ALS Stem Cell Trial Gets FDA Go Ahead

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ALS Stem Cell Trial Gets FDA Go Ahead29 Sep 2009

http://www.medicalnewstoday.com/articles/165528.php

<http://www.medicalnewstoday.com/articles/165528.php>

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, land.

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 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.

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 s 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.