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Lecture 13

The document provides an overview of therapeutic agents, including their classification, applications in chronic diseases, and the role of biologics and nanotechnology in modern medicine. It discusses the drug discovery process, advances in therapeutic agents, and challenges faced in the field. Additionally, it highlights the importance of biomarkers in disease diagnosis and monitoring, along with emerging trends and technologies in biomarker research.

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Maha Khan
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0% found this document useful (0 votes)
69 views26 pages

Lecture 13

The document provides an overview of therapeutic agents, including their classification, applications in chronic diseases, and the role of biologics and nanotechnology in modern medicine. It discusses the drug discovery process, advances in therapeutic agents, and challenges faced in the field. Additionally, it highlights the importance of biomarkers in disease diagnosis and monitoring, along with emerging trends and technologies in biomarker research.

Uploaded by

Maha Khan
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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Download as PDF, TXT or read online on Scribd
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Advanced Functional Materials

Dr. Abdur Rahim


Associate Professor

Date 18-12-2024
Therapeutic Agents?
Introduction
What are Therapeutic Agents?
• Compounds or substances used to treat, prevent, or cure diseases.
Importance in Modern Medicine
• Role in addressing critical health issues.
• Examples include small molecules, biologics, and nanomedicine.
Objective of the Presentation
• Overview of therapeutic agents.
• Applications in diseases and ongoing developments.

Biologics, also known as biological products, are complex pharmaceutical substances derived
from living organisms or their cells. They are used in the prevention, diagnosis, or treatment of
diseases. Examples: Vaccines, Monoclonal Antibodies, Recombinant Proteins, Gene Therapies,
Blood Products
Classification of Therapeutic Agents
Based on Origin
• Natural: Plant extracts, microbial metabolites.
• Synthetic: Chemically synthesized drugs.
• Semi-synthetic: Modified natural products.
Based on Mechanism
• Enzyme inhibitors (e.g., aspirin).
• Receptor modulators (e.g., beta-blockers).
Examples:
• Small molecule drugs (e.g., ibuprofen).
• Biologics (e.g., monoclonal antibodies).
• Nanoscale formulations (e.g., liposomes).
Therapeutic Agents for Chronic Diseases
Beta blockers, also spelled -blockers, are a class of medications that are predominantly used to
manage abnormal heart rhythms

Cancer enzymes responsible for the


activation of many proteins by signal
• Chemotherapeutic agents (e.g., doxorubicin). transduction
• Targeted therapy: Tyrosine kinase inhibitors (TKIs).
• Immunotherapy: CAR-T cells and monoclonal antibodies.
Beta blockers, also spelled -blockers, are a class of medications that are
Cardiovascular Diseases predominantly used
Beta blockers, to spelled
also manage-blockers,
abnormalareheart rhythms
a class
predominantly used to manage abnormal heart rhythms
of medications that are

• Anticoagulants, statins, beta-blockers. used to manage abnormal heart rhythms


to lower cholesterol

Diabetes low blood sugar


• Insulin therapies, oral hypoglycemic agents.
Biologics as Therapeutic Agents

What are Biologics?


• Proteins, antibodies, or nucleic acid-based therapies.
Examples
• Monoclonal antibodies (mAbs): Rituximab, Adalimumab.
• Recombinant proteins: Insulin, growth hormones.
• Gene therapy: Viral vector-based therapies.

Advantages and Challenges Beta blockers, also spelled -blockers, are a class of medications

• Specific targeting, low toxicity vs. high cost and stability issues.
that are predominantly used to manage abnormal heart rhythms
Role of Nanotechnology in Therapeutics

What is Nanomedicine?
• Use of nanoscale materials for drug delivery and diagnostics.

Examples
• Liposomal drugs: Doxil (liposomal doxorubicin).
• Nanoparticles for targeted therapy.
• Polymeric nanocarriers for controlled drug release.

Advantages
• Improved bioavailability and reduced side effects.
Drug Discovery and Development

•Steps in Drug Development Phase 1:

• Target identification. Participants: Small group (20–100 healthy volunteers or


patients).
• Preclinical studies. Purpose: Assess safety, dosage, and side effects.
Focus: How the drug interacts with the body
• Clinical trials: Phases I–IV. (pharmacokinetics and pharmacodynamics).

• FDA approval and commercialization. Phase 2:


Participants: Larger group (100–300 patients).
Purpose: Evaluate efficacy and further safety.
Focus: Does the treatment work in the targeted

•Challenges
condition?

Phase 3:
• High cost and time constraints. Participants: Large group (1,000–3,000 patients).
Purpose: Confirm efficacy, monitor side effects, and
• Safety and efficacy testing. compare with standard treatments.
Outcome: Data for regulatory approval (FDA, EMA, etc.).

Phase 4 (Post-Marketing Surveillance):


Participants: General population after drug approval.
Purpose: Long-term safety and effectiveness monitoring.
Advances in Therapeutic Agents

•Targeted Therapy
• Precision medicine using biomarkers.

•CRISPR and Gene Editing


• Correcting genetic disorders.

•mRNA Therapeutics
• Example: COVID-19 vaccines (Pfizer, Moderna).

•Artificial Intelligence in Drug Design


• Accelerating discovery of new drugs.
Future Trends
Personalized Medicine
• Patient-specific therapies.

Immunotherapy for Cancer and Autoimmune Disorders


•Smart Drug Delivery Systems
• Controlled and triggered release of drugs.
Theranostics
• Combining therapy and diagnostics in a single agent.

Theranostics is a combination of the words "therapy" and "diagnostics" and refers to an


integrated approach that combines diagnostic and therapeutic capabilities in a single platform or
agent. The goal of theranostics is to provide personalized medicine by simultaneously diagnosing
and treating diseases, often with a high degree of specificity and efficiency. Examples:
Nanoparticles, Radioisotopes, Biomarker-Driven Therapies.
Challenges and Limitations

•High cost of development.

•Drug resistance in diseases.

•Regulatory hurdles.

•Ethical concerns in gene editing and advanced therapies.


Conclusion

•Therapeutic agents are critical for disease management and treatment.

•Advances in biologics, nanotechnology, and AI are shaping the future of


therapeutics.

•Challenges remain, but innovation continues to drive the field forward.


Biomarkers

•What are Biomarkers?


• Biological molecules that indicate normal or abnormal processes, or
disease states.
• Biological indicators used for disease detection, monitoring, and
prognosis.

•Importance of Biomarkers
• Diagnosis, prognosis, and treatment monitoring.
predicting the likelihood or expected development of a disease

•Objectives of the Presentation


• Discuss the types of biomarkers.
• Highlight potential biomarkers for various diseases.
• Explore emerging trends and challenges in biomarker research.
Biomarker Classification
•Types of Biomarkers
• Diagnostic biomarkers: Identify presence of disease.
• Prognostic biomarkers: Predict disease outcome or progression.
• Predictive biomarkers: Assess treatment response.
• Monitoring biomarkers: Track therapeutic effects over time.

•Examples
• Proteins (e.g., PSA for prostate cancer).
• Genes (e.g., BRCA1/BRCA2 mutations).
• Metabolites (e.g., glucose for diabetes).
Biomarker Discovery Process

•Stages in Biomarker Discovery


• Sample Collection: Blood, urine, tissue, saliva, etc.
• Identification: High-throughput omics approaches
(proteomics, genomics, metabolomics).
• Validation: In-vitro, in-vivo, and clinical trials.
• Translation: Clinical application.

•Tools and Techniques


• Mass spectrometry, ELISA, PCR, NGS (Next-Generation
Sequencing).
Biomarkers for Cancer
•Cancer Biomarkers
• Proteins: CA-125 (Ovarian Cancer), HER2 (Breast Cancer).
• Genetic Biomarkers: p53 mutations, EGFR mutations in lung
cancer.
• Circulating Tumor DNA (ctDNA): Emerging tools for liquid
biopsy.

•Case Study:
• HER2 in breast cancer for targeted therapy (Trastuzumab).
Biomarkers for Cardiovascular Diseases
•Key Biomarkers
• Troponins (cTnI, cTnT): Gold standard for myocardial
infarction.
• C-reactive protein (CRP): Inflammation marker for heart
disease.
• B-type Natriuretic Peptide (BNP): Heart failure indicator.
•Clinical Application
• Early detection and prognosis.
• Monitoring therapy effectiveness.
Biomarkers for Neurological Disorders
Neurodegenerative Diseases
• Alzheimer’s Disease:
• Beta-amyloid (Aβ) plaques.
• Tau protein (CSF biomarker).
• Parkinson’s Disease:
• α-synuclein accumulation.
Multiple Sclerosis (MS)
• Neurofilament light chain (NfL) in cerebrospinal fluid.
• Glial fibrillary acidic protein (GFAP).
Emerging Biomarkers and AFM Applications

•Cancer Biomarkers
• Detection of circulating tumor DNA (ctDNA), exosomes, and
specific proteins.
• Role of AFMs: SERS, electrochemical sensors with MOFs.

•Neurological Biomarkers
• Alzheimer’s disease: Aβ42 peptide detection using QDs and
plasmonic materials.

•Inflammatory Biomarkers
• IL-6, TNF-α using advanced electrochemical platforms.
Biomarkers for Infectious Diseases
COVID-19
• IL-6: Inflammatory marker for disease severity.
• D-dimer: Coagulation marker for thrombotic risks.
HIV
• CD4 T-cell count: Disease progression and treatment
monitoring.
• Viral load: Therapy effectiveness.
Tuberculosis (TB)
• IFN-γ release assays (IGRA).
Emerging Biomarkers and Technologies

Omics Approaches
• Genomics, Transcriptomics, Proteomics, Metabolomics.

Circulating Biomarkers
• Circulating tumor cells (CTCs).
• Extracellular vesicles (exosomes).

Artificial Intelligence and Machine Learning


• AI-based biomarker prediction and analysis.
nhanced Sensitivity and Specificity
Miniaturization and Point-of-Care
Testing
Challenges in Biomarker Research Multi-Modal Detection
Drug Delivery and Theranostics
•Key Challenges Biomarker Isolation and Enrichment
Disease Prognosis and Monitoring
• Lack of reproducibility across studies.
• High cost and time for clinical validation.
• Ethical issues in genetic biomarker applications.

•Solutions
• Standardized methodologies.
• Integration of multi-omics data.
Wearable Sensors for Biomarker Detection

•Integration of AFMs in Wearable Devices


• Flexible, biocompatible materials: Graphene, nanocomposites,
and polymers.

•Examples
• Sweat sensors for electrolyte or cortisol detection.
• Skin patches for glucose monitoring.

•Advantages
• Real-time, non-invasive biomarker tracking.
Advantages of AFMs for Biomarker Detection

•High Sensitivity
• Low detection limits for early disease diagnosis.

•Selectivity
• Functionalization ensures targeted biomarker detection.

•Scalability
• Potential for miniaturized and portable devices.

•Versatility
• Compatible with electrochemical, optical, and biological sensing
platforms.
Future Trends
•Personalized Medicine
• Biomarkers for individualized treatment plans.

•Wearable Biosensors
• Real-time biomarker monitoring (e.g., glucose, cortisol).

•Non-Invasive Biomarkers
• Liquid biopsies for cancer.

•Integration of AI and Big Data


• Faster discovery and validation processes.
Conclusion
•Biomarkers are critical tools in disease diagnosis, prognosis, and treatment monitoring.

•Advances in omics technologies and AI are driving biomarker discovery.

•Overcoming current challenges will enhance clinical translation and patient outcomes.
•Advanced functional materials play a significant role in enhancing biomarker
detection for diagnostics.

•Nanomaterials, MOFs, and hybrid systems provide sensitivity, selectivity, and


versatility.

•Emerging technologies promise to transform disease diagnostics and personalized


medicine.

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