RECOMBINANT PROTEIN THERAPEUTICS
Academic year and teacher
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- Versione italiana
- Academic year
- 2022/2023
- Teacher
- GIORDANA FERIOTTO
- Credits
- 2
- Didactic period
- Secondo Semestre
- SSD
- BIO/10
Training objectives
- Develop expertise on:
a) the specific features of protein therapeutics produced by biotechnological means
b) the important role that these biopharmaceuticals have taken in every area of medicine and the enormous potential for development in the coming years, thanks to the new protein engineering strategies
c) the most important and current problems encountered in the clinical use of these biologics; d) knowledge of the main commercially important class of protein therapeutics. Prerequisites
- Having attended and achieved the training objectives of the first year and the first semester of the second year of the Degree in Medicine and Surgery.
Course programme
- Protein therapeutics: what they are and how they differ from drugs of chemical synthesis. Hints at methods of production with the recombinant DNA technology. To date, several hundred therapeutic proteins have been approved by the FDA and many more are currently being studied in clinical trials.
The most important therapeutic areas of application are: oncology, autoimmune and inflammatory diseases, hormonal deficiencies, haemostasis, protein/enzyme hereditary deficiency.
The Recombinant Protein Therapeutics own strengths (1) and limits (2):
1) efficiency of production processes, low cost and no quantitative limit (vs purification from biological material); the molecules, being species specific, are more active and less immunogenic; therapies are safer because you avoid the risk of transmission of pathogens; recombinant technology can change the molecules, to improve the therapeutic efficacy, or to create new molecules with the biological activities of interest.
2) The treatment with protein therapeutics is more expensive than that with small molecules; protein therapeutics cannot be administered orally; sometimes they have limits of absorption, distribution and stability (glycosylation, PEGylation and conjugation with an antibody subunit (Fc) are some strategies used to overcome these problems); the therapeutic use of certain proteins can trigger immune reactions against the drug (it’s the case of the murine or chimeric antibodies that have been replaced by human antibodies, much less immunogenic). The best example of evolution in the application of protein therapeutics: recombinant human insulin, a history that began in 1982 and is not yet completed. Protein therapeutics are enzymes, growth factors, hormones, interferons, interleukins, thrombolytics, anticoagulants, blood factors, antibodies .
The classification is based on their pharmacological activity.
GROUP I. Proteins with enzyme activity or regulatory function (to compensate for the lack of a protein, to strengthen a physiological process, to provide a new biological activity).
GROUP II. Proteins that act on a specific molecular target (interfere with a molecule or with an organism; convey other compounds or proteins on specific therapeutic targets).
GROUP III. Recombinant protein vaccines for the prophylaxis (protects from infections) or for therapy (autoimmune disease, cancer). Emerging technologies are greatly increasing the number and the optimization of protein therapeutics, while new strategies to provide engineering molecules are increasingly aimed at the individual patient. Pharmaceutical companies are currently experimenting with new routes of administration for protein therapeutics to make easier and more acceptable therapeutic treatment. Biosimilar therapeutics. Didactic methods
- Recorded Lessons delivered in the classroom.
Learning assessment procedures
- Written report (to be loaded into a course drive) on two general topics chosen (from the course contents) by the teacher and assigned to each student. If the sufficiency is reached, the score will derive from the assessment of the achievement of the objectives in terms of knowledge (2 points / topic) and ability to understand (2 points / topic) and clarity of description (2 points / topic) of the topics covered.
Reference texts
- Will be provided by the teacher during the course:
- the PDF files used for the lessons and the movies of the lessons; - some reviews of the most significant scientific publications on the subject.
