BIG TIME CHANGES FOR CLINICAL TRIALS:

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The treatment of cancer is changing dramatically. The new drugs target a cancer gene fundamental to tumour growth. This is known as the molecular target.

Many authorities see this as the start of the genetic age in cancer research and personalized cancer treatment.

One big change is that clinical trials of new cancer drugs will involve patients with many types of cancers. The source of the cancer ( breast, lung, ovary) is less important than the molecular target that is identified.

Normal cells have a mechanism that tells them to die if their DNA is too badly damaged to repair. The p53 gene instructs the damaged cell to die.

Cancer cells which have very damaged DNA do not die because p53 has been turned off. Cancer cells disable p53 with either a genetic mutation or with a blocking protein.  This blocking protein is MDM2.

Researchers have found an alternative route to activating p53 by inserting a small molecule to separate p53 and MDM2. By separating p53 and MDM2, p53 is activated.

As reported in the New York Times ( Dec 23 2012), Dr Debussche from Sanofi had focused his research on p53 for over 20 years. Recently, his team found a molecule that will activate p53.

Clinical trials of this new molecule will involve patients with many types of cancer. These cancers might range from liposarcoma to acute myelogenous leukemia (AML). This is a big change for clinical trials.

Patient advocacy groups (PAGs) will have to change their approach. For decades they have sought funding for their disease based on tissue type: breast, lung, colon, ovary, neuro-endocrine, etc.

Now with many types of cancers in one clinical trial and with the focus on molecular targets and not tissue types, the PAGs will have to change their approach and will be wise to collaborate with other PAGs.

If they act together to lobby for more funding for research on molecular targeting and for new drug development, then they will be great advocates for their members.