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Top 10 experimental cancer drugs - 2013

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Human colon cancer cell--Courtesy of Annie Cavanagh, Wellcome Images

With the help of open-minded regulators, pharma companies have ditched the traditional march through three phases of clinical trials for some cancer drugs. Trials of targeted drugs offer rapid proof of efficacy against molecularly defined malignancies, giving the authorities a basis for approvals before late-stage studies to prove that therapies prolong the lives of patients.

In our third annual report on 10 exciting cancer drugs, there are plenty of R&D programs zipping through trials with the blessing of the FDA, which has awarded "breakthrough" status for expedited development to treatments in oncology more than any other field. Take Novartis' LDK378, one of the featured treatments in this report. In March the next-gen ALK inhibitor joined the "breakthrough" club amid Phase II trials, and Novartis ($NVS) said it would seek FDA approval next year with only three years of development.

This explains why we've dropped "late-stage" from the title of this report, as several featured programs are in midstage development.

Oncology is the most crowded area of drug research. So the shorter development cycle helps limit companies' risk of chasing the same or very similar targets as a bevy of other companies, wagering on their program to come out on top. Novartis might have the frontrunner with LDK378, but it is one of no fewer than four experimental compounds against ALK in lung cancer. These drugs home in on misfit proteins in an ALK-positive subgroup that represents as little as 3% of the NSCLC population.

The FDA is opening the inside track to more genetic therapies in response to overwhelming patient need, as the efficacy of the first wave of gene-targeting drugs fades after clever cancers adapt and find new pathways to grow out of control. Roche ($RHHBY) and other companies also face patent expirees to existing drugs, and the Swiss drugmaker has mounted an effort to make one of its best drugs, Rituxan, even greater with a glycoengineered successor called GA101.

Targeted or not, none of these drugs is a silver bullet, and patients must still endure side effects. Yet there's hope for some cancers to become manageable chronic illnesses as opposed to death sentences. Bristol-Myers Squibb ($BMY), Merck ($MRK) and others in this report have also advanced drugs based on discoveries of how to restore natural immune attacks on cancer. In fact, three immunotherapies that travel the programmed death pathway to kill cancer have made our top 10 this year.

Please read the report and share your thoughts on the programs by tweeting links to the report along with the hashtag #10CancerDrugs. Feel free to tweet about these programs during the ASCO meeting, which begins this week. -- Ryan McBride (email | Twitter) | John Carroll (email | Twitter) | Emily Mullin (email | Twitter)

For more:
FDA oncology chief Pazdur vows to accelerate 'breakthrough' drug R&D
ASCO confab highlights immunotherapies, 'breakthrough' drugs and small victories
Top 10 Late-Stage Cancer Drugs – 2012
10 promising late-stage cancer drugs - 2011