A team of maverick scientists is trying to build a bootleg version of a million-dollar drug

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Last weekend in Las Vegas, Gabriel Licina sat on the edge of a stage at the Biohack the Planet 2019 conference and held up a small package filled with vials of bacteria containing a pirated copy of a human gene.

He offered to give a free sample to anyone with access to a biology lab who was prepared to take his nascent gene therapy and run with it.

"This was developed in a shed in Mississippi, a warehouse in Florida, a bedroom in Indiana and a computer in Austria," he told the audience of self-described biohackers. They're science enthusiasts who experiment with biology outside the scientific mainstream, often working in home laboratories and sometimes testing their inventions on themselves.

"Step zero is to not inject yourself with this thing," Licina told the audience."Please, for the love of god, stop stabbing yourself."

Licina doesn't like the term "biohacker." He has a degree in molecular biology and considers himself an independent biologist. "We're not just random chumps here," he said.

He calls his creation "Slybera," a play on the original name of "Glybera," the world's first approved gene therapy, which also became one of the world's most expensive drugs.

The saga of Glybera was the focus of a special CBC News report that explored how a Canadian medical breakthrough that was 30 years in the making became the world's most expensive drug — and then quickly disappeared.

• The million-dollar drug | CBC News Interactives
• The million dollar drug that patients can't access| The National

Glybera was developed by Canadian scientists to treat a rare but devastating disease called lipoprotein lipase deficiency (LPLD). The disease affects about one or two out of every million people, but in the Saguenay region of Quebec, as many as one in 50 people are carrying the gene mutation.

Patients with the disease are unable to process dietary fat. Their blood turns white from the accumulation of fat molecules. And the disease causes devastating and potentially fatal attacks of pancreatitis.

Even though Glybera was shown to be effective in preventing the pancreatic attacks, the breakthrough treatment was abandoned after only two years on the market because patients could not afford the $1-million US price for a dose.

They are comfortable watching people suffer and die because of money.- Gabriel Licina, Scihouse Inc.

Glybera was sold only once, to a German woman, after her doctor fought to have a private insurance company pay for it. A few remaining doses were given away for one euro each. Then the drug was pulled from the market.

The Dutch company, uniQure, which still holds the Glybera patent, did not respond to CBC's request for a comment on the biohacking activity.

The idea that this treatment worked but is not available because of a corporate financial decision infuriates Licina.

"That's super f--ked-up," he said. "They are comfortable watching people suffer and die because of money." 

Licina runs a non-profit community laboratory called Scihouse, in South Bend, Ind. The Slybera project started about two months ago when he got a call from a fellow scientist in Austria who suggested that they try to reverse engineer Glybera.

Using the original gene sequence that was published in the scientific literature, Licina and his colleagues spent $300 to have a professional genetic lab generate a copy of the DNA. And when Licina tested his genetic prototype in mammalian cells, it started working, producing the LPL protein that is deficient in patients who suffer from LPLD, he said.

None of this work on Slybera has been published in peer-reviewed literature. Instead, Licina is offering the genetic material to anyone with access to a biology lab who can take the discovery further. 

He estimates he's spent about $7,000 on Slybera. The long-term dream, he says, would be to make a drug that patients can afford. 

"There's absolutely no reason that Glybera should have cost that much," he said. 

In my opinion, biohacking is the symptom and the consequence of a huge societal concern.- Dr. Daniel Gaudet, LPLD specialist, University of Montreal

Dr. John Kastelein is a Dutch scientist who was part of the team in British Columbia that developed Glybera. He told CBC News in an email that the gene therapy is "completely not suited for a biohack" because it is "too complex," particularly with patents involved.

"Testing the end product [is] extremely difficult, so all in all, a hopeless initiative."

Dr. Daniel Gaudet is an LPLD specialist who conducted the original Glybera clinical trials at his clinic in Chicoutimi, Que., which is home to one of the largest populations of LPLD patients in the world. He understands the impulse to provide an affordable drug. He said biohacking is a response to an unsustainable situation.

"In my opinion, biohacking is the symptom and the consequence of a huge societal concern," Gaudet said. "How can we assure, in the future, access to new, safe and effective treatments in the precision medicine era?"  

But he, too, believes gene therapy is too complex for a biohacking approach. 

"Bringing their solution to the patient is another world," said Gaudet. "The process is extremely complex. It involves guarantees of safety and efficacy."

As for the abandoned drug, Glybera?  

"Glybera was too expensive, no doubt," Gaudet said. "For Glybera, it's over."

That means there are no available treatments for LPLD. But Gaudet says a next-generation gene therapy for LPLD is in development along with several other new treatments for the rare disorder.

And he is also working on strategies to ensure patients will have access to those new drugs at an affordable price once they are on the market.

"From the patient's perspective, knowing that drugs exist that can be very important for saving your life but they are too expensive, it's terrible."

• The million-dollar drug | CBC News Interactives
• The million dollar drug that patients can't access| The National

Meanwhile, back in his community laboratory in Indiana, Licina said hopes that someone else who has the ability to scale up his bootleg version of Glybera will bring it to patients through the appropriate regulatory channels.

"We're not really interested in breaking any laws," Licina said. 

"Nobody can make any money off of it, but you might do something good," he told the biohackers at last weekend's conference, as he offered them a chance to be part of his project.

He gave away about ten samples of Slybera and now he's waiting to find out if anyone comes back with any data. 

"Hopefully, if we can get enough people who are willing to make good data, then we can find some people that are willing to do good with that data."

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