Previous research discovered that alterations to the enzyme ACVR1 are present in approximately 25% of diffuse midline gliomas. These alterations results in the enzyme becoming overactive and causing tumour growth.
Researchers at Northwestern University in Chicago, showed that an altered version of ACVR1 cooperates with another group of proteins called histones. Histone proteins act like a spool for DNA, as they help condense long strands of DNA. Histones also help to regulate the way in which the DNA code is 'read'.
The combination of ACVR1 and histone changes were shown to play an important role in initiating tumour growth.
This study also showed that reducing the activity of ACVR1 has the potential to slow tumour growth and increase survival in pre-clinical models of diffuse midline gliomas.
Diffuse midline glioma is a type of primary, high-grade brain tumour that occurs in children. It is one of the most fatal paediatric brain tumours, with less than 1% of patients diagnosed surviving more than 5 years.
The dismal prognosis of these tumours can be blamed on its location within the brain, the brain stem. The brain stem is difficult to access, making it difficult to remove the tumour using current surgical techniques.
The current standard of care to treat diffuse midline glioma is radiation therapy. However, this is not a cure and only reduces the symptoms temporarily, making new and effective treatments for this tumour type critical.
The results of this study are encouraging and the researchers aim to continue further research on this drug before advancing it into clinical trials.
Doing similar work is Structural Genomics Consortium (SGC), who are working in partnership with The Charity to screen various drugs to block the activity of ACVR1. The SGC work closely with a biotech company called M4K Pharma who have found a compound, called MK42009, that has the ability to block the function of ACVR1.
While further research is needed before this compound can be tested in children, the compound had shown to be selective, can be administered orally, and has the ability to cross the blood brain barrier.
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