Kyoto Univ. to Start iPS Clinical Trial for Parkinson’s by Year-End

July 31, 2018

Kyoto- Kyoto University said Monday that it will launch the world's first clinical trial for transplanting neural precursor cells made from induced pluripotent stem, or iPS, cells into the brains of Parkinson's disease sufferers.

The Japanese university will select seven patients for the trial and carry out the transplant surgery on the first of them by year-end at the earliest. The patients will be those aged between 50 and 69 whose conditions are not serious although their medicines are working less effectively than before.

If the clinical trial turns out to be successful, the university will aim to develop new drugs for the intractable disease in collaboration with Sumitomo Dainippon Pharma Co. and file an application with the government on the new drugs as early as in fiscal 2022.

Parkinson's disease, stemming from a fall in the number of nerve cells that create dopamine, a substance to transmit signals in the brain, causes sufferers to tremble or become unable to move their limbs properly.

A Kyoto University team, including Prof. Jun Takahashi, will use iPS cells made from the blood of people with a special immune type that rarely causes rejection. The iPS cells will be cultured into neural precursor cells, and the cultured cells will be injected into the brain. The transplanted cells will develop into nerve cells, which are then expected to start releasing dopamine, according to the team.

In the clinical trial, the team will transplant a total of about five million cultured cells into two areas close to the center of the brain.

To check the safety of the operation, the first patient will have an interval of six months between the cultured cell injections into one of the two areas and the other.

For two years after the operation, the team will examine the possible emergence of tumors and the degree of improvement in the patient's symptoms.

Kyoto University's program will be the third for transplanting iPS-derived cells into patients, after a clinical research done by government-affiliated institute Riken in 2014 for an intractable eye disease and a clinical research planned by Osaka University for a heart disease.

Takahashi said that he hopes the planned treatment "will become an effective option to cure Parkinson's disease."

"We expect the treatment will be covered by health insurance programs," he added.

The team obtained the government's approval for the clinical trial after submitting the plan in June.

The planned start of the clinical trial is very good news, said Shinya Yamanaka, head of Kyoto University's Center for iPS Cell Research and Application, or CiRA.

"We will move ahead with our research carefully and at the same time promptly so we can use the iPS-based treatment for Parkinson's disease patients as early as possible," said Yamanaka, who won the Nobel Prize in Physiology or Medicine in 2012 for inventing iPS cells, which can develop into almost any type of tissue. Jiji Press