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Trends in Biotech-Derived Health Products: Carol Nonkwelo and Romilla Maharaj

This document discusses trends in biotechnology-derived health products. It covers areas like therapeutic agents, vaccines, diagnostics, and gene therapy. Top biotechnologies for developing countries include affordable diagnostics, vaccines, drug delivery, sanitation technologies, and genetically modified crops. The EU prioritizes new diagnostics, prevention, therapeutics, medical genomics, communicable diseases, and more. Biological defense technologies focus on detection, therapeutics, vaccines, and remediation. Therapeutics research includes pharmacogenomics, bioinformatics, proteomics, SNPs, tissue engineering, and novel antibiotics. Diagnostics research analyzes assays and biomarkers. Vaccine challenges include safety, efficacy, and delivery methods. Gene therapy shows

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49 views41 pages

Trends in Biotech-Derived Health Products: Carol Nonkwelo and Romilla Maharaj

This document discusses trends in biotechnology-derived health products. It covers areas like therapeutic agents, vaccines, diagnostics, and gene therapy. Top biotechnologies for developing countries include affordable diagnostics, vaccines, drug delivery, sanitation technologies, and genetically modified crops. The EU prioritizes new diagnostics, prevention, therapeutics, medical genomics, communicable diseases, and more. Biological defense technologies focus on detection, therapeutics, vaccines, and remediation. Therapeutics research includes pharmacogenomics, bioinformatics, proteomics, SNPs, tissue engineering, and novel antibiotics. Diagnostics research analyzes assays and biomarkers. Vaccine challenges include safety, efficacy, and delivery methods. Gene therapy shows

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param540
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© © All Rights Reserved
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TRENDS IN BIOTECH-DERIVED

HEALTH PRODUCTS
Carol Nonkwelo and Romilla Maharaj

BIOTECHNOLOGY IN HEALTH
Uses human bodys own tools i.e.
proteins, enzymes, antibodies and other
naturally produced substances.
Also uses other living organisms e.g. plant
and animal cells, viruses and yeasts for
large scale production.
Four primary areas in health care
namely: therapeutic agents, vaccines,
diagnostics and gene therapy

HEALTH: State of Well-being


Prevention

Amino Acids
Vitamins
Vaccines

Health
Treatment

Diagnosis

Drugs

Molecular Diagnosis

Antibiotics

Monoclonal Antibodies

Therapeutic Proteins

Forensic Medicine

Gene Therapy
Tissue / Bone Engineering

Top Ten Biotechnologies for Improving


Health in Developing Countries
1. Modified molecular technologies for affordable,
simple diagnosis of infections diseases.
2. Recombinant technologies to develop vaccines
against infectious diseases.
3. Technologies for more efficient drug and vaccine
delivery systems.
4. Technologies for environmental improvement
(sanitation, clean water, bioremediation).
5. Sequencing pathogen genomes to understand
their biology and to identify new antimicrobials.

6. Protection against sexually transmitted diseases,


both with and without contraceptive effect.
7. Bioinformatics to identify drug targets and to
examine pathogen-host interactions.
8. Genetically modified crops with increased nutrients
to counter specific deficiencies.
9. Recombinant technology to make therapeutic
products (for example, insulin, interferon) more
affordable.
10.Combinatorial chemistry for drug discovery.
Daar et.al. 2002

European Union Priorities


Promoting technological platforms for new
diagnostics, prevention & therapeutics
Applying medical genomics to:
- diabetes, cardiovascular & rare diseases
- combating resistance to drugs
- neurological diseases
- human development & ageing
Combating cancer
Major communicable diseases linked to
poverty

BIOLOGICAL DEFENSE
TECHNOLOGIES

Detection & Diagnosis

Rapid detection (30 min) of infectious


diseases.

Portable biosensors to detect pathogens in the


air.

Remediation technologies e.g. enzymes to


decontaminate infectious areas.

Molecular barriers for infectious agents.

Therapeutics

Antibodies to bind to anthrax toxin

DNA or RNA based therapeutics against


infectious diseases

Drug delivery technologies e.g. Lozenge


containing interferon and drug and vaccine
patches

Artificial skin products

Vaccines

Developing a third generation anthrax


vaccine

Single oral dose, rapid protection and long


lasting

THERAPEUTICS

1.

Scope of Pharmacogenomics

Human genome has 30-40 000 genes


Produces 60-100 000 proteins
Potential 3-10 000 targets for therapeutic agents
Potential for personalised medicine

More drugs for smaller patient populations


Smaller cohorts for clinical trials
Fewer side effects and better compliance

Essential element:
Routine gene testing to identify responders
& those susceptible to adverse reactions

2.

Bioinformatics

Biology

Medicine

Bioinformatics
Maths &
Physics

Computer
Science

Application of tools of computation and analysis


to capture and interpret biological data

Contributing to functional understanding of the


human genome

Potential for enhanced discovery of drug targets


and individualised therapy

E.g. Imatinib mesylate (Gleevec) which


interferes with the abnormal protein made in
chronic myeloid leukemia.

PROTEOMICS
Analysis of the total amount of proteins
expressed by a cell
Investors no longer as eager to invest in
proteomics.
Want tangible therapeutic and
diagnostics products

Genomics/Proteomics Diagnostics
Prediction

growth
in
development
of
genomics/proteomics diagnosis will surge ahead of
new therapeutics.
Will take 3-4 years for these diagnostics to be
commonplace compared to 6-8 years for the
therapeutics to be developed.
Focus is on mainly on infectious diseases, e.g. TB
diagnostics.

3.

Single Nucleotide Polymorphisms

SNPs are single-base differences in DNA between


individuals in a population
Most frequent type of genetic variation
Attractive biomarkers for drug discovery &
development
Challenge is to identify SNPs that influence or
change pharmacokinetics, pharmacodynamics
and/or clinical end points

4.

Tissue Engineering

Most promising for skeletal tissues that have the


capacity to regenerate.
Products produced in the laboratory from human
& animal tissue are being developed to bring about
tendon and cartilage repair.
Bone morphogenic proteins have been successfully
used in periodontal bone regeneration.

5.

Vital Organs

Difficult to achieve and costly


LIFE initiative established with 60 international
participants pooling their expertise and
capabilities

6.

Novel Antibacterial Agents

Current trend:
A target based approach based on bacterial
genomes e.g. a peptide that inhibits the enzyme
deformylase
New drugs based on host/pathogen interactions
e.g. the drug Xigris for the treatment of sepsis

7.

Monoclonal Antibodies

The prediction is that more than half of new active


substances developed in the next 10-15 years will
be the result of antibody research.
Reason shorter development time for MAbs.
MAbs have been developed for a range of diseases
including cancers, organ rejection & rheumatoid
arthritis.

8.

HIV/AIDS

New approaches to complement highly active


retroviral treatment (HAART)
Entry inhibitors e.g. T-20 which aligns itself with
part of viral gp41 and prevents the virus from
fusing with the host cell.
Immune based therapies designed to optimise the
host immune response to keep the virus at bay.

DIAGNOSTICS

Biological Assays
Biological assays are needed to support biotechnology
derived products.
Biological assay measures biological activity based on a
specific functional, biological response of a test system.
Examples
In vitro tissue based.
In vitro cell line based.
Reporter gene based.
Biosensors that detect binding of cells to
immobilised ligands.
Kinase receptor assay that allows for more rapid
assessment of immunogens.

VACCINES

Challenges for Vaccine Development


Challenges for vaccine development is the same for
many infectious diseases
Vaccines need to be:
Safe
Effective
Broad-spectrum
Simple, transportable vectors
Easily administered
Provide long-term immune memory
Provide protection at the site of infection

AIDS Vaccines
Several vaccines have been developed
70 phase I trials
5 phase II trials
2 phase III trials

Current AIDS Vaccine Research


Enhancing the immunogenicity of specific HIV
peptides.
Enhancing antibody responses to regions of HIV
proteins protected from immune detection.
Developing new vectors and more efficient methods
for producing vectors.
Developing more efficient methods to determine Tcell receptor specificities & cytokine profiles.

Identification of host genes that confer resistance to


infection to determine which vaccines are best suited
to which populations.
Understanding the response of the human immune
system to candidate vaccines.

Malaria Vaccine Research


Receives roughly 10-fold less funding than HIV
vaccine development.
Cost of developing a malaria vaccine is estimated at
$500 m.
Several international initiatives to support the
development of a malaria vaccine e.g. European
Malaria Initiative, Gates Foundation, US NIH &
Department of Defense.

Several malaria vaccines are in or close to clinical


trials. The main obstacle is the capacity for
producing clinical grade material for protein-based
vaccines & for performing field trials for all types of
malaria vaccines.
At lease 5 000 candidate proteins have been
identified.
There is a lack of validated models that reliably
predict the degree of protection in humans.

GENE THERAPY

The retina is potentially the best candidate for gene


therapy.
Gene therapy has been successfully used to reverse
blindness in 3 dogs with a rare inherited disease.
The amount of vector virus injected into the eye is
0.001 of that used for systemic diseases.
Blood-ocular barrier separates the eye from the rest
of the body and the virus is less likely to have a
systemic effect.

DELIVERY SYSTEMS

Protein Transduction Technology


Developing field with potential applications in:
Gene therapy where it is preferable to deliver the
gene product.
Vaccine delivery e.g. the VP22 technology being
used to develop hepatitis & HPV derivatives.
Drug delivery e.g. synthetic peptide derivatives or
peptoids can be covalently linked to small drugs
for delivery & uptake at different sites in the body.
Peptoids are more cost effective, easier to
synthesise, more resistant to proteases than

Reproductive Medicine
Diagnostics

Highly Controversial

Pre-implantation genetic diagnosis (PGD)


used to screen early embryos for heritable genetic
defects or age-related genetic abnormalities

Therapeutics
Stem cell engineering for therapeutic and regenerative
medicine
Embryonic stem cells (5-6 days old) may be used
to reconstitute defective organ systems

Reproduction
Reproductive cloning
Twin cloning via embryo splitting

Academia & Industry

Much of therapeutic innovation in recent


decades resulted from biotechnology and
pharmaceutical industry sponsored
efforts.

Increasing pressures, competition, less


funding in public sectors has led to new
challenges in industry-sponsored clinical
research.

Predicted Genomic Impact by Therapeutic Area


Therapy Area

Share of Genomics (%)


2000
2005

Central nervous system


Cardiovascular diseases
Autoimmune diseases
Infectious diseases
Metabolic diseases
Cancer

20
25
15
20
25
25

40
30
30
55
40
50

Investors no longer as eager to invest in proteonomics


Viewed as low profit commodity
Eager to see tangible therapeutic & diagnostic products

6.

Artificial Cells

Artificial cells will make it possible to combine


nanoscale efficiency, self-organisation &
adaptability for therapeutic & diagnostic
applications.
Many individual components have been developed
The main challenge is to encapsulate components
in a single compartment & to have sequential and
controlled activities.

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