Clinical Trials

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Clinical Trials
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Clinical Trials

• Advanced Cell Technology embryonic stem cells,
  hemangioblasts (FDA approved)
• Macular degeneration, vascular repair, skin
  repair
• Cytori Adipose stem cells
• breast reconstruction, Heart repair
• Geron Embryonic stem cells (FDA approved)
• Spinal repair, heart repair, diabetes,
  osteoarthritis, osteoporosis
• OncoMed cancer stem cells
• Solid tumors
• Osiris Mesenchymal stem cells
• Graft-vs-Host disease, heart repair, bone and
  cartilage repair
• Stem cells Inc. Fetal neural stem cells (FDA
  approved)
• Batten Disease (lysosomal storage disorders)
• Tengion Tissue engineering

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Clinical Trials
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Clinical Trials
Most clinical trials fail
Ismail Kola John Landis Nature Reviews Drug
Discovery 3, 711-716 (August 2004)
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Clinical Trials

• Advanced Cell Technology
• Terminally differentiated cells made from human
  embryonic stem cells. Retinal pigment epithelial
  cells for Stargardts Macular Dystrophy and
  macular degeneration.
• Animal studies no tumors.
• Detection sensitivity less than 11,000,000 cells
  (in vitroin vivo?)
• 50,000 cells per transplant
• Twelve patients enrolled, none transplanted

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Clinical Trials

• Stem Cells Inc.
• Neural cells made from fetal neural stem cells
  for Batten Disease, a fatal condition with
  cognitive disorders. Bore holes into the skull,
  into which stem cells are injected.
• Subjects for the trial were children pre-teen and
  younger, raising issues about informed consent
  and expectations of benefit in a safety trial.
• Such trials for patients must not be greater
  than minimal risk. Placebos in high-risk trials
  are controversial, so-called sham surgeries.
• Six patients enrolled, one died from disease
  progression. Trial terminated in April because of
  lack of enrollment in Phase 1b.

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Clinical Trials

• Geron Corporation
• Oligondendrocytes derived from H9, a human
  embryonic stem cell line
• Human subjects 18-65 years old, recently injured
  with single spinal contusion (not severing or
  penetration). Must have procedure between 7 and
  14 days of administration. 50,000 people suffer
  spinal injuries every year in US
• 2,000,000 cells administered.
• Three procedures Stabilize the injury at a
  trauma center, receive the transplant, undergo
  intensive physical therapy

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Clinical Trials
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Clinical Trials
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Clinical Trials Heart Disease
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Diseases of heart muscle tissue

• Diseases of cardiac muscles
• Congestive heart failure, or ineffective pumping
  due to cardiomyocyte dysfunction, affects 4.8
  million people in U.S.
• Heart attack, also known as myocardial
  infarction
• Heart arrhythmias, or abnormal activity
  (palpitations)
• 1 of live births display a congenital heart
  defect

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Fill in the blankmyocardial cells hematopoetic
cells
?
?
?
?
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Heart and bloodSharing an ancestor
Lineage restriction
Differentiation
FGF8
Cardiogenic mesodermal cell
Anterior lateral plate mesodermal cell
Cardiomyocyte
Fetal Stage
Adult Stage
Lateral plate mesodermal cell
Posterior lateral plate mesodermal cell
Hemangiogenic mesodermal cell
Blood cells
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Clinical Trials Heart Disease

• Autologous (from you, to you) transplants into
  patients with end-stage cardiac disease
• Cells used from bone marrow
• First Brazil, Portugal, then the UK (BOOST)
  trial in 2004
• Highest number of stem cell trials of any type
  (not counting cancer trials)

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Clinical Trials Heart Disease
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Clinical Trials Heart Disease
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Clinical Trials Heart Disease

• Phase 0 or 1 studies
• Not all studies are FDA approved
• Most studies in other countries
• Heart HSC trials increasing
• About 140 clinical trials now

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Discussion discoveries, companies, and products

• What motivates scientists? Businesses?
• How do biotechnology companies start? Survive?
• What obligations do biotechnology companies have
  to society?

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Activity 2 Public Policy Panel Discussion

• Consensus statements and discussion grading
  scheme
• Presentation and Organization state position
  clearly present argument in logical order
  respectful and engaging writing and presentation
  style.
• Science Understanding Use accurate scientific
  vocabulary, facts, and concepts present
  information to support argument both
  scientifically, ethically, and socially
  understand implications of science policy
• Logic Reasoning Give arguments with sound
  reasoning draw on references from assigned
  readings and research use strong concise points,
  use supporting evidence interpret evidence
  correctly address challenges with counter
  arguments

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