Gene therapy, stem cell therapy trials underway

Stem cells and gene hold promising treatment options for Parkinson's, mandate doctors across the globe, including from Mumbai. Eleven trials to test stem cell and gene therapy for treating Parkinson's are underway currently of which the one in Mumbai had to be put on hold due to regulatory hurdles.

Currently, neuro-augmentative therapies such as usage of drugs or deep brain stimulation (DBS) are being used to treat Parkinson's disorder. "The future holds hope for neuro-restorative therapies like that of stem cells or gene infusion in the Parkinson's disorder treatment. It involves restoration of brain function to normal. In the next five to seven years, this may pave the way for future," said Dr Paresh Doshi, neurologist at Jaslok Hospital, Peddar Road in Mumbai.

Regulatory hurdles and resource constraints though have led to these trials being held up in Mumbai. Dr Doshi said that trials of Duodopa therapy which involves infusion of an active ingredient gel called Levodopa in the intestines has been kept on hold at the moment at privately-run Jaslok Hospital due to regulatory hurdles. The hospital was the only centre in entire South East Asia to have been running the trial.

"Levodopa gets converted into dopamine in the body. Normal levels of dopamine control Parkinsons disorder," said Dr Doshi.

Trials to infuse stem cells from the patient's body in the patient itself had been underway in small group of patients in India, but due to inability to recruit more patients, the trial was stopped. "We could only recruit four patients for two years. However, a similar trial is underway in China and another trial which explores adipose tissue stem cells in treating Parkinson's disease is underway in South Africa," said Dr Doshi.

In January this year, medical journal The Lancet reported that after sixteen years of trials, gene therapy is showing promising results in humans. "Three genes that promote the formation of dopamine generating cells in the brain were injected in the brain bound with a viral vector in fifteen patients. The genes are intended to boost the production of dopamine, a chemical that becomes deficient in patients withParkinson's," said The Lancet report.

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Gene therapy, stem cell therapy trials underway