Treatments currently undergoing Clinical Trials
Phases of clinical trials are as follows:
Phase 1: Safety study to see if the procedure or drug is well-tolerated.
Phase 2: Larger study to measure effectiveness of the new treatment against a control group.
Phase 3: Even larger study to compare the effect of various parameters such as dose and administration, and to monitor side effects prior to market release.
Phase 4: Post-marketing studies to learn even more about risks, benefits, and optimal use.
The therapies in clinical trials are a mix of Allogeneic and Autologous modes of treatment.
Auto-Immune
Graft-versus-Host Disease (GvHD)
Phase 3 Allogeneic Trial – pediatric: PROCHYMAL[tm] mesenchymal cells
Phase 3 Allogeneic Trial – adult: PROCHYMAL[tm] mesenchymal cells
Kidney transplant plus stem cells from donor’s allogeneic bone marrow
Diabetes, Type 1 (formerly called Juvenile Diabetes)
Published Phase 1 Trial: autologous cord blood stem cells (follow up trial of expanded Treg cells)
Enrolling Phase 1 Trial: autologous cord blood stem cells
Enrolling Phase 2 Trial: autologous cord blood stem cells
Enrolling Phase 2 Trial: allogeneic cord blood stem cells in the “Stem Cell Educator”, see publication
Approved Phase 2 Trial: Allogeneic PROCHYMAL[tm] mesenchymal cells (granted FDA Orphan Drug Status)
Crohn’s Disease
Phase 2 Allogeneic Trial: placental stem cells, (this was 1st placenta cell trial approved by the FDA)
Phase 2 Autologous Trial: stem cell transplants
Phase 3 Allogeneic Trial: PROCHYMAL[tm] mesenchymal cells
Phase 3 Autologous Trial: ASTIC
Ulcerative Colitis Phase 2 Allogeneic Trial: MultiStem adult stem cells prepared by Athersys
Rheumatoid Arthritis
Phase 2 Allogeneic Trial: placental stem cells prepared by Celgene
Lupus
Autologous Trial: peripheral blood stem cells,
Lab study in mice: Allogeneic MSC from cord blood press & publication
Multiple Sclerosis (MS; note that this disorder could be categorized as either auto-immune or neurological)
Cardiovascular
Cardiac repair & recovery
Review article: Adult applications of Autologous bone marrow
Cardiomyopathy Case Report: Allogeneic cord blood & placental MSC
Developing treatment of heart attack with allogeneic cord blood news and publication
Phase 3 trial: autologous bone marrow UK NHS, REGEN-AMI
Phase 3 trial: autologous bone marrow Eli Lilly et al., REPAIR-AMI
Phase 3 trial: autologous bone marrow Miltenyi Biotec GmbH, PERFECT
Phase 3 trial: autologous bone marrow FCB-Pharmicell, SEED-MSC
Phase 3 trial: autologous bone marrow Mexico Natnl. Heart Inst., TRACIA
Phase 3 trial: autologous bone marrow U. Oulu Finland
Phase 3 trial: autologous bone marrow Royan Inst. Iran
Phase 3 trial: autologous bone marrow Baxter’s Renew
Trial: Allogeneic MSC for heart attacks Mesoblast’s Revascor(TM)
Congenital heart defects (the most common birth defect)
First clinical trial of vascular grafts grown from autologous cells, and news report on first patient
Trial: Autologous cord blood for neurolgical side effects of Hypoplastic Left Heart Syndrome (HLHS)
Published trial: Autologous cord blood for open heart surgery within hours of birth
Critical Limb Ischemia
Phase 3 Trial: Autologous Bone Marrow prepared by Aastrom Biosciences
Phase 1 Trial: Allogeneic Cord Blood
Buerger’s Disease
Phase 1 Trial: Allogeneic Cord Blood
Ischemic Stroke
Review Article
Phase 2 Trial: Autologous Mesenchymal Stem Cells
Phase 2 Trial: Allogeneic Mesenchymal Stem Cells
Phase 2 Trial: Allogeneic placental stem cells prepared by Celgene
Phase 2 Trial: Allogeneic Multistem stem cells from bone marrow and other adult tissues prepared by Athersys
Phase 1 Trial: Allogeneic Cord Blood
Neurologic
Cerebral Palsy
Published Phase 1 Trial: Autologous Cord Blood
Enrolling Phase 2 Trial in Duke Mdeical Center, US: Autologous Cord Blood for spastic CP
Enrolling Phase 2 Trial in Medical College of Georgia, US: Autologous Cord Blood
Enrolling Trial in Monterrey, Mexico: Autologous Cord Blood & Placental Blood for Hypoxia
Phase 2 Trial waiting for approval in Australia: Autologous Cord Blood
Enrolling Phase 2 Trial in Korea: Allogeneic Cord Blood
Hypoxic Ischemic Encephalopathy (HIE)
Phase 1 Trial: Autologous Cord Blood within 2 weeks of birth
Autism
Phase 1 Trial: Cord Blood
Traumatic Brain Injury (TBI is the most common cause of death in young children)
Phase 1 Trial: Autologous Bone Marrow
Phase 1 Trial: Autologous Cord Blood
Spinal Cord Injury (SCI)
Published Phase 1 Trial: Autologous Bone Marrow
Case Report & Review: Allogeneic Bone Marrow
Planned Trial: Allogeneic Cord Blood
(Aside: the first clinical trial using embryonic stem cells is for this condition)
Hearing Loss (acquired sensorineural)
Phase 1 Trial: Autologous Cord Blood
previous research in animals: medical journal, news report
Amyotrophic Lateral Sclerosis (ALS)
Phase 1 Trial: Autologous Mesenchymal Stem Cells (MSC) from Bone Marrow
Review Article
Published case report Allogeneic cord blood
Scleroderma
SCOT Study
Alzheimer’s Disease
Phase 1 Trial: Allogeneic Cord Blood
Misc. “Degenerative Conditions”
Published safety study: Allogeneic Cord Blood
Orthopedic
Cleft Palate Repair (alveolar)
Published study: seeding scaffold with autologous bone marrow stem cells is preferred to bone graft
Cartilage Repair
Phase 3 Trial: Allogeneic Cord Blood prepared by Medipost
Stem Cells & Gene Therapy for Inherited Disorders (all therapies are Autologous)
Cerebral Adrenoleukodystrophy
Cystic Fibrosis
Fanconi Anemia
Severe Combined Immunodeficiency (SCID)
Sickle Cell Anemia
Thalassemia
Wiskott-Aldrich Syndrome
Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Stem Cell Transplantation for Additional Inherited Disorders (all therapies are Allogeneic)
Epidermolysis Bullosa
Lysosomal Storage Diseases (in utero transplant)
Stem Cell Transplatation for New Indications (all therapies are Allogeneic)
HIV “the German patient” medical journal, CNN report