Neuralstem Inc. is planning to make its phase II cell therapy for treating amyotrophic lateral sclerosis (ALS) available in Colorado, after the state became the first to enact right to try legislation, designed to give seriously ill patients access to novel, non-approved therapies.

"This is something the entire industry will have to deal with," said Richard Garr, CEO of Neuralstem. "Patient access is becoming a really big issue in the U.S., with patients going to social media to demand they get treatments."

The existing mechanisms for providing expedited access to products in development are really inadequate to the need. The FDA sanctions compassionate use about 100 times a year in ALS, whereas there are 5,000-6,000 new diagnoses per year.

"I have 5,000 emails from people diagnosed who want to get into our trial," Garr told delegates.

As it is, Neuralstem, of Rockville, Md., has treated 30 patients to date in clinical trials. "Clearly a different tool is needed to address patient access," said Garr.

Garr has worked with the Goldwater Institute, which has a stated mission of protecting freedom, in advancing the right to try legislation. On May 17, John Hickenlooper, governor of Colorado, became the first to sign this into law.

The law states that people diagnosed as having a fatal disease can be treated with an unlicensed product, as long as it has completed a phase I safety trial and is in phase II. The treatment must be prescribed by a physician. Similar legislation also has been passed in Missouri and Louisiana, and Garr said he expects 25 states to have taken it up by the end of the year. (See BioWorld Today, May 22, 2014.)

The measure has received support from across the political spectrum. "No one has voted against," Garr noted.

Neuralstem will start to train surgeons in Colorado to use its NSI-566 human spinal cord stem cells, which are administered by a series of lumbar and cervical injections. In animal models these cells made synaptic contact with the host motor neurons and express neurotrophic growth factors, which are protective of motor neuron cells.

"It will take time to put together a team," Garr said.

Under right to try, companies can charge for their products, setting their own prices. The law does not allow for reimbursement through the Centers for Medicare & Medicaid Services.

It will not be possible to use data from patients treated under right to try as part of a regulatory package, and Garr said that in that sense the scheme is the worst of both worlds.

"We will follow patients if we can; if something bad happens we will have to tell the FDA; but these patients cannot be part of a trial," he said.

In the phase I trial, NSI-566 was safe and well-tolerated when injected up to 15 times, at doses of 100,000 cells per injection. None of the 18 patients deteriorated over two years, in a disease that is usually fatal within two to four years. One patient was much improved.

The phase II 15-patient dose-escalation trial started in September 2013. The last of five cohorts has started, in which patients will get up to 40 injections, of 400,000 cells per injection. Preparations are in hand to start a phase III trial in 2015.

The influence of social media is highlighted by Garr in his blog on Neuralstem's website, where he points to four patients in the ALS trials who have written blogs about their experiences. For Garr, this is positive.

"The ALS community is best served by this free flow of information," he writes. The patients, "should be applauded for telling their stories and bringing hope where precious little exists."