TOKYO – With the introduction of new legislation that makes approvals easier, Japan aims to be the global leader in regenerative medicine.

Stem cell therapies use lab-grown stem cells to replace or repair failing organs and spinal injuries, including treating incurable conditions and diseases. Japan, one of the world's biggest potential markets for that class of treatment, has passed new legislation that could bring it to the forefront of regenerative medicine.

Japan's Upper House approved revised regenerative medicine regulations in November 2013, accelerating approval for cell and gene therapies. The new rules that took effect Nov. 25, require the sponsor to show safety and efficiency in a relatively small pool of 12 patients in order to get a seven-year conditional approval.

The new rules include two pieces of legislation. One is the "Act Concerning Safety Assurance of Regenerative Medicine," which allows for "conditional" approval for stem cell therapies based on probable efficacy and safety in small-scale trials. Another is a revised Pharmaceutical Affairs Act, which introduced an accelerated drug approval program with conditions and time limits based on the characteristics of regenerative and other medicines.

Prime Minister Shinzo Abe has said he regards regenerative and cellular therapeutic products as one of the most promising innovative fields to be cultivated in Japan. He has directed offices to assure governmental support for the acceleration of regenerative medicine, including regulatory aspects.

"It is our government's policy to support cell and gene research with financial aid and quicker approval, to bring Japan to the forefront of regenerative medicine," Masahiro Sasaki, director of the gene and cell support division of the Health Ministry, told BioWorld Asia.

Kyle Murphy, the managing director and founder of KMG Japan, who has lived and worked in the Japanese health care market for more than 25 years, agreed that the changes will accelerate progress.

"It is a major step, and the fundamental change in regulations allows reimbursement of autologous stem cell or regenerative medicine," he said. "Rules for allogeneic products already existed."

Autologous stem cells are taken from the patient being treated, and those taken from a different person are known as allogeneic stem cells. Japan has focused on the former, while the U.S. has focused on the latter.

Many different autologous start-up companies already exist inside and outside Japan. Japan Tissue Engineering Co. Ltd. (JTEC) already has two approved autologous products: one for skin and the other for knee cartilage, both approved on limited clinical data.

"The major players are now starting to look at this therapy area, either by investing in Japanese ventures and developing their products or by looking overseas for opportunities to in-license," Murphy said. "In that sense there is an impact on capital allocation toward this sector."

Prior to those regulatory changes, autologous therapy could only be done on a research or limited-patient basis.

"There was no regulatory pathway for getting a general approval for a drug, and then [for] reimbursement under the medical insurance scheme. The new regulations are to enable both to happen," Murphy noted.

THE WORLD IS WATCHING

A number of products, for the most part, are already developed. The new rules allow companies to commercialize them.

"This is a very important first step," said Fumiyoshi Sakai, a pharma analyst at Credit Suisse in Tokyo. "The next step will be to commercialize stem cell [and gene] therapies through doctors and specialized hospitals, and the costs must be reimbursed by the government through [National Health Insurance]."

For that to happen, the private sector must be attracted and get involved.

Already in Japan, billboards advertise regenerative medicine beauty clinics, claiming to restore aging skin. In many of those clinics, however, the efficacy and safety of the products on offer are still unproven, wrote Toshi Miyata, a surgeon who has been involved in the Revision of the Pharmaceutical Affairs Law at the Ministry of Health, Labor and Welfare, in local press.

Japan's regulation of regenerative medicine has been loose, he pointed out, and that has attracted people from countries with stricter regulations, such as South Korea. Unfortunately, that led to one case where a South Korean patient died suddenly upon returning home following treatment at a Kyoto clinic.

According to Miyata, "cases like this have caused the world to distrust Japanese regenerative medicine as a whole, which necessitated swift corrective measure as Japan is trying to push stem cell research forward."

The new law reduces those risks, but the world is now watching to see what Japan can do in regenerative medicine.

The country has been promoting regenerative medicine for a number of years. A Japanese scientist, Shinya Yamanaka, received a Nobel Prize, along with Sir John Gurdon, in 2012 for his discovery that mature cells can be reprogrammed and made pluripotent, which then can become a number of different tissue types.

"Thank goodness for Nobel Prizes; otherwise we would be waiting another 10 years for something like this to happen," KMG's Murphy said.

Hiro Fujita, a 35-year-old amyotrophic lateral sclerosis sufferer in Tokyo, said: "Whatever progress happens is surely welcome in this culture where cautiously standing still is preferred to taking a step forward. I wish Abe will boldly state, 'We will cure 10 diseases with stem cells by 2016' . . . similar to JFK's, 'We will land on moon,' statement."