Intl. Stem Cell readies for Parkinson's study

Posted: February 19, 2014 at 3:57 am

A proposed Parkinson's disease treatment from stem cells is on track for an application to be filed with federal regulators around the start of 2015, International Stem Cell Corp. said Tuesday.

The Carlsbad-based company said the U.S. Food and Drug Administration had met with researchers for guidance on what steps need to be taken before filing an application. The meeting went about as expected, said Simon Craw, executive vice president. Ongoing animal studies must be completed, along with safety testing. FDA officials discussed details such as the number of animals to be tested and how the safety studies are to be conducted, Craw said.

International Stem Cell's treatment consists of progenitor cells that mature into neural cells, including those making the neurotransmitter dopamine. These neurons are destroyed in the disease, causing progressive paralysis.

The progenitor cells are to be implanted into the brains of Parkinson's patients, where they are expected to make dopamine and protect the remaining dopamine-making neurons. They are derived from unfertilized, or parthenogenetic, human egg cells, made to grow without fertilization.

Human parthenogenetic neural stem cells in the process of differentiating into dopamine-producing neurons. / International Stem Cell Corp.

Interim data from tests in primates are expected in March, and final results later in the year. The Investigational New Drug application, or IND, is expected to be filed a short while thereafter, Craw said. Filing before year's end "would be a stretch," Craw said; the filing is more likely to take place in early 2015.

Shares of International Stem Cell closed Tuesday after the announcement at 24 cents, up 7 percent for the day. The company is working with outside scientists; including D. Eugene Redmond Jr., who is leading a pharmacology/toxicology study. Parkinson's expert Mark Stacy at Duke University will lead the clinical trial. Evan Y. Snyder, a stem cell expert at Sanford-Burnham Medical Research Institute, assists as a scientific advisor.

The company's approach is similar to one being studied by scientists at The Scripps Research Institute led by Jeanne Loring. Moreover, both are expected to ask approval to begin trials around the same time.

Loring's group gets its cells from the patients to be treated, "reprogrammed" back to an embryonic-like state and called induced pluripotent stem cells, then differentiated into the neural progenitor cells. That treatment's main advantage is the use of the patients' own or autologous cells, expected to minimize any possible immune reaction.

The two groups know each other. Craw said International Stem Cell has hired some of Loring's students, and they have collaborated on some research.

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Intl. Stem Cell readies for Parkinson's study

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