Cystic fibrosis drug spurs hope for future

For the first time in Mia James’ six-year life, her mum Kayla Hawkins is daring to feel hopeful for her future.

After hospital stays too many to mention, stints on life support, and the daily grind of clearing the airways, the prospect of access to a life-changing drug is emerging as something more than a dream.

Mia was an early baby, who quickly became desperately ill, with the devastating diagnosis of cystic fibrosis.

The genetic disorder causes people with the condition to produce thick, sticky mucous which clogs the respiratory and digestive systems.

Each day begins with time on a nebuliser and physiotherapy to clear the airways. It puts Mia at risk of infections, requiring routine hospital stays for intravenous antibiotics, and triggering several ‘‘crashes’’ which required time on life support to ensure she got enough oxygen to survive.

On good days, she is a pupil at Longburn School in the Manawatu. On others, she has to be distant from anyone who might be sick, especially anyone else with cystic fibrosis. Often, she is in hospital. Cystic fibrosis is life shortening, with only half of sufferers living past 16. An optimistic life expectancy is 36.

But with news in the last few days that Vertex, the suppliers of Trikafta, will apply to MedSafe and Pharmac simultaneously for registration and potential funding, there is hope of a normal life.

Hawkins has already done her part to help raise awareness of the devastating impact of cystic fibrosis.

Her family story was one of several that helped successfully advocate for funding of the drug Kalydeco, which has this year been added to the Pharmaceutical Schedule.

But at something like $383,000 a year, it is a drug that can only benefit about 35 people in New Zealand, and Mia is not one of them.

The majority, about 450, have the mutation that would respond to Trikafta.

But the fact it is an option being considered is ‘‘amazing news’’ for Hawkins.

For Mia, she would still have cystic fibrosis – it would not be a cure in that sense. But Trikafta would turn a life-threatening condition into a manageable one.

But now, even though she does not know what is coming, the hope was there that Mia might get the chance to grow up to be a regular teenager, to have grown-up relationships, and even her own family.

‘‘When I think of what she has been through ... she is amazing. And she has taught us some pretty good life lessons.’’