History of CNS drug development

Biomarkers are ubiquitously used within drug development programs in both nonclinical species and in humans to assess safety and efficacy of novel compounds. To routinely apply such novel biomarkers with certainty, a well-defined data package is necessary for review and endorsement by regulatory agencies including the US Food and Drug Administration, European Medicines Agency, and Japanese Pharmaceuticals and Medical Devices Agency. This type of endorsement is known as regulatory qualification. Novel approaches are being applied to speed the process, lower the resource intensity, and increase the accessibility of biomarker qualification data and it is likely that consortia will continue to play a fundamental role in the qualification process by bringing together like-minded stakeholders focused on specific tools to accelerate drug development. This article will focus on learnings from the previous three nonclinical biomarker qualification projects, as well as discuss the progression of preclinical biomarker projects into the clinical qualification space and the current strategy for the use of nonclinical biomarker data in the translational qualification of clinical biomarkers; much like nonclinical information is used in the approval of drug development candidates. Impact statement This minireview provides an overview of the history of preclinical biomarker qualification by summarizing the three examples of this type of qualification with US Food and Drug Administration, European Medicines Agency, and Japanese Pharmaceuticals and Medical Devices Agency. In addition, an overview of the biomarker qualification process is included to educate key stakeholders with links to relevant white papers that provide information on current evidentiary considerations. The manuscript also provides new information on the evolution of the role that preclinical qualification plays in clinical qualification of biomarkers and the novel approaches that are being utilized to improve the process.

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Two Cases of Possible Familial Chronic Myeloid Leukemia in a Family with Extensive History of Cancer

Acta Haematologica ◽

10.1159/000513925 ◽

2021 ◽

pp. 1-6

Author(s):

Marisa J.L. Aitken ◽

Christopher B. Benton ◽

Ghayas C. Issa ◽

Koji Sasaki ◽

Musa Yilmaz ◽

...

Keyword(s):

Chronic Myeloid Leukemia ◽

Drug Development ◽

Fusion Protein ◽

Reciprocal Translocation ◽

Genetic Variants ◽

Myeloid Leukemia ◽

Familial Occurrence ◽

Familial Association ◽

History Of ◽

History Of Cancer

CML is defined by the presence of an oncogenic fusion protein caused by a reciprocal translocation between chromosomes 9q and 22q. While our molecular understanding of CML pathogenesis has revolutionized drug development for this disease, we have yet to identify many predisposing factors for CML. Familial occurrence of CML has been rarely reported. Here, we describe 2 cases of CML in a 24-year-old woman and in her 73-year-old maternal great aunt. We describe genetic variants in these patients and report on their environmental exposures that may have contributed to CML pathogenesis. The possible familial association of these 2 cases of CML warrants further investigation into more definitive etiologies of this disease.

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A History of Antidementic Drug Development in Japan

Taiwanese Journal of Psychiatry ◽

10.4103/tpsy.tpsy_33_20 ◽

2020 ◽

Vol 34 (4) ◽

pp. 152

Author(s):

Masatoshi Takeda ◽

Shinji Tagami

Keyword(s):

Drug Development ◽

History Of

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The Indolent Natural History of Essential Thrombocythemia: A Challenge to New Drug Development

Mayo Clinic Proceedings ◽

10.4065/80.1.97 ◽

2005 ◽

Vol 80 (1) ◽

pp. 97-98

Author(s):

Ayalew Tefferi

Keyword(s):

Natural History ◽

Drug Development ◽

Essential Thrombocythemia ◽

New Drug ◽

History Of ◽

New Drug Development

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Quest for COVID-19 cure: integrating traditional herbal medicines in the modern drug paradigm

Applied Science and Technology Annals ◽

10.3126/asta.v1i1.30275 ◽

2020 ◽

Vol 1 (1) ◽

pp. 63-71

Author(s):

Prativa Pandey ◽

Angisha Basnet ◽

Aatish Mali

Keyword(s):

Drug Development ◽

Traditional Medicine ◽

Treatment Approach ◽

Herbal Medicines ◽

Economic Loss ◽

Modern Medicine ◽

Traditional Medicines ◽

Modern Drug ◽

History Of ◽

Rich History

The world is facing one of the biggest public health tragedies of our time, both in terms of socio-economic loss and death tolls due to the coronavirus COVID-19 pandemic. In a frantic race to find treatment for COVID-19, many interventions to discover drugs and vaccines are being expedited. Similarly, traditional herbal medicines are also being explored to find a cure for COVID-19. There are many traditional medicines that have exhibited promising antiviral and immuno-modulating properties against a plethora of infectious diseases like influenza, malaria, tuberculosis, and even COVID-19. Traditional medicine is an integral part of culture and practices in many countries with a vast and rich history of treating diseases. However, scientific research-based drug development approaches and effective regulatory mechanisms, on par with modern medicine, should be implemented to ensure safety, efficacy and overall validity of traditional medicine. Incorporating evidence-based traditional medicines in modern drug development paradigms can help assure affordability, accessibility and acceptability of the treatment approach. Furthermore, it can create pharmacological synergism to tackle drug resistance. Altogether, every country should create a roadmap for modernization and revival of traditional knowledge to improve the health care system and be better prepared for health crises.

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