Rising Mucormycosis Cases Drive Efforts for Spotlight on Treatment Research
Ashraf S. Ibrahim, PhD, outlines safety studies and the potential of this antibody therapy for treating this rare fungal infection, which is seemingly becoming less rare.
Researchers are advancing the development of a novel immunotherapy for mucormycosis, a rare and often fatal fungal infection primarily affecting immunocompromised individuals. Ashraf S. Ibrahim, PhD, senior investigator at the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, discussed ongoing research efforts to establish the safety and efficacy of this potential treatment.
“What we're doing right now is we're trying to establish whether the treatment is safe,” Ibrahim explained. “So we've done something called tissue cross-reactivity studies with human tissues. We don't see a huge amount of binding, so cut it, I should say, is unique to the mucoralis fungi, which is the fungi that cause mucormycosis. You see a little bit of binding, cytoplasmic binding, which is not on the cell surface. For antibodies to be toxic, they have to bind to cell membrane proteins. So there is no cell membrane protein binding at all.”
Mucormycosis, caused by Mucorales fungi, presents a severe threat to individuals with weakened immune systems, such as those with uncontrolled diabetes or undergoing organ transplants. Traditional antifungal therapies have limited efficacy, underscoring the need for novel approaches like monoclonal antibodies.
The research is supported by funding from the National Institutes of Allergy and Infectious Diseases (NIAID) through an SBIR grant awarded to Vital X Biosciences, a company founded by Ibrahim. “We’ll be able to do GLP toxicity studies in rats, and we would basically try to obtain further funding to do GMP production and go into phase one clinical trial,” he stated. However, additional financial backing is required to move forward. “We still haven't gotten funds for that, but we've just submitted a grant in response to the broad agency announcement to try to obtain funds to take it into two different phase one clinical trials—one in adults and the other one in seniors. If we get awarded, then we probably can be in humans within two, two and a half years, including maybe two to three years, and then concluding the phase one clinical trials within four and a half to five years.”
Mucormycosis is becoming increasingly recognized due to rising case numbers worldwide. “The most important thing in rare diseases is that we basically have a good spotlight on them because these are really ignored diseases,” Ibrahim emphasized. “The problem is, you know, it is not as rare as it used to be. I've known a couple of people who had mucormycosis—friends, daughters, or sons of friends. When you get to that stage, then it is not as rare as you think. It is rare, meaning probably you see one case in a lifetime, but there are increasing numbers.”
Current estimates suggest approximately 4,000 cases occur annually in the United States, with a comparable burden in Western Europe. However, in endemic regions such as India, the numbers are significantly higher. “India in particular is endemic to the disease—200,000 cases per year at least,” Ibrahim noted. “I'm hearing Vietnam is also probably going to be in that region as well, not necessarily 200,000, but definitely in the thousands of cases. One tertiary hospital in Vietnam has seen, in the last year, 70 cases. That's a lot. We usually see in transplant centers between 10 to 15, maybe 20 cases per year. When you have 70 cases in one hospital that is not necessarily transplant-related, mainly diabetics, then you know that the burden of the disease is very high.”
Raising awareness and advancing new treatments remain crucial steps in addressing mucormycosis. “Trying to have a spotlight on the disease is probably helpful, at least, to try to get this treatment to be FDA-approved, hopefully, and available for patients to benefit from,” Ibrahim concluded.
As research progresses, this novel monoclonal antibody therapy could offer a significant breakthrough in the fight against mucormycosis, providing a much-needed option for patients with limited treatment alternatives.
