Neuralstem wades into murky water with plan to offer unapproved therapy

Thanks to Colorado's recently passed "right-to-try" law, patients in the state with terminal diseases can test out therapies yet to receive FDA approval, and Maryland biotech Neuralstem ($CUR) plans to make available its treatment for amyotrophic lateral sclerosis, or Lou Gehrig's disease.

As CEO Richard Garr told BioCentury, Neuralstem is yet to decide whether it will charge for the stem cell therapy, but it is already in the process of training Colorado surgeons on how to administer the treatment. In a Phase I study published in Annals of Neurology this year, implantation of NSI-566's human spinal cord stem cells demonstrated to be safe, and, per Colorado law, that's all that's necessary to offer the treatment to ALS sufferers in the state.

When the bill passed last month, many figured drug companies would avoid taking advantage of it in fear of endangering the regulatory futures of their in-development drugs. States can pass any bills they like, but the FDA still regulates drug development. However, as Garr tells BioCentury, he believes any right-to-try administrations of NSI-566 are parallel to the drug's ongoing Phase II studies and won't affect its trial protocol.

However, whether this will truly benefit patients is an open question. Without mid-stage efficacy data, it's unclear whether NSI-566 has any positive effect on ALS.

Neuralstem's move comes as little surprise considering Garr's role as chairman of the libertarian Goldwater Institute's Right to Try National Advisory Council, and the broader effort is a rejection of the current "compassionate use" system, which has come under fire in recent months. As it stands, patients can petition the FDA for access to unapproved drugs, but if the treatment's developer gives it out to one patient, it must do the same for any other valid askers, an expensive prospect that is often impossible for venture-backed biotechs on a budget.

Meanwhile, similar bills are up for vote or gubernatorial signature in Louisiana, Missouri and Arizona, and they've enjoyed some public support. In Colorado, right-to-try backers dubbed the legislation "the 'Dallas Buyers Club' bill," borrowing the name of an Academy Award-winning dramatization of AIDS patients' efforts to obtain unapproved therapies in the 1980s.

- read the BioCentury story