Many new cancer drugs don't save lives

Hello and happy Saturday! Here's this week's roundup of eclectic and under-the-radar health and medical science news. If you haven't subscribed yet, you can do that by clicking here.

When researchers in the U.K. took a close look at the effectiveness of new cancer drugs approved by the European Medicines Agency between 2009 and 2013, they discovered that only about half showed "a significant improvement in survival or quality of life."

The finding published in the BMJ follows a study in JAMA last March that also found less than half of the new cancer drugs approved in the U.S. and Europe between 2003 and 2013 increased overall survival by more than three months. And many of the drugs have toxic side-effects.

It's been clearly shown that "progression-free survival" does not predict ...overall survival.- Dr. Christopher Booth, oncologist, Queen's University.

What does this research mean for patients? "That's the million-dollar question," said Queen's University oncologist and researcher Dr. Christopher Booth.

"We've always thought in medicine that any medical intervention should help people live longer and better lives, which means any new treatment in a clinical trial should be shown to increase survival and improve quality of life."

The problem is that many trials evaluate the success of the drug using "surrogate endpoints." That means instead of measuring whether patients live longer, they measure whether their cancer progresses or how many patients experience tumour shrinkage. It's sometimes called "progression-free survival."

"It seems counterintuitive," Booth said. "But it's been clearly shown that 'progression-free survival' does not predict or is not associated with overall survival."

Booth said it raises challenging questions for oncologists and regulatory agencies about whether it's acceptable to expose patients to very small gains for very toxic and expensive drugs.

"Our group is starting to do some work with patients to find out what they value," Booth said.

PM directs new health minister to crack down on extra-billing

It's illegal for Canadian doctors to charge user fees and extra-bill patients. That's what Prime Minister Justin Trudeau reminded Ginette Petitpas Taylor as she assumed her role as Canada's new health minister.

In a mandate letter, Trudeau has added a new priority for Petitpas Taylor — to "promote and defend the Canada Health Act to make it absolutely clear that extra-billing and user fees are illegal."

Trudeau has ordered his new health minister to work with the provinces and territories to "improve verification and recourse mechanisms when instances of non-compliance arise."

The prime minister has made it clear that user fees are not acceptable.-Statement from the office of Ginette Petitpas Taylor, minister of health 

But how are user fees and extra-billing defined? And is Ottawa prepared to hold back transfer payments to provinces if there are violations? These are questions we asked the new health minister who replied with this statement:

"The prime minister has made it clear that user fees are not acceptable, and that Canadians should have access to health-care services on the basis of need — and not ability to pay. Our government will always defend this principle."

"We are committed to working with provinces and territories to end extra-billing practices."

So is the menu of items and their prices posted at the office of one Canadian family doctor recently (see above image) an example of extra billing? It depends, said health-care policy analyst Karen Palmer, with Simon Fraser University.

"If you look at the law, anything that is a barrier to obtaining medically necessary hospital and physician care can be considered a violation," she told us. "So this is a matter of interpreting whether something is a violation."

Meanwhile Ottawa and B.C. are taking a closer look at extra billing in that province.

"We are now involved with the province of British Columbia in an audit of extra-billing practices," her office said in an email.

The audit follows a Globe and Mail investigation into extra billing by some of that province's doctors.

Scientists discover the brain's drain

It's time to rewrite the neuroscience textbooks, after one of the basic tenets of neurology was overturned this week, opening up a whole new area of research into diseases of the brain.

This week a team of researchers at the U.S. National Institutes of Health revealed they had discovered the brain's drain pipes. For years experts insisted the brain and the vessels of the lymphatic system were not directly connected.

This is a confirmatory study that proves the dogma of 10 years ago isn't true.- Dr. Jason Karamchandani, neuropathologist, McGill University

(Lymphatic vessels are similar to veins and circulate white blood cells and cellular fluid.)

"It was dogma that there was no lymphatics in the brain," said McGill neuropathologist Dr. Jason Karamchandani, who was not involved in the research. "It's amazing to see in 10 years how we went from challenging dogma to confirming the actual truth."

The brain is constantly creating waste that needs to be cleared, but scientists were never sure how exactly that happened.

Then in 2015 a team injected dye into the brains of mice and watched as it drained into vessels and then into the neck. They reported their findings in the journal Nature, and when Dr. Daniel Reich saw the discovery, he wondered if he could see the same thing in humans, using MRI.

After a series of experiments and post-mortem studies of brains, he found what he was looking for.

"We very quickly saw what I thought were lymphatic vessels, but it took us two years to fully convince ourselves," Reich told us. And once he identified, for the first time, how the human brain drains into the lymphatic system, he had to make a decision — to tell or not to tell.

He considered holding off on the news until he had done enough research to offer some explanations about the relevance of the finding. But instead he decided to publish the finding immediately so other scientists could start asking questions.

Already scientists are calling him asking about how they can implement his technique.

"I hope we do find that this is important to human disease," he said. "I think that the lymphatic vessels and their dysfunction might contribute in important ways to diseases of the brain that involve inflammation and buildup of toxic proteins."

The next step is to take a close look at exactly what kind of communication is happening between the brain and the immune system through the lymphatic vessels. At McGill University, Karamchandani says the discovery opens up new areas of study.

"We probably underestimate the role of the immune system in many neuropathologies including neurodegenerative pathologies like Parkinson's, ALS and Alzheimer's," he said.

"This is a confirmatory study that proves that the dogma of 10 years ago isn't true, and it's a novel way of linking the immune system and the central nervous system."

The mystery of the shrimp with the big brain

Researchers have long been trying to recreate the evolutionary story of the brain.

Primitive creatures like sea anemone and corals have basic nervous systems, neurons that are connected.

"But it's very hard to look at their nervous systems and say it was a 'brain,''' said Wayne Sossin from the Montreal Neurological Institute, who studies the molecular mechanisms of memory in Aplysia, a type of sea slug.

"The first reason to have a nervous system was to co-ordinate movements, to have fast movements, to be able to connect the outside environment to the constriction of muscles," he said. "The first time there were predator/prey interactions where speed was essential."

Eventually creatures developed clusters of neurons organized into structures that were more brain-like. But those evolutionary links are hard to see.

That's why scientists were interested when a team of U.S. and Australian researchers reported the curious finding of an insect-like neurological structure called a "mushroom body" in the brain of a mantis shrimp, a creature known for its sophisticated vision and expert hunting skills.

The mushroom body is believed to be important for memory. But how did the mantis shrimp develop this brain structure?

"They prove very nicely that these shrimp have a mushroom body. But they're left with a quandary at the end," Sossin said. The neural structure might have been passed down from an ancestor, but was only preserved in certain creatures, like the mantis shrimp, which needed it for vision and hunting.

But the researchers also argue that it might have evolved independently, because it just happens to be a good way to design a brain — something scientists call 'convergent evolution."

The authors admitted that they found both possibilities equally fascinating. "We are obliged to conclude that there is, as yet, no definitive conclusion," they wrote.

"I like the convergent argument much better," Sossin told us. "The idea that brains that have to do a common task find common solutions throughout evolution, suggesting that there are networks that are better at doing things than others, and that evolution will come to the same solution, is a more interesting thing to think about."

"People have made analogies between the mushroom body and the hippocampus region in our brains that is important for storing locations and making memories," Sossin said. "So it suggests that evolution will reach this same solution multiple times."

"It would suggest that this would be a good, good architecture for neuronal networks," Sossin said, a finding that could  have implications for designing artificial intelligence systems.

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