Game Changer for the Human Genome Sector?

By Valorie Sands  AUG 31, 2012 10:30 AM

Hopes for a cure for Lou Gehrig's disease have emerged in recent trials where neural stem cells injected into the spine during experimental surgery have shown to slow the progression of ALS' devastating symptoms.


MINYANVILLE ORIGINAL The  long-awaited, even miraculous breakthrough represents a major evolution in stem cell research that could spur interest in the sector.

Current big pharma players in stem cell research include GE Healthcare (GE) where Stephen Minger, head of GE's R & D captured the evolving story: "When you see companies like Pfizer (PFE), GlaxoSmithKline (GSK), Johnson & Johnson (JNJ), and GE invest in stem cells and regenerative medicine, it suggests a level of maturity.  It is still high risk, but it is a calculated risk."

Pfizer and  Athersys (ATHX) have partnered to develop a stem cell therapy to treat inflammatory bowel disease (IBD).  Another Pfizer study is looking at a stem cell-based treatment for macular degeneration.  Stem cell studies for the treatment of diabetes and cancer are also underway at Johnson & Johnson in partnership with Novocell, Inc, a privately held biotech firm. 

"Nothing Short of Miraculous": Hope Looms Large on the ALS Horizon
Now the world watches as great news for the ALS community continues to emerge.  Up until now, 'hope' has never been a word associated with the disease.  Known to be progressive and fatal, it strikes with unrelenting ferocity, attacking a patient's nerve pathways to the muscles, until eventually a victim is left paralyzed and robbed of all mobility.  Fatality occurs when the muscles that control breathing can no longer function.
Yesterday the spotlight turned once again to patient Ted Harada as he went  home to recover from experimental breakthrough surgery that could halt, even cure, his ALS.  He continues to attract widespread attention.  To date, Harada's progress has been featured on CNN, CBS, FOX, Crain’s, Gizmodo, Newsweek magazine, and many other media networks.
Last year, in an interview with CNN, Harada said, “It’s been nothing short of miraculous. I cannot begin to explain the difference it has made.”
Mr. Harada, a 37-year-old father of three, is the third person this summer to receive as many as three doses of highly specialized neural stem cells.  The trials, conducted by Emory Univesity in Atlanta, GA,  utilize specialized neural stem cells injected into the spinal cord to provide enough support, by releasing growth factors, to prevent motor neurons from dying.
Harada's dramatic improvement could be the game-changing catalyst that will involve Big Pharma's serious interest in the stem cell therapy industry.  Up until now, concerns about safety and efficacy have deterred companies from entering the arena with more than only nominal investment capital.
The cells used in Emery's surgical injections are produced by a Rockville, Maryland-based company, Neuralstem (CUR), noted for its success in isolating embryonic stem cells from the brain and spinal cord. “These cells can nurture the dying motor neurons back to health, or make them healthier and slow down the degenerative process," says Richard Garr, CEO of Neuralstem. 
In his first surgery, Harada safely received 10 injections of the neurostem cells.  Each dose delivered  about 100,000 cells into the sides of his lower spinal cord.  Most encouragingly, post-op, he was able to move his limbs with strength and dexterity that surpassed his abilities before the surgical treatment.  Typically, some ALS patients do see occasional brief periods of small improvements or stabilization.   However, the degree of recovery reported by Harada and his medical team is considered amazing and is unheard of.  Still, in the last few months, Harada says his abilities have slowly been regressing, although at a slower pace than before the treatment.
Most ALS patients eventually die of respiratory failure.  So in this week's procedure, instead of injecting cells into Harada's lower spine, surgeons will place cells directly into his upper spinal cord, the anatomical region where the large nerve cells control breathing functions. Researchers hope the finely targeted treatment will protect motor neurons in the upper spinal cord and prevent or slow the loss of lung function.
"We have found that the procedure is extremely safe," says Eva Feldman, a neurologist at the University of Michigan and the lead investigator of the trial. "In a subset of patients, we seem to see that the disease is no longer progressing," but it is too early to know if the result from that small number of patients is meaningful,” she said.
In another ALS trial underway at the Mayo Clinic, a patient's own stem cells were  isolated from their own fat tissue and injected into his/her spinal fluid.  So far, two patients have undergone the procedure.
With  ALS, where the average survival is two to three years after diagnosis and it is uniformly fatal, investigators and the FDA think it's ethical to try these more desperate approaches that carry potentially higher risk," says Anthony Windebank, a neurologist who heads the Mayo Clinic trial. Still the FDA and the ALS community are watching these trial operation's very closely to gauge safety, as well as efficacy.
Researchers at Mayo are also awaiting conclusions on the trial procedures' safety, however once the procedure proves safe, one of the next steps would be to genetically modify the cells to produce specific growth factors likely to prevent motor neuron death, says Windebank. "If there's any sign of efficacy with these approaches, then translation into the clinic would happen very rapidly."
Neuralstem's cells are said to be unique because unlike most harvested cells, they have a defined fate. That may translate into greater success due to the fact that by taking cells from a fetus at a particular gestational stage, they can still divide and regenerate into a specific cell type, such as spinal cord cells. This unique property of Neuralstem's cells have also enabled the company to test potential drugs in relation to specific central nervous system cell types. It is also currently searching for drugs that can protect and nurture neurons from the hippocampus, a part of the brain critical for forming and storing memories, as well as targeting other central nervous system conditions.
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