NEW YORK – The basic strategy of the international health organizations dealing with the current outbreak of the deadly Ebola virus in Guinea is to contain it by isolating the people infected.
The World Health Organization and other global bodies agree no medical treatment or vaccine exists for Ebola, which can prove lethal in 90 percent of cases.
A treatment that appears effective, however, against Ebola, other hemorrhagic fevers and HIV/AIDS, has been in various stages of testing since 2001.
The journey of Cyanovirin-N through the testing and approval process illustrates the difficulty of bringing promising treatments to the market.
Retired entrepreneur Jack Kneifl told WND he obtained a $5-million commitment in March 2001 from an “angel investor” to obtain an FDA license to manufacture a drug incorporating Cyanovirin-N, which is a protein.
It was discovered by a group headed by Dr. Michael R. Boyd at the National Cancer Institute of the National Institutes of Health near Washington, D.C.
Kneifl co-founded a pharmaceutical company with three other individuals called OmniViral Therapeutics LLC, located in Germantown, Md., near the National Institutes of Health.
“Within a matter of months we negotiated the license and began the pre-clinical trials,” he told WND.
WND was not able to reach Boyd, who retired last month as director of the University of South Alabama Mitchell Cancer Institute.
Boyd is the co-author of an academic article published in March 2001 by the Antimicrobial Agents and Chemotherapy Journal and posted online by the National Institutes of Health that states Cyanoviron-N potently inactivates diverse strains of HIV-1, HIV-2, simian immunodeficiency virus, and feline immunodeficiency virus.
The bottom line
Kneifl said that preliminary research on Cyanovirin-N by Boyd’s group had shown that, due to its Method of Action, or MOA, it was 100 percent effective in the treatment of HIV/AIDS. In the treatment of all known strains of influenza, including Ebola and other hemorrhagic fevers, it showed at least 90 percent efficacy, he said.
“The bottom line,” Kneifl said, “is Cyanovirin-N demonstrated potential applications to microbicide development.”
Microbicides are chemical substances that reduce the infectious nature of various viruses or bacteria, in comparison to vaccines, which have biological agents that improve immunity to a particular disease.
Simply understood, Cyanovirin-N is a protein that prevents viruses and bacteria from attaching to and infecting healthy cells.
Based on the success of Boyd’s initial research, Kneifl began approaching potential investors to raise the funds needed to transform the discovery of Cyanovirin-N into a commercially viable medical treatment.
“By mid-2003 and some $5.5 million later, OmniViral had completed the pre-clinical studies which demonstrated an extraordinary level of success,” he recalled.
Pre-clinical trials, conducted on animals to determine toxicity and efficacy, are typically followed by more costly Phase 1 trials with a small human population to test toxicity and Phase 2 trials with a small human population to test efficacy.
Kneifl said the chief scientific officer working with OmniViral indicated that Cyanovirin-N “had greater potential than any other drug he had ever tested.”
The next step, he said, was to complete Phase 1 and Phase 2 clinical studies at an estimated cost of $8.5 million.
“I went back to my angel investor requesting him to continue funding the operations,” Kneifl said. “He turned into a chicken and pulled his funding. I believe it was because he was an industrialist, and this was his first venture investing in pharmaceuticals.”
Kneifl said the investor “didn’t realize how expensive FDA drug testing is during development phases.”
Had Cyanovirin-N passed Phase 1 and 2 clinical tests, Kneifl believed large pharmaceutical companies would have been interested in investing the $50 to $100 million needed to complete large-scale Phase 3 tests on human populations.
“Due to lack of funding, OmniViral, for all practical purposes, was closed down at the end of 2004,” he said.
FDA spokeswoman Stephanie Yao told WND she could not speak specifically about Cyanovirin-N, but, in general, she said, “efficacy found during pre-clinical testing does not always translate to clinical testing.”
“Discovering a new molecule that is effective, can be administered and can be tolerated by humans is not an easy task,” Yao said. “Many drugs encounter problems during the development process that prevent their further development.
Boyd and his colleagues announced the discovery of Cyanovirin-N in a professional article published in a peer reviewed medical journal in July 1997.
In subsequent publications in peer reviewed medical journals, Boyd and his colleagues conducted tests demonstrating Cyanovirin-N was highly effective in combating HIV/AIDS, moderately effective treating herpes and various strains of the hepatitis virus, as well as highly effective against almost all strains of the influenza A and B virus.
The findings of the Microbicide Initiative, a Science Working Group founded by the Rockefeller Foundation in 2000, called for the development of microbicides to combat HIV/AIDS. The group argued microbicides were likely to become available more widely and much more rapidly than vaccines and also act at an earlier stage of the infection. They may also be able to interrupt the spread of other sexually transmitted infections that an HIV-specific vaccine would be unlikely to affect.
“Investment in this global priority is now needed as never before,” the Rockefeller report concluded. “Since large-scale pharmaceutical industry involvement is unlikely until a phase 3 trial has demonstrated human efficacy, this investment will be required from public agencies and from private philanthropies. Prioritizing an investment in the microbicides field offers a realistic, manageable, and near-term chance to seize a powerful opportunity to promote the public good.”
The National Institutes of Health confirmed to WND that OmniViral had patent license agreements with the NIH for Cyanovirin-N technology that are now expired or terminated.
An author of the initial discovery paper, Dr. Barry O’Keefe, deputy chief of molecular biology at the National Cancer Institute’s Molecular Targets Laboratory, has worked with the protein for several years and tested it against both influenza and Ebola.
O’Keefe said he was not personally aware of the results of Omniviral’s preclinical studies. He affirmed, however, that scientific studies were performed on Cyanovirin-N at the National Cancer Institute.
He said the studies found Cyanovirin-N was active against influenza in mice at levels up to 100 percent In the Ebola studies, systemic treatment with Cyanovirin-N only delayed the time until death for treated mice. None of the animals survived.
An April 2013 article in the Brazilian magazine Pesquisa, published by the Sao Paulo Research Foundation, explained that work is still going on to develop Cyanovirin-N, at least for HIV prevention.
The article cites O’Keefe, who explained that Cyanovirin-N was at the pre-clinical development stage and hadn’t been tested on humans yet.
The article noted O’Keefe spearheaded a study published in 2003 that showed that the protein was also active against some strains of the influenza virus.
O’Keefe was taking part in studies, the article said, to obtain the protein through plants, such as soybean seeds.
“We still lack a commercially viable, low-cost way to achieve large-scale production of Cyanovirin-N,” O’Keefe said, “and plants are a good means to that end.”
The researchers’ first stumbling block, the article said, was obtaining large quantities of the protein.
The latest challenge has been to improve the protein extraction process in order to purify larger quantities of Cyanovirin-N from soybean seeds.