- A new coronavirus drug type is in clinical trials, with researchers studying its efficacy at blocking the virus from infecting cells.
- PIKfyve kinase inhibitors like vacuolin-1 and apilimod have been found in lab tests to block the SARS-CoV-2 virus from infecting human cells.
- Scientists are now looking to replicate the same results in human trials.
Scientists from all over the world have come up with all sorts of ways to fight the novel coronavirus, targeting the pathogen with various existing drugs and new compounds in an attempt to prevent it from infecting cells. Some of these drugs failed in key tests, with hydroxychloroquine being the most prominent failure so far. Others have shown some efficacy, like remdesivir, dexamethasone, and blood thinners. Several new treatments are still in testing and could yield results in the coming months. It’s not just vaccines the world needs to end the pandemic, after all. Effective COVID-19 therapies that can significantly reduce complications and deaths are also needed to help those people who will continue to get infected.
Researchers are now studying vacuolin-1 and apilimod, two similar drugs that might be able to block the novel coronavirus from infecting cells.
The drugs aren’t brand new, Harvard Medical School explains. But they’ve been repurposed for treating COVID-19.
Vacuolin-1 and apilimod were developed years ago and they target an enzyme called PIKfyve kinase. This enzyme has a role in the COVID-19 infection, which is why the drugs might work. Tomas Kirchhaused, professor of cell biology in the Blavatnik Institute at HMS and professor of pediatrics at Boston Children’s, discovered vacuolin-1 16 years ago. Apilimod was developed by LAM Therapeutics. The two drugs are similar and they can both block the Ebola virus, researchers found a few years ago. Those studies were continued once the novel coronavirus arrived, as Kirchhausen realized that the kinetics of cell entry in Ebola and COVID-19 are similar.
Published in PNAS, a study on the matter explains that PIKfyve kinase inhibitors could prevent infection from either SARS-CoV-2 or the Zaire ebolavirus.
“Our findings show that targeting this kinase through a small-molecule antiviral against SARS-CoV-2 may be an effective strategy to lessen the progression or seriousness of COVID-19,” study co-senior author Kirchhausen said. “Within a week, we knew apilimod worked extremely well in preventing SARS-CoV-2 infection in human cells in the lab. We found that like apilimod, vacuolin-1 is a very strong inhibitor for viral infection in the lab.”
Separately, a paper in Nature published a list of 12,000 clinical-stage or FDA-approved small molecules that could inhibit the replication of the novel coronavirus. Apilimod was one of the drugs included on that list.
AI Therapeutics tested apilimod in Phase 1 and 2 trials for the treatment of autoimmune conditions, but that drug failed to show any efficacy. However, the tests proved the compound did not produce significant side effects even after more than a year of high doses.
The company received FDA approval this spring to see if apilimod can reduce the severity of COVID-19, using data from Kirchhausen’s early study that was initially published in pre-print form in bioRxiv, as well as other drug screens. AI Therapeutics then announced the start of a randomized, double-blind, placebo-controlled study with apilimod in late July— the LAM-002 study.
The drug will be tested on 140 COVID-19 patients, with the company looking to assess its safety, tolerability, and efficacy at reducing the viral load in patients.