What is Gene Therapy and Can it Prevent Addiction?

Groundbreaking advancements in the experimentalarea of gene therapy have the potential to revolutionalize the landscape ofaddiction treatment as well as a broad range of other diseases.

The first gene therapy was approved in the EU in 2012, and in thelast few years development of gene therapies has accelerated dramatically. Genetherapy research institutes can be found all over the world, with studies underway in the United States andbeyond testing a new form of therapy that can potentially treat geneticconditions, cancer, and HIV/AIDS.

What is gene therapy?

In its simplest sense, gene therapy involves theintroduction, removal or change in the content of a person’s genetic code withthe intended goal of treating or curing a disease.It has the potential to unlock the underlying cause of bothgenetic and acquired diseases by inserting a gene into a patient’s cells ratherthan using drugs or surgery.

Genes contain DNA, the code that controls thefundamental form and function of the body. Genes that don’t aren’t workingproperly may cause disease.

How does gene therapy work?

Researchers are currently testing severalapproaches, mostly on rodents, including:

• Replacing a mutated gene that hascaused disease with a healthy copy of the gene
• Inactivating a mutating gene thatis functioning improperly
• Introducing a new gene to helpfight a disease

Gene therapy has already been shown to reduce use of cocaine by up to 75 percent inrodents trained to self-administer it through increasing the brain level ofreceptors for dopamine. Cocaine, for example, builds up lots of thepleasure-boosing chemical dopamine in the brain. The brain wants more dopamine;that is unless you alter the brain’s response to a chemical like dopamine.

Transferring the genetic material - whether it bereducing levels of disease-causing versions of a protein, increasing theproduction of disease-fighting proteins, or creating new proteins altogethercan be an incredibly useful tool in treating addiction and changing the brain’sdesire to consume while preventing relapse.

The protein BChE, for example, is producednaturally by the liver and breaks down compounds called esters, which allowmuscles to relax while resetting activated neurons. It can disrupt nervefunctions and break down toxins like cocaine. Advancements in gene therapy arespeeding up the protein’s structure to be faster and more efficient in treatingaddictions and overdoses, with some modifications of the protein able toinactivate cocaine within seconds of it showing up in one’s bloodstream.

ClinicalTrials.gov lists over 1000 varyingtypes of gene therapy available in clinical trials.

Since any gene in the human genome can betargeted, the potential for new therapies is huge. Gene therapy can helpsomeone quit a drug by literally decreasing the drive to use it by altering itseuphoric effects. Even years into recovery, if a person slips up and does takethe drug again, they wouldn’t feel its’ effects, completely changing theirrelationship with not only the drug but themselves.

The practice is still considered experimental forhumans, but so far there seem to be no negative side effects. Rodent models ofaddiction are generally accurate but not perfect, and researchers want to makesure they’re completely confident that these modified proteins wouldn't haveunexpected effects on the human brain. Tests with BChE have been safe andeffective from rodents to nonhuman primates, so it isn’t impossible to say theymake their way to human medicine in the coming decades.

Some barriers that stand in the way of genetherapy becoming a preferred mode of treatment include targeting the correctcells, potentially unwanted immune system reactions, and infection caused bythe virus.

The FDA, for its part, has already begun to approve a number of gene therapyproducts and devices, with the Center for Biologics Evaluation and Research(CBER) providing regulatory oversight of clinical studies and scientificguidance to researchers and manufacturers. Clinical trials of the therapy havealready begun to show success in treating diseases such as severe combinedimmune deficiency, hemophilia, blindness caused by retinitis pigmentosa, andleukemia. Together with the National Institutes of Health, the FDA closelymonitors the clinical trials underway to the safety of the process.

All in all, the tool itself has countlessapplications and we’re just getting started with discovering alternative waysto fight addiction and save lives.

Ethos Structured Sober Living is an all-malecommunity designed to empower our brothers in recoveryand beyond. We are happy to answer any and all questions you may have as aresource on this journey towards a happier and healthier life.

Contact us today at (323) 942-9996.