A California-based biotech company says it's successfully implanted lab-made neurons in Parkinson's patients' brains to stimulate a dopamine response — and if it works as intended, it could be a substantial advance in fighting the disease.
As MIT Technology Review reports, the early stem cell experiment, which was meant to test whether the procedure is safe, appears to have succeeded at that goal.
The trial included 12 people with Parkinson's, a debilitating progressive disease characterized by a shortage of dopamine, and was run by BlueRock Therapeutics, a subsidiary of the pharmaceutical giant Bayer. The lab-made neurons were implanted for a year before results were taken, and as researchers told attendees of the International Congress for Parkinson’s Disease and Movement Disorder in Copenhagen at the end of August, the implanted cells seem to have survived — and, in a particularly exciting twist, there are indications that they may be reducing the patients' symptoms, too.
Brain scans from the experiment's subjects show, the report notes, both an increase in dopamine cells and a decrease in times when the patients felt "off" or incapacitated by their symptoms. As Reuters noted, the study's leaders told the Copenhagen conference that the "off time" was lower for those patients who had been given higher doses of the experimental stell-cell neurons.
At this stage, the small trial's most significant success is its apparent safety, but University of California, Irvine neurologist Claire Henchcliffe, who led the study, told Tech Review that the ultimate ideal outcome is far loftier.
"The goal is that [the lab-grown cells] form synapses and talk to other cells as if they were from the same person," she said. "What’s so interesting is that you can deliver these cells and they can start talking to the host."
But according to Roger Barker, a University of Cambridge Parkinson's expert who was not involved in the study, the current results are a "bit disappointing."
Because researchers cannot see cells inside patients' brains once they're implanted, they injected them with a radioactive dopamine precursor that can be imaged on a PET scan during its uptake. Those scans showed some increases in dopamine, but as Barker noted, it is "still a bit too early to know" whether the transplanted cells actually began repairing patients' brains.
Nevertheless, "it is encouraging that the trial has not led to any safety concerns and that there may be some benefits," the Cambridge researcher told Tech Review.
While there is medication to help control Parkinson's symptoms, there is no cure for the progressive disorder that leads to increasing motor difficulties and causes tremors and limb rigidity. Cell therapy, like the one BlueRock is developing with the lab-grown cells, could radically alter the trajectory of the disease by replacing the broken neurons instead of just treating its symptoms.
"The potential for regenerative medicine is not to just delay disease, but to rebuild brain functionality," said Seth Ettenberg, the president and CEO of BlueRock. "There is a day when we hope that people don’t think of themselves as Parkinson’s patients."
BlueRock will now be moving forward with Phase 2 trials, and should they work, the test of the controversial regenerative technology may finally bring it further into the mainstream.
More on regenerative medicine: Harvard/MIT Scientists Claim New "Chemical Cocktails" Can Reverse Aging
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