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Decades ago, “personalized medicine” meant “don’t give penicillin to the person who is fatally allergic to it.” Today, the phrase is shorthand for the ambitious but achievable concept of targeting medications to a specific group of people, based on genetic information, disease progression, biomarkers, and other factors. Still, there’s a small but growing force in the biomedical community that takes the notion of “personalized medicine” much further. For them the term is used literally—they aim for treatment options custom-crafted for the unique snowflakes that we are.
The dream of individualized treatment is a non-starter under current economic and regulatory models for drug development. Estimates of how much it costs on average to bring a drug to market—from earliest drug discovery to assembling teams of experts to review pricey clinical trials—range from $1 billion to about $5 billion. It’s expensive in terms of time, too: 10 to 15 years to successfully launch an individual drug. To put that in depressing perspective, the time and money required for each new therapeutic undertaken by a conventional pharmaceutical company is roughly equivalent to what it took to complete the Human Genome Project.
Naturally, people seeking to get away from the blockbuster drugs of big pharma also envision getting away from its intrinsic costs. Veterans of drug development often reply that a new therapeutic costs just as much and takes just as long whether it works for one person or 1 billion people; the cheaper-faster-better mantra hasn’t made a dent in their industry.
Through that lens, attempts to develop custom-tailored medications or treatment plans for a single person may seem quixotic. But if some new approaches prove successful, we all stand to benefit.
Andrew Hessel’s Pink Army Cooperative is a nonprofit biotech organization founded in 2009 to design completely customized, virus-based cancer treatments—and then provide them at no cost, so long as patients agree to share medical data about their experience with the community. Each treatment is based on a sample of that patient’s cancer cells, which are screened against possible therapies to find ones that eradicate cancer but leave healthy cells intact. Hessel spoke at the recent Techonomy Bio conference about his ideas.
Another example is a company called N-of-One (the name is a reference to the number of people in a clinical trial, usually referred to as “n”; an n of 1 means there’s only one person being studied). Launched in 2008 and also focused on cancer, N-of-One works with medical providers and diagnostic companies to deliver custom profiles and interpretations of each patient’s tumor. That information is merged with data about therapies, including those in ongoing clinical trials, to design a tailored treatment plan for the patient.
In hospitals and clinical research institutes, the custom approach has gotten a boost from animals called “avatars.” These organisms are developed to mirror a person’s tumor or disease, to evaluate the effects of treatment options and study disease progression. Fruit flies can be injected with cells from a patient’s tumor and then exposed to various medications to see which ones shrink that tumor. Just a handful of these fly avatars have been made across the globe, but promising results might turn them into a mainstay of personalized medicine. Mice have also been avatars: On a mission to understand his young daughter’s one-of-a-kind syndrome, clinical geneticist Hugh Rienhoff worked with scientists to recreate her particular genetic variant in mice. In addition to helping assess treatment options, these mice can shed light on how Rienhoff’s daughter’s disease may progress in the future.
Even if all of these efforts succeed, though, it remains unclear how regulatory agencies will respond to individualized treatments. Many public health officials have been champions of personalized medicine (check out this recent blog post from the CDC’s director of the Office of Public Health Genomics), but good intentions are unlikely to make for a smooth transition from current requirements. Clinical trials, for instance, are a necessary cornerstone of the FDA’s review process; but therapies for just one person by definition cannot be tested on lots of other people to ensure they’re safe and effective.
There is no doubt that personalized medicine is gaining traction in the biomedical industry—but just how personalized treatments will get, and how soon, remains to be seen. In the meantime, stakeholders around the world should be joining the conversation, lobbying legislators and regulatory agencies to pave the way for individualized treatments, and educating themselves about alternatives to traditional drug development. Only if demand soars, and resources follow, will these upstarts get their big chance.