Researchers have announced “promising” results from an experimental compound that might provide a treatment for MLL-rearranged leukaemia in infants.
Rearrangement of the MLL gene – also known as KMT2A – is involved in up to 80% of cases of acute leukaemia in infants, according to the developers in Michigan, USA. Leukaemias with this mutation are often resistant to standard treatments, and as a result patients have poorer chances of survival compared to other types.
The new compound, named MS-41, targets and destroys ENL, a protein believed to be essential for MLL-rerranged leukaemia. This prevents the leukaemia cells proliferating and spreading, the researchers report in Science Advances.
MS-41 is a PROTAC degrader compound, a class of drug which, instead of blocking proteins and inhibiting their activity, tags the protein for destruction and leads to their complete removal from the cell.
MS-41 has been tested on laboratory mice and seemed not to harm healthy cells, the researchers say. They are planning to test the compound on other kinds of ENL-related leukaemias.
Study co-leader Professor Hon Wen, of the Van Andel Institute, Grand Rapids, said: “Up to 80% of acute leukaemias in infants are linked to problems with the MLL gene, yet there are few effective treatments for MLL-rearranged leukaemias.
“MS-41 is a new, experimental compound that effectively targets and degrades a central survival mechanism in leukaemia cells. Our early results are promising, and we’re excited to continue developing MS-41 as a potential leukaemia treatment.”
Source:
Xue Z, Qin L, Xuan H, Luo K, Huang M, Xie L, Su Y, Xu L, Harsh J, Dale B, Shi X, Chen X, Kaniskan HÜ, Jin J, Wen H. (2024) “A potent and selective ENL degrader suppresses oncogenic gene expression and leukemia progression.” Science Advances, 28 August 2024, doi: 10.1126/sciadv.ado1432.
Link: https://www.science.org/doi/10.1126/sciadv.ado1432
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