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January 8, 2018

UMass Medical researchers conducting global research

UMass Medical School researcher Raffi Van Aroian shows visitors a microscoptic look at a hookworm in his lab.
Haitian mothers waiting on diphtheria vaccinations.
Centers for Disease Control responds to a diphtheria outbreak in Indonesia.

Their work may not yield patents worth billions or save millions of American lives, but UMass Medical School researchers are conducting ambitious work trying to solve problems on the other side of the planet.

In one lab at the UMass Medicine Science Park, Raffi Van Aroian is working away at a topic few have the stomach to tackle: hookworms. The insects are tiny but vicious, infecting most often in India, Africa and other developing communities.

Mark Klempner, the head of the medical school's MassBiologics center, is working on solving another issue few in the United States need to worry about today: diphtheria.

Both doctors say they're tantalizingly close to bringing their work to the market.

“We're going to make this medicine,” Klempner said of his drug for diphtheria, an infection that's most common in war-torn areas like Syria, Bangladesh and Yemen.

“I'm compelled to do it,” Klempner continued. “I lose sleep every night knowing there's a kid dying of diphtheria, and I have the solution.”

$5M to save children

Klempner doesn't hide his frustrations in being unable to land a relatively small amount of funding to obtain regulatory approval. He's been working for about seven years on a diphtheria antitoxin drug using cell cultures.

“It's been very hard,” Klempner said.

He has the drug simply sitting in his freezer waiting for the next step: More funding to help him obtain U.S. Food and Drug Administration permission to bring the drug to market.

Klempner estimates he needs about $5 million to get to that stage – a drop in the bucket, he says, compared to big pharmaceutical companies spending upwards of $1 billion bringing certain drugs to market to treat diseases like cancer.

Pharmaceutical companies no longer produce treatments for diphtheria, he said, because the market for is so small.

Until routine vaccations began in the United States, diphtheria killed up to half of those who got the disease, according to the Centers for Disease Control and Prevention.

In 2016, just over 7,000 diphtheria cases were reported worldwide, according to the World Health Organization. That's a fraction of the number two decades ago thanks to immunization efforts WHO estimates covers nearly 90 percent of the world's population.

Diphtheria, which is transmitted through coughing and sneezing, strikes the nose and throat and can suffocate a child in a matter of days, said Klempner. The disease is a problem today largely only in areas with displaced populations hard for agencies to reach.

“Where do you even begin to try to sell it to someone who's in Yemen?” Klempner asks.

Treating rabies in India

Klempner has already proven such efforts can work with other diseases Americans rarely need to worry about. A drug treating rabies, called Rabieshield, was released to big fanfare in India in the fall.

“We felt like rock stars there,” said Klempner, who attended an event with UMass Medical School Chancellor Michael Collins.

Rabies kills an estimated 20,000 people every year in India, according to MassBiologics, the vaccine-manufacturing division of the UMass Medical School of which Klempner is the executive vice chancellor.

The non-lethal problem

It has been a similar effort for Aroian to try tackling hookworms.

Hookworms enter the body through fecal contamination – a common risk in areas without modern plumbing – or by penetrating through skin, which is more routine in places where people might not wear shoes as often.

Approximately 1.5 billion people – or about one of five worldwide – are infected with soil-transmitted worms such as hookworms or roundworms, according to the WHO.

It is rarely deadly – and “that's the tricky part,” Aroian said. “If they did kill, we'd probably pay a lot more attention to them.”

Hookworms typically live only a year or two, Aroian said, but detecting symptoms can take 50 or 60 days. Victims feel lethargic, a problem Aroian said compounds poverty in often much poorer areas where hookworms pose the highest risk.

In a presentation of his work, Aroian shows an old black-and-white photo of two boys who look to be middle-school age and asks how old they are. It's a trick question. One boy is 12 and the other is actually a 21-year-old man whose development was stunted by hookworm-caused malnourishment.

Market barriers

Aroian is a Worcester native who moved back to his home city three years after after a stint at the University of California San Diego because he said the UMass Medical School financially supported his work. Now at UMass, Aroian said he's taken a protein used in pesticides and has proven it can be successful in treating hookworms in dogs, pigs and rodents. He's been working in collaboration with another UMass researcher, Gary Ostroff.

A drug, known by the acronym IBaCC, would be given to those where hookworm is deemed most prevalent.

Hurdles remain in bringing the drug to market.

It needs to be made cheap, safe, easily scaled up to large quantities, stable during transportation and able to sit on a shelf for years, possibly in high temperatures and humidity, Aroian said.

IBaCC – pronounced eye-back – is safe, but it still needs to meet those other challenges. Getting to FDA approval could take another $1 million to $5 million, Aroian said.

“This is really about the children in the world who through no fault of their own are so badly harmed,” Aroian said. “You don't get a fair shot at life, period.”

If things fall into place, Aroian said, the drug could hit the market within a year or two.

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