Keywords

Introduction

Stem cell therapy, also known as regenerative medicine, promotes the repair response of diseased, dysfunctional, or injured tissue using stem cells or their derivatives. One of the main requirements for cell-based therapy is to test the cells in different phases in humans, called clinical trials. There are four different clinical trial phases, such as Phase-1, Phase-2, Phase-3, and Phase-4 trials, respectively. These clinical trials are performed in healthy persons and patients, which aim to evaluate potential benefits. Such trials’ primary objectives are to find out if the cell-based therapy is safe (healthy person) and therapeutically effective (patients). Often, clinical trials are used to learn if a new cell-based therapy is more effective or has less harmful side effects. In the year 2014, cell-based therapies clinical trials were either sponsored by academic institutions roughly constitute 70% of total clinical trials, and 30% of clinical trials were sponsored by private companies (Bersenev, 2015). It has been observed that a combination of public and private funding was strongly encouraged in the clinical trials conducted by the Californian Institute for Regenerative Medicine, California, USA (Trounson et al., 2010). The investments for stem cell clinical trials have been heavily based on the successful and positive clinical trial results. The primary concern of any investments toward stem cell-based clinical trials is to get clear information about the trial’s outcome. Over the past few years, attempts have been made to better evaluate and monitor the stem cell-based trials at different intervals. It has often been observed that investors have retracted the funding due to non-getting satisfactory trial results. The outcome of clinical trials must be shared with investors with the technical team to decide whether to continue the trials.

There have been reports on clinical trials (Ratcliffe et al., 2013; Trounson et al., 2011), where different stem cells have been used in the clinical trials. Most of the publicly available data on stem cell-based therapy trials either available at the National Institute of Health, USA, and European databases. The outcome of stem cell-based clinical trials has also been published in peer-reviewed journals, and they are available at the National Center for Biotechnology Information, USA. However, some clinical trial data are not available in the public domain. As stem cell-based therapy is a new therapy, it is essential to have the outcome of all clinical trials to be able to develop effective cell-based therapy. Among different stem cells, limbal stem cells and neural stem cells showed significant promising results in regenerative repair. The application of pluripotent stem cells and mesenchymal stem cells (MSCs) is the most widely used cell types for clinical trials. There is also interest in placental-derived stem cells in regenerative medicine. The application of stem cell-based therapy is mostly used in eye-related degenerative diseases. One of the reasons is that small numbers of cells required in the eye regeneration, and tissue is easily accessible for surgery.

Concerning the type of stem cell transplantation, autologous stem cell transplantation is most widely used as there are many benefits of using autologous transplantation such as easily accepted by the body, no immuno rejection issue, and cell survivability and functionality are better. In the case of allogeneic cell transplantation, few clinical trials are conducted, but due to high immuno rejection and low cell acceptability, allogeneic cell transplantation is less favored than autologous cell transplantation. Different types of stem cells, such as pluripotent stem cells and induced pluripotent stem cells, are extensively used in clinical trials (Cyranoski, 2014; Kushner et al., 2014; Schwartz et al., 2012, 2015).

Clinical Trials of Bone and Muscle Diseases Using Stem Cell Therapy

Stem cells have been tested as the treatment for various bone and muscle-related diseases. Many clinical trials are currently in progress or have been completed where stem cells have been used in bone and muscle disease. The summary of clinical trials is listed in the tables.

Bone Disease and Regenerative Medicine

Forty clinical trials are being performed using different types of stem cells to treat bone diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The list of trial which has been conducted during the year 2009–2020 is listed in Table 9.1.

Table 9.1 Bone disease and stem cell therapy
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Clinical Trials in Joint Diseases and Stem Cell Therapy

As per the clinical trial record, more than 110 clinical trials are being performed using different types of stem cells to treat joint diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The list of trial which has been conducted during the year 2019–2020 is listed in Table 9.2.

Table 9.2 List of clinical trials in joint diseases
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Musculoskeletal Disease and Stem Cell Therapy

One hundred sixty clinical trials are being conducted using different stem cells to treat musculoskeletal diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The list of trial which has been conducted during the year 2020 is listed in Table 9.3.

Table 9.3 List of clinical trials in musculoskeletal disease
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Neuromuscular Disease and Stem Cell Therapy

There are 40 clinical trials being conducted using different types of stem cells to treat neuromuscular diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The trial list conducted during the year 2017–2020 is listed in Table 9.4.

Table 9.4 List of clinical trials in neuromuscular diseases
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Clinical Trials of Brain Diseases and Stem Cell Therapy

Stem cells have been tested as the treatment for various brain-related diseases and injuries. Many clinical trials are currently in progress or have been completed where stem cells have been used in bone and muscle disease as per the USA clinical trials portals.

List of Clinical Trials in Brain Injuries

Forty-six clinical trials are being conducted using different types of stem cells to treat brain injuries as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The trial list conducted during the year 2017–2020 is listed in Table 9.5.

Table 9.5 List of clinical trials in brain injuries
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List of Clinical Trials in Brain Diseases

One hundred ten clinical trials are being conducted using different types of stem cells to treat brain diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The list of trial which has been conducted during the year 2017–2020 is listed in Table 9.6.

Table 9.6 List of clinical trials in brain diseases
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List of Clinical Trials in Spinal Cord Injuries

Fifty-two clinical trials are being conducted using different types of stem cells to treat brain diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The trial list conducted during the year 2016–2020 is listed in Table 9.7.

Table 9.7 List of clinical trials in spinal cord injuries
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List of Clinical Trials in Peripheral Nerves Diseases

Forty-six clinical trials are being conducted using different types of stem cells to treat peripheral nerve diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The list of trials conducted during the year 2016–2020 is listed in Table 9.8.

Table 9.8 List of clinical trials in peripheral nerve diseases
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Clinical Trials of Heart Diseases with Stem Cell Therapy

Stem cells are tested as the treatment for various heart-related diseases and injuries. There are many clinical trials either currently in progress or have been completed where stem cells have been used in heart diseases as per the USA clinical trials portals.

List of Clinical Trials in Myocardial Infarction

Ninety-six clinical trials are being conducted using different stem cells in the treatment of myocardial infarction as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The list of trial which has been conducted during the year 2017–2020 are listed in Table 9.9.

Table 9.9 List of clinical trials in myocardial infarction
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List of Clinical Trials in Stroke and Related Diseases

There are 66 clinical trials being conducted using different types of stem cells in stroke and related disease treatment as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The trial list conducted during the year 2017–2020 is listed in Table 9.10.

Table 9.10 List of clinical trials in stroke and related diseases
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List of Clinical Trials in Digestive System Diseases

There are 90 clinical trials being conducted using different types of stem cells to treat digestive system diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The trial list conducted during the year 2017–2020 is listed in Table 9.11.

Table 9.11 List of clinical trials in digestive system diseases
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List of Clinical Trials in Eye Diseases

There are 90 clinical trials are being conducted using different types of stem cells in the treatment of in eye diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The list of trial which has been conducted during year 2017–2020 is listed in Table 9.12.

Table 9.12 List of clinical trials in eye diseases
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List of Clinical Trials in Kidney Diseases

There are 47 clinical trials being conducted using different types of stem cells to treat kidney diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The trial list conducted during the year 2017–2020 is listed in Table 9.13.

Table 9.13 List of clinical trials in kidney diseases
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List of Clinical Trials in Skin Diseases

There are 47 clinical trials in skin diseases as per the USA clinical trials portals (https://www.clinicaltrials.gov/) and (https://stemcellsportal.com/stem-cells-translational-medicine-clinical-trials-portal). The trial list conducted during the year 2017–2020 is listed in Table 9.14.

Table 9.14 List of clinical trials in skin diseases
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