Groundbreaking advancements in the experimental area of gene therapy have the potential to revolutionalize the landscape of addiction treatment as well as a broad range of other diseases.
The first gene therapy was approved in the EU in 2012, and in the last few years development of gene therapies has accelerated dramatically. Gene therapy research institutes can be found all over the world, with studies underway in the United States and beyond testing a new form of therapy that can potentially treat genetic conditions, cancer, and HIV/AIDS.
What is gene therapy?
In its simplest sense, gene therapy involves the introduction, removal or change in the content of a person’s genetic code with the intended goal of treating or curing a disease.It has the potential to unlock the underlying cause of both genetic and acquired diseases by inserting a gene into a patient’s cells rather than using drugs or surgery.
Genes contain DNA, the code that controls the fundamental form and function of the body. Genes that don’t aren’t working properly may cause disease.
How does gene therapy work?
Researchers are currently testing several approaches, mostly on rodents, including:
- Replacing a mutated gene that has caused disease with a healthy copy of the gene
- Inactivating a mutating gene that is functioning improperly
- Introducing a new gene to help fight a disease
Gene therapy has already been shown to reduce use of cocaine by up to 75 percent in rodents trained to self-administer it through increasing the brain level of receptors for dopamine. Cocaine, for example, builds up lots of the pleasure-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 be reducing levels of disease-causing versions of a protein, increasing the production of disease-fighting proteins, or creating new proteins altogether can be an incredibly useful tool in treating addiction and changing the brain’s desire to consume while preventing relapse.
The protein BChE, for example, is produced naturally by the liver and breaks down compounds called esters, which allow muscles to relax while resetting activated neurons. It can disrupt nerve functions and break down toxins like cocaine. Advancements in gene therapy are speeding up the protein’s structure to be faster and more efficient in treating addictions and overdoses, with some modifications of the protein able to inactivate cocaine within seconds of it showing up in one’s bloodstream.
ClinicalTrials.gov lists over 1000 varying types of gene therapy available in clinical trials.
Since any gene in the human genome can be targeted, the potential for new therapies is huge. Gene therapy can help someone quit a drug by literally decreasing the drive to use it by altering its euphoric effects. Even years into recovery, if a person slips up and does take the drug again, they wouldn’t feel its’ effects, completely changing their relationship with not only the drug but themselves.
The practice is still considered experimental for humans, but so far there seem to be no negative side effects. Rodent models of addiction are generally accurate but not perfect, and researchers want to make sure they’re completely confident that these modified proteins wouldn’t have unexpected effects on the human brain. Tests with BChE have been safe and effective from rodents to nonhuman primates, so it isn’t impossible to say they make their way to human medicine in the coming decades.
Some barriers that stand in the way of gene therapy becoming a preferred mode of treatment include targeting the correct cells, potentially unwanted immune system reactions, and infection caused by the virus.
The FDA, for its part, has already begun to approve a number of gene therapy products and devices, with the Center for Biologics Evaluation and Research (CBER) providing regulatory oversight of clinical studies and scientific guidance to researchers and manufacturers. Clinical trials of the therapy have already begun to show success in treating diseases such as severe combined immune deficiency, hemophilia, blindness caused by retinitis pigmentosa, and leukemia. Together with the National Institutes of Health, the FDA closely monitors the clinical trials underway to the safety of the process.
All in all, the tool itself has countless applications and we’re just getting started with discovering alternative ways to fight addiction and save lives.
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