Time: 2024-07-06
The search for a drug to effectively treat Sickle cell disease ( SCD ) has been a long - standing challenge in the field of hematology and drug discovery . Researchers at the Novartis Institutes for BioMedical Research ( NIBR ) have made a significant breakthrough by identifying a small - molecule drug candidate that has the potential to treat SCD patients by increasing the levels of fetal hemoglobin ( HbF ) . The study , led by Pamela Ting , PhD , has been published in Science , with the support of Jay Bradner , MD , PhD , a former president of NIBR who recently joined Amgen.
The research at Novartis falls under the category of molecular glue pharmacology , where slight modifications to drug compounds can impact the degradation of targeted proteins . The team at Novartis conducted a screen of nearly 3,000 compounds targeting cereblon , a component involved in protein degradation . Through various experiments and tests , they identified a promising drug candidate , dWIZ-2 , that could trigger targeted protein degradation of the WIZ transcription factor , leading to the upregulation of HbF. This new approach shows potential in treating SCD patients by inducing fetal hemoglobin production.
While the results of the study are promising , there are still unanswered questions and challenges that need to be addressed . Specificity of inhibition and potential side effects need further study before progressing to human clinical trials . Researchers like Vivien Sheehan and Vijay Sankaran emphasize the need for quantification data and safety assessments before moving forward . The team at Novartis remains committed to advancing the research and developing a drug that can benefit SCD patients around the world.
The development of a small - molecule drug for SCD treatment could have a significant impact on patients worldwide , especially in medically underserved communities and low - income countries . The potential affordability and accessibility of such a drug are crucial for reaching millions of patients in Africa and beyond . However , ensuring global access to drug therapies remains a challenge that requires careful consideration and planning . The future of SCD treatment holds promise , but further research and collaboration are essential to address the unmet needs of patients with this debilitating disease.
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