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.
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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