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Some cancer patients may benefit from being treated with “off-label” drugs that are not routinely prescribed for their exact tumor type, according to new research conducted as part of the Drug Rediscovery protocol. Findings were published in Nature September 30, 2019.



“There are potential opportunities for patients to benefit from drugs that already exist, and it may not actually be necessary to develop new drugs. Therefore, you can start testing these existing drugs almost immediately – that was the basis for starting this study,” explains Emile Voest, senior author of the study.



The Drug Rediscovery protocol – An adaptive, precision-oncology trial

To be eligible for the trial the cancer patients had to either:

or No longer benefit from a standard anticancer treatment

Have a cancer for which no such treatment is available or indicated.



Eligible patients with a potentially actionable genetic or molecular variant were matched to one of the “off-label” anticancer drugs in the study.



“The good news is, is that there are already many, many drugs on the shelf that are approved by the [US Food and Drug Administration] (FDA) or [European Medicines Agency] (EMA). For the drugs that have been developed, we know that certain drugs work in certain tumor types. By having a national program of large-scale sequencing, we identified that 13% of patients have a molecular target where we have such a drug,” says Voest.



What does “off-label” mean?



Before a drug is approved and made available to patients, it undergoes rigorous preclinical and clinical testing to confirm that it:

is effective at treating a specific medical condition

works the way it is expected to

is safe to use



Evidence of this testing is submitted to the regulatory authorities (e.g. the US Food and Drug Administration (FDA)) which is then considered, and approval is granted if they are satisfied with the information provided. The drug is then given an official “drug label” – defining its approved indication, recommended dosage, and method of administration.



When a drug is given to a patient in a format that deviates from the drug label it is said to be “off-label” use.



Findings from the first 215



“Every patient with metastatic cancer, deserves to have diagnostic work or whole genome sequencing performed to make sure that we can identify old targets that are potentially helpful to the patient,” say Voest.



Driving precision medicine forward



The Drug Rediscovery protocol aims to identify early signals of activity in these patients, accelerate the clinical translation of these insights into the use of drugs outside of their drug label, and create a repository of data that can be used to guide future treatment decisions.

Emile Voest, PhD, was speaking to Laura Elizabeth Lansdowne, Science Writer for Technology Networks.