When Robert “Buddy” Allen could no longer run after a Frisbee, he thought the cause was an old childhood injury -- when he got hit by a car. A neurologist, a chiropractor and an orthopedic surgeon all weighed in on Allen’s symptoms, which included tightness in his right leg and an overall stiffness in his body, but no one could pinpoint the culprit.



Then Allen, of Ormond Beach, came to UF Health Shands Hospital in Gainesville, and after several MRIs and a spinal tap, he was diagnosed with multiple sclerosis.

First came the walking stick, then the wheelchair. That was in 2005, and nearly 10 years later, Buddy, now wheelchair-bound, has gotten progressively worse.

“Time is not necessarily my friend,” said the 51-year-old. “I can’t really walk at all.”

After Buddy’s diagnosis, he signed up for Google Alerts on everything regarding MS -- especially treatment information. He shared that information with his doctor, Dr. Daniel Kantor, the director of the Comprehensive Multiple Sclerosis Center at UF Health Shands in Jacksonville.

“Dr. Kantor would actually talk to me about each one,” Allen said.

And then one drug emerged that got both Allen and Kantor excited. The drug itself was not actually brand-new -- once called Campeth, it was initially used as a cancer drug. But the parent company, Sanofi, repurposed the drug for MS patients, calling it Lemtrada.

Trials of the drug -- on both early-stage MS patients and those who had relapsed -- showed the drug’s efficacy. Most patients’ disability scores were stable or improved, and more than 50 percent of patients were relapse-free, said Kantor, one of the trials’ lead investigators.

“I’ve seen people where their MS had been almost melted away” by the drug, Kantor said.

Scientists think the drug works by resetting the immune system.

“When you have MS, the body attacks itself,” Kantor said. “If you reset the immune system, you can put MS into strong remission.”

In 2013, the drug was approved in several countries throughout the world -- including the EMA, the European Medicines Agency), which is the FDA’s counterpart in Australia, Canada and Mexico.

But the FDA rejected the drug in December, saying that the studies to test the drug’s efficacy were flawed.

The FDA hadn’t actually liked the study design from the start, Kantor said. Originally designed as a double-blind, placebo-controlled study -- which compares the trial drug to a placebo, and makes sure that neither the patients nor the investigators know which is which -- the researchers instead replaced the placebo with a standard drug called Rebif. The patients knew which drug they were taking, while the investigators did not.

“The patients knew because they would’ve known anyway,” Kantor said.

He added that the FDA, given its disagreement with the trial design, could have stopped the trial. Instead, the FDA let the trial go on.

“How could they allow the study to go on if there was no hope that it was going to be approved?” Kantor asked. “From an efficacy point of view, there was no question that (Lemtrada) worked.”

In response to the FDA’s decision, MS patients around the country started a petition that so far has more than 7,700 signatures. The goal is 10,000.

Meanwhile, the FDA’s decision has created what Kantor calls “two worlds of MS patients: People in other countries doing well, and people in the U.S. being sick.”

“That’s what we’re setting ourselves up for,” Kanto added.

Already, websites have popped up advertising treatment trips aligned with cruises for sick Americans, Kantor said.

“People are gonna go with rogue companies who will take them on cruises to get them their medications,” Kantor said. “It’s to me unconscionable that we’re not going to have this medication … that patients will have to go to Mexico to get it.”

“They say you can go to Mexico to get it, but you don’t even drink the water in Mexico,” Allen added.

But any place an American would go for the drug poses safety concerns, Kantor said, simply because taking the drug involves constant monitoring that would be hard to coordinate.

And going abroad for treatments is also too costly for most patients to consider. Allen said he couldn’t afford it. He already struggles to pay for his biweekly physical therapy and acupuncture sessions.

“None of those are covered by insurance,” Allen said. “I’m afraid to stop any of it.”

As for Lemtrada, “We’re sitting around waiting,” he added.

There is some hope, however, that the wait time might get shorter than anticipated. Two weeks ago, Sanofi announced it would resubmit its application for the drug to the FDA -- instead of its initial reaction to file an appeal. The decision followed “constructive discussions” with the FDA, according to news reports.

“We’re all very hopeful,” Kantor said. “It’s at least a good sign that we’re moving forward.”

“I am just hoping for something ... a more regular day,” Allen added. “I would love to walk around my yard; go for a walk in the woods; actually hold my wife’s hand and walk down the street.”

Labels: Lemtrada