The Importance of Clinical Trials in Research For Cystic Fibrosis

Clinical trials remain crucial to further developing our understanding of Cystic Fibrosis and how it can be treated, as there is no cure for the disease.

In 2007, screening newborn babies using the Newborn blood spot test was made mandatory. Implementing such measures further emphasises how scientists and medical specialists agree on the importance of being able to diagnose the disease as soon as possible. The more clinical trials that are conducted and the more results that are gathered from trials, the greater the chance of lowering early deaths and avoiding long-term complications for individuals.

With the disease having effects on the respiratory system, clinical trials involving Respiratory Therapy allow medical professionals to constantly test new treatments to find new ways of alleviating symptoms.

What is Cystic Fibrosis?

Cystic Fibrosis (CF) is one of the most common life-limiting diseases in the UK. It is an inherited condition that can be passed down from parents to their children.

The disease causes mucus to build up in the lungs and digestive system, causing lung infections and issues with digesting food. If not diagnosed at birth, the disease gets worse over time. The damage to a person’s lungs gets more severe, and the lungs can stop working properly.

Anyone diagnosed with Cystic Fibrosis has a shorter life expectancy. The sooner the disease can be diagnosed, the quicker treatments that help improve someone’s quality of life can be administered.

Symptoms of Cystic Fibrosis

• Persistent coughing.
• Frequent lung infections, typically Pneumonia or Bronchitis.
• Recurring chest infections.
• Persistent inflammation of the airways.
• Wheezing and shortness of breath.
• Serious bowel obstructions in newborns.
• Jaundice.
• Problems putting on weight and growing.
• Diabetes from late childhood or early adulthood.

Causes and Treatments for Cystic Fibrosis

Cystic Fibrosis occurs when a child inherits a copy of an altered (changed) gene from both parents. The disease is caused by a faulty gene that affects the movement of salt and water in and out of cells in the body. 

Medicine is the preferred form of treatment for issues with the lungs. This includes:

• Antibiotics to prevent and treat recurring chest infections. 
• Dornase Alfa or Hypertonic Saline to thin mucus in the lungs.
• Ivacaftor to reduce the levels of mucus in the body.
• A combination of 3 medicines (Kaftrio) to treat the root cause of the disease (in patients over the age of 12).

In very serious cases, when the lungs are struggling to work properly, a lung transplant can be considered.
Additionally, physiotherapy to help clear the airways is a common form of treatment to help lessen the symptoms of Cystic Fibrosis. A physiotherapist can recommend Autogenic Drainage, which is a series of controlled breathing techniques that aim to clear mucus from the lungs, or Active Cycle of Breathing Techniques (ACBT) to help with consistent coughing. In some cases, treatment can consist of the use of a breathing aid such as a positive expiratory pressure device.

Cystic Fibrosis Awareness Month 2025

Since it was formed in 1955, The Cystic Fibrosis Foundation has raised vital funds for research into Cystic Fibrosis. The work carried out by the foundation has brought about critical changes to help diagnose the disease sooner and determine new ways to treat symptoms of the disease.
Every year in May, the foundation hosts Cystic Fibrosis Awareness Month. The foundation encourages people to share stories of those with and those affected by the disease, and raise funds for research. Each year has a theme, and this year the theme is “resilient”.

How to Get Involved in Cystic Fibrosis Awareness

Anyone can get involved with Cystic Fibrosis Awareness Month. The foundation provides a social media toolkit to make it easy for people to promote the month and any events they wish to organise or be involved in. 

Some of the main ways you can get involved include:

• Sharing awareness posts.
• Donating.
• Fundraising.
• Encouraging diagnosis.
• Taking part in an event. 
• Attending workshops or webinars.
• Volunteering.

Clinical Trials & Scintigraphy For Cystic Fibrosis

The importance of clinical trials in the diagnosis and treatment of Cystic Fibrosis cannot be overstated. Clinical trials assist scientists in diagnosing the condition from birth, advancing existing treatments to improve their effectiveness, and exploring new therapies.

Scintigraphy

Scintigraphy is a medical scanning procedure used to help better understand how a disease acts within the body, and how the body reacts to different drugs. In the case of Cystic Fibrosis patients, Scintigraphy provides detailed scans of the structure of the lungs. 

The procedure involves injecting a small amount of radionuclide into a vein or having it swallowed by the patient. This allows professionals to produce images of blood flow within the body. These images allow professionals to analyse how the lungs and blood react to different medications and how the disease travels through the body. The detailed scans allow specialists to compare the effects of treatment at different stages and from patient to patient. 

Being able to collect this data enables specialists to diagnose the disease, determine what stage of the disease someone is at, and monitor the disease more accurately.   

CRO Services With Simbec Orion

Simbec Orion offers full clinical trial management services that suit all types of clinical development projects. We are a complete clinical research organisation (CRO). Our laboratory services and data processing abilities allow for patient assessment, subject trials, and pharmacovigilance practice.

We tailor the services we provide to ensure they remain goal-orientated but considerate of individual needs. Our services cover all stages of a clinical trial. We have categorised our services into phase 1, phase 2, and phase 3 to give a thorough breakdown of what we can provide.

Inquire about how we can assist with your clinical trial and see why we pride ourselves on offering world-class services that are both attentive and adaptable.