Cancer patients who have exhausted their treatments are hoping a growing practice of personalized medicine can be their last hope.
Cancer is a difficult ailment to cure because it often mutates and can become resistant to treatments. That is why scientists have started looking at drugs that aren’t approved for a specific cancer.
Mount Sinai Hospital in New York is doing a pilot study of about 100 patients of multiple myeloma. The patients in the trial have multiple myeloma and have been given most of the existing treatments for that specific type of cancer.
The concept is to take a patient’s tumor and do an entire genome sequencing of that tumor. Then researchers compare the results to an existing database of drugs to see if anything might work on that specific tumor’s mutation.
The database doesn’t sort drugs by the type of disease it treats such as heart disease or diabetes. Instead, it sorts the drugs by the mechanism of action. For instance, you can look for a drug that doesn’t treat breast cancer, but instead treats a specific mutation that may be in another type of cancer, said Dr. Steven Burakoff, director of the Tisch Cancer Institute at the hospital.
The pilot study already has seen some promising results.
After analyzing a tumor, researchers found that another type of drug used to treat melanoma could work for a patient. The patient was given that drug and has been in remission for four to five months, Burakoff said.
“We don’t know how long that will last,” he told the Washington Examiner.
This type of treatment is called precision medicine, in which treatments are more tailored for a specific patient. President Obama’s budget included $200 million in extra funding for the National Institutes of Health to pursue personalized medicine projects.
A big reason why personalized medicine could become a reality is the low cost of genomic sequencing.
Burakoff said the first human genome was sequenced for more than $1 billion and the process took several years. Now it can be done for about $1,000 and performed in a matter of weeks.
Mount Sinai isn’t the only center studying such a method. Another study in Seattle is developing tests for treatments for pediatric brain tumors.
A trial in 2010 used a malaria drug alongside a newer drug to treat lung cancer.
While repurposing drugs shows promise, there appear to be several caveats to the idea.
A big one could be cost. Since a repurposed drug is being used for something it was not approved for, it is considered “off-label” use.
This is a bit of a gray area in healthcare. Doctors are allowed to prescribe a drug for an off-label use, but insurance typically doesn’t cover it.
Pharmaceutical companies are not allowed to promote off-label uses for their products, but the Food and Drug Administration has recently showed a willingness to relax its oversight of the practice.
However, patients also could save money by getting a more cost-effective treatment.
“Instead of giving them drugs indiscriminately, we will have data that will allow us to use medicine in a more cost-effective manner,” said Dr. Samir Parekh, associate professor of hematology-oncology at the Icahn School of Medicine at Mount Sinai.
Pharmaceutical companies that own the drug may not be willing to pursue approval for a new indication since it might be difficult to get a new patent, Burakoff said.
Parekh said the result of the trial is to use the same genome sequencing process for patients that come in and have had little success of getting rid of their cancer.
“The hope is that we can make a greater and more specific panel of drugs for treating patients, particularly those patients that are out of options,” he said.
