Drug Discovery and Evaluation for Treatment of Autoimmune Diseases

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Medicinal Chemistry".

Deadline for manuscript submissions: closed (15 February 2023) | Viewed by 3852

Special Issue Editor


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Guest Editor
Department of Molecular Biology, National Institute of Geriatrics, Rheumatology and Rehabilitation, Spartańska 1, 02-637 Warsaw, Poland
Interests: genetics; epigenetics; molecular biology; personalized medicine; T cells; autoimmunity; inflammation
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Special Issue Information

Dear Colleagues,

Over the past decade, for many autoimmune diseases there have been numerous successes in developing targeted therapies that have led to an improved range of choices for clinical treatment. The success of biological therapies for autoimmune diseases, coupled with rapid advances in basic research, has validated the targeting of many immunology-relevant signaling pathways and uncovered new intracellular molecules as potential new drug targets. However, there are still unmet clinical needs in these diseases. In autoimmune diseases, loss of B-cell or T-cell tolerance is frequently observed, and indeed, the human leukocyte antigen (HLA) locus is commonly associated with an increased risk of disease susceptibility. While a deep understanding of signaling pathways in autoimmune diseases is challenging, basic research is very informative for the development of new therapies. Thus, this Special Issue aims to address the pressing need for the development of new drugs or the repositioning of drugs based on a molecular and clinical understanding of the signaling pathways of autoimmune diseases in combination with high-throughput analysis of integrated datasets.

Dr. Agnieszka Paradowska‑Gorycka
Guest Editor

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Keywords

  • genetic
  • epigenetics
  • multi-omics technologies
  • immunology
  • clinical studies
  • cellular therapy
  • biological therapy
  • immune therapy

Published Papers (1 paper)

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Research

15 pages, 1096 KiB  
Article
Real-World Evidence for Favourable Quality-of-Life Outcomes in Hungarian Patients with Relapsing-Remitting Multiple Sclerosis Treated for Two Years with Oral Teriflunomide: Results of the Teri-REAL Study
by Krisztina Bencsik, Enikő Dobos, Zita Jobbágy, Adrienne Jóri Birkás, Krisztina Kovács, Mária Sátori, Gyula Lencsés, Gabor Bartok, Erika Losonczi, László Vécsei and on behalf of the Teri-REAL Investigators
Pharmaceuticals 2022, 15(5), 598; https://doi.org/10.3390/ph15050598 - 13 May 2022
Cited by 5 | Viewed by 2998
Abstract
Relapsing-remitting multiple sclerosis (RRMS) is a degenerative, inflammatory disease of the central nervous system in which symptoms and disability progression vary significantly among patients. Teri-REAL was a prospective, real-world observational study that examined quality-of-life (QoL) and treatment outcomes in a Hungarian cohort of [...] Read more.
Relapsing-remitting multiple sclerosis (RRMS) is a degenerative, inflammatory disease of the central nervous system in which symptoms and disability progression vary significantly among patients. Teri-REAL was a prospective, real-world observational study that examined quality-of-life (QoL) and treatment outcomes in a Hungarian cohort of RRMS patients treated with once-daily oral teriflunomide. QoL was assessed at baseline, 12, and 24 months with the Multiple Sclerosis Quality of Life-54 (MSQoL-54) questionnaire. Other measurements included disease progression (Patient Determined Disease Steps [PDDS]), clinical efficacy (relapses), fatigue (Fatigue Impact Scale [FIS]), depression (Beck Depression Inventory [BDI]), cognition (Brief International Cognitive Assessment in MS [BICAMS]), persistence and safety. 212 patients were enrolled (69.1% female, 50.5% treatment naïve), with 146 (69%) completing the study. Statistically significant improvements in subscales of the MSQoL-54 versus baseline were found at Month 12 and Month 24. Significant improvements were also observed for individual components of the BICAMS score at 24 months, while PDDS, FIS and BDI scores remained stable. The mean annualised relapse rate was 0.08 ± 0.32. There were 93 safety events, most of which were mild to moderate. Improved QoL and cognitive outcomes in teriflunomide-treated patients over 2 years offer a unique perspective to this real-world study. Full article
(This article belongs to the Special Issue Drug Discovery and Evaluation for Treatment of Autoimmune Diseases)
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