Gregory A. Hall

The (Louisville, Ky.) Courier-Journal

LOUISVILLE, Ky. -- Kentuckian Rita Wooton has tearfully shared the story of son Eli's frequent seizures — and the promise of marijuana oil to treat it — with dozens of people, ranging from doctors to journalists to legislators.

Earlier this year, her story helped pers

uade the Kentucky General Assembly to unanimously — and somewhat speedily — pass a bill approving the first medicinal use of marijuana in Kentucky.

But now doctors and researchers are saying it could take years to begin trials treating epileptic children such as Eli with marijuana oil.

"We're really, really heartbroken," Wooten said. "It's just really sad that everyone put that much time and effort and energy into it and now it's going absolutely nowhere. For people like us and Eli, who have intractable epilepsy, seizures that are nearly impossible to even control or get a handle on, our hope is gone."

Some of issues stalling the trials are the availability of marijuana oil, the possible need for approval from the Food and Drug Administration and the cost of studiesthat preliminary estimates say could be $10,000 per participant.

Still, doctors hoping to conduct the trials are optimistic.

"I am extremely excited about it," University of Louisville Dr. Karen Skjei, who specializes in pediatric epilepsy, said, citing anecdotal reports of the medicine reducing seizures where traditional medications, diets and surgery can't. Supporters say the oil, administered under the tongue, can provide relief to children who have severe cases of epilepsy. The oil contains low amounts of THC, the perception-altering ingredient in marijuana.

"I suspect that it probably won't live up 100 percent to the hype," Skjei said, but for some, "it could be a life-changing experience. ... (F)or these kids who have no other options, we have to remain optimistic."

The new law allows trials of marijuana oil at the Universities of Louisville and Kentucky medical schools or through FDA trials.

Winning legislative approval for the bill is "just the initial part of the puzzle," saidDr. Christopher Shafer, who specializes in adult epilepsy.

Shafer and Skjei said there will be no problem finding participants for the trial they are working to develop at the University of Louisville. Shafer said three of the five patients he saw in one day last week wanted to try the medication.

"Dr Skjei and I want this for our patients, probably, almost as badly as the patients want it themselves," Shafer said. "And it's really discouraging for us to be not be able to tell them that we have it available. ... It's going to take some time."

A University of Kentucky spokesman expressed the same concerns.

"While there is certainly interest in this initiative, there are significant issues that remain to be addressed," spokesman Jay Blanton said. "Additional research, the securing of funding and support, as well as support from the appropriate regulatory body, all would be required before a trial could be conducted. That process could likely take months, if not years."

Details of the Louisville trial are still being determined, but researchers say it would involve several phases, including one where doctors would give half of the patients the oil and the other half a placebo before switching.

The final phase of the study would be another year where all the patients get the oil.

The new state law doesn't specify age limits on patients but testimony before the legislature focused almost exclusively on young children because severe seizures in children are so much worse than in adults, Skjei said.

Children "can be so incredibly debilitated by their seizures. ... and sometimes these kids don't even make it into adulthood. ... We are seeing the sickest of the sickest on the pediatric front lines that the possibility of something to help these children is just incredible," she said.

Currently, marijuana oil isn't being manufactured in Kentucky, and there are numerous hurdles to consider when shipping it across state lines.

Discussions are ongoing with pharmaceutical companies and others about possibly setting up a Kentucky dispensary, Skjei said.

"As of right now, there's really nowhere to get it," she said.

Then there's the issue of the FDA.

Skjei said she and Shafer hope the FDA would accept existing data related to multiple sclerosis and other disorders regarding the safety of marijuana oil that would clear the way for the advanced trials Louisville is considering. Skjei said trials could be done without the FDA approval, but doctors would have to have the oil.

Lastly, there's the cost, which Skjei said is dependent on the study's structure, timing of phases and number of subjects. If the study had 60 patients, Skjei said, "then that's an awful lot of money right there."

"I feel their desperation," Shafer said, .

Skjei said, "I still have patients that are talking about moving to Colorado," where marijuana is legal, "because they understand it's going to be a while before this is up and running,"