"Gliber" from biohackers
Biohackers promised to create a "pirate" gene therapy a hundred times cheaper than the original
Polina Loseva, N+1
An international team of biologists gathered to copy a drug to correct a rare genetic disease – lipoprotein lipase deficiency. The original is called Glybera and costs patients a million dollars per treatment session. The "pirated" version, according to the creators' forecasts, will be able to cost less than seven thousand dollars. The MIT Technology portal was the first to draw attention to this story, and the authors of the project presented the pilot results the other day at the Biohack the planet conference.
In 2012, Glybera became the first gene therapy method approved for use in clinics in Europe and the USA. It allows you to correct the insufficiency of lipoprotein lipase. This enzyme breaks down complex fats (eg, triglycerides) into individual fatty acids so that cells can capture them from the surrounding fluid. In the absence of this enzyme, lipoproteins – complexes of proteins and fats - accumulate in the blood. When there are enough of them, they are deposited under the skin and in the walls of various organs. The result is disorders of the liver and pancreas, severe pain and diabetes. It is possible to cope with this disease only with the help of a strict diet, but it does not help to avoid some side effects, such as the inability to have children.
Glybera is a neutralized virus that carries a "healthy" lipoprotein lipase gene. Penetrating into the patient's cells, it is embedded in the genome, but it cannot reproduce, therefore it serves as a carrier of the desired gene. In 2015, it was still the most expensive medicine in the world – about a million dollars per therapy session. Now this palm has been intercepted by a new drug Zolgesma against spinal muscular atrophy, which already costs $ 2.1 million. But, having lost its leadership position, Glybera did not become more accessible. For several years, only a few dozen patients received this treatment, and in 2017 the drug was withdrawn from sale, considering that it did not pay for the costs.
An international group of biohackers led by Gabriel Licina told an MIT Technology correspondent about their plans to create a "pirated" version of the drug under the pilot name Slybera. Their plan looks much simpler than the original: they took a sequence of a healthy gene from open articles that were published during the development of Glybera, and sent it to a commercial laboratory that synthesizes DNA molecules to order. As a result, it turned out to be a ring DNA molecule with the right gene, which the authors of the project propose to use as an alternative medicine.
This plan has its pros and cons. The main plus is the price: according to Lisina and his colleagues, a dose of alternative gene therapy will cost less than seven thousand dollars. Biohackers plan to save on viruses, since their drug is not a neutralized virus carrier of a gene, but a gene sequence in its pure form. However, this may also be a minus – experts fear that such a drug may be much less effective: it will be worse to penetrate into cells and integrate into the genome. But this does not stop biohackers, they offer to compensate for low efficiency with a low price. "It's like if you wanted to dig a pool or pond," says one of the project participants. "You can buy an excavator and do it in one day, or you can take a shovel and do it in a few months for free."
This project may also face legal barriers. The FDA does not approve the sale of homemade drugs for gene therapy, and UniQure, the manufacturer of Glybera, may sue biohackers for infringement of intellectual property rights. However, this is still far away – at the moment Slybera exists only in a draft version, and at the conference "Biohack the planet" the authors of the project were going to announce a fundraiser to test their "pirated" version on animals.
Gene therapy is gradually gaining momentum around the world. They are trying to treat not only hereditary genetic defects, such as hemophilia, but also diseases for which the genetic cause has not been definitively proven, for example, Parkinson's disease.
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