iPS細胞さいぼうを用もちいたパーキンソン病びょう治療ちりょう 治験ちけんで“有効ゆうこう性せい” 京都大きょうとだい

A research team at Kyoto University, which is developing a new treatment method of transplanting cells created from iPS cells into the brains of Parkinsons disease patients, announced that the results of clinical trials involving seven patients demonstrated safety and efficacy.

The pharmaceutical company that cooperated in the clinical trial plans to apply for manufacturing and sales approval from the government in the future.

Parkinsons disease is a difficult condition in which the brain cells that produce a neurotransmitter called dopamine are lost, causing tremors in the limbs and immobility. It is estimated that there are approximately 250,000 patients in the country.

Currently, treatments such as medication administration and implanting electrodes in the brain are being conducted, but there is no fundamental cure.

The research team led by Professor Jun Takahashi at the Kyoto University iPS Cell Research Institute announced the results of a clinical trial aimed at improving symptoms by transplanting dopamine-producing nerve cells made from human iPS cells into patients brains.

In the clinical trial, 5 million or 10 million cells were transplanted into the brains of seven male and female patients aged 50 to 69, and no major health issues were observed in any of the patients.

It was confirmed that dopamine was produced from the transplanted cells in all of the six patients who were monitored over a period of two years.

In addition, in tests to examine the severity of symptoms, improvement in motor function was observed in 4 out of 6 people, and the research team stated that safety and efficacy have been demonstrated.

Sumitomo Pharma, a pharmaceutical company in Osaka that cooperated in the clinical trial, plans to apply to the government for manufacturing and sales based on this data.

Professor Takahashi said, The improvement of symptoms through cell transplantation is revolutionary for researchers.

We want to obtain the countrys approval and deliver treatment to patients as soon as possible, they said.

Hopes from patients and families for new treatment methods Voices of hope for new treatment methods using iPS cells were heard from patients with Parkinsons disease and their families.

The group formed by Parkinsons disease patients and their families in Kyoto City regularly holds information exchange sessions on treatment and study sessions on new treatment methods.

The 66-year-old wife of the patient says, Many years have passed since my husband developed the condition, and he is experiencing more falls and has difficulty speaking.

The emergence of treatments using iPS cells is a new hope for families.

I had been waiting for this, they expressed with anticipation.

On the other hand, a 75-year-old male patient said, I am very pleased with the good results from the clinical trial and deeply appreciate the efforts of the doctors, but I wonder if all patients will be able to receive the treatment.

Okada Takashi, the branch manager of the Kyoto branch of the Japan Parkinsons Disease Association, said, Its like a dream.

I just hope they can do something quickly with a groundbreaking treatment.

Ive heard that it is a treatment that is more effective for younger people, but since there are many elderly patients with Parkinsons disease, Im hoping that it will eventually become a treatment that is effective for all patients, they said.

Expert: Reduced medication frequency and improved quality of life expected. Regarding the results of this clinical trial, Professor Nobutaka Hattori, a specially appointed professor at Juntendo University who is well-versed in Parkinsons disease, said, I think it is good news for patients in that it may become a new treatment option.

If this treatment method is established, it may be possible to reduce the number of times medication is taken, or in some cases, eliminate the need for medication altogether. For patients, this could lead to an improvement in their condition while sleeping, reduce the risk of forgetting to take medication, and ultimately improve their QOL Quality of Life.

In addition, Looking at the clinical trial results, not all patients have dramatically improved, but there are patients who showed high effectiveness.

Transplanting at an earlier stage may lead to long-term stabilization of symptoms, but it is necessary to verify which patients will benefit in the future.

In this clinical trial, there were few patients whose effects were examined, and there are still many unknown points, so it is also necessary to clarify more effective methods, he said.

[Details] Improvement of patients motor function This clinical trial was conducted on 7 patients, but for one person, only safety was confirmed, and the treatment effect was investigated in 6 people.

To determine how much the patients motor function has improved, one of the international indicators for assessing the severity of symptoms in Parkinsons disease patients was used.

This index evaluates each item on a 5-point scale from 0 to 4, such as whether the speech is clear when speaking, or whether support is needed when standing up from a chair, with higher numbers indicating more severe symptoms.

As a result of conducting a motor function test when the effect of the Parkinsons disease medication wore off, the numbers improved for 4 out of 6 people after 2 years, with some showing a significant improvement of 32 points.

Out of the four people, the condition of two improved from moderate to mild, and one improved from severe to moderate.

On the other hand, the two peoples numbers deteriorated by a few points, but this was the same level of deterioration as those who were receiving medication during the same period.

According to the research team, patients who showed significant improvement were relatively young and had mild symptoms. The research team considers this treatment to be suitable for young patients with low severity.

Professor Jun Takahashi of the Center for iPS Cell Research and Application at Kyoto University stated, We have also begun to see directions such as the optimal dosage and which patients can be expected to benefit, so I believe it is a very meaningful result. He added, Since this is the first clinical trial in humans, we are starting by transplanting a smaller number of cells into patients who have severe symptoms or are older.

We aim to eventually supply enough dopamine solely through cell transplantation by gradually increasing the number of transplanted cells for patients who are expected to benefit more, so that medication will no longer be necessary, it says.