World-first stem-cell therapy for Parkinson’s approved in Japan

Japan has approved pioneering stem-cell treatments for Parkinson’s disease and severe heart failure, a manufacturer and media reports said on Friday, with the therapies expected to reach patients within months.

Pharmaceutical firm Sumitomo Pharma said it had received approval to manufacture and sell Amneal, its treatment for Parkinson’s disease that involves transplanting stem cells into a patient’s brain.

Japan’s health ministry has also authorised ReHeart, a treatment consisting of heart-muscle sheets developed by medical start-up Cuorips that can stimulate the formation of new blood vessels and help restore heart function, according to media reports.

The therapies could reach the market and begin being administered to patients as early as this summer, the reports said, citing the health ministry. They would become the world’s first commercially available medical products using induced pluripotent stem (iPS) cells.

Japanese scientist Shinya Yamanaka was awarded the Nobel Prize in Physiology or Medicine in 2012 for his research into iPS cells, which have the potential to develop into any type of cell in the body.

“I hope this will bring relief to patients not only in Japan but also around the world,” Health Minister Kenichiro Ueno said at a press conference.

“We will promptly complete all necessary procedures to ensure it reaches patients without delay.”

In a statement, Sumitomo Pharma said it had obtained “conditional and time-limited approval” to manufacture and market Amchepry under a regulatory framework designed to deliver such treatments to patients more rapidly.

The approval effectively acts as a provisional licence, The Asahi Shimbun reported, with the safety and effectiveness of the therapy evaluated using data from fewer patients than typically required in standard drug trials.

A trial led by researchers at Kyoto University indicated that the treatment was safe and showed potential in improving symptoms.

The study involved seven Parkinson’s patients aged between 50 and 69, each receiving either five million or 10 million cells implanted on both sides of the brain.

The iPS cells, derived from healthy donors, were developed into precursor cells capable of producing dopamine — brain cells that are lost in people suffering from Parkinson’s disease.

Patients were monitored over a two-year period, during which no major adverse effects were reported, the study said. Four participants showed improvements in symptoms.

Parkinson’s disease is a chronic and progressive neurological disorder that affects the body’s motor system, often causing tremors and difficulties with movement.

Around 10 million people worldwide live with the illness, according to the Parkinson’s Foundation.

Existing treatments, the foundation notes, can help alleviate symptoms but do not slow or stop the progression of the disease.

iPS cells are created by reprogramming mature, specialised cells back into a juvenile-like state – effectively enabling cell regeneration without the use of embryos.

These cells can then be transformed into a wide range of cell types, making them a major focus of medical research worldwide.