Cville Company Uses Genetics and Big Data to Find Better Medical Treatments

May 28, 2019 | Company, News


Getting approval for new drugs takes years and costs companies millions of dollars. What’s more, clinical trials sometimes fail, leaving firms with nothing to show. Now, however, a Virginia company is taking a different approach – looking at drugs that already have FDA approval to see what other conditions they might treat.

Amrie Grammer spent ten years working at the National Institutes of Health before she and a colleague founded AMPEL – a Charlotteville firm that studies drugs approved to treat one thing, to see if they might be effective against another.

“Lupus has been our focus for the last five years – there’s only been one drug approved in the last 60 years,” she says.

This disease occurs when a person’s immune system attacks their own tissues and organs. Inflammation caused by lupus can affect joints, skin and blood cells, the brain, heart, kidneys and lungs. So AMPEL began by studying the genetics of lupus – figuring out which genes cause what symptoms.

“The genes that are expressed in lupus nephritis are very different than the genes expressed in lupus skin for example,” Grammer explains.

Working with doctors nationwide, AMPEL compiled a sizable database and used computers to search for patterns that could help doctors more precisely diagnose and treat their lupus patients.

“We have the largest lupus gene expression database in the world with over 3,000 samples now and growing, and we have deep clinical data, knowing what medications the patients are on, do they have lupus nephritis? Do they have skin lupus? And to be able to find the right drug for the right patient at the right time,” says Grammer.

A medication called Stelara had been approved to treat other auto-immune disorders — Crohn’s disease and plaque psoriasis. AMPEL thought the drug might also help people with Lupus, so the manufacturer – Janssen Pharmaceuticals – partnered with the company to find out.

“So we did a trial with them, got positive results last November. Now physicians feel comfortable in writing a prescription for that drug.”

AMPEL also came up with a genetic test that gives doctors a better understanding of individual lupus patients and allows drug companies to select participants most likely to respond to a study medication.

“Up to half of the patients that are being enrolled in trials did not have the protein that the drug inhibited. We assist pharmaceutical companies in enrolling patients that have the opportunity to respond to their drug. That significantly increases the chance that the trial will work. Our hope is it will decrease trial failure, increase the number of drugs that are coming to patients and, at the end of the day, big pharma also benefits, because it will cut their losses and their costs for R&D.”

Grammer says the company will keep working to find custom treatments for lupus, but it also hopes to make progress in fighting Alzheimer’s Disease.

“Alzheimer’s, like lupus, is a very complicated disease that’s genetically based. + In addition, there are quite a few FDA approved drugs that are out there in the neurological space, and we have preliminary data that some of those could be very effective for Alzheimer’s patients.”

All of this is done without touching a test tube or a laboratory animal, through a kind of research known as in silico– referring to the silicon chip found in computers. By combining genetics and big data AMPEL offers a whole new way drug companies can provide new medications and doctors can prescribe them.


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