The Gene Therapy Research Institution will start the development of gene therapy in the CNS (including Parkinson’s disease, ALS and Alzheimer’s disease) and research for phase I and phase II clinical trials with The Institute of Medical Science, The University of Tokyo. Shin-ichi MURAMATSU, board member of the Gene Therapy Research Institution (Extraordinary professor, Jichi Medical University), takes a position as an extraordinary professor at The Institute of Medical Science, The University of Tokyo as of November 1, 2014.
This clinical research targets intractable diseases in the area of the CNS, such as 1) Parkinson’s disease, 2) ALS and 3) Alzheimer’s disease and will be carried out at the Research Hospital, The Institute of Medical Science, The University of Tokyo. This research will use adeno-associated virus (AAV) for gene transfer for the treatment of Parkinson’s disease, ALS and Alzheimer’s disease. This research will attempt to confirm the safety and efficacy of this treatment method. In the United Sates and Europe clinical research using AAV for gene transfer has already started. It has become the standard method for gene transfer. The field of research and development of the Gene Therapy Research Institution and The Institute of Medical Science, The University of Tokyo are congruent with the development of gene therapy in the area of the CNS using AAV. Therefore, we agreed to sign this collaborative research agreement.
The Gene Therapy Research Institution’s gene therapy is based on a special technique using AAV which is capable of safe and effective gene transfer in selective nerve cells (Patent pending by Jichi Medical University. The Gene Therapy Research Institution signed the agreement in order to use this patent.). Our objective is to deliver genes from the brain and spinal cord to as many nerve cells as possible. Especially, for neurological disorders such as ALS and Alzheimer’s disease. One potential advantage of this technique is to positively affect the efficacy of the treatment.
However, to take advantage of this technique, we need to establish an efficient large-scale production method and register it as intellectual property. The production cost of current AAV for treatment is prohibitive, but by utilizing a new method (baculovirus-based chimeric vector method which is a large-scale production method of AAV which implants the target genes to microorganisms in a large tank. This is the latest technique used to produce vaccines such as the influenza vaccine on a large-scale.), it is possible to reduce production costs by several hundred times. Therefore, we need to develop this production method as soon as possible.
The Gene Therapy Research Institution plans to outsource the development of this production method and will prepare to begin clinical research to confirm the treatment’s safety and efficacy at the Research Hospital, The Institute of Medical Science, The University of Tokyo. We will able to conduct our research under ideal circumstances in the Center for Translational Research which is located in the Research Hospital. The Center for Translational Research includes the Core Facility for Therapeutic Vectors (CFTV) where we can produce vectors and genetic recombinant viruses. We will also able to adjust vectors which are needed for gene therapy and administer them to cells based on GMP (Good Manufacturing Practice). We plan to discuss the use of these facilities in the future.
Accordingly, we will move forward with this clinical research and the development of production methods for AAV as well as the application of clinical trials and advanced medical care. After confirming the research results, we will act on the early implementation of gene therapy.
■Outline of the collaborative research
Research performer: The Institute of Medical Science, The University of Tokyo
Collaborative researcher: Gene Therapy Research Institution
Research subject: Development of gene therapy in the CNS (including Parkinson’s disease, ALS and Alzheimer’s disease) and the research for phase I/II clinical trials
Research content: Clinical research of gene therapy utilizing AAV for 1) Parkinson’s disease, 2) ALS and 3) Alzheimer’s disease
1. Develop protocol of clinical research for each disease
2. Develop AAV which implants target gene transfer for each disease
3. Applications for clinical research
4. Perform phase I/II clinical research
5. Analysis and evaluation
Research term: November 1, 2014 to March 31, 2017
Number of cases (plan): about 6 cases for each disease