The state's $1 million investment toward finding a way to prevent ticks from spreading Lyme disease is paying off, a UMass Medical School researcher said Thursday, and an additional investment could move an antibody proven in labs to protect mice against Lyme closer to a human trial.

As Lyme, a tick-borne bacterial infection that can cause neurological problems if left untreated, has spread in both the number of cases and affected geography, Dr. Mark Klempner from the MassBiologics division of UMass Medical School has been leading a team to develop "a novel approach" to preventing the disease.

"It's really been a spreading, rising tide of cases that are concerning," he said, noting that the 35,000 to 40,000 reported annual cases of Lyme disease in the United States is "very underreported" and is likely closer to 300,000 annual cases. Since the mid-1990s, he said, the ticks that can carry Lyme disease have increased in concentration and spread from New England and the mid-Atlantic to the upper Midwest.

Klempner's team secured a $1 million appropriation in the current state budget and, paired with federal grants from the National Institutes of Health, developed a "pre-exposure prophylaxis" in which antibodies injected into a human could block the release of the Lyme bacteria if the human is bitten by an infected tick.

"It's really based on some very simple notion that the bacteria, before it comes to you, is stuck in the gut of the tick" and must make a complex trip through the tick before it can infect a human, he said Thursday at a briefing. "Our approach is to take advantage of this very complicated pathway for the bacteria to get out of the tick and into you ... a medicine that will circulate in you that when the tick drinks it, the blood will contain something that will kill the bacteria in the midgut or for sure prevent it from getting out of the gut so that none of this can happen."

The team ran an initial test on mice in which the mice were injected with the antibodies and then exposed to six Lyme-infected ticks each. At a dose as low as 5 mg per 2.5 pounds of body weight, the mice were found to be 100% protected from Lyme disease, Klempner said. The team also tested the preventative treatment on a monkey and was able to protect the monkey from Lyme disease exposure.

The development hit a snag, Klempner said, when his team realized that a single injection of antibodies would not protect a human for the duration of tick season.

"Our problem is that we've invented a drug that will be there for only a few weeks and yet your time of risk of exposure is many months, typically we say seven to eight months in our area around here," he said.

Researchers found a way to modify the antibody treatment and extend its usefulness by up to three-and-a-half times in mice, he said, but have not yet been able to test on humans to find out how long the antibodies would sustain in a human body.

That's where a new state investment would come in, according to UMass Medical School Vice Chancellor for Government Relations John Erwin. The school is seeking $800,000 in the fiscal 2020 budget, he said, which along with more than $2.1 million from the school would help Klempner's team test the safety and stability of its treatment, draft and file a new drug application with the Food and Drug Administration, and begin to conduct the first human trial.

Klempner said he is hoping to get FDA approval to conduct modified studies for the second and third phases of the treatment's development, which would be cheaper and possibly get the treatment to market sooner. The second phase study, he said, would typically cost up to $50 million but could be done for closer to $5 million if the FDA approves the modified test plan.

"If not, I'm going to have to partner with big pharma. It's that simple," Klempner said. In the most optimistic scenario, he said, the treatment could be available in about three-and-a-half years.

If or when the treatment becomes commercially available, the state could expect to recoup its investment. Under the terms of the funding, Klempner and Erwin said MassBiologics is required to make the treatment available to Massachusetts residents at a low cost and to pay back the state funding.

Though other states fund Lyme disease education efforts, Klempner said Massachusetts is the only one actively trying to come up with a solution to it.

"I don't know any other state that is funding drug development," he said. "I think this is totally unique."