Drug development: A complicated path

Humphries, Courtney

doi:10.1038/515s4a

Cited by 5 publications

(7 citation statements)

References 2 publications

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“…As a consequence, there is ischemia, pain, damage to various organs and tissues, necrosis and chronic hemolysis. In addition, the regional hypoxia process itself may intensify sickling of new erythrocytes, increasing ischemic damage (2,(7)(8) .…”

Section: Introductionmentioning

confidence: 99%

Importance of health guidance for family members of children with sickle cell disease

et al. 2018

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Objective: To know the main health guidance needs of family members of children with sickle cell disease. Method: Qualitative research, developed in a pediatric reference hospital of Ceará State, between April and May 2017, through the participation of 12 family members of children with sickle cell disease. The data were collected through semi-structured interviewees and analyzed according to the Bardin’s Thematic Categorical Analysis. Results: The relatives had divergent opinions about what this pathology would be and expressed the expectation of being broadly guided, from general information (signs and symptoms) to more complex ones about the disease, including major complications and ways of preventing them. Final considerations: The health fragility due to the punctual and/or meager guidance provided to family members reflects the importance of increasing knowledge and clarifying doubts of these relatives about the disease, which makes it urgent to develop health education strategies by multiprofessional teams.

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Section: Introductionmentioning

confidence: 99%

Importance of health guidance for family members of children with sickle cell disease

et al. 2018

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“…Patients with sickle cell anemia (SCA) display higher levels of HbF than healthy individuals, whose HbF concentration is less than 1% of total hemoglobin. HbF does not interact with HbS and limits polymerization of HbS within red blood cells (RBCs) [1], which provides a sound rationale for the development of HbF inducers as treatment options for SCA [2].…”

mentioning

confidence: 99%

A thalidomide–hydroxyurea hybrid increases HbF production in sickle cell mice and reduces the release of proinflammatory cytokines in cultured monocytes

Lanaro

Franco‐Penteado

Silva

et al. 2018

Experimental Hematology

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Fetal hemoglobin (HbF) induction by hydroxyurea (HU) therapy is associated with decreased morbidity and mortality in sickle cell anemia (SCA) patients, but not all patients respond to or tolerate HU. This provides a rationale for developing novel HbF inducers to treat SCA. Thalidomide analogs have the ability to induce HbF production while inhibiting the release of tumor necrosis factor-alpha. Molecular hybridization of HU and thalidomide was used to synthesize 3- (1,3-dioxoisoindolin-2-yl) benzyl nitrate (compound 4C). In this study, we show that compound 4C increases HbF production in a transgenic SCA mouse model and reduces the production of pro-inflammatory cytokines by SCA mouse monocytes cultured ex vivo. Therefore, compound 4C is a novel drug designed to treat SCA with a unique combination of HbF-inducing and anti-inflammatory properties.

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“…This in turn worsens the disease and causes a complication called vaso-occlusive crisis (VOC). VOC leads to intense pain that requires hospitalization and over time causes permanent organ damage [24]. According to Paul Frenette, director of the Gottesman institute for Stem Cell and Regenerative Medicine Research at Albert Einstein College of Medicine in New York City, there is still no specific therapy for VOC which is responsible for most of the symptoms of SCD [24].…”

Section: Challengesmentioning

confidence: 99%

Sickle cell Anaemia: The Need for Increased Drug Development in Africa

Olukorede

Farayola

Badmus

et al. 2022

Annals of Public Health Issues

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Sickle cell anaemia is a life-threatening genetic disease that causes damage to red blood cells by polymerisation of deoxygenated haemoglobin. It is highly prevalent in Africa especially in regions with high prevalence of malaria. Over the years, hydroxyurea had been the only promising drug used in the management of sickle cell anaemia; however, it has been found to be unaffordable and not readily available to the affected poor people in rural areas. Several challenges face drug development efforts in Africa yet there remains a significant need for the development and standardisation of newer, cheaper, and effective anti-sickling drugs that would be readily affordable and available to meet the needs of the African populace.

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Drug development: A complicated path

Cited by 5 publications

References 2 publications

Importance of health guidance for family members of children with sickle cell disease

Importance of health guidance for family members of children with sickle cell disease

A thalidomide–hydroxyurea hybrid increases HbF production in sickle cell mice and reduces the release of proinflammatory cytokines in cultured monocytes

Sickle cell Anaemia: The Need for Increased Drug Development in Africa

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