Saturday, August 2, 2008

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.

2 comments:

RiverPoet said...

I'm waiting for nanotechnology to solve some of these issues. My husband follows it closely, and he is especially up on Kurzweil's theories. My sister and I are two of those frustrating patients who seem to develop allergies and rare side effects to most drugs. There are very few we can take, especially her.

Due to severe lymphocytic colitis, she can't take antibiotics without serious risks. At 57 with lupus, she's afraid an infection is going to be the end of her. I would love to see something developed to help her. I'm still hoping.

Peace - D

Unknown said...

Personalized medicine seems to be the latest in mass-customization. Unfortunately, we need a regulatory structure that allows for the reduction in testing that allowed many of our cheaper medicines to be approved in the pre-blockbuster era.

How exactly does one justify medicines optimized to small populations if the total addressable market won't pay for the combination of development and risk?

I think personalized medicine will first be deployed as personalized care and treatment. Optimizing care for individuals with needs that also include those outside of medicine will improve the rather poor showing we have with real-world engagement or "adherence". Its no wonder in the era of visits 3 weeks out, with an hour wait, for a 5 minute conversation. After all, nobody really worries about Google adherence or "Coca-cola adherence". Making the medical system fit and optimize against segmented consumer needs-- that will be an interesting, and nearer term, journey.