High-tech health care only for the ultra-rich? B.C. toddler’s case spotlights inequality of gene therapy

Researchers have made incredible advancements in recent years treating rare illnesses—but what happens when there isn’t enough funding to pay for them?

When Gurmoh Gill is playing with his favourite dump truck in his favourite park, there’s no sign the Surrey, B.C., toddler is experiencing mobility loss due to a rare neurodegenerative disease that could be cured — at a steep price.

Researchers at McGill University’s The Neuro institute are ready to take treatment for Gurmoh’s spastic paraplegia-4 (SPG4) from the lab into clinical trials and the treatment room, but they need up to $8 million to make that a reality.

With every provincial government running a considerable deficit and desperately stretching each tax dollar to serve as many patients as possible, it’s likely only the federal government has the resources to contribute to life-altering treatment.

The three-year-old’s walk is already stilted and his parents only recently got the devastating diagnosis after seeing several doctors who’d been unable to determine why he had trouble standing when he was a year old.

“(SPG4) is going to keep on damaging his nerves and then they said eventually he will lose his ability to walk,” said his mom, Navpreet Gill. “Then there are cases where literally everything like arm (usage), speech, mental capacity — it will take everything away from him.”

Not an isolated case

There are hundreds of Canadians diagnosed with SPG4, the most common type of spastic paraplegia, with youngsters having the best chance of responding to gene therapy since the erosion of their mobility is still in its early stages.

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From a cost-benefit perspective, the more patients who would benefit from trialling and developing the treatment, the more sense it makes for government to invest in the process. Whether leaving patients paraplegic or quadriplegic, the degeneration of their nerve fibres will add considerable cost to the public health-care system without treatment.

“We have the research, we have the technology, we have doctors, researchers, and everybody is saying we have everything set up ready,” said Gurmoh’s dad, Stalin Gill. “All we need is the governments or the big organizations to help utilize those developed technologies.”

The family has launched a fundraiser to try and crowdsource the initial funds in the hope the federal government will step in once the process is underway.

Who should develop the treatments?

The federal government’s agency for funding such pediatric research, RareKids-CAN, is bombarded by requests from families desperate for cutting edge technology to cure their youngsters, or just halt the progression of their illness.

The treatments are essentially tailor-made, since they’re so rare, which sounds “a little science fiction, but it can be done,” according to Dr. Massimo Pandolfo, a McGill neurologist and researcher at The Neuro.

He explained that there are many sub-types of spastic paraplegia that would each require their own clinical trials and testing, even though the underlying technology is the same.

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“Obviously, this is quite expensive and usually the way to go has been to try and find support via foundations, via fundraising,” he said. “I think the appropriate public control on this things is the way to go (because we) cannot allow and we cannot accept astronomically high prices.”

Pharmaceutical companies are profit-driven and focus their research dollars and efforts on developments that are most likely to return their investment, which is why there’s comparatively little work done on conditions that affect a small portion of the population.

That essentially makes effective but costly gene therapy the domain of the ultra-wealthy for the years it’ll take for the costs to come down and become more manageable for public health-care systems and insurers.

That injustice is felt deeply by affected families, including the Gills.

“It’s just the money holding it up right now,” said Navpreet. “No child should be deprived of a cure or a treatment just because of money.”