Clinical Trial Underway to Test MicroRNA Cancer Therapy

(NIST.gov)

Mirna Therapeutics Inc., a biotechnology company in Austin, Texas, recently began a clinical trial to test its lead candidate, MRX34 that simulates the tumor-suppression action of microRNA, as a therapy for cancer. The trial is an early-stage study mainly of MRX34’s safety with patients suffering from cancer of the liver or advanced or metastatic cancer involving the liver.

Mirna Therapeutics develops therapies based on the actions of microRNAs or miRNAs, which are genetic molecules serving as regulators of the genome. They start out small, but evolve into more complex molecules that interact with another type of RNA — messenger RNA — to repress the expression of genes that respond to various proteins. More than 1,500 miRNA genes control some 30 percent of all human genes, and can regulate the expression of tens to hundreds of genes, involving such functions as embryonic development, immune and related inflammatory responses, and cellular growth and proliferation.

The company’s technology uses a process called miRNA replacement therapy, to introduce material that simulates functioning miRNA, replaces the non-functioning miRNAs that have allowed cancer cells to proliferate, and reactivates pathways encouraging a therapeutic response.  Mirna Therapeutics says its miRNA replacement material has the same genetic sequence as the naturally occuring miRNA, replacing the depleted miRNA and addressing the same genes as the original miRNA. Thus, says the company, the miRNA replacement  is less likely by itself to produce an adverse effect.

The clinical trial began in April enrolling up to 48 adult patients at medical centers in Dallas and San Antonio with liver cancer or cancer in advanced or metastatic stages affecting the liver. MRX34 will be given with an IV twice a week for three weeks, with one week off, over a period of 28 days. Doses of the drug will be escalated, with safety indicators monitored, as well as activity of the drug in the bodies of the patients.

Mirna Therapeutics was founded in 2007, and gained investment backing from venture companies Sofinnova Ventures and New Enterprise Associates active in the life sciences, as well as Pfizer’s venture capital subsidiary. The clinical trial itself is funded by a grant from the Cancer Prevention and Research Institute of Texas.

Read more:

• AstraZeneca Licenses Messenger RNA Therapy Technology
• Patent Awarded for RNA Process of Inhibiting Gene Expression
• RNA Nanoparticles Advanced for Cancer Drug Delivery
• Compound That Repairs RNA Defects Identified

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