A Special Place to Address Rare Diseases
Harnessing the tools of drug discovery in a nonprofit, academic setting

The Journey from Molecule to Medicine – A Photo Tour

Click on the arrows to see the path a discovery takes toward becoming a potential treatment.

Every Cure Begins With Understanding

Louis Scampavia, PhD, senior director of the UF Scripps High Throughput Molecular Screening Center
Louis Scampavia, PhD, is senior scientific director and co-director of the Wertheim UF Scripps High Throughput Molecular Screening Center.

It’s early on a Wednesday morning The team working in the robotic drug screening lab at The Wertheim UF Scripps Institute gets the text they’ve awaited. They jump out of bed and rush to the lab.

A patient with a rare form of brain cancer has consented to donate tissue from their cancer surgery to the institute. The precious tissue is carefully packed in dry ice at the hospital, and sent on its way to Jupiter, via courier, so it can be grown in culture and coaxed to form microscopic spheroids fit for tiny test tubes. Meanwhile, the cancer tissue’s genome is sequenced.

The institute is an unusal place, a nonprofit, academic research center with the expertise and technology of a pharmaceutical firm. Part of the University of Florida and its academic health center, UF Health, The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology owns a collection of every cancer drug tested in humans. It also has a robotic compound screening system that enables researchers to test all of the compounds against the tumor spheroids, alone or in new combinations, in a matter of hours.

The data gained will eventually be published in scientific journals so that cancer patients everywhere will benefit from the new understanding. It will also be used to engineer potential new treatments that act in novel ways.

The institute is made of more than 35 independently managed research labs, each headed by a scientist who is a leader in their field. They research the basic science of health and disease in areas that include brain development, learning and memory, brain degeneration, cancer, viral and infectious diseases, and genetic diseases including myotonic dystrophy, ALS, Fragile X Syndrome, Alport Syndrome, SYNGAP1 disorder and more. And because the institute possesses the expertise of a drug company, the scientists can go much further when they learn something new about a disease. They can collaborate on a possible treatment.

In the two decades since the institute launched on 30 acres off of Donald Ross Road and Central Boulevard, its scientists have contributed to the discovery of drugs and potential drugs for multiple sclerosis, glioblastoma, ALS, breast cancer, lymphomas, Parkinson’s, Alzheimer’s and more. People like Michael Cameron, Ph.D., have been key to these successes.

Evaluating the drug-like properties of compounds at UF|Scripps.

The journey from discovery to new medicine involves many steps, Cameron explains.

After the screening group identifies a promising compound, a team of medicinal chemists headed by Thomas Bannister, Ph.D., collaborates with Cameron to iterate a potential drug that’s potent, safe and selective. Having all of these experts together at one institute is rare and powerful, he says.

“Here, we can very quickly respond to new findings about disease, and see if we can come up with therapeutic interventions,” Cameron says. “Because we’re a nonprofit, we can leverage the expertise in biology, chemistry, drug design, and go after areas of unmet medical need, to try to make the biggest impact we can.”

Cameron’s area of expertise is drug metabolism. If a compound shows potential, he investigates how it should be dosed. Many questions must be considered: How can it be designed to reach the brain if that’s what’s needed? How can it be tested to ensure it is absorbed and breaks down in appropriate ways?

“Having this kind of expertise in an academic setting is almsot unheard of,” Cameron says. “We build this into our project teams so that we can react quickly. We can do everything in-house.


Further Reading:

Glioblastoma Drug OKd for Clinical Trials

New Grant Will Accelerate Research on Rare Cause of Childhood Epilepsy and Intellectual Disability

Chemists Honored With Top ACS Award for Myotonic Dystrophy Work

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