HD is living in an era of target-specific drug development with emphasis on the mechanisms related to mutant Huntingtin (HTT) protein. Examples include antisense oligonucleotides (ASO), splicing modifiers and microRNA molecules that aim to reduce the levels of mutant HTT protein. After initial negative results with ASO molecules Tominersen and WVE-120101/ WVE-120102, the therapeutic landscape continues to expand, with various trials currently under development to document proof-of-concept and safety/tolerability. Immune-targeted therapies have also been evaluated in early-phase clinical trials, with promising preliminary findings. The possibility of quantifying mHTT in CSF, along with the development of an integrated biological staging system in HD are important innovations applicable to clinical trial design that enhance the drug development process. Although a future in HD with DMTs remains a hope for those living with HD, care partners and care providers, the therapeutic landscape is promising, with various drug development programs underway following a targeted approach supported by disease-specific biomarkers and staging frameworks.
Keyphrases
- clinical trial
- healthcare
- palliative care
- open label
- lymph node
- quality improvement
- pet ct
- double blind
- protein protein
- phase ii
- single cell
- pain management
- small molecule
- binding protein
- study protocol
- wild type
- phase iii
- randomized controlled trial
- affordable care act
- cancer therapy
- health insurance
- hepatitis c virus
- human immunodeficiency virus