Personalized Medicines

James Weintrub, a Providence, Rhode-Island, plastic surgeon and coding entrepreneur called today. He asked what I written about personalized medicine in my blog. James had seen Charlie Rose speak to Francis Collins, MD, America’s leading DNA and genome expert talk of the infinite prospects for personalized medicines, unique drugs tailored to each individual.

Nothing, I’m embarrassed to say. Genome-based medicines, or personalized medicine are the most eagerly awaited next step in the genetic revolution. The prospect of examining a person's entire genome to make individualized risk predictions and treatment decisions may soon be within reach.

It sounds logical that to have one-of-a-kind drugs tailored, customized, and targeted for each patient based on phenotypes, genetics, and environment. The excitement has grown since science completed sequencing the human genome in 2003. Many institutions are pursuing personalized drugs for each individual. Harvard and Duke, among others, are seeking just the right drug, for just the right person, at just the right time. You can find more about the quest for personalized medicines at (www.personalizedmedicinecoaltion.org)

But noble as these goals are, progress is slow.

• First, there’s scalability and sustainability. Developing drugs for individuals one-on-one is a very expensive proposition, and no one yet has come up with a feasible and affordable scheme to find the right patients and sufficient money to support the clinical trials necessary.

• Second, the results have been patchy and inconclusive so far – Herceptin for breast cancer patients whose genes express HER2 and Gleevec for patients with chronic myelogenous leukemia with certain genotypes.

It may be, of course, researchers will come up with a comprehensive broad database with magical algorithms to unlock keys to individual or cancer tissue drug responses. It may be genetic profiling will be routine in every clinical workup, and clinicians will diagnose and treat with pinpoint personalized precision, and computer stimulation can predict what personal drug to use for each individual and each cancer.

It will require cross-institutional research and massive clinical trials – both prohibitively expensive - to find why patients respond differently to the same drug. The differences may due to individual genetics for arthritis, hypertension, or high cholesterol, or to genetic variations in cancer tissues. Sorting out these differences will not be easy since each individual genome and each cancer tissue genome is made up of more than one billion genes. Because of expense and complexity, a broad breakthrough is not likely soon.

But research races are not always won by the swift, but to those plodders who work out the kinks, and sort out the drugs and the patients, one by one.