REGENERATIVE MEDICINE

-THE VANGUARD OF 21ST

CENTURY HEALTHCARE
BY
KINNAR DIXIT
Roll no : 7,SEM-1
M.B.A.(PHARM)
NIPER S.A.S Nagar
Agenda
 What is Regenerative Medicine?
 How Regenerative medicine works?

 Why do we need Regenerative

Medicine?
 What is the current state of

Regenerative medicine?
 Market Opportunities for Regenerative

Medicine
 Government Policies Around The Globe
 Conclusion

What is Regenerative
Medicine?
 Regenerative medicine is the process of
creating living, functional tissues to
repair or replace tissue or organ
function lost due to age, disease,
damage, or congenital defects
 Holds the promise of regenerating
damaged tissues and organs in the
body by stimulating previously
irreparable organs to heal themselves

Source : National Institute of Health , US

Contd..
 Also empowers scientists to grow tissues
and organs in the laboratory and safely
implant them when the body cannot
heal itself
 Regenerative medicine has the potential
to solve the problem of the shortage of
organs available for donation compared
to the number of patients that require
life-saving organ transplantation
How Regenerative medicine
works?
 Regenerative medicine is the application
of tissue science, tissue engineering,
and related biological and engineering
principles that restore the structure and
function of damaged tissues and organs
1.Using therapies that prompt the body to
autonomously regenerate damaged
tissues
2.Using tissue engineered implants to
prompt regeneration
Regenerative Medicine
Opportunities
Tra ch e a
M u scle

B re a s t
V a lv e
L iv e r
V e sse ls
N e rv e

K id n e y
U re te r
B la d d e r
B one
O th e r g e n ito u rin a ry
S k in
C a rtila g e
Neo-bladder
A Model of Regenerative Medicine

A surgeon takes a Scientists isolate The isolated cells The cultured cells are
small, full- urothelial and smooth are cultured properly seeded onto a
thickness biopsy muscle cells that are separately until biodegradable scaffold
from the patient’s capable of there are a shaped like a bladder.
bladder. regeneration. sufficient quantity.

The biodegradable
scaffold dissolves
and is eliminated The body uses the The neo-bladder Scientists ensure that
from the body, neo-bladder construct is the cells attach and grow
leaving a construct to implanted by the properly throughout the
functioning bladder regenerate and surgeon using scaffold. After about 8
made only of the integrate new standard surgical weeks, the neo-bladder
patient’s own newly tissue, restoring techniques. construct is returned to
regenerated tissue. the bladder’s the surgeon for
functionality. implantation.
Why do we need


Regenerative
Medicine?
Need of Regenerative
Medicine

 Regenerative Medicine is a cure, not

a treatment
 Many of today’s increasing healthcare
costs are from recurring treatments for
chronic diseases and their subsequent
complications
Example
 One such example is insulin therapy for
type 1 diabetes, and glucose therapy
for type 2 diabetes
 While insulin and glucose can help
patients manage diabetes, these
therapies do not cure diabetes, nor do
they prevent long-term complications,
such as kidney failure
 Underlying cause of more than 68,000
deaths and the contributing cause of
death in more than another 141,000
individuals
Contd..
 Through regenerative medicine, insulin-
producing pancreatic islets could be
regenerated in vivo or grown in vitro
and implanted, creating the potential
for curing the patient and completely
eliminating the need for future
treatments
Some other examples
 Other potential regenerative medical
advances include the ability to improve
myocardial (heart) functions, which
would help combat heart failure
 Regenerative medicine will enable
doctors to grow new blood vessels
through vascular endothelial growth
factor (VEGF) techniques, and by
improving myocyte growth. With these
techniques, heart damage could be
repaired, saving countless lives
 One of the greatest needs for
regenerative therapy is in the field of
whole organ replacement
 In 2002 alone, there were:

Ø 12,800 organ donors (deceased and

living)
Ø 24,900 life saving transplants
Ø 88,242 patients still on the waiting list at
the end of the year
Ø 6,439 people who died while waiting for a
transplant
Source :Website of US Dept. of Health and Human services : www.hhs.gov

 In total, the cost of all organ replacement
therapies in the U.S. is estimated to
exceed $100 billion per year
 Organ demand is a major health care
issue that is growing in magnitude
 Over the past 10 years, while organ
donations have increased, the waiting
list has grown even more

Source :Website of US Dept. of Health and Human services : www.hhs.gov

 The current world market for replacement
organ therapies is in excess of $350
billion, and the projected U.S. market
for regenerative medicine is estimated
at $100 billion

Source :Website of US Dept. of Health and Human services : www.hhs.gov

 An example of some conditions and diseases
that could be easily cured by regenerative
medicine and their current cost of
treatment include:
Ø 250,000 patients receive heart valves, at a
cost of $27 billion annually

Ø 950,000 people die of heart disease or

stroke, at a cost of $351 billion annually

Ø 17 million patients with diabetes, at a cost of

$132 billion annually
Source
 :Website of US Dept. of Health and Human services : www.hhs.gov
 What is the
current state of
regenerative
medicine?
Current state of
regenerative medicine
 At a functional level, regenerative
medicine researchers have been able to
grow heart arteries and create artificial
blood in the laboratory
 Engineered bladders, ligaments and stem
cell therapies are in various stages of
preclinical and clinical tests
 However, the only FDA-approved and
available products are much simpler
tissues, such as dermal and joint
substitutes, and bone marrow for
orthopaedics
 In India
 Sri Ramachandra Medical college in
Chennai, Kokilaben Dhirubhai Ambani
Hospital and Medical Research Institute
in Mumbai are some names which offers
stem cell therapy
 Reliance Life sciences Pvt. Ltd, Life cell
International Pvt. Ltd, Regenerative
Medical Services Pvt. Ltd and Cryosave
Pvt. Ltd offer cell culture to above
hospitals
The U.S. President Barack Obama
was in the news recently for
signing the Executive Order
reversing the former President’s
August 9, 2001 policy of
providing limited federal
funding for embryonic stem cell
research.

Source : The Hindu April 7, 2009

Market


Opportunities for
Regenerative
Medicine
Building an Industry
 > 350 Private or Public corporations in EU,
Asia, and US developing regenerative
medical products
 Small/large molecule, scaffold, cell, combination


 6 major therapeutic categories

 Cardiology, Dermal, Dental, Neurology, Orthopedic, Urology


 Industrial Pipeline – worldwide

 >100 companies currently with active R&D product program
 ~50-75 different product programs within each therapeutic
category
 60% Research/Preclinical Development, 40% Clinical


 Financials
 >$5B Market Capitalization (US)
 ~33% of Venture Capital’s
 In India

 At least 60 clinical trials for stem cells

therapies are under way currently and
many more trials will start with the
approval of Drug Controller General Of
India


S o u rce : M in t A u g u st 4 , 2 0 0 9
 Exploration In India

S o u rce : M in t A u g u st 4 , 2 0 0 9
Selected Examples of
Building an Industry
 1996: Integra's Artificial Skin approved by the FDA
 Cell based/natural scaffold product
 1997: Genzyme’s Carticel approved by FDA.
 Cell based/scaffold free product
 1998: Organogenesis’ Apligraf Skin Equivalent approved by FDA
 Claim: "first manufactured living human organ"
 1998: DePuy’s Restore Orthobiologic Soft Tissue Implant approved
by FDA
 Scaffold based/cell free product
 2006: Osiris’s PROCHYMAL™ Fast-Track by FDA/Orphan status by
EMEA
 Cell based/scaffold free product
 2006: Tengion’s neo-bladder Phase II IND accepted by FDA.
 Cell based/synthetic scaffold product

 

Anticipated Industry
Revenue Growth
S o u rce – C a p e g e m in i re p o rt
 

 Therefore, Pharma companies need to

define their own position now and decide
if or when they will enter the field of
Regenerative Medicine
S o u rce – C a p e g e m in i re p o rt
Government Policies Around
The Globe
 India's federal government has launched
a national stem cell research program
that will establish six research clusters
in six cities across the country
 The Federal Initiative for Regenerative
Medicine (FIRM) is proposed by U.S.
Dept. Of Health & Human Services
 China has invested heavily in its key
project “Stem Cell and Tissue
Engineering for Regenerative Medicine”

 The Kobe Medical Industry Development
Project by Japanese Govt. aims to
nurture an industry in the fields of
regenerative medicine
 The plan includes spending a total of
$831 billion by the year 2010
NCRM
 NCRM is India’s biggest regenerative
medicine institute developing protocols
in regenerative medicine
 Nichi-In* Centre for Regenerative
Medicine (*Nichi = Japan, In = India in
Japanese), abbreviated as NCRM is an
Indo-Japan joint venture institute
 NCRM has the uniqueness of Japanese
cutting edge technological strength and
core Indian Clinical & Research Skills
 The FDA has created the Office of
Combination Products and the Office of
Cellular, Tissue and Gene Therapies
 These new offices solve the problem of
regulating complex combination
products that typically fell under two or
more of the FDA’s traditional offices
 These changes help prepare the FDA for
regenerative medicine product
regulation and product approvals
 The Brazilian government, meanwhile,
has invested $US4.3 million to conduct
a three-year clinical trial of autologous
stem cell therapy for the treatment of
heart disease
 However, a recent study also identified
regenerative medicine activities to
varying degrees in 31 low- and middle-
income countries, including, for
example, Cuba, Argentina, South Africa,
Egypt, Iran, and Malaysia
 Investment by Pharmaceutical
companies
 In November 2008 Pfizer formed a new
dedicated research unit, Pfizer
Regenerative Medicine, which will
explore the use of stem cells to develop
future treatments with the goal of
delivering new medicinal products using
cells as therapeutics


S o u rce : P fize r w e b site

 New Reliance regenerative medicine
centre in Thane
 One of the labs selected by the US
government's national institute of
health for stem cell research, would
have this large centre as an expansion
of the embryonic stem cell research
programme that has been carried out
by the RLS for the last little over a year

S o u rce : T h e Tim e s O f In d ia , 1 st September 2001

 Manipal Acunova, promoted by the
Manipal Group, is investing $10 million
to position itself as a biotech and
clinical research company and branch
into stem cell research
 For stem cell research, it is setting up
facilities designed by Zanders of
Germany in Bangalore

Source : Biospectrum Asia website

Current barriers to
progress
 The lack of research related to the
fundamental building block areas of the
science
 The lack of interdisciplinary study. There
is very little collaboration and
communication among biologists,
clinicians, engineers, biochemists,
materials scientists and other related
fields due to the emphasis on private,
product-focused funding
 Cost of research
Future Thought
 The 2000 census report finds that there
are 35 million Americans aged 65 or
older
 In 10 years, this number is expected to
increase dramatically as the 56.6 million
Americans who are currently aged 55-
64 join the senior citizen age group
 By 2030, as the last baby boomer
becomes a senior citizen, the population
of senior citizens over the age of 65 in
the U.S. will be double today’s number,
for a total of 70 million
 Current national healthcare costs are in
excess of $1.5 trillion annually, or 13
percent of Gross Domestic Product (GDP)
 Accordingly, as much as 25 percent of the
U.S. GDP would be devoted to healthcare
by 2030
 The majority of these projected costs are
from recurring treatments for diseases that
arise from tissue failure commonly seen in
the elderly
 Because regenerative medicine focuses on
functional restoration of damaged tissues,
not abatement or moderation of
Future aspects
 Within 5 years, organ patches that repair
damaged tissues would potentially be
available
 Within 15 years, full organ regeneration is
a strong possibility
 With a $350 billion global industry already
built on first generation tissue and organ
therapy products and substitutes,
regenerative medicine has potential to
exceed $500 billion in the next 20 years
Conclusion
 Furthering this field would create jobs and
grow a new sector of the healthcare
industry while creating a new
generation of life-saving products
 Therefore, Pharma companies need to
define their own position now and
decide if or when they will enter the
field of Regenerative Medicine
 Thank You