Discovery at MIDD Leads to Human Trials

For the first time, a compound discovered at the Milwaukee Institute for Drug Discovery, or MIDD, has been approved to enter human clinical trials. The drug is under development by Damona Pharmaceuticals, a biopharmaceutical company developing drugs to reverse cognitive deficits associated with brain disorders. It’s the first step of testing the drug in humans that, if successful, paves the way for the drug to become a medicine available for patient treatment.

The drug was discovered in part by Jim Cook, who has emeritus status but still performs research as an adjunct professor in UWM’s Chemistry & Biochemistry Department. He is also one of the founders of MIDD. Operating under the umbrella of the Chemistry & Biochemistry Department, MIDD advances research and late-stage development of new drugs. The institute draws its members from various departments across UWM.

Cook is retired, but when he worked at MIDD, he developed compounds that were derivatives of benzodiazepines. That’s the same category of drugs that includes anxiety medications like Valium and Xanax – “Both of which have bad side effects,” he said. So, Cook and his colleagues tried to design compounds without those side effects by targeting a different receptor in the brain than the receptors that previous drugs bind to. They came up with several iterations and even licensed some to pharmacological companies, including Bristol Myers Squibb, but for various reasons, the drugs were never brought to full trials.Then one of Cook’s compounds caught the eye of a researcher named Etienne Sibille, who is the deputy director and senior scientist in the Campbell Family Mental Health Research Institute at the University of Toronto and the Chief Scientific Officer and a co-founder of Damona Pharmaceuticals. Together, they tweaked the design to increase its efficacy until they came up with a candidate that achieved impressive results in preclinical studies.

Damona is initially focused on developing the compound to treat cognitive deficits associated with depression and Alzheimer’s disease, but Cook is hopeful that it may eventually be a treatment for schizophrenia as well. For now, the FDA has approved Damona’s application to study its safety in treating cognitive deficits associated with depression.

Going through FDA trials means a lot of risk with many roadblocks, but the reward is high. If the drug were to pass clinical trials and become available for the consumer market, UW-Milwaukee and the UWM Research Foundation and MIDD would receive royalties from drug sales which would be distributed to all inventors, though the lion’s share would go to the drug company that will ultimately sell and market the compound. But more than the potential payday, Cook and his colleagues stay motivated through the hard work, the bureaucratic red tape, and the numerous roadblocks to success because they want to help people.

Even if their drugs don’t make it out of trial or end up curing patients, researchers can still publish their findings in academic papers. Their work might help another chemist make a groundbreaking discovery that will someday aid patients.