NZCR a Key Participant in New Cystic Fibrosis Treatment

They say “good things take time”, and never has that been more true than in the case of Trikafta, a new treatment for the symptoms of Cystic Fibrosis, now funded by Pharmac.

10 years ago, NZ Clinical Research (or Auckland Clinical Studies as it was known then) was a key partner in phase 2 patient trials of a potential treatment for Cystic Fibrosis. Cystic Fibrosis (CF) is the most common inherited life-threatening genetic condition affecting New Zealand children. It is a genetically inherited condition some people are born with that mainly affects their lungs and digestive system.

The treatment NZCR ran trials on, has now been launched in 30 countries, under the name Trikafta, a medicine manufactured by Vertex. Pharmac NZ is now funding this vital medication for people with cystic fibrosis, aged 6 years and above, who meet certain eligibility criteria.

Lisa Burns, Chief Executive of Cystic Fibrosis NZ said, “This decision is nothing less than monumental, it’s a significant announcement that will dramatically change the lives, and life expectancy, of so many Kiwis with CF. The last two years have been tough, with the months since the preliminary announcement being especially difficult. Emotions and anxiety have been running high for our families and we’re delighted that they now have certainty.”

The NZCR team is humbled and proud to be involved in such a groundbreaking treatment that will give many New Zealanders with CF the chance of a healthier future. Our thanks to all the participants in the trial all those years ago, who can be proud to be involved in helping improve the lives of Kiwis with CF.

Symptoms of Cystic Fibrosis

(Source reference: Cystic Fibrosis NZ website)

Mucus becomes thick and sticky and blocks the tiny tubes of organs. In the lungs, the sticky secretions are difficult to cough up and viruses, bacteria and fungus can become trapped under the mucus. This can cause inflammation and infection, making it harder to breathe and often causing a chronic cough and repeated chest infections. People with CF also have a much higher risk of developing serious complications from bugs that are generally harmless to other people.

In the digestive system, thick secretions also block the flow of digestive enzymes from the pancreas to the duodenum (top part of the intestine), where they are needed to help break down food. Without these enzymes, fats and vitamins can’t be absorbed causing problems with malnutrition and poor weight gain.

There is no cure, but the gene that causes CF has been identified and new research and medications that focus on ways to repair or replace the gene are very promising.

For more info on CF and Trikafta visit the CFNZ website.