Newly Presented Preclinical Data Shows Potential for Novel Gene Therapy To Treat Opioid Use Disorder – Second Year of NID/ANIH Grant Support Will Enable Further Exploration of Hypothesis
- Dr. Barbara Waszczak, Professor of Pharmacology at Northeastern University's Bouvé College of Health Sciences, and collaborators from Cummings School of Veterinary Medicine at Tufts University and Tufts University School of Medicine, are Principal Investigators.
- Copernicus Therapeutics, Inc. supplied the DNA nanoparticles that are the basis for the gene therapy approach used in the study.
- Northeastern University and Copernicus Therapeutics, Inc. receive second year of Phase 1 funding through a grant from the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH) through the Helping to End Addiction Long-term Initiative, or NIH HEAL Initiative to develop a gene therapy approach for treating opioid use disorder.
- With these data and further proof-of-concept studies underway, Copernicus will be commercializing its technology for additional diseases, including rare targets, with the goal of advancing gene therapy treatment.
CLEVELAND, OH / ACCESSWIRE / May 18, 2023 / Copernicus Therapeutics, Inc., today announced preliminary data, in collaboration with Northeastern University and Tufts University, that demonstrated plasmid GDNF DNA nanoparticles (pGDNF NPs) delivered intranasally bypass the blood-brain barrier to allow gene expression in brain cells, which ultimately results in a reduction in drug-seeking behaviors in rats. With further study, this novel approach, which utilizes proprietary DNA nanoparticles as the basis for a non-viral technology platform, is expected to restore normal functioning of the brain's reward system, reduce drug craving, and thus enable people in recovery from opioid use disorder to avoid relapse. Intranasal testing of these pGDNF NPs in primates is currently underway with results expected in early 2023.
"These data take us one step closer to being able to show that we can deliver therapeutic doses of gene therapy into the brain in a non-invasive way," said Dr. Barbara Waszczak, Professor of Pharmacology at Northeastern University's Bouvé College of Health Sciences, a Principal Investigator in the study. "We are encouraged that this approach could be part of a solution to this significant global health crisis and look forward to further study in this area and in other brain disorders."
Opioid use disorder is a serious public health issue in the United States and throughout the world with 5.6 million people in the United States experiencing opioid use disorder in 2021 according to the Substance Abuse and Mental Health Services Administration. The Centers for Disease Control and Prevention estimates that the total "economic burden" of opioid misuse in the United States was $1.02 trillion in 2017, with Forbes updating that figure to $1.3 trillion in February 2022. Although opioid use disorder prevalence has increased significantly in the past decade, few seek treatment or maintain their recovery. "These data further illustrate a path to a new gene therapy approach to treat opioid use disorder, as well as a template for exploring gene therapy approaches for other substance use disorders, and the cellular and molecular mechanisms that are involved in this novel and promising approach," said Mark J. Cooper, MD, Senior VP of Science and Medical Affairs at Copernicus. "We are pleased to continue to work with these talented academic scientists and believe this is a critical proof point toward moving our patented nanoparticle platform forward into human trials for opioid use disorder as well as other indications in the brain, lung and eye."
About the Study
One of the neuroadaptive changes associated with repeated drug use is a neural dopamine (DA) deficiency in the brain's reward circuit, which has been long recognized as a significant underlying mechanism of drug craving and seeking behaviors. Targeting this mechanism, a gene therapy for OUD utilizing glial cell line-derived neurotrophic factor (GDNF) was designed based on the hypothesis that GDNF treatment can correct the DA deficiency induced by chronic opioid use, thereby attenuating drug-seeking behaviors during abstinence.
For the current application, plasmid GDNF DNA nanoparticles were delivered intranasally, bypassing the blood-brain barrier to allow GDNF transcription and translation in dopamine-containing brain regions. The main goal was to determine whether intranasal infusion of pGDNF NPs could reduce drug-seeking behavior in a robust animal model of opioid addiction. The newly presented results confirmed their ability to significantly reduce relapse-like behavior in both male and female rats. A second aim, currently underway, will assess whether these effects are associated with correction of the neural DA deficiency following chronic opioid self-administration.
The study was recently presented at the 2022 Society for Neuroscience annual congress by co-Principal Investigators, Dr. Barbara Waszczak, Professor of Pharmacology in the Bouvé College Department of Pharmaceutical Sciences at Northeastern University, and Drs. Elizabeth Byrnes, Professor of Comparative Pathobiology at Cummings School of Veterinary Medicine at Tufts University, and Emmanuel Pothos, Associate Professor of Immunology at Tufts University School of Medicine.
About the NIDA/NIH Grant
The Phase 1 segment of the NIDA grant, through the Helping to End Addiction Long-term Initiative, or NIH HEAL Initiative, awarded $6.7 million to Northeastern University, to fund the first two years of this research project. Upon successful completion of Phase 1, Northeastern University will be eligible to receive an additional approximate $7.5 million in funding from NIDA to further advance this therapeutic approach towards human clinical trials.
About Copernicus Therapeutics, Inc.
Copernicus Therapeutics, Inc. is a U.S.-based privately held clinical stage company, with a non-viral, nucleic acid gene therapy platform technology enabled by patent protected nanoparticles, which deliver gene therapy directly to target sites to address diseases of the brain, eye, and lung.
Pilot data research was done with the support of awards from the Brain Research Foundation and Tufts Clinical Translational Science Institute.
This research is supported by the National Institute on Drug Abuse of the National Institutes of Health, through the NIH HEAL Initiative, under award number 1UG3DA050942-01A1. The content presented in this release is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
The Helping to End Addiction Long-term® Initiative, or NIH HEAL Initiative®, is an aggressive, trans-NIH effort to speed scientific solutions to stem the national opioid public health crisis. Launched in April 2018, the initiative is focused on improving prevention and treatment strategies for opioid misuse and addiction, and enhancing pain management. For more information, visit: https://heal.nih.gov.
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For More Information, contact:
Robert C. Moen, MD. PhD
President & CEO
Copernicus Therapeutics, Inc
(216) 231-0227 ext 026
SOURCE: Copernicus Therapeutics
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