Helping Samuel and kids with cystic fibrosis

A bunch of us in P&P are either doing "Movember" (Wendell, Dhaya, Marshal and Daniel) or running/walking 60 km in November, to raise money for cystic fibrosis.

Cystic fibrosis (CF) is the most common life threatening genetic disorder affecting New Zealand children. It is a genetically inherited condition some people are born with that mainly affects their lungs and digestive system. There are around 500 people in NZ with CF.

When we don’t have CF, our bodies make mucus that is thin and slippery and works as a lubricant to help protect us from infections and to keep the inside of our body working well. If we have CF, our mucus becomes thick and sticky and blocks the tiny tubes of many of our 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.

There is currently 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. Kalydeco is one of those medications - it is used to treat a strand of cystic fibrosis called G551D.

Kalydeco is the first medication that fixes the underlying cystic fibrosis defect, essentially turning off cystic fibrosis. Kalydeco represents a major breakthrough in cystic fibrosis research; current medications only treat the symptoms.

“Kalydeco has been hailed as a miracle drug – and with good reason. It has massive health benefits. Current life expectancy for those with cystic fibrosis is 37 years, but this drastically improves with Kalydeco. Every day that Kalydeco remains unfunded in New Zealand, irreversible lung damage is occurring” says Cass Byrnes, Cystic Fibrosis NZ's Medical Spokesperson.

The evidence backing Kalydeco is unequivocal. New Zealand is the only country in the OECD (with a significant cystic fibrosis population) that has not funded Kalydeco for those with G551D cystic fibrosis.

There is currently a major advocacy push by CFNZ and Kalydeco for Kiwis to try and get this drug funded in NZ.

Geri's 17 month old son has the G551D gene - so Kayldeco would help him.