Researchers at Emory University are studying a drug intended to fight multiple sclerosis as a potential tool to help the immune system fight off HIV.
Dr. John Altman, a researcher at the Yerkes National Primate Research Center and the Emory Vaccine Center, recently received a $100,000 grant from the Bill & Melinda Gates Foundation to continue testing Fingolimod, code-named FTY720. The drug is currently undergoing clinical trials to prevent relapses of multiple sclerosis.
“The way that it is thought to work in the context of multiple sclerosis is it blocks a receptor that lymphocytes need to get out of lymph nodes, and in the context of autoimmune diseases that’s a good thing because you’re locking disease in the lymph nodes,” Altman said.
Altman will examine how FTY720 affects HIV in apes. Earlier this year the drug proved effective in rodents against the lymphocyte choriomeningitis virus (LCMV), a high-level, persistent, rodent-borne viral infection. The rodent immune system tries to fight LCMV but gets worn out and is not able to completely eradicate the infection. This is similar to the way some non-human primates respond to the simian immunodeficiency virus (SIV) and how humans react to HIV.
“I will emphasize that this whole thing is very risky. I would probably not be able to get any NIH [National Institutes of Health] funding at this stage without doing some pilot experiments, and that’s what this money from the Gates Foundation will allow me to do,” Altman said.
Altman is the only Emory researcher to receive a $100,000 Grand Challenges Explorations grant from the Bill & Melinda Gates Foundation this year. This award is part of the first round of funding for the Grand Challenges Explorations, an initiative to help lower the barriers for testing innovative ideas in global health.
The funding will provide for a year of non-human testing. At that point, Altman said they should know whether FTY720 should move on to a larger ape trial.
“We should have results within a year,” he said.
If the drug proves effective against SIV, then human trials could begin. Altman noted the drug is years away from possibly being shown effective in humans. He also cautioned against HIV-positive people using FTY720 as an “off label” product to fight HIV should it be approved safe and effective for multiple sclerosis.
“That would be very dangerous, and could potentially cause damage in someone who has HIV,” he said.
‘RISK WORTH TAKING’
Altman and his team stumbled across this use of FTY720 while testing another theory. A researcher in his lab was examining whether the drug would be effective in repressing infections in a depressed immune system, only to find it helped the immune system in some cases.
“So this exhausted immune system somehow removes the virus,” Altman said.
The drug stops disease from spreading by inducing lymphopenia, which causes a lower level of lymphocytes, which slows down the rate of infection and gives the immune system a better chance of responding.
The drug was initially tested as a transplant anti-rejection drug, but is currently in phase two and three human trials to test its effectiveness against two kinds of multiple sclerosis.
Potentially, FTY720 could be used to halt the infection in those recently exposed to HIV, but Altman hopes there are broader applications.
“The potential for translations for humans, is to use this in people who are chronically infected,” he said. “Where it fits in with respect to current anti-retroviruses therapy remains to be seen.”
Altman considers his research risky in the sense that it might not pan out, and is very careful to not raise the specter of false hope. Still, he has faith he is making the right gamble with his time.
“I wouldn’t bet the house on this,” he said. “But it’s a risk worth taking.”
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