Right To Try Act Would Allow Greater Access To Experimental Treatments

ANNAPOLIS – In August 2010, after seeing a doctor about a pain in his left leg, Ted Harada was given a grim diagnosis: ALS, or Lou Gehrig’s Disease, with two to three years to live.

“I’ll never forget what my doctor told me,” Harada said. “‘There’s no cure. There’s nothing you could have done to get it. There’s nothing you could have done to not get it.’”

In the months after, Harada began walking with a cane, he said, and the pain spread to his right leg.

On Thursday, five and a half years after his diagnosis, Harada walked on his own into a Senate committee meeting in Annapolis after undergoing an experimental treatment for ALS that could be made more accessible to terminally ill patients under the Right to Try Act.

Maryland’s bipartisan-supported Right to Try Act would allow doctors to administer experimental treatments not yet approved by the FDA to terminally ill patients who sign a consent form.  Similar Right to Try laws have already been passed in 24 states, Kurt Altman, a lawyer with the Goldwater Institute, said Thursday.

Currently, patients can only undergo experimental medical treatments in clinical trials or as part of the FDA’s expanded access programs. Otherwise, they will have to wait the eight to 10 years it takes for a drug to go to market, said state Sen. Bryan Simonaire, R-Anne Arundel.

“Patients don’t have time,” Simonaire said.

With a disease like cancer, which spawns around 1 million new patients a year, 40,000 of those patients will apply to clinical trials for experimental drug treatments, Simonaire said.

But clinical trials are “costly, cumbersome and time consuming,” said Simonaire, and only about 3 percent of patients who apply are accepted.

Companies running the clinical trials set specific restrictions on who they will accept, Altman said, and patients “have to be sick enough, but … not too sick.”

The FDA’s expanded access programs accept a majority of their 700 to 800 yearly applicants, but that still leaves thousands of cancer patients who tried applying for experimental drug treatments with clinical trials and were not accepted, Simonaire said.

Some patients resort to traveling to Europe for non-FDA approved treatments, said Simonaire, but not everyone can afford the airfare.

Harada, who lives in McDonough, Georgia, was accepted into a clinical trial for a stem cell treatment conducted by Neuralstem in Germantown. He met the drug company’s trial requirements, based on applicants’ ages and progressions of the disease, he said.

In the months after his first treatment in March 2011, Harada already began regaining strength in his legs, he said.

“Every night I prayed this wouldn’t just go away when I woke up,” he said.

He now travels state to state as a public speaker, sharing his story and advocating for Right to Try legislation.

“I’m very passionate about speaking for those that can’t speak for themselves, and that’s the terminally ill,” Harada said. “I’ve been given hope, and I want everyone to have the same hope I’ve been given.”

The bill, sponsored on the House side by Delegate Karen Lewis Young, D-Frederick, would grant patients access to medical treatments that have passed the first phase of the FDA’s three-part drug testing process.

The first phase of testing is usually conducted on small groups of volunteers and is primarily concerned with how the treatment is metabolized in the human body, including the treatment’s side effects and toxicity, said FDA spokeswoman Sandy Walsh.

“During phase one, there’s very little known about safety and dosing,” said Walsh.

The experimental drugs covered by the bill are by no means a guaranteed treatment, Altman said, but they can give patients hope.

“These patients know where they are, they know that this isn’t a magic cure-all. They want some control at this stage in their life,” Altman said.

Under the bill, patients would have to sign a letter of informed consent indicating their understanding of their diagnosis and of the treatment.

The Right to Try Act would require manufacturers to provide the experimental drugs at no profit, whether for cost of production or for free, and insurance companies would not be required to cover the treatment.

The bill would provide patients another option for treatment, said David Fishkin, a 28-year chiropractor in Montgomery County.

“Do you wish to die in the usual FDA-approved way, or do you want to fight to live?” he said.

–CNS correspondent Eliana Block contributed to this story.