Cancer drug is the first potential therapy to emerge from 23andMe and GlaxoSmithKline collaboration

The pharmaceutical giant GlaxoSmithKline is starting human trials of the first medicine, a cancer drug, that has emerged from its two-year-old collaboration with consumer genetics firm 23andMe.

The novel partnership focuses on using 23andMe’s massive genetic database, composed of the test results and self-reported health data from 12 million consumers who have taken its tests to learn about their ancestry and a smattering of disease-related genes, and who have said their samples could be used in research. GSK uses the database to try and validate genes and proteins that could be what researchers call targets for new drugs — that is, the proteins and other molecules whose functions medicines block or otherwise change.

The new experimental drug is an antibody targeting the CD96 receptor, and is aimed at enabling the immune system to attack cancer cells. The receptor is expressed on so-called natural killer cells, and the drug is designed to keep tumors from attaching to the receptor to thwart the tumor-killing actions of the natural killer cells. It has entered phase I trials, with the first patient dosed earlier this month. GSK will announce the development as part of its quarterly earnings call this morning.

Anne Wojcicki, the co-founder and CEO of 23andMe, called the step “a major milestone” for the company. “We’ve always had this goal of helping our customers benefit from the human genome,” Wojcicki said. “And I think this collaboration with GSK has really accelerated that.”

23andMe began working in drug discovery in 2015. It and GSK now say they are working on 30 different research projects involved in their collaboration, up from eight when it began two years ago.

The effort is “exceeding, I think, everyone’s expectations,” said Hal Barron, GlaxoSmithKline’s chief scientific officer. In addition to using 23andMe’s genetic database to evaluate potential drugs, the companies are continuing to use it to help identify patients who could participate in clinical trials, Barron said.

The 30 targets span oncology, cardiovascular disease, and metabolic diseases, Barron said. “We just put forth the utmost compelling targets for the most significant unmet medical needs and [based on] the tractability of the target in terms of creating a drug,” Barron said.

A “stretch goal” for the collaboration was to start testing a drug based on the collaboration, Barron said. The 23andMe database might not seem an obvious fit for cancer research, because mutations that drive cancer are often not inherited and instead arise during a person’s life. But Barron and Wojcicki said that it contains data from many patients who have happened to get cancer, and that genetic differences in the immune system were something that could be probed by 23andMe’s proprietary algorithms.

Unlike newer technologies, like whole genome DNA sequencing, the tests run by 23andMe provide only limited information about how people differ on very specific genetic markers — think of it as skimming the genome quickly instead of reading every word. And the self-reported data might be incomplete.

Wojcicki pointed out that electronic medical records can have errors, too. And Barron, who pointed to the power of a “massive, huge, biggest-in-the-world data set,” recounted one example where genetic markers that were thought, based on other genetic studies, to indicate whether people were morning people or night people correlated tightly with the results in the 23andMe database.

One criticism leveled against 23andMe is that using data from its customers to collaborate with a large pharmaceutical company might in some way violate privacy or that drug companies should not profit off of consumers’ curiosity about genetics. But Wojcicki, who points out that 23andMe is not yet profitable, said that this perception misunderstands the desires of people who purchase its product.

“I have found over years of talking to people who are critically ill that the number one thing people want is, they say either, I want there to be some kind of treatment for me, or I want to understand whether or not this is going to impact my family,” Wojcicki said. “And part of the reason why we’re set up the way we are is we want to give our customers the best chance possible of either living a healthy life or of having their information used to [develop] a treatment.”

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