The first drug to treat an aggressive form of multiple sclerosis has won approval from the Food and Drug Administration, a significant medical development with ties to the Tampa Bay area.
Local patients helped test the safety and effectiveness of the drug in clinical trials at the University of South Florida's Multiple Sclerosis Center, said Dr. Derrick Robertson, the center's director.
"We were one of the top enrolling sites in the country for this medication," Robertson said. "We have lots of patients who have been part of the science that led to this drug getting approved."
The 10 patients included Jeffrey Frank, a 39-year-old software engineer who lives in Tampa. Frank enrolled in the trial last year, and said the treatment has kept his condition from worsening.
"When I first got diagnosed, it really seemed like there wasn't a good drug treatment out there," he said. "This will be hugely helpful, especially for people who are newly diagnosed."
The drug, approved late Tuesday, will be sold under the brand name Ocrevus. It will be available to patients across the United States within two weeks.
"I don't think it is an understatement that this is a game changer," Robertson said. "This is really amazing progress for the development of therapies to help control a daunting, damaging disease."
Multiple sclerosis is chronic condition that occurs when a person's immune system attacks the nerve cells. It affects an estimated 400,000 people in the United States and has no known cure.
Common symptoms include muscle weakness and fatigue. Sometimes the disease affects vision and balance. In the most severe cases, it can lead to paralysis or permanent disability.
"What sometimes gets overlooked is that MS often afflicts those just starting their lives," Robertson said, pointing out how symptoms don't often start until people are in their 20s or 30s. "It's a gradual decline."
Drugs can help control the most common form of the disease, known as relapsing multiple sclerosis. But before the FDA action this week, there was nothing for the more aggressive primary progressive multiple sclerosis, which affects between 10 and 15 percent of patients.
Ocrevus is given through an infusion every six months. It works by "wiping out" certain white blood cells that can malfunction and direct parts of the immune system to attack healthy cells, Robertson said.
Studies published in the New England Journal of Medicine found the treatment helped slow the progression of the relapsing form of the disease, and, to a lesser extent, the more severe form.
That it helped at all in those cases is encouraging, Robertson said.
"It starts to open the door to how we can help that type of patient," he said. "They've been largely forgotten about and really difficult to treat because of the aggressive nature of their disease."
Patients in the trials were more likely to catch a cold after having the infusion, the studies found. But other than that, side effects were minimal.
Ocrevus isn't cheap. Its list price without insurance: $65,000 a year, according to the pharmaceutical company Genentech. But that's still 25 percent less than an existing drug to treat relapsing multiple sclerosis.
Frank, the Tampa software engineer, had tried other drugs after being diagnosed with relapsing multiple sclerosis in 2011. But the disease seemed to get worse, he said, leading him to believe he has the more aggressive form of the disease.
That brought him to the USF Multiple Sclerosis Center last year.
Frank went in for his latest infusion a few days ago, he said. His most recent MRIs showed the condition hadn't progressed since he enrolled in the trial.
"It's nice to see that there's something that has this much of an effect on the disease," he said.
He still has some difficulty getting around the office, and often lacks the energy to go out on weekends. "But as long as it's not getting any worse, I take that as a good first step," he said.
Information from the New York Times was used in this report. Contact Kathleen McGrory at firstname.lastname@example.org or (727) 893-8330. Follow @kmcgrory.