Frequently Asked Questions
Thank you for your interest in our research. We continue to receive a considerable number of inquiries on the report we published in 2008 in the scientific journal Nature. This report received extensive press coverage, some of it misleading. We will briefly answer some of the most common questions that we have been asked, and particularly how our work relates to a new treatment for cold sores, caused by Herpes Simplex Virus 1 (HSV-1), or genital ulcers, caused by the related but distinct HSV-2.
1) What have you accomplished?
Our work provides insight into how HSV-1 establishes a life-long latent infection in the nerve cells of the face, and how it reactivates from latency to cause cold sores. We have also pioneered a potential treatment for HSV-1/HSV-2 based on direct cleavage of viral DNA genomes using the DNA editing enzyme CRISPR/Cas.
2) Have you developed a new treatment for cold sores?
The work we have performed provides a basis for the development of anti-HSV-1 and anti-HSV-2 treatments, based on CRISPR/Cas, that might be able to permanently clear these viruses from patients.
3) Are these treatments being used on people?
No, but we have initiated trials in HSV-1-infected mice to study the efficacy of this treatment in collaboration with Dr. David Bloom (UFL) and Editas Medicine, Inc.
4) When might this treatment reach the clinical trial stage in humans?
We anticipate several years of animal experiments in mice followed by approximately 1-2 years of toxicity studies in other animals, followed by small studies in healthy volunteers. This timeline is dependent on obtaining additional research funding, which we have not yet succeeded in doing. After that, assuming things go well, this drug might proceed to clinical trials in HSV-1 infected individuals. It is very possible that this treatment might fall out at any of these stages, due to lack of effectiveness or some unanticipated side effect.
5) How does this relate to HSV-2, which causes genital herpes?
Most of our work so far has been on HSV-1, the cold sore virus, but HSV-2 is quite closely related. We are endeavoring to see if the lessons we have learned in HSV-1 also apply to HSV-2. We hope to eventually start animal trials for an anti-HSV-2 drug, depending on the results with HSV-1 and our financial resources. Our initial data indicate that HSV-1 and HSV-2 may respond to distinct, but similar, treatments.
6) Would you be willing to accept a financial contribution to be used for your research focusing on HSV-1 and HSV-2?
Funding is a major constraint on our ability to advance the research, so I am therefore pleased to tell you that the National Institutes of Health has recently awarded a research grant to Editas Medicine Inc, my laboratory and that of our collaborator Prof. David Bloom at the University of Florida. This is still far short of what we would need to go to clinical trials. We are hopeful that additional experimental evidence further validating our model for the regulation of HSV-1 and HSV-2 latency will persuade NIH to support our drug discovery efforts, leading to more animal tests and finally, if successful, a clinical trial. Nonetheless, it still looks like it will be several years before any clinical trials will become possible. Given that pre-clinical trials are very expensive—probably >$1,000,000 would be required—donations are greatly appreciated.
If you are interested in making a contribution, you may do so in one of the
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7) What can I use to treat chronic HSV-1-induced cold sores or
HSV-2-induced genital ulcers now?
All treatment decisions should be made in consultation with your physician, and no general guidelines will apply to everybody. While there are no drugs that attack latent herpes viruses, three closely related prescription drugs (Zovirax/acyclovir; Famvir/famciclovir and Valtrex/valacyclovir) are potent inhibitors of active virus replication that work quite well and that you might discuss with your physician.
We hope this information answers your questions and is helpful. Please be assured that we are continuing to work on the problem of developing novel HSV-1 and HSV-2 treatment approaches.