First U.S. stem cells transplanted into spinal cord

Miriam Falco, CNN Medical News Managing Editor, January 21, 2010

In September 2009, Neuralstem Inc., a Rockville, Maryland-based biotech company, was granted approval from the U.S. Food and Drug Administration to conduct a safety based clinical trial in patients with ALS using stem cells derived from human fetal neurons.  The company recently announced the successful treatment administration in the first patient enrolled in the clinical trial.  At Emory University doctors injected neuronal stem cells that were derived from 8-week-old fetal tissue into the spine of a man in his early 60s who was suffering with ALS.  Stem cell administration was performed in the lumbar region of the spinal cord, the area that controls leg function.  This is because the majority of ALS patients first lose muscle function in their legs.  The injection procedure was uneventful, and according to the press release, this was the first use of stem cells for direct intraspinal cord injection in the United States. 

"We are entering a new era of cell therapeutics for ALS, and in my opinion, it is an new era of hope for patients with ALS," said veteran ALS researcher and University of Michigan neurologist Dr. Eva Feldman, who is acting as Principle Investigator of the clinical trial that is expected to treat 12 patients. 

"This is the first study to see if the invasive injection into the spinal cord is safe for the patient," said Lucie Bruijn, science director of the ALS Association.  She continued to state that there have been some small trials performed outside of the United States, but that this is the first of its kind to receive FDA approval.  She continued, "Our biggest hope for stem cells is to significantly slow the progression the disease".

ALS is a disease characterized by progressive loss of motor function, whose causes are not completely understood but are believed to be related to neuronal excitotoxicity.  To date the only drug approved for treatment of ALS is Riluzole, which has been demonstrated to delay the onset of ventilator-dependence or tracheostomy in selected patients and augment survival by approximately 3 - 5 months.  While Riluzole seems to inhibit progression of ALS to a minor extent, it has no effect on the processes associated with regeneration after the injury has been induced. 

The ability to use fetal derived stem cells for treatment of ALS has some problems besides the obvious ethical issues.  Firstly the cells may need to be matched with the recipient, or if not, immune suppression has to be administered.  Secondly, the cells need to be expanded in large quantities.  While it is relatively straightforward to grow cells in the setting of an Academic Laboratory, difficulties in generating large enough numbers of cells that retain their therapeutic properties has always been a dilemma. 

Being the first FDA study for neural stem cells, it is anticipated that this approval will help to accelerate the field of cellular transplantation.