After delay, ISCO's trial of Parkinson's stem cell therapy gets underway again

brain imaging
Analysts predict a phase 2 study could start in late 2017 or early 2018.

A second patient has now been treated in the first trial of a stem cell therapy for Parkinson's disease developed by California biotech International Stem Cell Corporation (ISCO).

The treatment involves transplants of millions of stem cells delivered by intracranial injection in a bid to replace dopamine-producing cells that have died off. In Parkinson's, that neurodegeneration disrupts the neural pathways that govern muscle movements and causes characteristic symptoms such as limb shaking.

So far, the two patients treated to date are showing no ill effects from the therapy, which is being studied in a phase 1 trial involving 12 Parkinson's disease sufferers conducted by researchers at the Royal Melbourne Hospital in Australia.  


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It's too early to see if there is any clinical effect of the ISC-hpNSC therapy just now, but there may not be too long to wait—the team will be checking to see if Parkinson's symptoms improve over the course of the 12-month trial and first data could be available in the first quarter of 2017, say analysts at Edison.

"We currently expect that the company will be able to progress to a phase 2 study in late 2017 or 2018 depending on the safety and efficacy profile discovered in the current trial," they said in a research note.

ISCO's trial got underway earlier this year with the first patient receiving the transplant in July, but additional surgical procedures were delayed by "a supply chain disruption of equipment critical to the operation," according to the firm's chief scientific officer Russell Kern. 

The trial is now resuming and will see the doses gradually increased from a level of 30 million cells in the first two patients to a maximum of 70 million cells. 

If safe and effective, stem cell therapy could be a massive step forward for Parkinson's disease patients, who are still treated mainly with the combination of levodopa and carbidopa, which replace the dopamine lost by the death of neurons.

Most of the clinical development of new therapies to date has focused on the reformulation of dopamine-replacing regimens and, to date, no disease-modifying therapies have been approved to help the 7-10 million people worldwide with the disease.

Other companies with cell- and gene-based therapies for Parkinson's in clinical testing include Living Cell Technologies, uniQure, Voyager Therapeutics and Oxford BioMedica.

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