World-First Clinical Trial For Parkinson’s Disease To Inject Special Stem Cells Directly Into Patients’ Brains

Parkinson's disease
Jne Valokuvaus / Shutterstock

A unique treatment for Parkinson’s disease is about to be tested in Japan, with the first clinical trial scheduled to begin on August 1. The announcement was made on July 30 by the Kyoto University Hospital, which started patient recruitment on the same day at 5 p.m. local time, reports Science Magazine.

This innovative treatment is based on a special type of stem cells known as induced pluripotent stem (iPS) cells, discovered in 2006 by Japanese researcher Shinya Yamanaka.

As the Inquisitr previously reported, pluripotent stem cells are extremely versatile and have the advantage of evolving into any type of cells once transplanted into the body. But while these are typically embryonic stem cells (used for a wide array of medical applications), Yamanaka has found a way to manipulate adult stem cells and infect them with a virus that restores them to their immature state.

This breakthrough, which eliminates the need to use embryonic stem cells in medical research, earned Yamanaka a Nobel Prize in 2012 and is now being used in a series of clinical trials that address brain tumors and the repairing of damaged hearts, notes New Atlas.

The same technology is also the basis for this novel Parkinson’s disease treatment, which aims to convert millions of iPS cells into so-called dopaminergic progenitors — neurons that can generate the dopamine neurotransmitter in the brain and replenish the low dopamine levels responsible for the onset of Parkinson’s symptoms.

The reprogrammed cells are to be injected directly into the patients’ brains with a specialized device that requires the drilling of two 12-mm (0.5-in) holes in the skull.

The ground-breaking clinical trial is being led by Jun Takahashi, from the Center for iPS Cell Research and Application (CiRA) at Kyoto University.

The neurosurgeon plans to test the stem cell Parkinson’s treatment on seven patients and transplant about five million iPS cells each into a specific area of the brain known as the putamen — a large, round structure located at the base of the forebrain and which regulates limb movement, explains Healthline.

Last year, Takahashi reported promising results of the iPS cell treatment in the journal Nature, following a two-year-long study on monkeys with Parkinson’s. According to the results, the animals experienced significant improvement after receiving injections with human iPS cells — thereby propelling the treatment into human trials.

Although Yamanaka’s technology allows researchers to make patient-specific iPS cells by harvesting adult stem cells from each test subject in order to minimize the risk of transplant rejection, CiRA has elected to use readily available stocks of iPS cells harvested “from healthy donors with specific cell types that are less likely to cause immune rejection,” notes Science Magazine.

“Using stocks of cells, we can proceed much more quickly and cost-effectively,” Yamanaka said in a 2017 statement.

At the time, his technology had just been reported safe in a trial for patients with age-related macular degeneration, led by Jun Takahashi’s wife, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, and described in the journal Science.

As an added precaution against transplant rejection, the Parkinson’s patients will also receive a drug called tacrolimus, designed to suppress the immune response and prevent the body from attacking the iPS cells.

Embryonic stem cells.
Embryonic stem cells. Giovanni Cancemi / Shutterstock

About 1 million Americans and another 9 million people worldwide are currently battling with Parkinson’s disease, reports Newsweek, citing The Parkinson’s Foundation. This degenerative neurological disease is triggered by a lack of dopamine in the brain and causes a decline in motor skills, leading to walking difficulty and involuntary tremors in the hands and feet.

While Parkinson’s symptoms typically occur in older adults, there have been cases when the disease was diagnosed in younger people. Such was the case of actor Michael J. Fox, who received a Parkinson’s diagnostic at the age of 29, the Inquisitr recently reported.

Other celebrities who have struggled with this debilitating disease include Robin Williams, Neil Diamond, Muhammad Ali, and Alan Alda, who announced this morning that he has been suffering from Parkinson’s for 3.5 years, per an Inquisitr report.