"A 12-month study would have taken three years in the U.S. because of the recruitment schedule the FDA is asking for," Craw said. Shameful!!
By Bradley J. Fikes JAN. 14, 2015
http://www.utsandiego.com/news/2015/jan/14/parkinsons-stem-cell-parthenogenetic/?#article-copy
A therapy for Parkinson's disease from Carlsbad's International Stem Cell Corp. is expected to get approval for testing in Australia as soon as February, the company said this week.
The publicly traded company has grown neural stem cells, which can mature into cells making the neurotransmitter dopamine, deficient in Parkinson's. The company plans to implant these stem cells into the the brains of Parkinson's patients, restoring dopamine production and normal movement in the patients.
If approved, the trial will be the first test of therapy with the company's cells, derived from unfertilized, or parthenogenetic human egg cells. The cells, which in theory can produce nearly all types of cells found in the body, are grown into neural stem cells. These cells will be implanted and mature in place.
Parthenogenetic cells have much the same potential as embryonic stem cells without the ethical objections some have, says International Stem Cell, which has 38 employees. In addition, these parthenogenetic stem cells are less likely to provoke an immune reaction, the company says.
International Stem Cell Corp. chose Australia for its first trial because its regulatory agency is more "interactive" than the U.S. Food and Drug Administration, said Simon Craw, executive vice president for business development. The FDA is inclined to give yes-or-no answers for proposed cell-based treatments, Craw said. In addition, patient recruitment takes place more slowly, which delays trial completion. The FDA does this for safety reasons.
The Australian agency helps guide companies through the application process, Craw said in an interview Wednesday at Biotech Showcase, an annual life science conference in San Francisco. Craw also gave a company presentation on Tuesday at the conference.
"We're in the process of submitting the (application)," to Australian regulators, Craw said. "We're going back and forth with them right now. We expect to hear back from them by the end of February."
The trial will primarily assess safety, but also look for evidence of efficacy, Craw said.
The trial will take place at Royal Melbourne Hospital, Craw said. The hospital is headquartered in Parkville, in the state of Victoria. The principal investigator, Dr. Andrew Evans, will recruit patients from his own practice.
A U.S. trial of the therapy is planned later on, Craw said. ISCO has been working with Duke University on preclinical studies. Duke University neurologist Mark Stacy introduced ISCO to Evans, Craw said.
The pace of patient recruitment will be decided by the hospital, Craw said, and the pace could potentially be accelerated with the help of Evans.
"A 12-month study would have taken three years in the U.S. because of the recruitment schedule the FDA is asking for," Craw said.
There's a precedent for cell therapy for Parkinson's, with implants of fetal brain cells. These cells produced mixed results. Some patients improved, some didn't, and some developed abnormal movements, apparently from an excess of the dopamine-making neurons, Craw said.
The parthenogenetic neural stem cells behaved better in monkey experiments, Craw said. The cells mature into various kinds of brain cells, including the dopamine-making neurons. Moreover, they generate the needed amount, implying there is some kind of communication going between the implanted cells and the monkey brains.
International Stem Cell won a significant victory in Europe last month, when a European court ruled that parthenogenetic stem cells can be patented. Embryonic stem cells cannot be patented in Europe, and the court ruled that parthenogenetic stem cells are not embryonic, and can therefore be patented.
A similar effort is being developed by the Parkinson's Association in San Diego and researchers at The Scripps Research Institute and Scripps Health. This effort could take about two years to enter therapy. It uses artificial embryonic stem cells called induced pluripotent stem cells. These cells are grown from each of eight Parkinson's patients taking part in the effort. More information is available at summit4stemcell.org.