Dec. 15, 2015
Australia has been chosen to host a world-first trial of revolutionary stem cell therapy for Parkinson’s disease.
The trial, by the Californian-based International Stem Cell Corporation, will be held at the Royal Melbourne Hospital.If first phase is successful, larger trials will start in Australia, Europe and the US in the next couple of years.
Melbourne neurologist Dr Andrew Evans said phase one was approved to be held in Australia because the country has a culture of innovation and a high standard in clinical research.“I think favourable research environment with regards to R and D [Research and Development] tax credits,” he said.
The trial involves 12 Australian Parkinson’s sufferers injected with neural stem cells.Those 12 patients’ stem cells will then be observed for one year, with Dr Evans and his team looking to see whether they are able to boost the brain’s capacity to produce and release the chemical messenger dopamine.
“Dopamine is one of the most critical neurotransmitters or chemical messengers in the brain that is lost in Parkinson’s disease, and the loss of this neurotransmitter is directly linked to the development of the cardinal features that include stiffness, slowness and shaking,” Dr Evans said.
“It’s hoped that through replenishment of the dopamine through these neural stem cells, which show some capacity to differentiate into obviously dopamine producing cells in the annual models, we are hoping to restore some of the functions in patients with Parkinson’s disease.”
Neural stem cells get around ethical minefields of previous trials
Previous Parkinson’s disease studies have used embryonic stem cells.
Dr Evans said that one of the major sticking points in terms of those technologies are the ethical minefields surrounding the use of those cells.So by using neural stem cells, Dr Evans said that problem was eliminated.
“[Embryonic stem cells] are fertilised and have a capacity to go on and develop into humans, whereas the parthenogenetic stem cells do not,” he said.
“Another major technological barrier has been the ability of these cells to differentiate well into neural cells, and that with the most recent technologies is becoming much less of a barrier.”
Cause of Parkinson’s disease remains the ‘holy grail’ for scientists
At the moment the Therapeutic Goods Administration in Australia has given the trial a conditional approval “There are a few caveats we have to work through, but I’m sure we will be able to do that within the next month or so,” Dr Evans said.
“They’re not high bars.”
Dr Evans said he was excited to be involved in the trial.
“The holy grail of finding out what causes Parkinson’s disease is still elusive for scientists, so we really need to focus our fight against Parkinson’s disease on two fronts,” he said.He said the first thing they need to work out is what causes Parkinson’s disease initially and then how to reverse that “trigger”.
“In the meantime we need to find treatments that do not necessarily rely on understanding or knowing what causes Parkinson’s disease, and perhaps replenishing or replacing some of the brain functions that are lost,” Dr Evans said.
“This is the way forward in the current research environment.”
By Rachael Brown
http://caringcare.info/?p=2585
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Parkinson’s Treatment Using Stem Cells to Be Tested in Phase 1/2a Clinical TrialDosing study to evaluate International Stem Cell's ISC-hpNSC in moderate to advanced PD patients
International Stem Cell Corporation announced that its subsidiary, Cyto Therapeutics, has been given regulatory permission to start a Phase 1/2a dose escalation clinical trial of its neural stem cells (ISC-hpNSC) in the treatment of Parkinson’s disease (PD). Clearance for the trial came the Therapeutics Goods Administration (TGA) of Australia.
The trial will test ISC-hpNSC, neural stems cells derived from human parthenogenetic stem cells, in patients with moderate to severe PD symptoms at the Royal Melbourne Hospital in Australia. “We are very pleased to start the first human study of ISC-hpNSC’s for the treatment of this debilitating disease. There is a large unmet medical need for new treatments that may halt or reverse the progression of Parkinson’s disease and we believe our human neural stem cells may fill this need for the millions of people with this disease,” Andrey Semechkin, PhD, CEO of International Stem Cell, said in a press release. “We look forward to reporting on the progress of the clinical trial over the coming months.”
The company has published positive results from preclinical trials on rodents and primates that found its proprietary ISC-hpNSC candidate to improve PD symptoms and increase brain dopamine levels after intracranial administration. Results showed that ISC-hpNSC provided neurotrophic support and cell replacement to dying dopaminergic neurons in the recipient PD brains.
The open-label clinical trial will evaluate three different dose regimens of 30 million to 70 million neural cells in 12 study participants. After transplantation, follow-up will occur at predetermined intervals for 12 months to evaluate the safety and biologic activity of ISC-hpNSC. A PET scan will be performed at baseline, and at six and 12 months post-surgery. After the administration of ISC-hpNSC, clinical responses will be compared with initial PET scan results.
“We are the first company in the world to conduct clinical trials of human pluripotent stem cells based product for the treatment of Parkinson’s disease. We believe the outcome of the study will produce findings in-line with our preclinical studies, where we demonstrated not only safety of our proprietary neural stem cells, but also their functional efficacy. The cells were able to successfully integrate into the brain and provide a significant increase of dopamine levels in the nigrostriatal system,” said Russell Kern, PhD, the company’s executive vice president and CSO.
Current Parkinson treatments, such as L-DOPA and dopamine agonists, are known to improve early disease symptoms. As the disease progresses and dopaminergic neurons continue to be lost, however, the drugs can become ineffective and cause side effects
http://parkinsonsnewstoday.com/2015/12/17/international-stem-cell-corporation-receives-authorization-to-initiate-phase-iiia-clinical-trial-of-isc-hpnsc-for-the-treatment-of-parkinsons-disease-2/
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