Speaking before an advisory committee with the Food and Drug Administration last week in Silver Spring, Md., Michael Tuohy of Prospect said the side effects of cancer drugs don’t scare him.
“The alternative, quite frankly, is death,” Tuohy told members of the committee who voted 11-0 with one abstention to recommend approval of a medication called carfilzomib for people with a form of multiple myeloma that does not respond to treatment.
Multiple myeloma is an incurable cancer that affects blood cells in bone marrow. Tuohy, who turns 48 next week, was diagnosed with it 12 years ago. At any one time, there are more than 100,000 multiple myeloma patients undergoing treatment in the U.S.
Michael and his wife, Robin Tuohy, who directs support groups for the International Myeloma Foundation, each gave a three-minute testimony in support of the new drug.
“The reason I am so interested in this is because there is no cure for multiple myeloma,” said Michael Tuohy during a phone interview Tuesday. “There will be a time when the drug I am on now will not work for me. It’s such an individual disease so patients need to have different options out there.”
Tuohy, the father of two teenagers, has gone through remissions, relapses and a stem-cell transplant. A drug he started as part of a clinical trial has kept the cancer at bay for the past seven years. He said his health right now is good.
“I have been in a nice long remission, but in a sense, it’s like living with a time bomb and waiting to come out of remission,” he said. “Each time you come out, it’s more difficult to get it under control and that’s why we need these drugs.”
Robin Tuohy said a new multiple myeloma drug has not been approved in six years.
“So it was really time and imperative for carfilzomib to be available for people,” she said.
Members of the FDA advisory committee decided the drug’s promised benefits of controlling progressive disease in patients who have exhausted all other treatments outweighed its risks, which include toxic side effects to the heart, lungs, liver and other organs.
The FDA has designated carfilzomib as eligible for accelerated approval, which means it would be approved based on limited data, but its manufacturer, Onyx Pharmaceuticals, must conduct more studies.
In the meantime, the Tuohys continue to lobby for state and federal bills that give reimbursement parity for oral cancer drugs.
“Last year, we lobbied in Connecticut that oral and IV drugs should be reimbursed at the same rate. Connecticut was one of the first states to do that,” said Robin Tuohy, adding that they are advocating for the same measure on the federal level. “This is so important because newer oral drugs today work better and they are easier for patients than having to go sit in a chair and do an IV.”