How Gene Therapy Could Revolutionize Addiction Treatment

How Gene Therapy Could Revolutionize Addiction Treatment

This episode of SciShow is sponsored by the
American Society of Gene and Cell Therapy. [♪ INTRO] If you’ve heard anything about gene therapy,
it’s probably how it can treat certain illnesses and disorders. But while that’s totally true — and amazing
— that is not the only way it can be used. Recently, scientists have been paving the
way for gene therapy to be used as a treatment for drug addiction and overdose. Most of these studies have specifically focused
on cocaine, and some of the more promising ones suggest that gene therapy might not just
be able to treat overdoses… it might also be able to help prevent relapse. This kind of treatment is still very early
in the development cycle, but if we can show it safely works in humans, it might just revolutionize
the way we treat addiction. Cocaine is one of the more common illegal
drugs out there, and it works by causing large amounts of dopamine to build up in the brain. Dopamine is one of the molecules involved
in the body’s reward system, so having a ton of it floating around causes feelings
like euphoria. Over time, though, the brain can get used
to having all that extra dopamine, which makes the drug hard to permanently quit. It also means that people need to take more
and more of the drug to get the same high. But in large doses, cocaine can be fatal. It can cause irregular heart rhythms, seizures,
trouble breathing, or strokes. And in 2017, there were an estimated 14,000
overdoses in the U.S. involving the drug. There aren’t any great treatments for this
addiction, either, although there are plenty in the works. Many involve changing the brain’s response
to chemicals like dopamine. But gene therapy treatments go even deeper. They typically center around a protein called
BChE. It’s made naturally by the liver and breaks
down compounds called esters, which sort of “reset” activated neurons and allow muscles
to relax. It can be also used to protect people against
poisons that disrupt nerve functions, like nerve gas. But it can break down cocaine, too. The problem is, BChE works way too slowly
to treat addictions and overdoses, allowing almost all of a typical dose of cocaine to
make it to the brain’s reward centers. So over the years, scientists have been working
to modify the protein’s structure so that it’s even faster and more efficient. Using methods like computer simulations, they’ve
been pretty successful so far, and some of their modified proteins can inactivate cocaine
within seconds of it showing up in the blood. But there’s still the whole issue of how
to deliver the new protein. And that’s where gene therapy really comes
in. In this method, scientists specially engineer
a virus called a vector, taking out the parts that cause disease and inserting information
that tells cells to start making modified BChE. So far, it hasn’t been tested in humans
— most of the work has been in rodent models — but there have been multiple experiments
in the last decade, and they’ve generally been really successful. Like, not only does this treatment seem to
work, but from what scientists can tell, it also doesn’t seem to have any negative side
effects. One of the more significant papers in this
field comes from the journal Vaccine in 2014. In the study, there were two groups of mice. One group was injected with trillions of vector
particles that told their bodies to produce an enhanced version of BChE. The other group was either injected with a
harmless saline solution or a vector without BChE as a control. Based on the team’s special mouse-monitoring
system, which looked at things like motor activity and oxygen consumption, the researchers
found that the mice didn’t seem to function any different with large amounts of modified
BChE in their bodies. But the real test is what happened when those
animals were injected with cocaine. The scientists gave them what would have easily
been a lethal dose of the drug, and waited to see what happened. And they found that… like, nothing happened. The mice that received the modified BChE vector
didn’t show any changes in activity, which likely meant the protein broke down drug molecules
too quickly for them to have any effect. What’s significant about this treatment
isn’t just that it can protect against overdoses, either. It may also be able to help people quit the
drug, by preventing someone from feeling its euphoric effects and, therefore, decreasing
their drive to use it. In the same 2014 experiment, the scientists
found that the levels of modified BChE remained high in the mice’s blood for 8 to 16 months
after the initial vector injection. If those results can be translated to humans,
it means that even if someone recovering from addiction does slip up and take the drug,
they wouldn’t feel its effects — maybe even years after the treatment. And that would likely help them quit for good. Right now, it’s definitely an “if”,
though. Rodent models of addiction are generally pretty
accurate, but they’re not perfect. And researchers want to make sure they’re
totally confident that these modified proteins wouldn’t have unexpected effects on the
brain. So for now, they’ll keep running tests on
animal models to see what happens. The good news is, experiments with BChE have
been effective and safe in everything from rodents to nonhuman primates. So it’s not impossible that these treatments
will make their way into human medicine at some point. Also, although it has been a major research
area, gene therapy isn’t just for cocaine. Researchers have been studying how to use
this technique to treat other addictions, like alcohol or meth. And similar treatments could even be used
to fight opioid addictions — although that problem is a bit more complicated, because
there are times when people do need strong painkillers. Also, it’s tricky because not all drugs
are broken down by BChE, so scientists have to develop different methods for each new
addiction. For example, a 2017 paper actually used gene
therapy to enhance the negative effects of drinking, like dizziness, in order to drive
mice away from it. Still, even though the exact way of using
gene therapy might change from case to case, the tool itself is valuable and has a ton
of applications. And if we learn it’s safe to start treating
addiction this way in humans, it could have the potential to save tens of thousands of
lives. If you’re interested in learning more about
developments in this field, or other types of gene therapy and how they work, you can
check out the new patient education portal from the American Society of Gene and Cell
Therapy. It’s a really comprehensive resource full
of clear explanations and easy-to-follow summaries — and it’s all completely free. And to no one’s surprise, we here at SciShow
are big fans of free online education around here, especially when the content is as interesting
as this. To check the portal out for yourself, just
head over to, or just follow the link in the description below. [♪ OUTRO]


  1. That is nice and all, but would these people during that time be also resistant to anaesthesia like lidocaine, benzocaine etc.? Since these are very closely related to cocaine in chemical structure and are even used to treat arythmia. Also what about other medication? Would it also break down quicker other drugs that people are using like for example to treat hypertensia?

  2. The reason we are loosing the war on drugs is because of all of the people fighting on the Drug's side. The infrastructure that distributes hard drugs is built on weed. If we weren't calling people who are willing to sacrifice the safety and security of everyone who depends on them to get high on something that is supposedly non-addicting "a victim", we would be a lot more able to fight hard drug and opioid addiction.

  3. the only problem with this is addiction is psycological and physical. also addicts can and will find a way around these "cures" and also the obligatory where does this stop

  4. People take drugs TO get high. You may as well be pushing a glass of water as a cure for drug addiction. If you have ever lived in an area surrounded by junkies, then you'd understand. The only real cure is death. It's not going to change anything just by treating the symptoms, you have to treat the cause. People are self-medicating to get through their crappy lives, an overdose is the best thing that can happen to these people. The only good junkie is a dead one.

  5. Bunno… um… seriously… you’re not being very sophisticated about addiction… lumping cocaine use in with serious addictions like opioids or meth makes me realize YOU have no idea what your talking about.

  6. You know what would be even crazier/better? if you made the vector/carrier actually contagious because junkies hangout with other junkies and you would probably want to give them short tails of junk DNA with a vital function near the end so they can only do something like 100 cell duplications but then all their friends /dealers would catch it and become non addicted. When I was little I used to think about putting something like that into a bee venom or scorpion venom so then you could just grow the scorpions and use them as live hypodermic needles. that would be messed up to do to the animals though…

  7. Universal soldier comes to mind, if this stuff breaks down chemicals that quickly we need to send Marty McFly back to WW1 and prevent mustard gas attacks doing any damage.

  8. Wait a minute, if cocaine is not working anymore they will just take another drug. This hasn't solved anything.

  9. i vaguely remember hearing about how they were growing lungs and other test animal organs in the labs over a decade ago and other stories about other potential medical breakthrough languishing in perpetual laboratory research. the only way this or any other "research breakthrough" would ever see the light of day is if the company in charge can secure absolute control over profitability and if it dosen't affect other profitable symptom treating industry. the modern medical industry isn't interested in healthy people. the real money is in sick people and that is well…..SICK

  10. Man, we are close to recreating the Les Enfant Terribles project and indirectly causing the sickest fist fight in military history.

  11. This would't work in an overdose though, you need to allow time for the body to produce the modified protein, that isn't really why I'm posting though, the reason people get addicted, as you stated, is that they feel low without the increased levels of dopamine, hence they seek out substances and activities that result in a boost, if we inject these people so that they can't gain the effect of a dopamine boost from cocaine, what will they do instead? Are we going to end up with them jumping out of planes to get that high or will they become suicidal because they can't get that high? It's all good and well fixing the symptoms but we really need to address the cause too and I would have thought that is how we currently treat this.

  12. I wouldn't be too sure, whether this can deliver satisfying results in humans. Wouldn't it be quite easy to use a drug that uses a different mechanism? That might be especially critical when we talk drugs that are highly similar to substances produced by the body itself.

  13. Can they work on food addiction next? There's nothing worse than being addicted to something you need to live.

    Also, I'm really tired of the war on opiate drugs because along the way, states are punishing people who are responsible and genuinely need regular opiate painkillers to treat chronic pain conditions that, because they're CHRONIC, will never just go away. My mother is one such person, with RA, fibromyalgia, and multiple other forms of arthritis. And no, don't tell me there's other forms of treatment. My mom's worked her way down the list, had them damage her heart, liver, kidneys, and pancreas, and cause allergic reactions that nearly turned into Steven Johnson Syndrome. She has permanent heart and kidney damage, nearly died from liver ischemia, had heavy metal poisoning from one treatment, has diabetes from pancreas damage, and had her skin all slough off. All because she's been on treatment for chronic pain for over forty years. So let her have her 2-3 opiate pills a day when she uses them "as needed" and has for years. She's never run out early.

    P.S. Yes, she tried some natural methods, like turmeric. Turmeric is a blood thinner and she's on a blood thinner from the heart issue causing blood clots.

  14. Why don’t you make a video on the alternative theories about the Universe on
    YouTube this would be interesting and good for the YouTube community!!!

  15. Increase negative effects? Immunity to drugs? Immunity to poisons? Carcinogens? Sounds like weaponization or more designer drugs. Drops tons of chemicals on a country, all troops immune thru gene therapy. Make populace more sensitive to drugs? Opium war 2.0. Want a drug that has all the good and none bad effects of several drugs? Make your dna get you super high. Region lock… race lock drugs and poisons… could be used to digest previous inedible foods.. think cellulose. Man… the future is crazy

  16. The problem here is that it’s rare anyone overdoses on cocaine, unless it’s laced with fentanyl. This is not going to be a substitute for psychotherapy

  17. I'm sorry, but that doesn't sound like a proper addiction treatment, it sounds more like an extreme form of forced abstinence!

    I mean, wouldn't blocking the drug's effects like that cause an addict to go through the worst withdrawal possible?
    BChE also affects a whole range of compounds. That may well include genuine prescription drugs you would need later in life, now rendered ineffective! And since it's gene therapy, it's permanent!
    Not to mention the risk of that altered gene being passed to your children, to whom it would be of no benefit, and to their potential detriment.

    I'm all for gene therapy when it comes to curing genetic diseases, maybe even improving our basic biology, but that kind of use seems clearly into the unethical territory to me!

    That really sounds like something only an Orwellian government would force on people…

  18. Could it stabilize chemical imbalances, like ebbing a pornography or sex addiction? Could we pump this thing into the sex offender list?

  19. Why not use it to counter the side effects, but keep the buzz? getting wasted without hurting your body seems much better to me as beeing sober my whole life

  20. this new research worriengly makes me worried that the government might someday modify people against their will, so they cant have fun with drugs XD

  21. Visit for more free resources on gene and cell therapy, including progress on a number of promising treatments already in development.

  22. I can think of a side effect of this. you're not capable of getting high anymore if you wanted to. Sure, cocaine is illegal, and addiction is dangerous, but if cocaine was legal, the addiction wouldn't be a problem, only overdose, and if it was legal, you could fund research to prevent overdoses without preventing addiction. Caffeine is addictive but you can start consuming it as a child with no restrictions what so ever. It's morally wrong to prevent someone from deciding to have an experience that you personally disagree with.

  23. Considering the mechanisms of action and the manifestation of the experience that results from any drug use are still obscure to bioscientists, I highly doubt that there aren't any negative side-effects for humans or rodents…

    …Lots of genetic code is interactive too, you might not even know where to look for side-effects…

    This is a really stupid idea and when you figure how that money could do such better work distributed amongst social workers and actual therapists in terms of helping people, no question, this is a huge waste of time and money.

  24. Cocaine binds to the same receptors as dopamine? How subtle is the BChE at removing cocaine vs. dopamine? Would higher BChE levels cause susceptibility to depression?
    It's not enough to remove the addiction. It is imperative that a person's mental health be left intact. A depressed or bipolar person might easily try self – medication and trade one addiction for another.

    You really have to think of all cases when it comes to the brain. So many things could miss the mark completely.

  25. I wonder if there is any treatment on this with regards to more psychological addictions, like behavioral therapies and such. Addictions work on more than just a chemical level (like phones and video games can be addicting)

  26. This is amazing, and a beautiful partnership with asgct! Thank you for the great news and content y'all keep us in. Much love from a weird 🙂

  27. Let's be clear: if you make it harder for someone to 'achieve' a cocaine high, they will simply buy a lot more. The problem doesn't go away.

  28. What we hope happens: This works on humans with no side-effects – thousands of lives saved from addiction!
    What will actually happen: Pharma sells it to rich people and stock traders for risk free drug trips.

  29. Is this reversible? I don't like the idea of permanently taking away someone's agency. What happens when parents force their kids to get this?

  30. "likely"
    Oh dear. I guess I'll wait for the real proof and peer-review before I count this as a win for science.

  31. The treatment for alcohol addiction has been mostly stagnant since the 1930's. Blind allegiance to the 12 Step program has obstructed medical research into the field of addiction.

  32. Cannabis already regulates the part of the brain that deals with the values associated with things. In studies, Heroin addicts were able to reduce the amount of Heroin they used by 40% per week. It's funny how police always say "Cannabis is a gateway drug" because they always found it at the houses of drug addicts and yet the reason it was there is that it is a substance that helps them restore some sanity and reduce the addiction. And for the record, the real enemy of Cannabis is Alcohol, Opiates and Big Tobacco – because those substances are among the leading causes of death in a majority of major cities and Cannabis frees people from the addiction to those substances by down-regulating the overly high values associated with consuming those addictive and harmful substances. So actually, I kind of laughed when I read the title because Cannabis has not been recognized as the revolutionary choice for addiction even though there is nothing more effective at treating addiction than Cannabis. And no, Cannabis is not addictive as it's half-life is upto 59 hours. To put that in Context, Cocaine has a half life of 30 minutes and addiction is determined by how short the half-life is. Alcohol is 2 hours. Digesting food is between ~30 to ~46 hours or so and technically you are more likely to become addicted to digesting food than you are Cannabis – if you want to go down that path of saying Cannabis is biochemically addictive. It can be behaviorally addictive but that is like saying that a diabetic is addicted to insulin because they have to inject it several times a day so they don't die. Likewise, people with disconnected brain components (Such as Asperger's, ADHD/ADD and the autism spectrum) will find relief with Cannabis and it's no coincidence that scientists claim that they believe they can reverse all the negative symptoms of Autism through science based on Cannabis – as it reconnects the parts of the brain that are disconnected. Michio Kaku claims that this is short-circuiting the brain however for these people, a short-circuit is more function-able than a disconnected brain.

  33. Z-DNA alert! The DNA in the thumbnail seems to be the wrong way around (left-handed instead of the natural right-handed version)

  34. As someone who has suffered from addiction most of my life and has many ,many family and friends who are addicted I hope this works.

  35. Maybe I'm just being pessimistic, but wouldn't the most likely response from a patient be to switch to another drug out of frustration and need for that euphoric feeling ?

  36. Pfft.. I love my drugs and if I had a 'dope of meme' I'd
    Be high as fudge right now
    .. so… yeah don't take that away people act like "filling the hole is a bad thing" quite the contrary it feels REALLY good. Lol but Drugs are bad Mkay

  37. Wait, so the researchers had an experimental group and control group of mice… did they give a lethal overdose to the control mice?? I know it's for science but that's pretty upsetting :'(

  38. The only thing that scares me about this is that one day, if it’s seen as having few/no side effects, it might become illegal not to be injected with treatments like this by a certain age as a way to stop ANYONE from potentially wanting recreational chemicals of any kind.

  39. Didn't cocaine used to be less refined and what about the Adverse Childhood Experiences that caused the problem

  40. Whatever happened to the drug that was meant to treat alcoholism but ended up isolating the h.i.v virus from cells instead?

  41. As a doctor, I'm very excited with future therapy. However, how would gene therapy be received when we still have anti-vaxxers? Vaccines use harmless versions of pathogens while current gene therapy uses modified, live viruses.

  42. Instead of modifying your genes so you cant get high, they should modify your genes so you dont get withdrawal.

  43. uh it possible in my thinking to cause the BcHE to turn into a prion by failing to create the intended results.

  44. When are we going to make gene editing do something useful like making an army of Captain America-like humans. Not affected by common Chemical Warfare agents, better strategic and battlefield planning, built in PPAR delta production boosts, etc.

    and we're waiting for life extension when?

  45. Looks like cruel and unusual punishment that could have side effects like lack of will, depression, Parkinson's and psychosis.

  46. Helps prevent relapse? I was hoping for treatment that allows you to use recreationally, yet you’re able to walk away. 🙁

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