People with rare genetic mutations may offer solutions for prescription drug makers

By Alex Heigl
Updated July 29, 2015 04:35 PM
Credit: Moviestore/Rex USA

Steven Pete has a rare genetic disorder that doesn’t allow him to feel pain. He inherited two different mutations, one from each parent, that combined to form his condition.

Timothy Dreyer has another quality that wouldn’t seem out of place in the pages of Marvel comics: He’s one of about 100 people with sclerosteosis, a condition that makes his bones so dense, they can withstand damage that would break those of ordinary people. Like Pete, he wound up with his rare condition because of mutations from each parent that combined in his DNA.

Dreyer and Pete have another thing in common besides their odd conditions: Their DNA is extremely valuable to pharmaceutical giants like Amgen. Andreas Grauer, global development lead for an osteoporosis drug Amgen is developing, told Bloomberg Business that Dreyer and Pete are “a gift from nature,” adding, “It is our obligation to turn it into something useful.”

Companies pay big money to find people like Dreyer and Pete. Amgen bought out DeCode Genetics in 2012 for $415 million, and part of the deal included genetic data on 160,000 Icelanders. Genetic testing company 23andMe has deals with over 10 drugmakers, including Pfizer and Genentech, that one could reasonably assume involves sharing data on 23andMe’s 1 million customers.

But the payoffs dwarf the costs to companies. Amgen is conducting final-stage trials on osteoporosis drugs it developed after three and a half years of research that involved people like Dreyer. The profits from the proposed drug could net Amgen $1 billion to $2 billion in sales per year, analyst Eric Schmidt told Bloomberg.