Pilot Clinical Study of Regenerative Medicine Products

  • CGS provide consulting services of the following early clinical studies of RM products;
    • Clinical Pharmacology
    • Early Clinical Trial (First in Human)
    • 1. Current Status of Japanese Clinical Research using Regenerative Medicine Cell
    • 　The clinical researches more than 80 within Japan using the transplant of somatic stem cell which is specific for earch organ or tissue have
      　performed and continued, based on "MHLW guidance regarding safety security of regenerative medicine" (which was construced on 3rd July,
      　2008) and "Law Art. 85 concerning safety security of regenerative medicine".
      　The most clinical researches had been performed using transplant therapy of the bone marrow-derived hematopoietic stem cells or
      　mesenchyma stem cells. In addition, the clinical research using the sheet form prepared by a somatic cell or a tissue stem cell was
      　performed. As for clinical research of human iPS cells, an autologous human iPS cell has been transplanted into the patient with age-wet
      　macular degeneration.
    • 2. Approximately 30 companies including mainly venture companies in the United States and a part of major pharmaceutical companies have
      　 performed GCP trials of the regenerative medicine products worldwide. Regenerative medicine products in phase III trials are the muscular
      　 tissue-derived autologous stem cell (Indication: Cardiac function), the product transferred a gene to hematopoietic stem cells / precursor cell
      　 (Indication: Chronic cardiomyopathy), the umbilical cord tissue-derived mesenchymal stem cell (Indication: Osteoarthritis), the adipose
      　 tissue-derived stem cell (Indication: Neck damage), etc. (Please see table1). Most are the clinical trials using somatic stem cells.
      　 In addition, the only clinical trial of the regenerative medicine product using the human embronic stem cell is ongoing as as indication of
      　 Type I diabetes mellitus (pancreatic β cell reproduction) in the United States.
      　 But GCP clinical trial of regenerative medicine product using human induced pluripotent stem cells is not performed yet worldwide so far.
    • 　 The human allogeneic marrow-derived mesenchyma stem cell (Indication: Graft versus host disease), which was imported from the United
      　 States, has been on Japanese market only from 2016 after getting regulatory approval in 2015.
  • No. Cell Indication Development Stage
    1 The pancreatic endoderm cell which was provided by the differentiation process of the human ES stem cell strain
    (Viacyte Inc. in US)    		 Type-I Diabetes Mellitus
    (Regeneration of pancreatic cell)

    Pancreatic endoblast cells are delivered within the body by drug delivery system with implantable medical devices.    		 Phase I/IIa
    2 Allogenic human bone marrow-derived mesenchyma stem cell
    Prochuymal®
    (OsirisTherapeutics in US)
    		 GvHD (Graft vs. Host Disease),
    		 Approvaed in Canada in 2012, New Zealand and Japan in 2015, Phase III in USA
    3 Allogenic human bone marrow-derived mesenchyma stem cell
    Prochuymal®
    (Osiris Therapeutics in US)    		 Crohn's disease
    (Fast Track),
    Restoring pulmonary tissue of chronic obstructive pulmonary disease,
    Restoring heart tissue during heart attack,
    Prevention of pancreatic is let cell in patients with Type-I Diabetes    		 Phase II
    4 Autologous CD+34 cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) enzyme,
    (Ex-vivo stem cell gene therapy)
    (Orchan、GSK）    		 Treatmen for ADA deficiency (severe combined immunodeficiency disease) 2016 submitted in European Union
    5 Human autologous placenta-derived stem cell
    (intraveous and intramuscular administration, CCT Inc.)    		 Crohn's disease
    Ischemic stroke
    Rheumatoid arthritis
    Multiple scleorosis
    Diabetic foot ulcer
    Peripheral arterial disease    		 Phase II
    6 CAR-T cell therapy
    (CRA: chimeric antigen receptor）
    (Iv infusion administration, Novartis)
    		 Acute lymphocytic leukemia
    Chronic lymphocytic leukemia
    Multiple myeloma    		 Phase II in EU, Canada and Australia
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