The Cancer Problem: What Needs to Be Done

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Cancer has become the leading world-wide cause of death from disease, according to the World Health Organization.

Mendelsohn.jpgSignificant progress is being made against cancer. The five-year survival rate for all forms of cancer combined has risen to 66%, more than double what it was 50 years ago. And the cancer death rate has been falling by 1% to 2% annually since 1990.

MD Anderson President John Mendelsohn, M.D., is a thought leader on what needs to be done to achieve more progress against cancer. Last year, he proposed 10 steps that can be taken to ensure cancer deaths decrease more rapidly, the ranks of survivors swell, and an even greater number of cancers are prevented in the first place.

This is the first in a series of posts on key actions outlined by Mendelsohn:

1. Focus therapeutic cancer research on targeting abnormally functioning genes and their products.

Cancer is a disease of cells that have either acquired or inherited abnormalities in critical genes and the proteins for which they code. Because most cancers involve several abnormally functioning genes -- not just one -- this makes understanding and treating cancer a complex challenge.

The good news is that screening for mutations and other abnormalities in genes and their products can be done with new techniques that accomplish in days what once took years. The costs of these tests, while not inexpensive, are falling rapidly.

This has made it possible to conceive of approaching cancer treatment in a far more rational and effective way.

Increasingly, a patient's cancer will be analyzed in real time (7-10 days) to detect the abnormalities in genes (such as mutations, extra copies, altered expression) and their products (such as malfunctioning proteins), along with abnormalities in the critical biochemical pathways that control cell proliferation and survival.

The results of these tests will be used to select experimental or approved therapies that target the particular genetic and molecular abnormalities in that patient's cancer.

We expect that responses to treatment will be far more likely to occur, and to be more effective, when therapies are selected on this basis. The data from a recent landmark clinical trial of four drugs, each targeting a different gene mutation in four groups of patients with lung cancer, presented by MD Anderson at the 2010 American Association for Cancer Research meeting, strongly support this expectation.

In most situations today, such an evaluation of a patient's cancer prior to prescribing treatment is considered research. Research during the next few years will aim to demonstrate efficacy of this approach so that it will become standard of practice for providers, patients and payors.

This new paradigm has major implications for drug discovery and the pharmaceutical/biotech industry. The pipeline now has more than 800 new drugs and monoclonal antibodies in clinical development, targeting many of the candidate genes and gene products that are likely to be causative in human cancer.

If clinical trials with these targeted agents are carried out after pre-screening cancers for the presence of the abnormalities that they target, clinical investigators can test them with patients whose cancers are more likely to respond to treatment. This has been demonstrated successfully at MD Anderson and other institutions in recent studies.

In addition to improving outcomes for patients, this will result in huge savings in time and dollars, because efficacy (or lack of efficacy) of an experimental treatment can be determined with smaller numbers of patients and trials.

Next: Develop better tests to predict cancer risk and enable earlier detection of cancer.

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