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Global impact of Japan's regenerative medicineLegal changes for one of the world leaders in stem cell research

January 21, 2014  Toshio Miyata

 This article first appeared in Japanese on JBpress on December 20. You can read it here.

Regenerative medicine sounds like something from science fiction: using lab-grown cells to replace or repair failing organs and spinal injuries and giving hope for treating hitherto incurable conditions and diseases. But although it is gradually becoming realistic, traditional medical standards for testing safety and effectiveness are difficult to apply. In an attempt to make standards that reflect this reality and to accelerate new breakthroughs, Japan recently changed and created relevant laws, as Dr. Toshio Miyata, who was involved in the process, writes.

If you cut off a newt's tail or leg it will eventually grow back as good as new. Regenerative medicine applies this principle.

In November 2013 the Diet revised the Pharmaceutical Affairs Law and enacted the Regenerative Medicine Law, thereby reforming the organization of regenerative medicine in Japan.

In response to numerous questions from overseas, I explained the new system to Harvard University, the National Institutes of Health, the Institute of Medicine (IOM) and the National Academy of Sciences (NAS) in the US. I would now like to explain it in print to a larger audience.

Impact of the revised Pharmaceutical Affairs Law

The Pharmaceutical Affairs Law aims to guarantee the safety and effectiveness of medicines and medical equipment in Japan, so what impact can it have on the rest of the world to warrant so much interest?

Because regenerative medicine is a new technology, internationally, it generally takes longer to approve it for use than for other medicines. The revised law will reduce the time required to evaluate their safety, permitting the effectiveness of treatments to be anticipated more quickly than in the past, ensuring faster effective deployment. This is being monitored closely by the rest of the world.

About the Regenerative Medicine Law

Meanwhile, the new Regenerative Medicine Law regulates regenerative medicines whose effectiveness and safety has not yet been established. Before its introduction insufficient regulation in Japan allowed some regular clinics take up the practice of regenerative medicine.

Riding a train in a Japanese city, you can look out the window and see billboards advertising regenerative medicine beauty clinics and their claims for reviving aged skin. At present, clinics like these, which use regenerative medicine whose efficacy and safety is unproven, abound.

At US workshops sponsored by the IOM and NAS cases were cited where clinics have grown cells, made them into cell sheets and used them as cosmetic face packs. Governments in the US and most other advanced countries are strictly regulating this practice.

Because of this, some people are traveling to countries with looser or no regulations to receive treatment as medical tourists, which is a big problem in terms of safety.

Japan's regulation of regenerative medicine has hitherto been loose, which had attracted people from countries with strict regulations, such as South Korea. Unfortunately, this led to one case where a Korean died suddenly upon returning home following treatment at a clinic in Kyoto.

Cases like this have caused the world to distrust Japanese regenerative medicine as a whole, which necessitated swift corrective measures as Japan is trying to push stem cell research forward. Under the new law these risks have been reduced, and the world is now watching to see what we can do in regenerative medicine.

What is regenerative medicine?

In Japan regenerative medicine immediately brings to mind Dr. Shinya Yamanaka, who received the 2012 Nobel Prize in Medicine for his work with stem cells. Regenerative medicine is a treatment that involves cultivating human cells outside of the body, changing or reproducing them, and then introducing them into a patient.

Stem cell technology makes it possible to create all types of cells, and in having the goals of creating cells and creating organs, is expected to become an extremely useful tool.

There are two types of cells used for these treatments: those taken from the patient being treated (autologous stem cells) and those taken from a different person (allogenic stem cells). Japan has focused on the former while the US has focused on the latter.

Initially, regenerative medicine is expected to provide new treatments for hitherto incurable diseases and broaden treatment options for diseases that offer a scarcity of them.

In Japan there is a great deficiency in donor organs for transplant, so people are talking about a dream for a future where regenerative medicine may enable the creation of various bodily organs for use as transplants.
Also, for patients with weakened heart muscles from heart attacks, experiments are being conducted with ‘cell sheets' made from stem cells that can be applied to the heart to rehabilitate the pumping function.

Additionally, during the process of experimenting with regenerative medicine, possibilities for treatments beyond organ regeneration may arise, such as for patients with brain hemorrhages or spinal injuries who were thought to be without hope of recovery. This is why the development of this branch of medicine is so highly anticipated by so many.

Topics in regenerative medicine

Hitherto most pharmaceuticals have been chemical compounds, which has made quality stable and led to set methods for their evaluation.

Meanwhile, medical equipment is primarily made of metal and electronic circuits and it is fairly simple to evaluate its capabilities, notwithstanding the technical ability of the doctors who use it.

However, with regenerative medicine it is no simple thing to mass produce identical products as you can with chemical products. Non-homogenized products also have less consistent effects, with different people having varying reactions.

The most important problem with the cell culturing process is with cells becoming cancerous; if there is one cancerous cell among the thousands of cells being cultured there is a danger that cancer will occur in the patient being treated.

In particular, the cells used in regenerative treatments for Japan's dominant method of autologous stem cell transplants are extremely unstable and non-homogenous, which makes conducting scientific evaluations of their effectiveness and safety difficult.

On the other hand, the allogenic stem cell transplant method favored in the West is able to produce comparatively stable and homogenous treatments, which enables scientific evaluation, although this evaluation is time consuming. However, because of problems with patients' bodies rejecting cells or organs made from allogenic stem cells, some are of the opinion that scientifically evaluated autologous stem cell transplants would be ideal.

Some believe that with new technological advances there is a possibility that immuno-suppressive drugs will cease to be necessary for conducting allogenic stem cell transplants. Furthermore, some have expressed concerns that the risk of developing cancer will increase with the use of stem cells and it is well nigh impossible to guarantee 100 percent safety.

The inspection standards set by the American FDA for regenerative stem cell treatment are extremely high and place the strongest emphasis on safety inspections. However, many of the patients seeking regenerative treatment have serious or incurable diseases, and some are very vocal in asking why they can't receive treatment using their own cells in their home country.

Next steps the government needs to take

With a specialized approval system for regenerative medicine the effectiveness of treatments can be anticipated, and if proven safe they will go on to have a global impact.

Currently, people like Dr. Masayo Takahashi of the Riken Kobe Institute and venture firms such as Helios are at the center of preparations for a first-in-human trial in patients with age-related macular degeneration, a serious condition of the retina, due to start in early 2014.

As global competition in regenerative medicine intensifies there is clearly a need for stronger public-private frameworks to support projects like this.
With Japan's new structure for regenerative medicine, it is necessary for relevant ministries to create a framework for more unified support and to merge their related budgets, make an effort to hire the best personnel, and create exit strategies for projects while managing their progress.

Why not also consider establishing foundations for collecting public-private funding and private donations? With Helios working to treat macular degeneration, the Greek myth where the blinded Orion is healed by the god Helios may end up coming true!

The author is a surgeon who has been involved in numerous innovations, including in the revision of the Pharmaceutical Affairs Law at the Ministry of Health, Labor and Welfare. Prime Minister Shinzo Abe appointed him to the Cabinet Secretariat Office for Healthcare and Medical Strategy in November 2013.

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