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Cystic Fibrosis Patients Breathing Easier Thanks To New Drugs
Jim Feldhausen, of Fitchburg, who turns 55 on Sunday, plays volleyball and is thinking about retirement and grandchildren.
Bethany Hawes, 36, of Montfort, west of Dodgeville, is going to her son's sporting events and no longer erupts in coughing fits after walking up the stairs.
Feldhausen and Hawes have cystic fibrosis, a genetic disorder in which mucus builds up in the lungs and digestive system, causing infections that often used to be fatal before adulthood. But after medical advances gradually increased life expectancy in recent decades, a breakthrough drug approved five years ago has transformed many patients' lives, boosting the expected lifespan from 46 years in 2019 to 61 years today.
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"I didn't think I was going to make it" to retirement, said Feldhausen, who is now looking at stepping down from his state government human resources job within three years and thinking he could be around for any children his 19-year-old daughter might have.
Jim Feldhausen with his wife, Colleen, right, and daughter, Josie, has lived much longer with cystic fibrosis than expected when diagnosed. A drug approved five years ago this month, involving a clinical trial at UW Health, has transformed the lives of many patients with the genetic lung disease.
AMBER ARNOLD, STATE JOURNAL"I can go up the stairs easily now," said Hawes, who is cheering on her son, Connor, 15, at his track meets instead of resting in the car like before. "I'm able to really be a present mom, which is huge."
The drug, Trikafta, has allowed many patients to reduce or break free from arduous daily routines of inhaled medications and chest vest therapy, in which an inflatable, vibrating vest helps clear thick mucus from the lungs.
While Trikafta can treat about 90% of the nearly 40,000 Americans and roughly 750 Wisconsinites with cystic fibrosis, it doesn't work on patients with some genetic variations. It costs more than $300,000 a year, typically covered by insurance, and has brought some reports of neurological side effects such as memory loss and brain fog.
At UW Health, which took part in the clinical trial for Trikafta, nearly all 350 or so patients with cystic fibrosis are on Trikafta or similar drugs, and four of them are in their 70s.
Jim Feldhausen demonstrates the chest vest vibrator he used for many years to dislodge mucus from his lungs. Thanks to new drugs for his cystic fibrosis, he no longer needs the device.
AMBER ARNOLD, STATE JOURNALThe need for lung transplants and hospitalizations has dramatically declined, said Dr. Andrew Braun, director of UW Health's adult cystic fibrosis clinic. Before Trikafta, he said, three to eight patients with the condition routinely needed inpatient treatment at any given time at UW Hospital.
"Now, it's usually zero to two. It's often zero," Braun said. "My dream job has always been to be a cystic fibrosis geriatrician, and it's looking like I'll be able to do that."
UW-Madison research
Nearly 40 years ago, UW-Madison played a pivotal role in treating cystic fibrosis.
In 1985, Wisconsin became the second state, after Colorado, to test newborns for the condition, through blood samples taken by pricking their heels. From 1985 until 1994, UW researchers studied babies whose parents were immediately notified of positive blood tests and those whose parents didn't get the news until the children reached age 4.
The study found that early diagnosis and early use of enzymes and other treatments led to increased height, weight and cognitive function, bolstering the argument for newborn screening. In 2004, the federal government cited the Wisconsin study as a key reason for recommending that all states begin such testing. By 2009, all states did.
In 1994, five years after scientists discovered the gene mutation that causes the condition, Pulmozyme, the first drug specifically to treat it, was approved. Other treatments came along, including aerosolized antibiotics and hypertonic saline, which reduced symptoms and helped patients live longer.
Trikafta, approved in 2019, treats the underlying cause of cystic fibrosis by correcting defective chloride channels in patients' cells, a defect that creates salt build up and leads to thick mucus.
AMBER ARNOLD, STATE JOURNALThe new era of treatment began in 2012, when the first pill to treat the underlying cause of the condition was approved for a small group of patients. Ivacaftor, or Kalydeco, corrects defective chloride channels in patients' cells, a defect that creates salt build-up and leads to thick mucus.
Kalydeco's approval was later expanded to more patients, and two similar drugs were sanctioned before Trikafta, a three-drug combination pill, was launched in October 2019.
All of the drugs were developed by Boston-based Vertex, in part through funding from the Cystic Fibrosis Foundation. The company has applied for approval of a new triple combination pill, for which UW Health has also participated in a clinical trial.
The turnaround in prognosis for patients on Trikafta and the other chloride channel modulator drugs has been so dramatic that children with the condition no longer automatically qualify for Make-A-Wish foundation support.
More patients with cystic fibrosis are working and becoming pregnant, Braun said. But as many live longer, more are developing diabetes and some feel so-called survivors' guilt for outlasting others. "We have a lot of mental health changes occurring," he said.
Breathing easier
When Hawes was diagnosed two months after birth, doctors told her parents the average lifespan was 16, she said.
Bethany Hawes, of Montfort, who struggled to manage symptoms of cystic fibrosis, has been more able to attend her son Connor's track meets thanks to a new kind of drug for the condition.
BETHANY HAWESHer parents had to learn how to give her enzymes and do chest therapy, pounding on her chest to release mucus before vibrator vests were widely available. Her father, Don Hawes, left his job in the mailroom at Land's End and became a nurse, eventually working at UW Hospital's unit for cystic fibrosis patients.
Bethany Hawes said she was frequently hospitalized, especially during high school, which was "really rough."
Her health improved enough to allow her to graduate from UW-Milwaukee, have her son, get a master's degree from Concordia University Wisconsin in Madison and work at the Iowa County Aging and Disability Resource Center. But by 2016, her lung function deteriorated to less than 40% of normal, requiring her to quit her job and causing her to cough repeatedly and become exhausted when walking up stairs.
In 2019, after Hawes started on Trikafta, she immediately felt better, she said. She still takes inhaled medications and uses the vibrator vest, but she isn't in the hospital or on intravenous treatments as often and her lung capacity is now more than 50%.
"Compared to what I had, it's amazing," she said.
Living longer
Feldhausen, who had symptoms as a child and young adult, wasn't diagnosed with the condition until he was 33, when he was told life expectancy was 36.
He and his wife, Colleen, blew their savings on a cruise. "I thought he was going to die," said Colleen, an administrator at UW Health.
They moved from Virginia to the Madison area to be near UW's clinic for adults with cystic fibrosis and closer to their families in the Green Bay and Chicago areas.
Jim Feldhausen, who has cystic fibrosis, said he wasn't sure he'd live long enough to see his daughter, Josie, graduate from high school. But she graduated this year and now attends Edgewood College.
AMBER ARNOLD, STATE JOURNALSince cystic fibrosis hinders male fertility, the couple adopted a child from China, not knowing how long Jim would be around. "I hoped I could see her graduate high school, but I thought that was a stretch," he said.
Josie, 19, graduated from high school this year and is a first-year, pre-med student at Edgewood College.
When Feldhausen started on Trikafta, he had what is known in the cystic fibrosis community as "the purge": "I sat over a trash can and coughed up about two cups of nasty goo," he said.
Soon, he stopped using inhaled treatments and the vibrator vest. With lung function of more than 60% today, he typically plays volleyball three times a week.
A recruitment director at the state Department of Administration, Feldhausen in recent years started saving for retirement — something he never imagined a decade ago — and plans to stop working within three years.
He still has lung scar tissue and other lasting damage, but he figures he has many good years left.
"I don't expect to live a normal lifespan, but 70 seems pretty realistic," he said.
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KITHER BIOTECH ANNOUNCES PHASE 1 CLINICAL STUDY OF KIT2014, A NOVEL INHALED PEPTIDE THERAPY FOR CYSTIC FIBROSIS
Study to evaluate the safety and tolerability of KIT2014, an inhaled peptide therapy designed to enhance Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) function, act as a bronchodilator, and reduce inflammation
KIT2014 is being developed as a CFTR enhancer, acting as an add-on treatment to existing CFTR modulators, targeting improved lung function and reduced inflammation in Cystic Fibrosis patients
TURIN, Italy, Oct. 30, 2024 /PRNewswire/ -- Kither Biotech, a clinical-stage biotech company focused on innovative therapies for respiratory diseases, today announced the start of a Phase 1 clinical study of KIT2014, a novel inhaled peptide therapy for Cystic Fibrosis (CF). KIT2014 is designed as an add-on therapy to existing CFTR modulators and works by enhancing CFTR gating, promoting bronchodilation, and reducing inflammation through PDE3/4 inhibition.
"The initiation of this Phase 1 study marks a significant step forward for Kither Biotech and the CF community," said Dr. Dimitrios Goundis, CEO of Kither Biotech. "KIT2014 addresses mucus obstruction, chronic inflammation, and bronchoconstriction-three critical challenges that affect CF patients even with current CFTR modulators. We are excited to begin assessing the potential of KIT2014 to improve treatment outcomes."
About the KIT2014 Study
This Phase 1 double-blind, placebo-controlled study will primarily evaluate the safety and tolerability of single and multiple doses of KIT2014 in healthy adult volunteers, followed by an assessment of pharmacokinetics. For more information, visit clinicaltrials.Gov (NCT06659757).
About Cystic Fibrosis
Cystic fibrosis is a life-limiting genetic disease that affects approximately 130,000 people worldwide. It is caused by mutations in the CFTR gene, which lead to defective chloride ion transport across cell membranes, resulting in the buildup of thick, sticky mucus in the lungs. This mucus impairs mucociliary clearance, promotes chronic bacterial infections, and triggers persistent inflammation, leading to progressive lung damage.
Unmet Medical Need for KIT2014
Despite significant advances from CFTR modulators, many CF patients continue to experience persistent inflammation and recurring infections, severely impacting lung function and overall health. Neutrophil-driven inflammation, marked by elevated levels of neutrophil elastase, remains a major contributor to lung damage in CF patients. KIT2014 addresses these issues with an innovative solution that directly targets this inflammation, reduces neutrophil activation, and provides an additional therapeutic benefit that complements existing CF treatments. KIT2014 has the potential to improve significantly long-term outcomes for CF patients.
Story Continues
Mechanism of Action of KIT2014
KIT2014 is a peptide-based therapy designed to target these fundamental issues by acting on multiple pathways in CF lung disease. Delivered through inhalation, it allows direct targeting of the lungs. By inhibiting phosphodiesterase 3/4 (PDE3/4), it elevates cyclic adenosine monophosphate (cAMP) levels. In bronchial epithelial cells, this mechanism enhances CFTR gating and improves chloride ion transport, while in airway smooth muscle cells, it acts as a bronchodilator. Additionally, KIT2014 reduces neutrophil-driven inflammation through PDE3/4 inhibition. Together, these mechanisms position KIT2014 as a promising add-on to existing CFTR modulator therapies.
About Kither Biotech
Kither Biotech is a clinical-stage biotech company focused on developing innovative therapies for respiratory diseases. Kither Biotech's lead program, KIT2014, is an innovative inhaled peptide therapy aimed at enhancing CFTR function, providing bronchodilation, and reducing inflammation in CF patients. By targeting multiple pathways in CF lung disease, Kither Biotech is committed to addressing unmet medical needs and improving the quality of life for patients suffering from severe respiratory conditions. The company has successfully raised $26 million to date from investors including Claris Ventures, 2Invest, 3B Future Health, CDP Venture Capital, Italian Angels for Growth, Club degli Investitori, Ersel and ACE Venture. For more information, please visit www.Kitherbiotech.Com and follow us on LinkedIn.
CisionView original content:https://www.Prnewswire.Com/news-releases/kither-biotech-announces-phase-1-clinical-study-of-kit2014-a-novel-inhaled-peptide-therapy-for-cystic-fibrosis-302290900.Html
SOURCE Kither Biotech
Crispr Therapeutics: Poised For A Major Turnaround
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