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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 4520

Special Issue Editor

Prof. Dr. Anna Rita Franco Migliaccio

E-Mail Website
Guest Editor
1. Institute of Nanotechnology, National Research Council (Cnr-NANOTEC), c/o Campus Ecotekne, Lecce, Italy
2. Altius Institute for Biomedical Sciences, Seattle, WA, USA
Interests: hematopoietic stem cells; erythropoiesis; thrombopoiesis; cell therapy; hemoglobinopathies; myeloproliferative disorders
Special Issues, Collections and Topics in MDPI journals

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

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Published Papers (4 papers)

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Review

16 pages, 925 KiB  
Review
Development of VLA4 and CXCR4 Antagonists for the Mobilization of Hematopoietic Stem and Progenitor Cells
by Peter G. Ruminski, Michael P. Rettig and John F. DiPersio
Biomolecules 2024, 14(8), 1003; https://doi.org/10.3390/biom14081003 - 14 Aug 2024
Viewed by 202
Abstract
The treatment of patients diagnosed with hematologic malignancies typically includes hematopoietic stem cell transplantation (HSCT) as part of a therapeutic standard of care. The primary graft source of hematopoietic stem and progenitor cells (HSPCs) for HSCT is mobilized from the bone marrow into [...] Read more.
The treatment of patients diagnosed with hematologic malignancies typically includes hematopoietic stem cell transplantation (HSCT) as part of a therapeutic standard of care. The primary graft source of hematopoietic stem and progenitor cells (HSPCs) for HSCT is mobilized from the bone marrow into the peripheral blood of allogeneic donors or patients. More recently, these mobilized HSPCs have also been the source for gene editing strategies to treat diseases such as sickle-cell anemia. For a HSCT to be successful, it requires the infusion of a sufficient number of HSPCs that are capable of adequate homing to the bone marrow niche and the subsequent regeneration of stable trilineage hematopoiesis in a timely manner. Granulocyte-colony-stimulating factor (G-CSF) is currently the most frequently used agent for HSPC mobilization. However, it requires five or more daily infusions to produce an adequate number of HSPCs and the use of G-CSF alone often results in suboptimal stem cell yields in a significant number of patients. Furthermore, there are several undesirable side effects associated with G-CSF, and it is contraindicated for use in sickle-cell anemia patients, where it has been linked to serious vaso-occlusive and thrombotic events. The chemokine receptor CXCR4 and the cell surface integrin α4β1 (very late antigen 4 (VLA4)) are both involved in the homing and retention of HSPCs within the bone marrow microenvironment. Preclinical and/or clinical studies have shown that targeted disruption of the interaction of the CXCR4 or VLA4 receptors with their endogenous ligands within the bone marrow niche results in the rapid and reversible mobilization of HSPCs into the peripheral circulation and is synergistic when combined with G-CSF. In this review, we discuss the roles CXCR4 and VLA4 play in bone marrow homing and retention and will summarize more recent development of small-molecule CXCR4 and VLA4 inhibitors that, when combined, can synergistically improve the magnitude, quality and convenience of HSPC mobilization for stem cell transplantation and ex vivo gene therapy after the administration of just a single dose. This optimized regimen has the potential to afford a superior alternative to G-CSF for HSPC mobilization. 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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16 pages, 2003 KiB  
Review
The History of Nerve Growth Factor: From Molecule to Drug
by Elizabeth Gavioli, Flavio Mantelli, Maria Candida Cesta, Marta Sacchetti and Marcello Allegretti
Biomolecules 2024, 14(6), 635; https://doi.org/10.3390/biom14060635 - 29 May 2024
Viewed by 864
Abstract
Nerve growth factor (NGF), the first neurotrophin to be discovered, has a long and eventful research journey with a series of turning points, setbacks, and achievements. Since the groundbreaking investigations led by Nobel Prize winner Rita Levi-Montalcini, advancements in the comprehension of NGF’s [...] Read more.
Nerve growth factor (NGF), the first neurotrophin to be discovered, has a long and eventful research journey with a series of turning points, setbacks, and achievements. Since the groundbreaking investigations led by Nobel Prize winner Rita Levi-Montalcini, advancements in the comprehension of NGF’s functions have revolutionized the field of neuroscience, offering new insights and opportunities for therapeutic innovation. However, the clinical application of NGF has historically been hindered by challenges in determining appropriate dosing, administration strategies, and complications related to the production process. Recent advances in the production and scientific knowledge of recombinant NGF have enabled its clinical development, and in 2018, the United States Food and Drug Administration approved cenegermin-bkbj, a recombinant human NGF, for the treatment of all stages of neurotrophic keratitis. This review traces the evolutionary path that transformed NGF from a biological molecule into a novel therapy with potential research applications beyond the eye. Special emphasis is put on the studies that advanced NGF from discovery to the first medicinal product approved to treat a human disease. 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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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
Cited by 1 | Viewed by 1497
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
Cited by 2 | Viewed by 1341
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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