The US Food and Drug Administration (FDA) said the first clinical trial to test a new stem cell treatment for the fatal neurodegenerative disease ALS (amyotrophic lateral sclerosis) this month (ALS) can go ahead.

The federal agency gave the green light earlier this month, allowing principal investigator and neurologist Dr Eva Feldman, the DeJong Professor of Neurology at the University of Michigan (U-M) Medical School to start Phase 1 testing of the safety and efficacy of an investigational new drug (IND) from Neuralstem, a company based in Rockville, Maryland.

The drug, which comprises the company’s own patented neural stem cells, is administered as a series of injections at different sites along the spinal cord.

Feldman, who directs the ALS clinic at U-M, and the U-M Program for Neurology Research & Discovery, worked with Neuralstem to develop the protocol for carrying out the injections.

The trial is expected to take place only at Emory University in Atlanta, Georgia, subject to approval from its Internal Review Board. If the board gives its approval, the site principal investigator of the trial is expected to be Dr Jonathan Glass, director of Emory’s Neuromuscular Laboratory and also its ALS Center which has a world-wide reputation.

Feldman told the press earlier this month that they were very excited to be starting the trial:

“This is a major stride forward in what still could be a long road to a new and improved treatment for ALS,” she added.

Feldman explained that ALS is a terrible disease that kills the patient by paralysis. She said work with animals showed that spinal cord stem cells worked in two ways: they protected motor neurons that were at risk and they also made new connections between them and the neurons controlling muscles.

“We don’t want to raise expectations unduly,” said Feldman, “but we believe these stem cells could produce similar results in patients with ALS,” she said.

The trial will study the safety of Neuralstem’s cells and the surgical procedures and devices required for multiple injections of the drug directly into the grey matter of the spinal cord.

Also known as Lou Gehrig’s disease, ALS affects about 30,000 Americans, with about 7,000 new diagnoses a year. The disease gradually destroys neurons or nerve cells that control voluntary muscles to the point where eventually patients can’t move or even speak.

There are currently no known treatments that can slow the progression of the disease.

For the first phase of the trial, which is being paid for by the drug company, Feldman and colleagues will be treating 12 of 18 recruited patients with varying degress of ALS. The 12 patients will receive between five and ten injections of stem cells into the lumbar region of the spinal cord. They will be examined regularly for up to two years after the operation at which point the data will be reviewed.

If the results are favorable, the drug will still have to undergo Phase II and Phase III trials and then win final FDA approval before the treatment is available to the public.

Richard Garr, Neuralstem’s CEO and President, told the press that:

“While this trial aims to primarily establish safety and feasibility data in treating ALS patients, we also hope to be able to measure a slowing down of the ALS degenerative process.”

He declared confidence in Feldman and Glass and their team, saying “there is no better team to conduct this study for us”.

In pre-clinical work Neuralstem’s cells have reportedly extended the life of rats with ALS and reversed paralysis in rats with Ischemic Spastic Paraplegia (there were two studies about this work, one in collaboration with Johns Hopkins researchers was published in Transplantation in 2006, and the other with researchers at University of California San Diego was published in Neuroscience in 2007.)

Neuralstem said that its patented technology enables for the first time, neural stem cells of the human brain and spinal cord to be made in commercial quantities, and it also controls the differentiation of the cells into mature, physiologically relevant human neurons and glia cells.

In addition to ALS, the company is hoping to use its new technology to target other major central nervous system diseases such as Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury and Huntington’s disease.

In a press statement, U-M said Feldman has no financial interest in or financial arrangement with Neuralstem.

Sources: UMHS Newsroom, Neuralstem.

Written by: Catharine Paddock, PhD