‘Hope is a huge thing’

Mother wins battle for lifesaving drug

By Emily Norman

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Allyson Lock never gave up hope.

Not when her lifesaving drug trial was cancelled.

Not when she had to travel to Brisbane every fortnight for treatment.

And not when a fellow Pompe disease sufferer died of respiratory failure last year.

Now, the Masterton mother of three has been stunned by a humanitarian act granting her free access to the treatment she needs to survive – administered just around the corner at Wairarapa Hospital.

Mrs Lock is one of 10 New Zealanders living with Pompe disease, a rare inherited degenerative disorder which is almost always fatal.

Her fate was left “swinging in the wind” last year when a drug trial, run by pharmaceutical giant BioMarin was cancelled — without the drug, she would die of respiratory failure.

She fought for months to have an alternative lifesaving drug funded by the New Zealand Government, but these attempts were futile, with government pharmaceutical agency Pharmac confirming they would not be funding it earlier this year.

Mrs Lock’s life changed last Friday however when she was told that American biotechnology company Sanofi Genzyme had granted her access to the drug – for free and to be administered at Wairarapa Hospital.

“It’s so exciting — I need to just about peel myself off the ceiling,” she said.

“Hope is a huge thing. I hadn’t given up. I was always hopeful.

“But how many times can you be kicked before you can’t get up again.”

Mrs Lock was diagnosed with Pompe disease in 2010, and since then has travelled thousands of kilometres for treatment, including a six-month trial in Florida, and fortnightly trips to Brisbane, which she did for three years.

The mother of three, who uses a wheelchair to get around, said the most noticeable symptoms of her Pompe disease were that it made her legs, and diaphragm very weak.

It took 16 years from the onset of symptoms at age 30, to be diagnosed and, because she has difficulty breathing, she must use a breathing machine at night.

A spokesperson for Sanofi Genzyme said Mrs Lock was one of four Pompe patients in New Zealand to have been accepted into the company’s international humanitarian programme which allowed for free access to the drug.

“In the case of patients in New Zealand, the Pharmacology Therapeutics Advisory Committee of New Zealand’s recent decision not to offer reimbursement for these patients, combined with each person’s individual health circumstances, meant that other treatment options weren’t available to them,” the spokesperson said.

“Pompe is a progressive and debilitating disease.

“In lieu of reimbursement, we could see that our humanitarian programme was their only hope for treatment.

“We are now working with their New Zealand-based physician to ensure that treatment is delivered as quickly as possible.”

The lifesaving drug, Myozyme, is funded in 76 countries.

New Zealand is now one of only three OECD countries that does not fund it.

While Mrs Lock is “definitely relieved” about gaining access to the drug for free, she said “it hasn’t let Pharmac off the hook”.

“I’m still going to carry on knocking on their door,” she said.

“We are lucky we have a treatment for Pompe, but unlucky we live in New Zealand.

“That’s what really gets to me. This is New Zealand. It should not be like this.”

“If Pharmac can’t fund it, then the government has to give them more money.

“Forget about their stupid flags, forget about million dollar treatments for murderers in prison.

“They haven’t got their priorities right as far as I’m concerned. Not when they’re letting people die.”

With Myozyme treatment, Mrs Lock expects to see a stabilisation of her Pompe symptoms, and “modest improvement.”

“It’s not only getting better physically, but mentally as well — knowing that you’re actually being helped.”