The advent of MicroRNA treatments

 Here is a link to the article, which I have also copied below. This approach to combatting an illness is elegant in its conception and precise in its application, perhaps the beginning of a new era in medicine.

AASLD: MicroRNA Drug Safe in Ongoing HCV Study
By Michael Smith, North American Correspondent, MedPage Today
November 12, 2011 

MedPage Today Action Points
Note that this study was published as an abstract and presented at a conference. These data and conclusions should be considered to be preliminary until published in a peer-reviewed journal.
• Explain that a novel hepatitis C virus drug (miravirsen) targeting genetic material in the liver was safe and well tolerated in a small, dose-finding clinical trial.
• Point out that in all of the doses evaluated, the drug resulted in a significant decrease in hepatitis C RNA levels, compared with placebo.
• Review

SAN FRANCISCO — A novel hepatitis C virus (HCV) drug targeting genetic material in the liver was safe and well tolerated in a small, dose-finding clinical trial, a researcher said here.

Given as monotherapy, the compound, dubbed miravirsen, induced rapid dosedependent reductions in the hepatitis C viral load, according to Harry Janssen, MD, of Erasmus Medical Center in Rotterdam, the Netherlands.

The reductions were sustained for more than a month after the end of therapy, Janssen reported at a late-breaker session during the annual meeting of the American Association for the Study of Liver Diseases. 

Miravirsen “has the potential to eradicate” hepatitis C virus either alone or as part of an interferon-free regimen, Janssen concluded.

The compound blocks a host microRNA — miR-122 — that is critical to hepatitis C accumulation in the liver, Janssen said. MicroRNAs play important roles in gene regulation and expression and Janssen said miravirsen is the first drug to exploit a microRNA target for therapy.

Indeed, of the research presented at the late-breaker session, this study is ” the most exciting because it is a whole new class of drug,” said Norah Terrault, MD, of the University of California San Francisco, who was not part of the study but who was one of the moderators of the session. 

For the study, Janssen said, researchers enrolled 36 patients with the difficult-to-treat genotype 1 of hepatitis C and assigned them to placebo or one of three doses of the drug — 3, 5, and 7 mg/kg. 

The patients, none of whom had been previously treated with pegylated interferon and ribavirin, were given five subcutaneous injections of the drug over four weeks and then followed for another 14 weeks. 

The primary endpoint was safety and tolerability, Janssen said, with viral response as a second endpoint. Not all patients have completed the study, so the researchers reported data up to week 10, six weeks after the final dose. 

Adverse events, he said, were “very much balanced” among the arms and over all “there were not a lot of side effects seen here.” Most such events were mild and none led to stopping treatment, he added. 

The only serious adverse event, in a patient receiving the high dose of the drug, was not considered related to treatment, he said. 

In all three arms, the drug resulted in a significant drop in hepatitis C RNA levels, compared with placebo. Specifically: 

The low dose (3 mg/kg) led to a 0.57 IU/mL decline in viral load, which was significant at P=0.0334.

• The medium dose (5 mg/kg) yielded a drop of 2.16 IU/mL, which was significant at P=0.007.

• The high dose (7 mg/kg) led to a decline of 2.73 IU/mL, significant at P<0.001.

Although some patients in the low- and medium-dose arms had been allowed to begin treatment with peginterferon and ribavirin three weeks after their last dose of miravirsen, those taking the high dose were not allowed standard therapy until week 10. 

Janssen said the drop in viral load seen with the high dose at week 10 “is a pure effect of miravirsen.”

The downside of the drug, Terrault told MedPage Today, is that “it has to be given by injection, and that’s always a challenging form of treatment.” 

On the other hand, it appears well tolerated and safe, with good efficacy, so “it might still be part of the mix” of treatment options. “I clearly get the sense it isn’t going to be used solo,” she said. 

The study was supported by Santaris Pharma. Janssen reported financial links with the company and one author was employed by the company. 

Terrault reported financial links with Gilead, Pfizer, Genentech, Roche, SciClone, BMS, Novartis, Eisai, and Vertex. 

Primary source: Hepatology

Source reference:

Janssen HL, et al “A randomized, double-blind, placebo (PLB) controlled safety and anti-viral proof of concept study of miravirsen (MIR), an oligonucleotide targeting miR-122, in treatment naive patients with genotype 1 (GT1) chronic HCV infection” Hepatology 2011; Abstract LB-6.

Disclaimer The information presented in this activity is that of the authors and does not necessarily represent the views of the University of Pennsylvania School of Medicine, MedPage Today, and the commercial supporter. Specific medicines discussed in this activity may not yet be approved by the FDA for the use as indicated by the writer or reviewer. Before prescribing any medication, we advise you to review the complete prescribing information, including indications, contraindications, warnings, precautions, and adverse effects. Specific patient care decisions are the responsibility of the healthcare professional caring for the patient. Please review our Terms of Use. © 2004-2011 MedPage Today, LLC. All Rights Reserved.

6 thoughts on “The advent of MicroRNA treatments

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