cystic Fibrosis (CF) and how a new drug that Bennet is helping advance – Kalydeco – is changing his life.
In 2012, Kalydeco was approved to treat a small percentage (4 percent) of CF patients – like Caleb – with a specific genetic mutation. The success of this drug on patients like Caleb has led to the creation of new drug treatments, pairing Kalydeco with another drug (Lumacafter), and also using Kalydeco on patients with different gene mutations. These treatments could treat as much as 65 percent of the CF community.
Earlier this year, these new therapies received a “Breakthrough Therapy” designation from the FDA, which serves to accelerate approvals of drugs with early, dramatically positive results. As a result, this Kalydeco combination treatment will be put on a faster track to getting fully approved by the FDA and ultimately, in the hands of patients faster.
This new “Breakthrough Therapy” pathway for drug approval is based on a provision written by Colorado U.S. Senator Michael Bennet in the FDA Safety and Innovation Act that passed into law last July.
“Groundbreaking drugs like Kalydeco hold the key to helping patients like Caleb lead longer, healthier lives,” Bennet said. “The Breakthrough Therapy provision in last year’s FDA reform bill expedites the approval process for these drugs, ultimately ensuring that they reach kids like Caleb as quickly and safely as possible.”
“Since beginning Kalydeco treatments last year, our world has completely changed,” Melissa Nolan, Caleb’s mom said. “Caleb is able to breathe more easily and is more active and energetic than he ever has been. It has truly been a miracle drug for us and we are grateful to Children’s Hospital Colorado’s Dr. Frank Accurso and Senator Bennet for their work in advancing the drug.”
Caleb is a routine patient at the Children’s Hospital Colorado, which houses the largest CF clinical care center in the United States, caring for more than 500 children and adolescents. In fact, Caleb was originally diagnosed with CF as a newborn because Dr. Accurso spearheaded state legislation to make CF screenings a regular part of the newborn screening process in Colorado –it’s now a standard procedure across the country.
In addition, Dr. Frank Accurso has been a national leader in the development of Kalydeco and was the principal investigator during phase 2 of the drug’s research trial. He is currently working on its use in combination with other drugs.
Beyond CF, Breakthrough Therapy designation is having a large effect on other areas of unmet medical need as well, including drugs that treat breast cancer, leukemia and Hepatitis C.
May is Cystic Fibrosis Awareness month and next Sunday, May 18 is the Cystic Fibrosis Foundation’s annual “Great Strides” fundraising walk in Denver, at Sloan’s Lake.
CF is a life-threatening genetic disorder that affects roughly 70,000 people worldwide and approximately 30,000 in the United States, including nearly 1,000 Coloradans. The average life expectancy for people living with CF is 37 years.