Masterton mother of three Allyson Lock is one of 11 kiwis with Pompe disease, a rare and incurable degenerative disorder. The drug trial she has been on for the past five years has been abruptly cancelled. Photo / Hayley Gastmeier
In a few weeks' time, Allyson Lock's fate will be left "swinging in the wind".
The Masterton mother of three has Pompe disease, a rare inherited degenerative disorder which is almost always fatal.
For the past five years Mrs Lock, 51, has been on a drug trial run by pharmaceutical giant BioMarin.
"The drug was giving me my life back," she said.
But last week she found out the trial had been canned.
"It halted the progression of the fatal disease, so without it I will die.
"It gave me more strength. I have just been getting better and better on it, even after five years I have continued to improve.
"And that's not just my opinion or based on how I feel - although how I feel is important - but all the tests show improvements too."
Mrs Lock had not long signed a 10-year contract to extend the BioMarin drug trial, before finding out it would come to "an abrupt halt" at the end of this month.
She is yet to hear the news direct from the company, instead finding out through the Canadian Association of Pompe.
"[Without the drug] my legs will get weaker and my diaphragm will get weaker and I'll die of respiratory failure."
There is one last hope left for Mrs Lock, Myozyme, a drug she said she had already applied for and been denied twice through Pharmac.
"It's a proven treatment for Pompe. It's been on the market for 10 years and is available in nearly 60 countries. Australia started funding it last year."
However, New Zealand refused to fund the drug, Mrs Lock said.
Next week she will try her luck a third time and apply for the life-saving treatment through Pharmac.
Her journey since finding out she had the disease in 2010 has not been easy, involving travelling thousands of kilometres for treatment.
"For the first six months of the trial I spend in Florida. Then I started fortnightly trips to Brisbane, which I did for three years, equating to about 80 trips.
"The last year and a half I have been going to Auckland City Hospital which started a trial site for the Kiwis on the trial."
Only 11 other New Zealanders are known to have the disease.
Mrs Lock, founder of the NZ Pompe Network, said many of them had also applied for treatment and had been denied.
"It's hard to stay strong when you have been beaten down that many times.
"We haven't got a big enough voice - there's only 11 people."
Mrs Lock, her husband, who is in the Navy, and her children, the youngest of whom is 13, have a "flippant" attitude towards her condition.
"We don't dwell on it, it's just our life," she said.
"Every day's a struggle but I try to focus on other things. I don't let that be my life because that's not just who I am."
BioMarin released a statement saying it "greatly appreciates the many patients, families, and physicians who have participated in our Pompe clinical trial program, as well as the ongoing support of the entire patient community".
"After careful deliberation, BioMarin has made the difficult decision to stop its reveglucosidase alfa development program [sic] in Pompe disease.
"On a regular basis, BioMarin prioritizes the resources it can put towards its development programs in rare diseases and is focusing on different rare diseases."
The statement said that BioMarin recognized that the situation was "difficult and challenging" for those who chose to participate in the clinical trial.
"We are grateful to the patients who have made sacrifices to participate in clinical trials."
Mrs Lock also said she was yet to hear the news direct from the company, instead finding out through the Canadian Association of Pompe.
BioMarin said a consent document signed by all trial participants stated that the trial could be stopped "at any time", and that it was not appropriate "for companies sponsoring clinical research to have contact with participating patients".
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