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Developing drugs as a treatment for myotonic dystrophy (360G-Wellcome-107562_Z_15_A)

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy in adults. It is a highly debilitating condition affecting more than 100,000 patients in developed countries with an average life expectancy of 58 years. DM1 is primarily a neuromuscular disorder, which also affects a range of other systems including the heart, brain, endocrine and digestive systems. Patients may also show specific patterns of psychological dysfunction and personality traits, cognitive impairment/mental retardation and excessive daytime sleepiness. All features show an obvious deterioration with time and difficulty swallowing and sucking food into the lungs in the later stages of the disease contribute towards chest infections and represent a major cause of morbidity and mortality. There is no treatment for DM1. DM1 is caused by a repeat expansion mutation in the 3' untranslated region of the DMPK gene. Unaffected people have 5 to 30 copies of this sequence whereas patients may have hundreds or sometimes thousands of copies. When expressed the DMPK expansion transcripts remain in the nucleus where they form distinct spots or foci. Professors Chris Hayes and David Brook at the University of Nottingham developed an assay to screen for compounds that might provide a treatment for DM1. They identified small molecules that target a novel protein and destroy the spots in DM1 cells, thereby leading to a significant reduction in the faulty RNA and other molecular features of the disorder. Their drug discovery approach, in collaboration with Argenta, a Charles River company, is based on targeting this novel protein, by refining the chemical starting points to make them more selective and more suitable for oral administration to patients. The multisystem nature of DM1 provides particular challenges but Professors Hayes and Brook anticipate that a successful drug would target most/all features of the disease


02 Oct 2016

Grant details
Amount Awarded 356400
Applicant Surname Brook
Approval Committee Internal Decision Panel
Award Date 2016-10-02T00:00:00+00:00
Financial Year 2016/17
Grant Programme: Title Seeding Drug Discovery Award
Internal ID 107562/Z/15/A
Lead Applicant Prof John David Brook
Other Applicant(s) Prof Christopher Hayes, Dr Rebecca Trueman
Planned Dates: End Date 2018-01-31T00:00:00+00:00
Planned Dates: Start Date 2017-01-31T00:00:00+00:00
Recipient Org: Country United Kingdom
Region East Midlands
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