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Biomolecules
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From the Past to the Present: Unveiling the Role of...
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From the Past to the Present: Unveiling the Role of Biomolecules in Clinical History

A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Medicine".

Deadline for manuscript submissions: 15 September 2024 | Viewed by 1441

Special Issue Editor

Prof. Dr. Anna Rita Franco Migliaccio

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Guest Editor
1. Unit of Microscopic and Ultrastructural Anatomy, University Campus Bio-Medico, Rome, Italy
2. Altius Institute for Biomedical Sciences, Seattle, WA, USA
Interests: hematopoietic stem cells; erythropoiesis; thrombopoiesis; cell therapy; hemoglobinopathies; myeloproliferative disorders

Special Issue Information

Dear Colleagues,

Biomolecules generated on the basis of human genetic information are routinely used in the clinic to treat several human disorders and, in some cases, have replaced the cumbersome therapies previously available. Their use is so widely spread that many of us cannot even imagine a time when these biomolecules did not exist. Even more of us do not recall the brilliant and perseverant work of the researchers in their own fields that allowed their identification and characterization, the isolation of their genes, the expression of these genes into suitable organisms for mass protein production and then the validation of their efficacy in animal models and finally the clinical trials that proved their efficacy. The articles included in this special series are meant to provide a historical perspective of the numerous gaps of knowledge that had to be overcome to arrive where we are today. We believe that a look back at what it took for the field to more forward will not only allow us to appreciate the effort and the advancements in knowledge that allowed us to realize these achievements, but will also effectively tame the false expectations that the general population currently has on what medicine may deliver.

If you are willing to write about the history of a specific biomolecule currently used in the clinic, please do not hesitate to contact us. To avoid duplication with papers already in the pipeline, please send a provisional title and an abstract to use by Sept. 30. We will be happy to let you know as soon as possible if the area you want to cover is within our interest.

Prof. Dr. Anna Rita Franco Migliaccio
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Biomolecules is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • biomolecules
  • clinical application
  • history
  • anemia
  • neurodegeneration
  • cancer

Published Papers (2 papers)

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Review

14 pages, 2086 KiB  
Review
Thrombopoietin, the Primary Regulator of Platelet Production: From Mythos to Logos, a Thirty-Year Journey
by Kenneth Kaushansky
Biomolecules 2024, 14(4), 489; https://doi.org/10.3390/biom14040489 - 18 Apr 2024
Viewed by 353
Abstract
Thrombopoietin, the primary regulator of blood platelet production, was postulated to exist in 1958, but was only proven to exist when the cDNA for the hormone was cloned in 1994. Since its initial cloning and characterization, the hormone has revealed many surprises. For [...] Read more.
Thrombopoietin, the primary regulator of blood platelet production, was postulated to exist in 1958, but was only proven to exist when the cDNA for the hormone was cloned in 1994. Since its initial cloning and characterization, the hormone has revealed many surprises. For example, instead of acting as the postulated differentiation factor for platelet precursors, megakaryocytes, it is the most potent stimulator of megakaryocyte progenitor expansion known. Moreover, it also stimulates the survival, and in combination with stem cell factor leads to the expansion of hematopoietic stem cells. All of these growth-promoting activities have resulted in its clinical use in patients with thrombocytopenia and aplastic anemia, although the clinical development of the native molecule illustrated that “it’s not wise to mess with mother nature”, as a highly engineered version of the native hormone led to autoantibody formation and severe thrombocytopenia. Finally, another unexpected finding was the role of the thrombopoietin receptor in stem cell biology, including the development of myeloproliferative neoplasms, an important disorder of hematopoietic stem cells. Overall, the past 30 years of clinical and basic research has yielded many important insights, which are reviewed in this paper. Full article
(This article belongs to the Special Issue From the Past to the Present: Unveiling the Role of Biomolecules in Clinical History)
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29 pages, 2164 KiB  
Review
Erythropoietin: A Personal Alice in Wonderland Trip in the Shadow of the Giants
by Anna Rita Migliaccio
Biomolecules 2024, 14(4), 408; https://doi.org/10.3390/biom14040408 - 27 Mar 2024
Viewed by 669
Abstract
The identification of the hormone erythropoietin (EPO), which regulates red blood cell production, and its development into a pharmaceutical-grade product to treat anemia has been not only a herculean task but it has also been the first of its kind. As with all [...] Read more.
The identification of the hormone erythropoietin (EPO), which regulates red blood cell production, and its development into a pharmaceutical-grade product to treat anemia has been not only a herculean task but it has also been the first of its kind. As with all the successes, it had “winners” and “losers”, but its history is mostly told by the winners who, over the years, have published excellent scientific and divulgate summaries on the subject, some of which are cited in this review. In addition, “success” is also due to the superb and dedicated work of numerous “crew” members, who often are under-represented and under-recognized when the story is told and often have several “dark sides” that are not told in the polished context of most reviews, but which raised the need for the development of the current legislation on biotherapeutics. Although I was marginally involved in the clinical development of erythropoietin, I have known on a personal basis most, if not all, the protagonists of the saga and had multiple opportunities to talk with them on the drive that supported their activities. Here, I will summarize the major steps in the development of erythropoietin as the first bioproduct to enter the clinic. Some of the “dark sides” will also be mentioned to emphasize what a beautiful achievement of humankind this process has been and how the various unforeseen challenges that emerged were progressively addressed in the interest of science and of the patient’s wellbeing. Full article
(This article belongs to the Special Issue From the Past to the Present: Unveiling the Role of Biomolecules in Clinical History)
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