US-based miRagen Therapeutics has started a Phase I trial of its anti-cancer product candidate MRG-106, a synthetic microRNA antagonist (LNA antimiR) of microRNA-155, to treat patients with lymphoma.
In haematological malignancy, microRNA-155 has major roles in the differentiation, function and proliferation of blood and lymph cells.
Therapeutic inhibition of microRNA-155 in lymphoma cells reportedly restores thier normal function and reduces the aberrant cell proliferation that is characteristic of cancerous cells.
The trial is being carried out in patients suffering from cutaneous T-cell lymphoma (CTCL) of the mycosis fungoides (MF) sub-type.
miRagen R&D executive vice-president David Rodman said: "miR-155 is pathologically increased in many forms of lymphoma, including cutaneous T-cell lymphoma (CTCL) and diffuse large B-cell lymphoma (DLBCL).
"In our laboratory tests, MRG-106 enters lymphoma cells and induces programmed cell death through inhibition of microRNA-155, and although our first-in-human trial is designed to assess safety, tolerability and pharmacokinetics, we will also explore the molecular signature of MRG-106 in the lesions of these MF patients."
MicroRNAs are thought to be single molecular entities that dictate the expression of fundamental regulatory pathways, and could be potential treatments for controlling many disease processes.
miRagen president and chief executive officer William Marshall said: "This is the first clinical trial in lymphoma patients of an antimiR that targets a well-known oncogenic microRNA.
"We believe this trial of MRG-106 will advance a mechanistically important, potential new therapy for lymphoma patients and is an example of miRagen's focus on developing innovative product candidates for diseases where there is a significant unmet medical need."
miRagen aims to discover and develop new microRNA (miRNA)-targeting therapies for diseases with unmet medical needs.
In conjunction with strategic collaborators, the company seeks to leverage in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to develop new technologies and product candidates.
Image: Hodgkin lymphoma, nodular lymphocyte predominant (low power view ) H&E. Photo: courtesy of Gabriel Caponetti, MD.