Unit:
Κατεύθυνση Κλινικές Μελέτες: Σχεδιασμός και ΕκτέλεσηLibrary of the School of Health Sciences
Supervisors info:
Ευστάθιος Καστρίτης, Καθηγητής, Ιατρική Σχολή, ΕΚΠΑ, Επιβλέπων
Ιωάννης Ντάνασης - Σταθόπουλος, Επιστημονικός συνεργάτης, Θεραπευτική Κλινική & Ομώνυμο Εργαστήριο, ΓΝΑ «Αλεξάνδρα»
Μαρία Γαβριατοπούλου, Αναπληρώτρια Καθηγήτρια, Ιατρική Σχολή, ΕΚΠΑ
Original Title:
Κλινική ανάπτυξη μονοκλωνικών αντισωμάτων έναντι των ινιδίων του αμυλοειδούς στην συστηματική Αμυλοείδωση
Translated title:
Clinical development of anti-amyloid monoclonal antibodies in systematic Amyloidosis
Summary:
The term of systematic Amyloidosis stands for the disease that arises from the misfolding of specific proteins that aggregate and deposit on target organs, affecting their normal function. Light-chain Amyloidosis arises after the accumulation of immunoglobulin’s light chains, while transthyretin related Amyloidosis arises after the accumulation of transthyretin on the target organs. Until recently, the treatment of these two types of amyloidoses aimed to eliminate the amyloidogenic protein thus limiting cell damage and preventing further amyloid accumulation. Nevertheless, the need for amyloid removal and its cell toxic action from organs is vital to improve patient’s quality of life and survival. Pre-clinical data suggest that this could be done effectively by using monoclonal antibodies that target the amyloid fibrils. Several molecules have been tested through clinical trials not only for AL Amyloidosis but also for ATTR Amyloidosis. The aim of this thesis is to describe these clinical trials and critically discuss their design. Data from the field of AL Amyloidosis are encouraging since there are already 3 phase III clinical trials ongoing. As far as ATTR Amyloidosis is concerned, the progression is not the same, with 2 phase I clinical trials publishing encouraging data recently. Lastly, the design of these trials is crucial in order to demonstrate efficacy results. Limitations in the design of the studies, mostly on the primary outcome measures and secondarily on the target population, have contributed to the failure of some of these trials.
Main subject category:
Health Sciences
Keywords:
Clinical trials, Amyloidosis, Monoclonal antibodies
Number of references:
126
