A Japanese drugmaker says the first patient has received its stem-cell treatment for Parkinson’s disease since the country approved it earlier this year. Sumitomo Pharma said on Friday that “the first transplant was performed at Kyoto University Hospital in October,” in a statement included in its earnings press release, without giving further details1. AFP described it as a world first following the treatment’s conditional approval in Japan1.
The company did not say who the patient is, how the surgery went, or how the person is doing. What is known about the treatment comes from the earlier approval and the small trial behind it.
What happened
Japanese regulators approved the treatment in March, following a clinical trial1. At the time, Sumitomo Pharma said it had received the green light to make and sell the product, called Amchepry, which transplants stem cells into a patient’s brain2. The approval was “conditional and time-limited,” the company said, under a system that is reportedly designed to get such products to patients as quickly as possible2.
The Japanese newspaper Asahi called it a kind of “provisional license,” because safety and effectiveness were judged on data from fewer patients than ordinary drug trials use2. According to local media, the treatment will gain full approval in Japan only if its safety and effectiveness are confirmed over the next seven years1.
When the approval was announced, reports said the first products could reach patients as early as the summer2. The first transplant under the approval came in October1.
What the treatment is
Parkinson’s disease is a long-term, worsening disorder of the nervous system that affects movement, often causing shaking and other difficulties1. It is tied to the loss of brain cells that make dopamine, a chemical messenger the brain needs to control movement3. The Parkinson’s Foundation says current therapies “improve symptoms without slowing or halting the disease progression”1.
Sumitomo Pharma’s approach tries to replace the lost cells. It starts with induced pluripotent stem cells, or iPS cells: mature, already specialized cells that are pushed back into a young state, from which they can develop into almost any cell type in the body1. Japanese scientist Shinya Yamanaka won the Nobel Prize in 2012 for his research on these cells2.
In this treatment, iPS cells from healthy donors are grown into the precursors, or early forms, of dopamine-making brain cells1. Millions of these cells are then transplanted into the patient’s brain1. Some general descriptions of iPS therapy talk about making cells from a patient’s own skin3, but the cells in Sumitomo Pharma’s product come from donors, not from the patient1. (Biohack has separately covered a different company developing a Parkinson’s therapy grown from patients’ own cells.)
The evidence so far
The approval rests on a trial led by Kyoto University researchers2. It involved seven people with Parkinson’s, aged 50 to 692. Each received a total of either 5 million or 10 million cells, implanted on both sides of the brain2.
The patients were followed for two years, and no major side effects were found1. Four of the seven showed improvements in their symptoms1. Reporting at the time of approval said the trial indicated the treatment was safe and improved symptoms2.
That is the full set of results described in the sources available to Biohack. The new announcement adds one more treated patient but no new data.
What it does not show yet
Several important questions remain open:
- Small numbers. Seven people is a very small group. The approval was granted on fewer patients than ordinary drug trials use, which is why it is conditional2.
- Not everyone improved. Four of the seven trial patients showed better symptoms1. The sources do not describe how large the improvements were or what happened in the other three.
- No comparison group described. The sources do not mention patients who got a placebo (a dummy treatment) or a sham procedure, so it is hard to separate the effect of the cells from other influences.
- Long-term effects. The trial followed patients for two years1. Whether benefits last longer, and whether any later safety problems appear, is what the coming years of conditional approval are meant to test1.
- The first patient. The company gave no details beyond the place and month of the transplant1.
Who is behind it
The treatment is made and sold by Sumitomo Pharma, which announced the first transplant in an earnings release, a financial report to investors1. The company says it is working to bring iPS-based cell therapies to market together with academic researchers, Sumitomo Chemical, and two companies called RACTHERA and S-RACMO4.
The cells are produced at the company’s Central Research Institute in Suita City, Osaka Prefecture4. Sumitomo Pharma has also described the challenge of shipping live, unfrozen cell products from that facility to a site in the United States within 24 hours, for products still under development4.
The sources do not say who funded the Kyoto University trial. The announcement of the first patient comes from the company itself, which has a commercial interest in the product.
How it fits the field
Japan approved two iPS-based treatments at the same time in March. Alongside the Parkinson’s product, the health ministry approved ReHeart, sheets of heart muscle cells developed by the startup Cuorips that can help form new blood vessels and restore heart function, according to media reports2. Reports said these would be the world’s first commercially available medical products made from iPS cells2.
The heart treatment reached a patient first. About a month before Sumitomo Pharma’s announcement, a hospital in Osaka carried out the first surgery using the heart muscle cell sheets1.
At the approval, Japan’s health minister Kenichiro Ueno told reporters:
“I hope this will bring relief to patients not only in Japan but around the world.”2
The scale of the disease explains the interest. The Parkinson’s Foundation estimates that more than 10 million people worldwide have Parkinson’s, according to AFP’s latest report1; AFP’s March report put the figure at about 10 million2.
What to watch next
Sumitomo Pharma says it plans to carry out transplants in 20 to 30 patients by March 20291. The company is also running a clinical trial in the United States1.
Things to look for in the coming months and years:
- any update on how the first patient treated under the approval is doing;
- the pace of further transplants toward the 20 to 30 patients planned by March 20291;
- results from the US trial1;
- whether data gathered during the conditional period confirm safety and benefit, which local media say is required for full approval within seven years1.
For now, the news is that the treatment has moved from a trial into use under Japan’s fast-track rules. Whether it helps people with Parkinson’s more broadly is still being measured.
