Transcript Human Organ

Future Pharmaceutical Sciences and
Recent Advancement of Medical Sciences
Kunio KAWAMURA, Ph.D.
Otsuka Pharmaceutical Co. Ltd.
Outline of this speech-1
Following two topics will be presented as representatives
Co.
(1) Application of iPS : iPS cells are engineered by
introducing specific genes into cells derived from the
skin, blood and other body tissues. They have the
potential capacity to develop into cells of various parts
of the body. The first clinical study has just started to
treat age-related macular degeneration, an intractable
disease of the eye. iPS cells turn into pigment epithelial
cells, and thereafter, are cultivated to multiple
applicable sheets for transplants to many patients. The
cultivated cell sheets will be a kind of medical device.
The application is now expanding to various medical
sciences.
Outline of this speech-2
(2 ) Clinical trials using therapeutic porcine cell
implants have already been at the final stage.
Microspheres containing live pig’s islet cells are
implanted into a patient's abdomen. Once implanted, the
encapsulated islet cells work by self-regulating and
efficiently secreting insulin and glucagon in response to the
patient's changing glucose levels. The capsules containing
islet cells are protected from the body's immune response
by the semi-permeable membrane, and also permit the
bidirectional diffusion of molecules essential for cell
metabolism and the outward diffusion of waste products.
This technology is now being extended to treat other
diseases. Those encapsulated cells will be categorized as
combination products
Human Organs/Cells Transplantation
1．Shortage of Donors
2．Utilization of iPS Cells
1) Use of Human Cells (ex. Conversion from
Human skin cells)
2) Generation of Human Organ in Animal by
Animal-Human Chimeric Embryo Technology
(ex. Human Blood in sheep. Human Heart and/or ,
spleen in Pig)
3. Make use of Animal Organ by an New Invention to
Eliminate Rejection Reaction．
Clinical trials are now progressing for 2-1) and 3. These have
need for new concept of pharmaceutical sciences.
Transplantation of Organ in
Human
Current situation
Human-Human Heart Transplantation in Japan
Waiting
Transplanted
Organ, Tissue and Cell Transplantation
1. Generation of Human Organ, Tissue
and/or Cells from iPS Cells
2. Generation of Human Organ, Tissue
and/or Cells in Animal by Elimination
Rejection
3. Making Use of Animal Tissue or Cell,
as it is, by Elimination Rejection
Mechanisms of Rejection Reaction
Lymphocytes/Immune cells attack the Transplanted Organ
Lymphocytes/
Immune Cells
Transplanted Organ
In order to Reduce Rejection reactions
There are two methods.
1) One is to use the same kind of biological
products. ex. Human – Human transplantation.
2) The second is to make change the differences to
harmonize, by make use of genetic technology.
ex. using iPS cells.
3) The third is to protect cells from the rejection
reactions by artificial barriers. ex. Protecting cells
by blocking higher molecular barrier.
Examples of New Type of
Pharmaceuticals and Medical devices
・Generation Retina sheet for multi-patients,
(under clinical trial)
・Generation of Blood/Blood cells in animals formultipatients,
・Encapsulated Insulin Secreting Pig cells
( under clinical trial)
Pharmaceutical science should cooperate and contribute the
completion of these technology, and have to care how to deliver,
storage and use.
ES Cell and iPS Cells
Embryonic stem cells are able to develop into any type of cell.
Embryonic stem cells (ES cells) are pluripotent stem cells derived from
the inner cell mass of a blastocyst, an early-stage embryo . Human
embryos reach the blastocyst stage 4–5 days post fertilization, at
which time they consist of 50–150 cells. Isolating the embryoblasto or
inner cell mass (ICM) results in destruction of the fertilized human
embryo, which raises ethical issues.
Induced pluripotent stem cells, abbreviated as iPS cells are a type of
pluripotent stem cell artificially derived from a non-pluripotent cell –
typically an adult somatic cell – by inducing a "forced" expression of
specific genes. Induced pluripotent stem cells are similar to natural
pluripotent stem cells, such as embryonic stem (ES) cells, in many
aspects. Induced pluripotent cells have been made from adult
stomach, liver, skin cells and blood cells.
How to manufacture iPS Cells
Four kinds of genes
Skin Cells
Reset
CELLS
Cells
Muscle cells
Nervous Cells
Skin Cells
White Blood Cells
Red Blood Cells
Application of iPS Cell Technology
to Medical science
The first case of iPS Cell thechnology to medical
sciences.
“ Age-Related Macular Degeneration：
iPS Cells-Derived Retinal Pigment Epithelium
Cell”
Medical Treatment of Age-Related
Macular Degeneration for Eye
Patients of Age-Related Macular Degeneration for Eye
Cells
10 months
Skin Cells
Genes into Cell
iPS Cells
Macula Cell sheet
Trans Plantation
Age-Related Macular Degeneration
ー iPS Cells-Derived Retinal Pigment Epithelium Cell
Sheey （シートを将来市販する：医療機器）
iPS Cells
Cultured
cells are
Transplanted
to Retina
Cultured to
Cell sheet
Retinal
Pigment
Epithelium
Cell
Eye
lens
Rtinal Pigment
Epithelium Cell
The second Case:
Xenotransplantation
Human-animal Transplantation
Animal-Human Chimeric Embryo
Embryo
Animal Embryo
Human Cells
Use of Animal Organ –
Xeno-trans-plantation1．How to Overcome Rejection/
Immune
2．Ethics of Life
Human Organ (Pancreas) cultivated in Pig
Human iPS
cells
Fertilized
egg
Genetically
manipulated Embryo
Birth of animal
with human
pancreas
Injection to
animal womb
and incubate
Method 3
Making Use of Animal Tissue or Cell,
as it is, by Elimination Rejection
Nutrition, Oxygen
Insulin
Anti-Body
Pig Islet Cells
Semi-permeable
Membrane
Cell Encapsulation
Animal Organ from Pig cells to be Transplanted
Get animation: Processing pig cells for transplants
Capsules Containing Cells
Islets isolated from
neonatal porcine
pancreas in alginatepolyornithine
Capsules for sale ( Before Use)
Label of Clinical Use Product
Diabetes Treatment Utilizing Implanted
Encapsulated, Insulin-Producing Cells
Would Replace Existing Type 1 Diabetic
Drugs
1. iPS cell technology is usefull and
promising in future,
2. Living cell encapsulation technology
will also have various future
application.
Thank you for your attention
Kunio KAWAMURA