Tama Hubbard has good days and bad days, just like any other 3-year-old child.
But lately, the bad are outnumbering the good. And things aren’t looking up.
The Taupō toddler has a degenerative disease called spinal muscular atrophy (SMA).
It is thought there are fewer than 100 people in the country with the condition.
On his good days, Tama is talkative and just wants to play.
He’s quite strong within himself on those days, his mum Lisa Geddes told the Rotorua Daily Post this week.
But on his bad days – like last week – Tama is tired, he struggles to eat, or even lift his hand to his mouth. He’s weak.
“If he drops his head, he’s unable to lift it back up himself,” Geddes said.
She herself sounded tired on the phone. Exhausted, actually. She talked slowly and quietly. She paused mid-sentence more than once, before returning to her train of thought.
Her kids – she has three sons, including Tama – could be heard chatting away in the background.
The family live in rural Taupō. Last week, Tama developed a small cough.
But, the thing is, there is no such thing as a “small” cough for Tama.
Geddes said because of how weak her son has become, and how fast he has deteriorated, Tama has lost the ability and the strength to cough.
He requires a cough-assist machine and a lot of physiotherapy.
Geddes took Tama to Taupō Hospital last week for a check-up, because of the cough, but he was transferred straight away to Rotorua Hospital.
His temperature had spiked, his oxygen levels had dropped. The next day, his temperature was even higher, and so was his heart rate.
Photos taken at Rotorua Hospital show just how small and vulnerable Tama is right now. He was breathing through a machine.
Geddes said she does everything she can to prevent Tama from getting sick.
“Because once he does, he just goes downhill really fast.”
Here’s the glimmer of hope: there is a treatment for Tama’s disease.
The drug is called Spinraza and it is publicly funded in many countries around the world, including Australia. But not in New Zealand.
Geddes said she can’t afford the treatment, which can reportedly cost hundreds of thousands of dollars a year.
The New York Times a few of years ago reported that Spinraza would, by some estimates, be among the most expensive drugs in the world.
“If he doesn’t get the treatment, he will get worse, until it takes his life,” Geddes said.
“In three short years, he’s already deteriorated really fast.”
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Spinraza, also called Nusinersen, is the only FDA-approved treatment for SMA sufferers and is said to have produced incredible results in youngsters taking the drug overseas.
Biogen, the company that supplies it, provides it free of charge to Type 1 SMA patients in New Zealand under the age of 18 through its extended access programme.
But Tama is considered Type 2, so he doesn’t qualify.
Biogen, an American drug company, recently reported a 7.1 per cent rise in third-quarter profit, according to Reuters.
That was boosted by higher demand for Spinraza.
Net income attributable to the company rose to US$1.55 billion – or US$8.39 per share – in the quarter ended September 30, from US$1.44b – or US$7.15 per share – a year earlier.
Reuters reported that the company’s total revenue rose to US$3.6b from US$3.44b.
In July, Reuters reported that Spinraza sales rose 15.4 per cent from a year earlier to US$488 million.
Biogen has been approached for comment about Tama’s case and the price of Spinraza.
Pharmac, New Zealand’s medicine-buying agency, started evaluating an application for Spinraza funding in August last year.
The rare disorders subcommittee met in late September this year to consider more information provided by Biogen about the drug.
“We will publish a record of the subcommittee’s advice and recommendations on our website when it is available,” Pharmac said today in a statement.
Its operations director, Lisa Williams, said the agency works to get the best health for New Zealanders that it can, “by funding medicines from within the available budget”.
She said having a fixed budget means Pharmac needs to make careful and considered funding choices in the interests of all New Zealanders.
“Pharmac will continue making the best choices we can, expanding available treatments for all New Zealanders using a robust, evidence-based approach.”
Health Minister David Clark said he absolutely understood why New Zealanders suffering from SMA and their families want affordable publicly funded access to the latest treatments for the disease.
“However, the Government respects the independence and impartiality of Pharmac. It is not for us as politicians to second guess the experts at Pharmac about which drugs it purchases.”
Meanwhile, Tama and his family wait.
Geddes said when Tama was younger, he used to be able to sit in the crawling position.
While he has never been able to physically crawl, he would sit up on his hands and knees and rock back and forth. He could sit like that unsupported.
“He would bum shuffle around and play with his toys,” she said with a light laugh.
“And now he struggles to hold his head up himself. He can no longer sit unsupported. He can’t sit in a crawling position at all; his arms are now too weak.”
Tama no longer has the strength to hold himself up.
He needs the help of others. And so does his mum.
Another SMA sufferer from Rotorua, 3-year-old Heath Sutherland, also doesn’t qualify for Biogen’s extended access programme.
His family is in the process of trying to relocate to Australia, where Spinraza is funded.
However, the Sutherlands will still have to privately fund the cost of Heath’s equipment and therapy while there.
They are trying to raise money for those costs and their relocation through crowdfunding site Givealittle, and have so far raised more than $13,500.
What is spinal muscular atrophy?
Spinal muscular atrophy (SMA) is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle).
It involves the loss of nerve cells called motor neurons in the spinal cord and is classified as a motor neuron disease.
Source: Muscular Dystrophy Association