Researchers plan trial for Lou Gehrig's disease therapy
The mice at the top of a column of stacked plastic bins at Q Therapeutics are shivering so hard they seem to be jumping.
Their nonstop shivering and seizures are caused by a genetic defect that robs the mice of the crucial myelin sheath that surrounds nerve cells and helps them send signals. Because of the defect, the mice are soon paralyzed and die prematurely.
It is a related problem -- loss of this myelin sheath -- that in humans causes the progressive loss of function in multiple sclerosis and several other diseases that can cause paralysis in humans.
And that's why what has happened to the mice is so promising: After being treated with an adult stem cell therapy developed at Q Therapeutics, they are no longer shivering.
The product, called Q-Cells, also may be applicable to such neurodegenerative diseases as Parkinson's, Alzheimer's and amyotrophic lateral sclerosis, or ALS -- better known as Lou Gehrig's disease.
Now, the National Institutes of Health have awarded a $5 million grant to Q Therapeutics, the University of Utah's Cell Therapy Facility and Johns Hopkins University School of Medicine, which as a team has had success in animal models of ALS.
The funding will help support work needed to get permission from the Food and Drug Administration to start human clinical trials at Johns Hopkins. If efforts to raise additional funds are successful, those trials would begin next year.
"This type of therapy can bring about a major change in modern health care," said Deborah Eppstein, CEO of Q Therapeutics. "It's not just a little step. It's a pole vault change, a going to the moon change."
About 30,000 people in the U.S. have ALS, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
As the cells deteriorate, they become incapable of sending signals to move muscles that control speech, breathing, limb movement and other functions. Respiratory failure leads to death, typically within two to five years of diagnosis. Around 6,000 Americans contract the disease each year.
A genetic defect is responsible for less than 10 percent of cases; the rest have an unknown cause. The disease is diagnosed in military veterans at twice the rate as the rest of the population, Eppstein said, but researchers don't know why. Speculation includes exposure to toxins and active duty related stress.
"One in 800 deaths of all causes in the U.S. is due to ALS," said Eppstein. "I find that number staggering."
To treat neurodegenerative diseases, some researchers are focused on replacing damaged nerve, or neuron, cells, which Eppstein calls a much more difficult challenge. Q Therapeutics has taken a different approach, based on replacing cells that repair and support those neurons, potentially slowing -- and even preventing -- the progression of such diseases.
"If you can restore the health of a neuron before it dies, you don't have to worry about forming a new neuron which requires making all the proper connections," Eppstein said. "That is why we think these cells, Q-Cells, can treat a wide range of neuro-degenerative diseases."
Q-Cells, identified by company co-founder Mahendra Rao, work like emergency first responders.
"They migrate and when they find diseased tissue, they differentiate into these mature support cells and start doing good things," Eppstein said. "They are doing everything that needs to be done by interacting with the local environment. If nothing is diseased right now, they may sit in a quiescent state. If they encounter diseased cells, they can start to repair them."
The researchers will test whether a single treatment of Q-Cells injected into the spinal cord could provide enough support cells to protect neurons in people with ALS and slow the disease's progression.
"In humans, we would hope to achieve a few years increased survival, and potentially additional treatments could provide still further benefit," she said.
Their initial efforts also are targeting transverse myelitis, a devastating autoimmune disease. A single episode can so badly damage neurons that a person is paralyzed in a matter of hours to days.
Unlike ALS, about a third of those patients recover, but another third are wheelchair dependent after a single attack, Eppstein said. In transverse myelitis, Q-Cells would be used to restore myelin.
"Our plan is to look at studies in this group and after initial success, then go into other demyelinating diseases like MS," Eppstein said. "Q-Cells have the potential to treat the whole gamut of things that cause degeneration in the central nervous system."
Q Therapeutics was founded in 2004 by Dennis Farrar and Mahendra Rao. Rao identified what the company named Q-Cells, cells that support and repair neurons. The company is using Q-Cells to develop a therapy that holds promise for treatment of amyotrophic lateral sclerosis -- Lou Gehrig's disease.