Bulletin De L'Academie Nationale De Medecine[JOURNAL]

The concept of biosimilar medicine was launched by 2001 and 2004 European Directives. First European marketing authorizations were delivered in 2006. They are "copies" of biologically manufactured medicines, mostly prote... The concept of biosimilar medicine was launched by 2001 and 2004 European Directives. First European marketing authorizations were delivered in 2006. They are "copies" of biologically manufactured medicines, mostly proteins. Taking into account the intrinsic variability related to the biological manufacture process, some variation of the chemical structure of the finished compound may be observed. They impact especially the glycosylation residues but not the amino-acid sequence (for proteins). For this reason, the marketing authorization application dossier has to involve, as opposed to the generic medicine procedure, the demonstration of the therapeutic equivalence in at least one clinical indication of the princeps medicine. Introduction of biosimilar medicines of monoclonal antibodies has represented a remarkable event in the domain of rheumatology, gastroenterology and dermatology with infliximab, etanercept and adalimumab biosimilars and in cancerology domains with rituximab, trastuzumab and bevacizumab biosimilars. Biosimilar medicines availability reduces the risk of drug supply rupture of princeps but their main impact is the economic one allowing cost reduction of costly princeps biological medicines. With the acquired clinical experience, the initial fears concerning switch form princeps to a biosimilar for a given patient has progressively disappeared.

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[Update on treatments and innovations in systemic auto-immune diseases].

Guillevin L

Bull Acad Natl Med · 2020 Oct · PMID 32836291 · Full text

Many new drugs, sometimes promising, are now available to treat systemic auto-immune diseases. Treatment strategies and objectives are different according to each auto-immune disease. Major advances have been obtained in... Many new drugs, sometimes promising, are now available to treat systemic auto-immune diseases. Treatment strategies and objectives are different according to each auto-immune disease. Major advances have been obtained in vasculitides treatments. In systemic lupus erythematosus, many drugs have been evaluated, targeting one of the multiple pathogenic mechanisms of the disease. Unfortunately, new drugs remain rare on the market and no major advances have been obtained. In systemic sclerosis, symptomatic treatments improved outcomes but effective drugs targeting all disease manifestations are missing. Autologous hematopoietic stem cell transplantation could be one of the future innovative treatment of systemic sclerosis despite the occurrence of adverse events.

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[Human cells for therapeutics purpose: State of the art].

Guilhot F

Bull Acad Natl Med · 2020 Oct · PMID 32836290 · Full text

Patient-derived induced pluripotent stem cells as well as human embryonic stem cells are pluripotent and their derivation has been used for the understanding of numerous diseases. Currently they are also used for the tre... Patient-derived induced pluripotent stem cells as well as human embryonic stem cells are pluripotent and their derivation has been used for the understanding of numerous diseases. Currently they are also used for the treatment of neurologic disorders such as Parkinson disease or cardiac disorders. Gene therapy has been successful for the treatment of hemophilia A and B, hemoglobinopathies and immunodeficiencies. Hemopoietic stem cell transplantation is a well-accepted therapeutic strategy for Leukemias, whereas CAR-T cells is a new promising approach even for lymphomas and myeloma.

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