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Announcement of a clinical trial for amyotrophic lateral sclerosis (ALS)

1. Main Point
The Center for iPS Cell Research and Application (CiRA), Kyoto University, has announced a new Phase 1*1) clinical trial for the drug bosutinib to treat amyotrophic lateral sclerosis (ALS) at the Kyoto University Hospital (KUH). Leading the project from CiRA is Professor Haruhisa Inoue and from KUH is Professor Ryosuke Takahashi. This trial has received approval from the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).

2. Research Background
ALS is a disease in which motor neurons progressive die. Their death causes muscle atrophy and weakness, ultimately leading to patient death. The only approved treatments are riluzole and edaravone, which extend the patient's life at best a few months and relieve patient symptoms.

Prof. Inoue has been researching ALS by generating induced pluripotent stem (iPS) cells from ALS patients. Differentiating the iPS cells to motor neurons and testing a series of drug compounds led to the discovery that bosutinib could have a substantial effect on preventing motor neuron death. Bosutinib is currently used in the clinic to treat chronic myeloid leukemia. However, in motor neurons, Inoue found that it prevents the abnormal accumulation of proteins associated with ALS and improves cell survival.

3. Important dates

March 1, 2019 Submission of the clinical trial plan to PMDA
March 18, 2019 Commencement of the physician-led trial

※The patient recruitment start date will depend on the participating institutions.

Candidate patients are required to live in Japan, have Japanese public health insurance and understand the Japanese informed consent form.

4. Summary of the clinical trial

(1) Clinical trial name
Phase 1 Dose Escalation Study of Bosutinib in Patients with Amyotrophic Lateral Sclerosis (ALS)

(2) Purpose
The safety and tolerability of bosutinib has been confirmed in patients suffering from chronic myeloid leukemia. In this trial, the safety and tolerability will be investigated in patients with ALS

(3) Study design
An unblinded study*2) will be conducted at multiple institutes. Patients will receive the same dose of bosutinib once a day every day for 12 weeks.

(4) Number of patients
24

(5) Trial site
Four institutes including Kyoto University Hospital will participate.
The names of the other institutes will be announced later on the website.

(6) Websites
More information can be found at: UMIN-CTR (For medical staff)

5. Researchers and Organizations
  1. Center for iPS Cell Research and Application (CiRA), Kyoto University
    Professor Haruhisa Inoue, Department of Cell Growth and Differentiation
  2. Tokushima University Hospital
    Junior Associate Professor Yuishin Izumi
  3. EPS Corporation, Tokyo, Japan

6. Support
Financial support: The Japan Agency for Medical Research and Development (AMED) Clinical Research and Clinical Trials Promotion Project "Physician-led trial for the development of amyotrophic lateral sclerosis treatment using a patient registry" Investigational product: Pfizer Inc.

7. Notes
*1)Phase 1
The purpose of Phase 1 trials is to measure the safest dose (the dose with minimal side effects) of a drug on a small number of human subjects.

*2)Unblind studies
One or more parties (the researchers, patients, etc.) know which patients received which treatment.

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