Current status-phase I trial of cell-transplantation therapy with allogeneic adipose tissue-derived multilineage progenitor cells in severe familial hypercholesterolemia patients undergoing LDL-apheresis

2016 ◽  
Vol 252 ◽  
pp. e238
Author(s):  
M. Koseki ◽  
S. Ayami ◽  
T. Okada ◽  
T. Kobayashi ◽  
M. Asaji ◽  
...  
Keyword(s):  
Adipose Tissue ◽  
Phase I ◽  
Progenitor Cells ◽  
Familial Hypercholesterolemia ◽  
Cell Transplantation ◽  
Phase I Trial ◽  
Current Status ◽  
Ldl Apheresis ◽  
Cell Transplantation Therapy ◽  
Transplantation Therapy

Abstract 50: A Phase I Clinical Trial Evaluating the Safety of With Allogeneic Adipose Tissue-derived Multilineage Progenitor Cells-transplantation Therapy in Homozygous Familial Hypercholesterolemia Patients

2015 ◽  
Vol 35 (suppl_1) ◽  
Author(s):  
Masahiro Koseki ◽  
Shizuya Yamashita
Keyword(s):  
Clinical Trial ◽  
Liver Transplantation ◽  
Adipose Tissue ◽  
Progenitor Cells ◽  
Familial Hypercholesterolemia ◽  
Ldl Cholesterol ◽  
Ldl Receptor ◽  
Ldl Apheresis ◽  
Definitive Therapy ◽  
Transplantation Therapy

Familial hypercholesterolemia (FH) is an inherited disorder, mainly caused by defects in low-density lipoprotein (LDL) receptor gene. The patients are characterized by high LDL cholesterol levels in the blood and premature cardiovascular disease. Although most of heterozygous FH patients are usually treated with statin, ezetimibe and bile acid sequestrants, homozygous FH patients are resistant to drug therapy. Therefore, in Japan, many of homozygous FH patients are treated by LDL-apheresis. LDL-apheresis is a great procedure to remove LDL cholesterol from the blood and contribute to improve prognosis of homozygous FH patients. However, the effect of removing LDL cholesterol is temporary and still not enough. As a definitive therapy, liver transplantation therapy could be one of options to recover LDL receptor, but donor is limited in Japan. Therefore, based on the increase of the evidence about the safety of mesenchymal stem cells and percutaneous transhepatic portal approach in islet transplantation, we have developed a cell transplantation therapy with allogeneic adipose tissue-derived multilineage progenitor cells (ADMPCs), as an alternative treatment instead of liver transplantation. Our group has already proved that xenogenic transplantation of human ADMPCs into Watanabe heritable hyperlipidemic rabbits resulted in significant reductions in total cholesterol, and the reductions were observed within 4 weeks and maintained for 12 weeks. These results suggested that hADMPC transplantation could correct the metabolic defects and be a novel therapy for inherited liver diseases. Here, we report a protocol for the first-in-human clinical trial, which has been approved by the institutional review board and Ministry of Health, Labour and Welfare, Japan.

Abstract 602: Development of Cell Therapy in Severe Familial Hypercholesterolemia

2016 ◽  
Vol 36 (suppl_1) ◽  
Author(s):  
Masahiro Koseki ◽  
Ayami Saga ◽  
Takuya Kobayashi ◽  
Masumi Asaji ◽  
Takeshi Okada ◽  
...  
Keyword(s):  
Familial Hypercholesterolemia ◽  
Cell Transplantation ◽  
Ldl Cholesterol ◽  
Phase 1 ◽  
Safety Evaluation ◽  
Current Status ◽  
Ldl Apheresis ◽  
Rejection Reaction ◽  
First Case ◽  
Premature Cardiovascular Disease

Familial hypercholesterolemia (FH) patients are characterized by high LDL cholesterol levels in the blood and premature cardiovascular disease. Although most of heterozygous FH patients are able to be treated with statin, ezetimibe and bile acid sequestrants and anti-PCSK9 antibodies, homozygous FH patients are resistant to drug therapy. Therefore, many of homozygous FH patients used to be treated by LDL-apheresis. However, the effect of removing LDL cholesterol is still not sufficient and definitive therapies need to be developed. With the increase of the evidence about the safety of mesenchymal stem cells and percutaneous transhepatic portal approach in islet transplantation, we have developed a cell transplantation therapy with allogeneic adipose tissue-derived multilineage progenitor cells, as an alternative treatment instead of liver transplantation. We have generated a protocol for the phase 1 clinical study. The primary endpoint should be safety evaluation including allergic reaction, rejection reaction, thrombosis and complications in the blood vessel by cell-transplantation. The secondary endpoint should be the efficacy including changes in serum lipid such as LDL-C, TC, HDL-C, TG and Lp(a), and Secession possibility of LDL-apheresis. We transplanted the first case in February, 2016. Then we are collecting clinical data. We would demonstrate the current status and the issues to be solved for the following study.

scholarly journals Current Status and Future Development of Cell Transplantation Therapy for Periodontal Tissue Regeneration

2012 ◽  
Vol 2012 ◽  
pp. 1-8 ◽  
Author(s):  
Toshiyuki Yoshida ◽  
Kaoru Washio ◽  
Takanori Iwata ◽  
Teruo Okano ◽  
Isao Ishikawa
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
New Technology ◽  
Basic Research ◽  
Current Status ◽  
Periodontal Tissue ◽  
Research Risks ◽  
Cell Transplantation Therapy ◽  
Transplantation Therapy

It has been shown that stem cell transplantation can regenerate periodontal tissue, and several clinical trials involving transplantation of stem cells into human patients have already begun or are in preparation. However, stem cell transplantation therapy is a new technology, and the events following transplantation are poorly understood. Several studies have reported side effects and potential risks associated with stem cell transplantation therapy. To protect patients from such risks, governments have placed regulations on stem cell transplantation therapies. It is important for the clinicians to understand the relevant risks and governmental regulations. This paper describes the ongoing clinical studies, basic research, risks, and governmental controls related to stem cell transplantation therapy. Then, one clinical study is introduced as an example of a government-approved periodontal cell transplantation therapy.

scholarly journals Potential Therapies Using Myogenic Stem Cells Combined with Bio-Engineering Approaches for Treatment of Muscular Dystrophies

Cells ◽  
2019 ◽  
Vol 8 (9) ◽  
pp. 1066 ◽  
Author(s):  
Motohashi ◽  
Shimizu-Motohashi ◽  
Roberts ◽  
Aoki
Keyword(s):  
Stem Cells ◽  
Cell Transplantation ◽  
Genetic Disorders ◽  
Current Status ◽  
Muscular Dystrophies ◽  
Myogenic Cells ◽  
Myogenic Stem Cells ◽  
Bio Engineering ◽  
Cell Transplantation Therapy ◽  
Transplantation Therapy

Muscular dystrophies (MDs) are a group of heterogeneous genetic disorders caused by mutations in the genes encoding the structural components of myofibres. The current state-of-the-art treatment is oligonucleotide-based gene therapy that restores disease-related protein. However, this therapeutic approach has limited efficacy and is unlikely to be curative. While the number of studies focused on cell transplantation therapy has increased in the recent years, this approach remains challenging due to multiple issues related to the efficacy of engrafted cells, source of myogenic cells, and systemic injections. Technical innovation has contributed to overcoming cell source challenges, and in recent studies, a combination of muscle resident stem cells and gene editing has shown promise as a novel approach. Furthermore, improvement of the muscular environment both in cultured donor cells and in recipient MD muscles may potentially facilitate cell engraftment. Artificial skeletal muscle generated by myogenic cells and muscle resident cells is an alternate approach that may enable the replacement of damaged tissues. Here, we review the current status of myogenic stem cell transplantation therapy, describe recent advances, and discuss the remaining obstacles that exist in the search for a cure for MD patients.

scholarly journals Neural stem cells: involvement in adult neurogenesis and CNS repair

2008 ◽  
Vol 363 (1500) ◽  
pp. 2111-2122 ◽  
Author(s):  
Hideyuki Okano ◽  
Kazunobu Sawamoto
Keyword(s):  
Stem Cells ◽  
Neural Stem Cells ◽  
Cell Transplantation ◽  
Adult Neurogenesis ◽  
Embryonic Stem ◽  
Adult Brain ◽  
Therapeutic Interventions ◽  
Cell Transplantation Therapy ◽  
Transplantation Therapy

Recent advances in stem cell research, including the selective expansion of neural stem cells (NSCs) in vitro , the induction of particular neural cells from embryonic stem cells in vitro , the identification of NSCs or NSC-like cells in the adult brain and the detection of neurogenesis in the adult brain (adult neurogenesis), have laid the groundwork for the development of novel therapies aimed at inducing regeneration in the damaged central nervous system (CNS). There are two major strategies for inducing regeneration in the damaged CNS: (i) activation of the endogenous regenerative capacity and (ii) cell transplantation therapy. In this review, we summarize the recent findings from our group and others on NSCs, with respect to their role in insult-induced neurogenesis (activation of adult NSCs, proliferation of transit-amplifying cells, migration of neuroblasts and survival and maturation of the newborn neurons), and implications for therapeutic interventions, together with tactics for using cell transplantation therapy to treat the damaged CNS.

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