Stem Pharm Is Revolutionizing Drug Discovery for Brain Diseases
A team of scientists and entrepreneurs have developed a superior “brain-in-a-dish” platform that makes the development of therapeutics for diseases like Alzheimer’s and epilepsy much more likely to succeed.
The Challenge
Neurological diseases, such as Alzheimer’s, epilepsy, and brain cancer are devastating conditions with limited treatment options.
Pharmaceutical companies using traditional drug development methods have struggled to develop effective therapies for these conditions in part because the human brain is so complex. Specifically, the brain cells needed to do research have been so difficult to isolate and culture in the lab that neuroscience researchers have been forced to rely heavily on mice and rats. The results, historically, have been less than successful.
That’s where a startup out of Madison, Wisconsin, hopes to change the game. By developing a superior “brain-in-a-dish” technology, they aim to revolutionize neuroscience drug discovery, ultimately creating their own therapeutics for widespread brain conditions.
Origin Story
We pick up the Stem Pharm story over a cup of coffee in Madison, the picturesque college town home to the University of Wisconsin (UW). This was a few years ago when Steven Visuri, PhD, was an angel investor looking for investment opportunities. He sat down with the Stem Pharm co-founders, Bill Murphy, PhD, a UW professor, and Connie Lebakken, PhD, a cell biologist, who had just launched a company with technology from Dr. Murphy’s lab.
Both co-founders had close family members struggling with Alzheimer’s or dementia and were passionate about using discoveries and talents to get closer to a cure. Dr. Visuri had lost his father-in-law to Alzheimer's disease in 2018, and his sister has dementia and primary progressive aphasia, a brain condition that slowly degrades the ability to communicate.
“I'm very passionate about coming up with a therapeutic that's going to address her disease and symptoms,” says Dr. Visuri.
Drs Murphy and Lebakken are scientists but Visuri recalls they also keenly understood the business and social impact opportunity in front of them.
Impressed by the team and inspired by the vision – of finding a cure for Alzheimer’s and other disorders of the brain – Dr. Visuri signed on with the hopes of bringing Stem Pharm’s research into the world of pharmaceutical drug discovery. Shortly after he joined the company the team also brought on Ryan Gordon, PhD, who added deep domain expertise in pharma commercialization.
Under the Hood
If scientists want to do brain research – like discover a cure for Alzheimer’s – one thing they need is a proxy for the brain in the lab. So, they cultivate the cells necessary to approximate a piece of the brain in a petri dish. A newer approach taking hold in the field is to cultivate mixtures of brain cells together into a 3D structure, called neural organoids.
At the heart of Stem Pharm are a series of scientific breakthroughs related to these neural organoids.
“We and others will affectionately refer to this as brains-in-a-dish technology, but it is not a brain in a dish. Our platform approximates part of the function of the brain in a dish,” says Dr. Visuri. “In our case, we’re really good at modeling neuroinflammation. And the more you learn about it, the more you realize how complex that is.”
This is a rapidly emerging field today as dozens of teams from academic centers around the globe work to improve on the quality of neural organoids.
So what's unique about Stem Pharm’s approach? To put it simply, rather than simply housing neural organoids in a petri dish, they created a specific environment on which to grow the neural cells in a 3D structure. They build this structure on specialized synthetic biomaterials, and by doing so mimic the physiological environment of the brain and allow the growth of important cell types including neurons, astrocytes and microglia. The patents around their organoids and hydrogels give their work a helpful moat.
“By creating the right physical, chemical, and cellular environment for these cells, we can coax microglia cells to behave more like they're in the in vivo setting, that is, respond like they are in a human brain. That's what makes our model unique,” says Dr. Gordon.
“I think these complex models are probably the world's best way of studying neural inflammation in the laboratory,” says Dr. Visuri.
Next Steps
Recent successes in Alzheimer’s therapeutics, like those announced this year by Eli Lilly, have heated up the space and increased interest in Stem Pharm’s work. According to Drs Gordon and Visuri, one major opportunity is to work with leading pharma companies to use these superior brain organoids to develop treatments for brain diseases. And not just Alzheimer’s. These neural organoids are like “the goose that lays the golden egg,” says Dr. Visuri, because they can help researchers better understand all kinds of neural inflammation, which is related to a broader range of diseases.
“You can develop drugs to treat neural inflammation as it relates to Alzheimer's, Parkinson's, potentially ALS, MS, many, incredibly devastating neurologic diseases,” says Dr. Visuri. “It’s not a one-size-fits-all solution, but it’s a tremendous opportunity.” It’s helpful that each member of the leadership team has had startup company successes in pharma and life sciences and can bring those experiences to bear.
Stem Pharm works with larger pharmaceutical companies, but they are focused on developing their own therapeutics. It’s R&D-heavy work that will require a fair amount of upfront investment, which is part of their current calendar. Thankfully, the team has been very successful at raising grant funding to date – about six million dollars in total. They’ve got a long road ahead, and Stem Pharm is still a relatively small, scrappy team, but Dr. Gordon counts that as a strength.
“I think this is where the innovation happens in drug discovery,” says Dr. Gordon. “Small companies, new approaches, intriguing technology, changing the way drug discovery is being done. I think it's just really exciting that there's so much opportunity with this technology to make a huge difference.”
We couldn’t agree more. Join us in welcoming the Stem Pharm team to StartUp Health’s Alzheimer’s Moonshot.
Connect with Stem Pharm via email
Call for Alzheimer’s Innovation
With support from the Alzheimer’s Drug Discovery Foundation (ADDF) and Gates Ventures, we’ve launched a new global initiative created to develop a collaborative innovation community alongside leading companies, research teams, and stakeholders with a mission to accelerate progress in prevention, diagnosis, and management of Alzheimer’s disease and related dementias. Learn more and apply for an Alzheimer’s Moonshot Fellowship.
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Published: Aug 23, 2024