With 450 clinical trials initiated since 2018, outlook for CF ‘hopeful’
Consulting company report looks at trends in disease research, treatment
Biopharma and biotech companies have launched more than 450 clinical trials into potential new treatments for cystic fibrosis (CF) since 2018, according to a report from Novotech, a contract research organization that specializes in helping drug developers to run trials.
“There are ample opportunities presented by advancements in technology, personalized medicine, and the emergence of new therapies, all of which hold the promise of improving outcomes,” the report states, adding that “the future for individuals with CF is increasingly hopeful.”
CF is caused by mutations in the gene that encodes the CFTR protein. Without a functional version of this protein, the body produces abnormally thick and sticky mucus, which builds up in organs to drive disease symptoms.
A research focus is treatments for those ineligible for CFTR modulators
Before the 1980s, about half of all CF patients didn’t survive beyond young adulthood. But medicines developed since have revolutionized outcomes for people with the disorder.
In particular, in the last decade a class of therapies called CFTR modulators have come on the market. These medicines can work to increase the functionality of the defective CFTR protein in people with CF caused by specific mutations, and they have substantially improved care for eligible patients.
A notable drawback of these therapies, however, is that they don’t work for everyone — and the mutations that are most responsive to modulators are more common in populations of European descent.
“CFTR modulators have revolutionized CF treatment … However, individuals of Asian, Black, or Hispanic descent may not benefit from current CFTR modulators due to their specific gene mutations,” the report states. “This disparity could worsen existing health gaps in CF, rooted in societal injustices.”
In recent years, there’s been a particularly strong push to develop therapies that can benefit patients who aren’t eligible for modulators. Therapies in development include new treatments targeting the underlying cause of CF, as well as therapies to deal with disease complications like infections and thick mucus in the airways.
“Recognizing the need for diversity in clinical trials, ongoing research is dedicated to developing new CF treatments,” the report states, adding that “achieving fairness in CF care requires substantial efforts in research, clinical practices, and health policy.”
According to the report, there are more than 162,000 people living with CF worldwide. Data from the Cystic Fibrosis Foundation indicate that more than 90% of CF patients are white; countries with the greatest number of patients include the U.S., U.K., France, and Germany.
Majority of CF clinical trials taking place in the US or Europe
Most of the CF trials launched in recent years are being conducted in the U.S. or Europe, although quite a few are taking place in the Asia-Pacific region, especially in Australia. “Countries like the United States, the United Kingdom, Australia, and Germany emerged as leaders in conducting trials,” the report states.
CF trials in Europe tend to enroll patients faster than trials run in the U.S., which “may be attributed to factors such as a potentially more suitable patient population and higher CF prevalence in the region,” according to the report.
In addition to clinical trials, the report gives an overview of financial investments into potential CF treatments. The U.S. is by far the world leader in terms of venture capital invested in CF research: from 2019 to 2023, more than $900 million in venture capital investment was dedicated to the disease. Other countries leading investment into CF include Italy, Israel, and the Netherlands.
Most of these investments are funding early stage research, including preclinical testing, which the report says “can be attributed to the potential for groundbreaking discoveries and the promise of innovative treatments.” These investments “recognize the importance of combating CF, highlighting international collaboration in treatment and cure, with significant funding from the US for CF initiatives,” the report notes.
The report stresses that a lot of work still needs to be done to improve life for people with CF worldwide, including improving access to available therapies and minimizing treatment burden and side effects. Still, with so much research already underway, the outlook for CF care is increasingly optimistic.
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