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NIH Funding Nanoparticle Gene Therapy for Opioid Disorder

DNA puzzle

(Arek Socha, Pixabay)

1 Apr. 2022. National Institutes of Health is funding research on an experimental gene therapy delivered with nanoparticles to treat opioid use disorder. The project is lead by researchers at Northeastern University in Boston, adapting a nanoparticle delivery technology developed by Copernicus Therapeutics Inc., a biotechnology company in Cleveland, Ohio.

The opioid epidemic continues to plague the U.S., resulting in part from opioid-based drugs still prescribed for pain that in many cases lead to abuse or dependence. Abuse of opioid pain drugs in the U.S. continues at rates reaching emergency levels, along with heroin and fentanyl sold on the street. Overdose deaths from opioids, according to National Institute on Drug Abuse, reached nearly 50,000 in 2019, with the economic burden from abuse of prescription pain drugs totaling $78.5 billion a year.

People with opioid use disorder and their physicians have few tools to deal directly with the problem of dependence on these drugs. Current medications, say the researchers, do not address changes in the brain’s nerve cells that result in compulsive drug use. The Northeastern team, led by pharmacology professor Barbara Waszczak, seeks to develop therapeutics that target underlying neurologic causes of opioid use disorder, particularly dopamine and reward deficiencies, to reduce cravings and risks of relapse.

Waszczak and Northeastern colleagues Elizabeth Byrnes, professor of pathology in the university’s veterinary medicine school, and immunology professor Emmanuel Pothos, are studying use of DNA plasmids, circular nucleic acid particles, carrying genes that encourage healthy brain cell development, as a treatment for opioid use disorder. In this case, nanoscale DNA particles would deliver genes that code for glial cell line derived neurotrophic factors or GDNFs, proteins that support a range of human brain cells. And these nanoscale DNA particles would be inhaled through the nose, as a more direct delivery mechanism to the brain.

Alternative to adeno-associated viruses

Copernicus Therapeutics is part of the project team, offering a gene-therapy delivery technology based on nanoparticles, particularly as alternatives to adeno-associated viruses, the current workhorse for delivering gene therapies. Adeno-associated viruses or AAVs are benign, naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease, and may generate a mild immune response. Even with a mild response, says Copernicus, AAVs have limits, particularly for repeated dosages, and the company’s nanoparticle technology is designed for long-term treatments.

National Institute on Drug Abuse, part of NIH, awarded the Northeastern team $6.7 million for the first two years of the project that extends through August 2023. This exploratory phase aims to determine technical feasibility of the proposed technology, defined as reducing opioid cravings and dopamine deficiency in lab rats using DNA plasmids with genes encoded for GDNFs delivered through the nose. The researchers will also test the technology’s ability to produce two to three times more GDNFs in monkeys, with the team’s DNA plasmids given through the nose.

If the exploratory stage is successful, the project moves to a development stage, where the researchers plan to advance the technology to the point of an investigational new drug application or IND to the Food and Drug Administration, in effect a request to begin clinical trials.

Mark Cooper, senior vice-president at Copernicus Therapeutics says in a statement released through Cision that the company continues to work with “academic scientists in utilizing the DNA nanoparticles developed by Copernicus to treat serious brain disorders, such as opioid use disorder, and we believe this grant award enables the team to complete important steps necessary before this promising approach can be tested by Copernicus in human trials.”

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