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Therapeutics for Rare and Neglected Diseases

CMX001 for Treatment of Neonatal Herpes Simplex Virus

In the United States, approximately 1,500 babies are born each year infected with herpes simplex virus (HSV). In newborns, HSV infection, or neonatal herpes, can be life threatening. Untreated, the virus can spread throughout the body, including the brain and spinal fluid, causing seizures and even death. At least 40 percent of babies with virus in the central nervous system (CNS) are left with significant, lifelong neurologic damage. The purpose of this project is to develop a therapeutic regimen to clear HSV from the CNS and avoid this neurologic damage.

Scientific Synopsis

Despite significant advances in the treatment of neonatal herpes over the past 30 years, approximately 20 percent of babies with disseminated HSV disease die, and at least 40 percent of babies with CNS disease are left with significant, lifelong neurologic damage. Existing antiviral therapeutic options, such as acyclovir, have been maximized in terms of both amount and duration of administration. To accomplish the next series of therapeutic advances, new drugs with improved penetration into the CNS are required. The recent development of CMX001, the lipophilic derivative of cidofovir, provides for the first time a molecule with significant anti-herpetic activity that penetrates the CNS. Before a comparative trial of acyclovir + CMX001 versus acyclovir + placebo can be undertaken in neonatal HSV disease, the correct dose of CMX001 in neonates must be determined. In this project, a multi-institutional team of investigators, known as the Collaborative Antiviral Study Group, will define the pharmacokinetics and concentration response relationship of CMX001 in neonates with HSV CNS disease. TRND researchers will support the pre-clinical development studies of the pro-drug.

Lead Collaborator

University of Alabama at Birmingham
David Kimberlin, M.D.

Public Health Impact

 Despite significant advances in the treatment of neonatal HSV disease over the past 30 years, approximately 20 percent of babies with disseminated HSV disease die, and at least 40 percent of babies with CNS disease are left with significant, lifelong neurologic damage. This project seeks to develop a therapeutic regimen to clear HSV from the CNS and avoid this neurologic damage.

Outcomes

This project was carried out in conjunction with NIH’s National Institute of Allergy and Infectious Diseases (NIAID). TRND supported pre-clinical studies needed to enable successful filing of an Investigational New Drug (IND) application to the Food and Drug Administration (FDA). The FDA has cleared the IND, and NIAID will provide support for the clinical studies in collaboration with Dr. Kimberlin at the University of Alabama at Birmingham. This TRND project is complete.

Last updated: 09-06-2017
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