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May 12 2011

Conference Recap – Biomarker World Congress 2011

 

Biomarker World Congress 2011

Image via Biomarker World Congress 2011

Everywhere in the news today, we hear that we are in the era of personalized medicine – where new medicines will be tailor-made for individual patients.  Specifically, personalized medicine is dedicated to the concept hat the right drug is administered to the right patient at the right dose at the right time.  To get a pulse on the state of the personalized medicine today, I attended the Biomarker World Congress 2011 (02-04 May 2011, Philladelphia, PA); the conference where the frontlines of research and development of personalized medicines are presented and discussed among experts in the field.

 

In this era of personalized medicine, the physician designs the tailor-made treatments for the patients based on results of diagnostic tests.  These tests measure specific biomarkers within the patient that provide critical information on the molecular basis of the disease.  Biomarkers may be DNA, RNA, protein, metabolites, or even cells.  At this Biomarker World Congress, there were two major themes:

  1. Development of a diagnostic test takes more time to develop than originally thought
  2. Adoption of personalized medicine in real-life medical practice today has been slow.

Developing a diagnostic test for biomarkers is more challenging than originally anticipated.  More time is required to validate the diagnostic test and for the execution of a clinical trial with a large enough sample size to reach sufficient statistical power to attain regulatory approval of the diagnostic test.  Large pharmaceutical companies have found that development of the companion diagnostic test is often not in sync with the development of the drug – development of the diagnostic test often lags behind.  Pfizer and Novartis have realized that developing companion diagnostic devices for these innovative drugs cannot simply be “crow-barred” into the traditional scheme of drug development; it has been thought that development of the diagnostic test should begin at Phase 2a.  Now it is known that this timing is too late.

Genentech was the first company to market a personalized medicine (Herceptin and Herceptest for breast cancer in 2006) and has learned not to underestimate the task of developing a companion diagnostic.  Moreover, Genentech had the advantage of working with, and now being a part of Roche, a major device company.  Genentech’s current corporate strategy is to start companion device development early – instead of beginning at Phase 2a clinical trials, begin at-risk during Phase 1 clinical trials.

The second theme of the Biomarker World Congress was that personalized medicine has not been fully embraced by physicians in the real world.  The issues are:

  • Is the information from the diagnostic giving the physician enough information to act?
  • Is personalized medicine superior to exiting standard-of-care and does the cost of personalized medicine warrants its use?

Three case examples were discussed.  Irinotecan, approved by the FDA for the treatment of colon cancer, results in neutropenia as an unintended side effect in ten percent of the population.  This sub-population has an allele (UGT1A1P*28), that can be identified by an FDA-cleared genetic test.  Oncologists experienced with Irinotecan “know’ how to use the drug by empirically reducing the dose upon observation of severe neutropenia.  Physicians are accustomed to treating neutropenia and other cancer drug side-effects; in fact, the neutropenia associated with Irinotecan is often used as an indicator of efficacy.  Consequently, this diagnostic test has not been widely utilized.

A second example where adoption of personalized medicine has been slow is Oncotype Dx.  Oncotype Dx is a test that predicts the probability of relapse after surgery for breast cancer – it is intended to aid the oncologist to decide whether or not the patient should receive adjuvant chemotherapy in addition to surgery and hormonal therapy.  In using Oncotype Dx, there is a small statistical chance that the test may indicate not to treat a patient who, in reality, may benefit from the treatment.  Oncologists are wrestling with the consequences of the “wrong” decision and the legal implications.

In the third example, a diagnostic test for the enumeration of circulating tumor cells (CTC) has been cleared by the FDA.   In patients with metastatic breast cancer, a CTC count of 5 or more per 7.5 mL of blood is predictive of shorter progression free survival and decreased overall survival.  Dr. Minetta Liu of Georgetown University has found concordance with imaging and CTC in the prediction of progression-free survival in patients with metastatic breast cancer, with CTC being more sensitive and less expensive than conventional imaging methods.  Furthermore, she has educated her patients on the value of the CTC biomarker and has used it in her real-life clinical practice.  Based on CTC measurements, she has advised her patients on the scheduling of “drug holidays” from chemotherapy; this change in her current clinical practice has lowered cost as compared to imaging and has given an enormous improvement in her patients’ quality of life.

This last example demonstrates the potential fruition of personalized medicine.  Let’s check in next year at the Biomarker World Congress to see whether the research and development of biomarkers, drugs and diagnostics would yield more improvements in medical care.

This is a post by Will Lee, Ph.D. Will is the Director, Regulatory Affairs at Cato Research.