Personalized Medicine's Meaningful Impact

Personalized Medicine's Meaningful Impact

11.29.12 | By Gretta Stone

This week I'm in Boston attending the Harvard Personalized Medicine Conference. (Find out more on the basics of PM here.) Stepping back and listening to the discussion here, it is really inspiring to think about how our understanding of genetics is opening up vast new possibilities for treating and preventing disease. We are already seeing great strides in basic science, diagnostics, and new treatments.

One big question is how personalized medicine (PM) has impacted drug development. A quick audience response system showed that 84% of the more than 550 experts here from across the health care spectrum believe that PM is having a meaningful impact on drug development, echoing a 2010 survey by Tufts University which found that 94% of companies are investing in personalized medicine.

On a panel yesterday, researchers from three biopharmaceutical companies discussed how personalized medicine is changing drug development and the ups and downs of recent development programs. A new personalized cystic fibrosis (CF) drug provided a great example of PM in action. Researchers started with a daunting 1,700 mutations associated with this rare genetic disease. They grouped the mutations and used a detailed understanding of the cellular basis of the disease to perform high throughput screening, which yielded a compound, later called Ivacaftor. Researchers were able to select a very specific subset of patients with this type of genetic mutation and move efficiently through clinical trials and regulatory approval. The medicine was approved earlier this year and can be used in about 4% of CF patients.

In cases like this, researchers are seeing faster, more precise development processes, but they still need much more genetic knowledge to take a personalized approach in more disease areas. Ninety-seven percent of the audience today said that personalized medicine has the potential to impact drug development even more. I look forward to coming back to Harvard again next year and hearing how much closer we are to achieving the full potential of personalized medicine.

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