Challenges and Advances in Precision Drug Development for Non-Communicable Diseases | Chapter 6 | Achievements and Challenges of Medicine and Medical Science Vol. 13

Precision medicine focuses on targeted, personalized care. It uses Big Data to make disease or illness diagnoses, treatment therapies, and prevention more personalized, proactive, predictive, and precise. Precision medicine is “medical care designed to optimize efficiency or therapeutic benefit for particular groups of patients, especially by genetic or molecular profiling.” A somewhat vague outline of how precision medicine treats diseases suggests that physicians select treatments most likely to help patients. Drugs are available to treat infectious diseases. The situation is very different for noncommunicable diseases (NCDs); the essential part of applying precision medicine has frequently been ignored. Precision drugs developed based on omics information must be available for physicians to select meaningful treatments and therapies. However, molecular targets of NCDs needed for precision drug development have not yet been identified. For precision drug development, the necessary causes of NCDs need to be defined at the molecular level. Here, the essential requirements and tools for precision drug design, development, testing, manufacture, and clinical applications were examined. The findings revealed that Precision medicine is progressing; some 25% of all new therapeutics approved since 2015 have a precision or personalized component, according to the Personalized Medicine Coalition. This covers a wide range of modalities, including small molecules, antibodies, cells, and gene therapies, providing treatment alternatives for cancer and rare conditions such as spinal muscular atrophy (SMA), Duchenne muscular, and hemophilia. Such drugs are not yet available for the vast majority of NCDs. The study concluded that while the use of genomics in drug discovery and development generated excitement, the overall clinical efficacy of drugs developed so far has remained unimpressive, primarily due to the heterogenicity of diseases.

 

Author (s) Details

 

Karel Petrak
Research & Development, Matrx Pharmaceuticals, Inc., 555 Fifth Avenue, New York, NY 10017, USA.

 

Please see the book here:- https://doi.org/10.9734/bpi/acmms/v13/4515

Chylothorax Associated with Acute Lymphoblastic Leukemia | Chapter 7 | Challenges in Disease and Health Research Vol. 9

 Chylothorax is caused by lymphatic fluid accumulating in the pleural cavity from the ductus thoracicus. Chylous fluid has a murky, milky look due to the high levels of triglycerides it contains. Trauma or occlusion of the ductus thoracicus causes chylothorax. Malignancies, sarcoidosis, retrosternal goitre, amyloidosis, superior vena cava thrombosis, benign tumours, and congenital abnormalities such as lymphangioleiomyomatosis and hemangiomatosis are examples of nontraumatic etiologies.

We wanted to share a unique case of a woman who had shortness of breath due to chylothorax and was later diagnosed with acute lymphoblastic leukaemia. This report also includes chylothorax cases diagnosed with ALL from the literature.

Author (S) Details

Mehmet Can Ugur
Department of Hematology, Health Sciences University, Bozyaka Education and Training Hospital, Turkey.

Ulku Ergene
Department of Hematology, Health Sciences University, Bozyaka Education and Training Hospital, Turkey.

View Book :- https://stm.bookpi.org/CDHR-V9/article/view/2061