Several North Carolina firms are at the forefront of developing new treatments for the Ebola virus, but they face challenges in testing and delivering them, said executives from Chimerix, Quintiles, and GlaxoSmithKline on Monday at the NC Biotechnology Center.

Speaking on the panel discussion, “North Carolina Companies at the Forefront of Ebola Vaccines and Therapies” during a half-day briefing by experts on how the state is facing the Ebola crisis, Jeffrey Spaeder, M.D., chief medical and scientific officer at Quintiles summed up the crux of the problem.

“How do you bring a therapy to people who need it as quickly as possible, yet as safely as possible? It’s a challenge. Nothing is worse than getting a false signal. If you don’t do a robust analysis, you can expose patients to adverse events without benefits and make them think they’re getting care when they’re not. You just can’t make mistakes in the process.”

Normally, it can take from 10 to 12 years to develop and test new drugs and vaccines. Quintiles has argued for speeding up clinical trial timelines to streamline this process recently (Read WRAL TechWire’s report..

M. Michelle Berrey, M.D., CEO of Durham-based Chimerix, which reached an agreement with the U.S. Food and Drug Administration (FDA) for emergency use of its oral antiviral drug, Brincidofovir, has had encouraging results, but it hasn’t been used in randomized, controlled trials against Ebola. The drug has been tested in Phase III trials for virus infections in immune-suppressed individuals, so it already has a good safety profile.

Evaluating its effects in Ebola patients receiving it so far is difficult, she noted, because patients also received plasma from other patients who survived the disease. Another problem is determining if the drug is more effective if given at an early point in an Ebola infection. That can be another problem in West African nations where people are often reluctant to go to clinics early because the over-taxed health care systems there take days to test patients and get results.

Recent WRAL TechWire Coverage of Ebola:

  • North Carolina at forefront of Ebola fight
  • Lost amid layoff news: GSK’s Ebola vaccine progress 
  • Bill gates gives financial boost to Chimerix in Ebola fight
  • Quintiles calls for faster clinical trials process

“As we get some of these interventions in place, we’ll encourage folks to come to the clinics earlier. The earlier you can intervene, the better you can suppress the infection so patients recover more quickly.”

“Are we having a significant impact on mortality? We do have to do comparative trials,” Berrey said.

She said the company is having ongoing conversations with the Gates Foundation on conducting trials in Liberia and elsewhere. “We should have more information in coming months on its effectiveness against Ebola.”

What can we learn for the future?

Public-private partnerships created because of the Ebola crisis suggest the process can be hastened, at least in emergencies. GSK, which has a large footprint in developing vaccines for potential pandemics and in bio-security products, acquired an Ebola vaccine candidate when it bought Okairos in 2013.

“In August, when the Ebola epidemic became an international emergency, the World Health Organization asked us to begin trials, and through our collaboration with the government, we’ve moved into trials quickly while ensuring all safety and ethical protocols are in place,” said Donna Altenpohl, vice president U.S. policy with GSK. “This demonstrates the power of what can happen when public-private groups work together.”

Altenpohl said GSK plans to start three more Phase I trials, which test for safety and dosing, in “the next couple of weeks” and is getting ready to go into Phase II trials. “If those produce the response we want we’ll concurrently develop Phase III trials.” She added that while the vaccine is “very promising, we would not be progressing this far without this level of collaboration.”

But the whole process offers a chance to identify policies and procedures that can be applied to multiple diseases,” she said.

“The biggest opportunity here is to take this dire situation and ask, ‘what can we learn from what we’re going through right now?’ We don’t always want to be in a just-in-time situation, but rather, have a more sustainable, predictable platform. How can we work with the government to have more sustainable policies in place?”

Tremendous advantage in the Triangle

If it comes out of trials producing an effective immune response to the Ebola virus, the vaccine would go to front line workers first. Altenpohl said the Gates Foundation is helping with manufacturing of the vaccine and the need to keep it in very cold storage.

Answering a question from the audience, she said GSK does want some commercial opportunity from the therapies it develops, whether from the government or otherwise. In this case, however, she added, “We’ll make sure pricing is no barrier.”

Spaeder noted that one thing that should come out of facing this health care emergency is “Innovation not just in products, but the design of clinical trials as well. We have a tremendous advantage here in the Triangle, working closely with Duke and UNC.”

Panel moderator Chris LeGrand, of the GRM Futures Group, summed things up saying, “This emergency shines a light on the tremendous capabilities we have here in the triangle and NC with the public sector fueling the private in what it does best.”