A race against time: Teen fights for access to cancer drug

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Hope is what Zoey Jackson has lived on for years.

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Diagnosed with inoperable brain cancer at the age of six, Zoey first underwent nine months of chemotherapy. When that didn’t work, she received 30 rounds of radiation – the maximum number she could take – on her brain and spine.

And when that no longer kept the cancer from growing, Zoey went on the drug trametinib for two years, living with side effects like open sores and nausea in order to fight her recurrent, low-grade glioma.

But treatment options – and in turn, hope – are running low for the now 17-year-old Saint Johner.

Zoey’s best hope – a once-weekly pill called tovorafenib – isn’t approved for sale yet in Canada, leaving her with the options of obtaining the drug through either a clinical trial or Health Canada’s special access program.

But Zoey doesn’t currently qualify for any of the three trials of the drug underway in Canada, and at this point, the medication isn’t available through the Health Canada program.

That’s according to a pediatric neuro-oncologist working with the Jackson family.

As the tumor in her pituitary gland continues to grow, Zoey has been left asking why she can’t receive tovorafenib, telling her mother that she doesn’t want to die.

“All of our conversations up to this point were always filled with hope,” said her mother Pam Jackson. “You know, there’s this, we tried it, it worked for a little while and now we’re on to the next thing – we’ve come a long way in 10 years – and then when you get hit with this, what is my response? Sorry?

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“Well, I’m not going to stop at sorry. This is not just a cause, I’m advocating for her life.”

Jackson and her family have launched a petition calling for access to tovorafenib for children and young adults battling brain cancer in Canada. More than 4,100 signatures have been collected to date over the course of a few weeks.

It’s not clear how many other pediatric cancer patients are currently waiting for access to the drug in Canada and the complexity of their cases, but in the case of Zoey, it’s a race against time.

A recent scan has shown new lesions in the brain and the spine, said Jackson, who just wants to see her daughter, who attends St. Malachy’s Memorial High School and works part time at Canadian Tire, fulfill her dreams in life.

“We’re living high anxiety trying to get her the treatment that she needs before time runs out on any end – whether that be her aging out of pediatrics or just no other options.”

Doors closed

Fewer than five per cent of patients with Zoey’s type of cancer end up dying from the disease itself, but often these patients face shortened life expectancies due to the barrage of treatments they undergo and associated medical complications, according to Dr. Sébastien Perrault, a pediatric neuro-oncologist at CHU Sainte-Justine in Montreal.

These patients are prone to losing their quality of life over time because if the cancer continues to spread unchecked in the brain, they can lose vision, executive function skills and balance.

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Perrault, who is a clinician-researcher, has been working with patients who are receiving tovorafenib. Some of these patients have received it through drug trials, others through Health Canada’s special access program.

Out of the three current drug trials, Perrault confirmed Zoey doesn’t qualify for any of them. The first trial has reached capacity, the second trial is only for patients who haven’t received any treatment, and the third trial hasn’t expanded to Atlantic Canada.

That leaves the option of Health Canada’s special access program.

Under that program, a health-care provider can obtain drugs not yet authorized for sale in Canada on behalf of a patient. That patient must have “a serious or life-threatening condition where conventional treatments have failed, are unsustainable or are not available in Canada.”

But that program relies on the voluntary participation of the drug manufacturer.

In the case of tovorafenib, Day One Biopharmaceuticals – which developed the drug – was making the medication available through a special access program in Canada, Perrault said, “but they put a cap on the number of patients.”

“It’s a unique situation,” Perrault said of Zoey’s case. “We often have situations where we have no therapy that we can do because it’s a hard-to-treat tumor or it’s aggressive – we encounter that frequently – but to have a drug that is effective and we don’t have access, that’s unusual.”

Day One Biopharmaceuticals has recently licensed non-U.S. regulatory and commercial responsibilities for the drug to Ipsen Biopharmaceuticals.

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Brunswick News asked both Day One and Ipsen about the availability of tovorafenib through Health Canada’s special access program.

“There is currently no special access program available for tovorafenib to new patients in Canada,” Ipsen spokesperson Sally Bain wrote in an email. “Access to tovorafenib could be possible via an investigator-sponsored clinical trial and we’d encourage eligible patients to discuss this option with their treating physician.”

A total of 15 patients have received tovorafenib through the special access program (SAP) since 2022, according to Health Canada. The program allows for manufacturers to either sell or provide free of charge drugs that have yet to be approved in Canada.

“Manufacturers determine whether they will provide access to a drug, and manufacturers may decline or restrict access for any reason,” Health Canada spokesperson Joshua Coke said in an email.

“The SAP cannot compel a manufacturer to supply a drug, even if a request is authorized.”

‘Hope is all we want’

In April 2024, the U.S. Food and Drug Administration granted accelerated approval to tovorafenib for pediatric patients like Zoey who have a low-grade glioma with a BRAF gene alteration.

That approval means the drug is available for Canadian patients to purchase from the U.S.

“It’s possible to buy the drug, but it’s complicated and it’s really, really expensive, so it isn’t a sustainable way to access the medication,” said Perrault, who is trying to help the family find an alternative option.

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About $300,000 USD would likely be needed to cover Zoey’s cost of medication for 17 to 24 months if purchased south of the border, Jackson said.

That figure doesn’t include the cost of accommodations, testing and other expenses that wouldn’t be covered by insurance.

“We shouldn’t have to be medical refugees, going into another country,” said Jackson, who is also raising an 11-year-old son, Sawyer.

“We claim to have the best free health care, but do we have the best health care?”

In the U.S., Day One will provide tovorafenib – marketed as Ojemda – free of charge to “eligible families who don’t have health insurance or are underinsured,” according to the drug’s website.

It remains unclear when tovorafenib could be approved for sale in Canada.

Ipsen is preparing to file with Health Canada what’s called a new drug submission. It’s essentially the scientific case a company makes to Health Canada, which is responsible for approval of new drugs.

“Depending on Health Canada’s evaluation,” Bain said Ipsen would then work with Canadian provinces to seek reimbursement.

“We will continue working with different stakeholders, including Health Canada, to identify a regulatory path to make tovorafenib available in Canada in the future,” Ipsen said in an unsigned email to the Jackson family in early March.

In that email, Ipsen offered to speak with Zoey’s treating physician in the event that neither a clinical trial nor the purchase of the drug in the U.S. was an option for the family.

“We remain committed to helping where we can within the limits of existing regulations,” the email states.

After all her daughter has been through, Jackson said Zoey, who receives treatment in Halifax and has the support of her doctors there, is prepared to take a risk with a trial drug if it offers her a chance to extend her life.

“Hope is all we want – we’re not asking for guarantees – we’re just asking for some hope, and the pharmaceutical company and Health Canada is saying, ‘Sorry, we can’t give that to you.’”

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