TARGETED THERAPY

Presented by: Dr.Sushma sathya

Moderated by: Dr.Vandana
Introduction
• A targeted therapy is defined as process of attacking a
biologically important process central to a hallmark of cancer or
disease.

(i) Some block the action of certain enzymes, proteins or other

molecules involved in growth of cancer cells
(ii) some types help the immune system to kill cancer cells; and
(iii) most targeted drugs are small-molecule drugs or monoclonal
antibodies.

• Blocks the growth of cancer cells by interfering with specific

targeted molecules.
• Targeted therapies act on specific molecular targets, were
designed to interact with their target, and are often cytostatic
(block proliferation), while standard conventional chemotherapy
is designed to kill rapidly dividing tumor cells, may also affect
dividing normal cells, and is cytotoxic (kill tumor cells).

• Unlike traditional chemotherapy, which kills both cancer cells

and healthy cells, targeted therapy drugs are designed to attack
specific molecules that are involved in cancer cell growth and
survival

• Interfere with specific targeted molecules needed for

carcinogenesis and tumour growth rather than interfering with
all rapidly dividing cells
 Targeted therapy in cancer cells is based on the inhibition of crucial
enzymes/pathways in a cancer cell, for which two definitions can be
formulated:

• Firstly, high activity of a pathway indicates that it may be essential

“to drive the tumor” and its inhibition stops growth of (or
sometimes kills) the cancer cell.

• Secondly, a low activity indicates that the activity may be rate-

limiting and essential [according to Weber a ‘key’ enzyme] for
growth, and therefore its inhibition may kill the cancer cell . In this
case, the enzyme may be considered as the “engine”.
A Paradigm
Shift
Types of targeted therapies

1. Hormonal
2. Signal-transduction inhibitors (e.g. TKIs)
3. Gene-expression modulators
4. Apoptosis inducers
5. Angiogenesis inhibitors
6. Immunotherapies
7. Toxin-delivery molecules (targeted chemotherapy)
8. Gene therapy
Growth factor and
growth factor
receptor Inhibitors
Signal-transduction inhibitors (e.g. TKIs)
 The following four TKI drugs are approved as first-line treatment for
chronic phase CML:
• Imatinib mesylate (Gleevec®)
• Dasatinib (Sprycel®)
• Nilotinib (Tasigna®)
• Bosutinib (Bosulif®)

The following TKIs are approved

for the treatment of patients who
have received 2 or more TKIs or
who have the T315I mutation:
• Asciminib (Scemblix®)
• Ponatinib (Iclusig®)
Gene-expression modulators
Cell cycle regulators
The role of RB in
regulating the G1-S
checkpoint of the cell
cycle.
The role of p53
in maintaining
the integrity of
the genome.
Apoptosis
Inducers
Hormonal therapy

The structures of HER2

ECD and its complex with
Pertuzumab and
Trastuzumab fragment
Gene therapy
Ploting of genetic
alterations in a lung
cancer paient
AND MORE…..
Targeted
therapy in
Diabetes
Mellitus
Targeted therapy
in Dermatology
DRUGS IN TARGETED THERAPY
Challenges of targeted therapy

Targeted therapy also has some challenges, including:

• Resistance: Cancer cells can develop resistance to targeted therapy

drugs over time.

• This means that the cancer cells can become resistant to the drug
and continue to grow and spread.

• Cost: Targeted therapy drugs can be very expensive.

• Limited availability: Some targeted therapy drugs are only available

for certain types of cancer.
Future of targeted therapy
• Researchers are constantly developing new targeted therapy drugs
and new ways to use targeted therapy drugs to treat cancer.

• Targeted therapy is a rapidly evolving field, and it is likely to play an

increasingly important role in cancer treatment in the future.
Conclusion
• Targeted therapy is a promising new approach to cancer treatment.

• It offers several benefits over traditional chemotherapy, including

more targeted killing of cancer cells, fewer side effects, and
improved survival rates.

• However, targeted therapy also has some challenges, such as

resistance, cost, and limited availability.

• Researchers are constantly developing new targeted therapy drugs

and new ways to use targeted therapy drugs to treat cancer.

• Targeted therapy is a rapidly evolving field, and it is likely to play an

increasingly important role in cancer treatment in the future.
References
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Elsevier.

• Sharma, H.L. and Sharma, K.K .,Sharma \& Sharma's Principles of Pharmacology,.{1st edition},{2017}, Paras Medical Publisher

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10.1038/s41392-021-00572-w. PMID: 34054126; PMCID: PMC8165101.

• Yamaoka T, Ohba M, Ohmori T. Molecular-Targeted Therapies for Epidermal Growth Factor Receptor and Its Resistance

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• https://www.lls.org/leukemia/chronic-myeloid-leukemia/treatment/tyrosine-kinase-inhibitor-tki-therapy#:~:text=Tyrosine%
20kinase%20inhibitors%20(TKIs)%20are,less%20damage%20to%20normal%20cells

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