Pharmaceutical Development for Huntington’s Disease
- PMID: 21882420
- Bookshelf ID: NBK56003
Pharmaceutical Development for Huntington’s Disease
Excerpt
Huntington’s disease (HD) is a devastating neurological disorder caused by mutations in the human gene encoding the huntingtin protein, htt. Since the identification of the HD gene nearly 15 years ago, there has been enormous progress in understanding the molecular features of HD pathology at the cellular level, as well the development of a plethora of HD models in the mouse and other experimental organisms. These studies have led to the beginnings of a systematic process of target selection and validation in the service of formulating rational strategies for pharmaceutical development of HD therapeutics (see Chapter 4, this volume). Some of these targets have matured to the point of being the subjects of directed translational research programs in academia, biotechnology, and pharma organizations. This chapter will describe some of the features that have made the identification of treatments for HD a challenge and how the field is moving forward in the face of such challenges. In particular, we will focus on the activities of the CHDI Foundation, Inc. to bring disease-modifying treatments to the clinic.
Copyright © 2011 by Taylor and Francis Group, LLC.
Sections
Similar articles
-
Huntington's Disease Regulatory Science Consortium: Accelerating Medical Product Development.J Huntingtons Dis. 2022;11(2):97-104. doi: 10.3233/JHD-220533. J Huntingtons Dis. 2022. PMID: 35466945
-
Target Validation for Huntington’s Disease.In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 4. In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 4. PMID: 21882416 Free Books & Documents. Review.
-
Protein Interactions and Target Discovery in Huntington’s Disease.In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 3. In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 3. PMID: 21882413 Free Books & Documents. Review.
-
Value of Invertebrate Genetics and Biology to Develop Neuroprotective and Preventive Medicine in Huntington’s Disease.In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 6. In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 6. PMID: 21882411 Free Books & Documents. Review.
-
Biomarkers to Enable the Development of Neuroprotective Therapies for Huntington’s Disease.In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 11. In: Lo DC, Hughes RE, editors. Neurobiology of Huntington's Disease: Applications to Drug Discovery. Boca Raton (FL): CRC Press/Taylor & Francis; 2011. Chapter 11. PMID: 21882408 Free Books & Documents. Review.
References
-
- Bonelli RM, Hofmann P. A systematic review of the treatment studies in Huntington’s disease since 1990. Expert Opin Pharmacother. 2007;8:141–153. - PubMed
-
- Shults CW, Oakes D, Kieburtz K, Beal MF, Haas R, Plumb S, Juncos JL, Nutt J, Shoulson I, Carter J, et al. Effects of coenzyme q10 in early Parkinson disease: evidence of slowing of the functional decline. Arch Neurol. 2002;59:1541–1550. - PubMed
-
- Huntington Study Group. A randomized, placebo-controlled trial of coenzyme q10 and remacemide in Huntington’s disease. Neurology. 2001;57:397–404. - PubMed
-
- Murck H, Manku M. Ethyl-epa in Huntington disease: potentially relevant mechanism of action. Brain Res Bull. 2007;72:159–164. - PubMed
-
- Puri BK, Leavitt BR, Hayden MR, Ross CA, Rosenblatt A, Greenamyre JT, Hersch S, Vaddadi KS, Sword A, Horrobin DF et al. Ethyl-epa in Huntington disease: a doubleblind, randomized, placebo-controlled trial. Neurology. 2005;65:286–292. - PubMed
Publication types
LinkOut - more resources
Full Text Sources