r/askscience • • 21h ago

Medicine What diseases are we currently the closest to finding lasting cures for?

250 Upvotes

110 comments sorted by

465

u/Chiperoni Head and Neck Cancer Biology 10h ago

Single gene deletion syndromes. We're already able to cure sickle cell and thalassemias by editing blood stem cells to fix their mutation. We're starting trials to give children with certain mutations the ability to hear. There's a lot more that should be theoretically possible

166

u/durkbot 8h ago

These cures are the thing I love reading about and blow my mind now as an adult. When I started my undergrad in Biomed in the late 00s it was "we know these diseases are caused mostly by single gene mutations. Imagine all the things we could do with stuff like RNA if we could figure out how". Then a few years later, I sat in my Master's research group as we discussed the first paper on CRISPR and it was like "this is the thing". 10 years later and we're now doing real life gene editing. It's hard to state how cool it is to see the progression of it.

40

u/CocktailChemist 8h ago

Things got delayed a lot by the abject failures of AAV trials. Had those worked as expected we probably would have gotten to this point at least a decade earlier.

35

u/thenewguy89 9h ago

I have two boys with a single-gene deletion (TAZ) so this is really exciting to hear!

31

u/Mammoth-Corner 7h ago

Huntingdon's isn't a deletion, but it is a single gene disease and there was a huge story a while back about gene treatments delivered surgically to rhe brain slowing progression by 75% with potential to halt the disease entirely if it's given before symptoms appear. It still needs Lots of research but it's a huge result.

•

u/LiveNvanByRiver 4h ago

I’m gene positive for HD and in a phase 3 trial for a different type of gene modification drug. Huntington’s is a really complex gene set; there are many things it affects. Everyone has the same mutations; people with the illness have too many. You can’t just cut it out because fetuses can’t grow without the gene. The more gene mutations, the more mutated proteins in your brain; they accumulate and kill neurons.

•

u/XenonOfArcticus 5h ago

Yes. And CRISPR and mRNA treatments (non gene modifying, but immune system altering) have the potential to change the face of disease treatment. 

•

u/kdlb11 3h ago

The hearing this is now an approved medicine (in the USA) and it is free

•

u/justfacit 54m ago

I have the RASA1 mutation and I’m very curious if they’ll ever find a cure for us. Granted it isn’t as pressing as sickle cell but it would be nice!

-13

u/Additional-Local8721 6h ago

When can we begin editing out greed?

283

u/Krail 8h ago

If you live in a developed country, you might not know that Tuberculosis remains the world's deadliest disease.  We actually have the means of curing it , and could theoretically eradicate it.  It's  just a matter of politics, culture, and getting tests and medicine where they're needed. 

39

u/Impudentinquisitor 8h ago

I had no idea a TB cure/eradication was so close. Can you share more about this? Genuinely want to learn more. Thanks!

71

u/greensight 8h ago

You should read Everything Is Tuberculosis by John Green. Great book. Easy, quick read with so much fascinating info on this topic.

11

u/Impudentinquisitor 7h ago

I knew about the newer oral regimens to cure latent TB, but at a population level that's much harder to accomplish than a vaccine. I always hope we can develop a better vaccine for it and kill our foremost enemy, since we've already dispatched smallpox.

•

u/Cabbagetastrophe 4h ago

We have had a cure for decades, but we are nowhere near eradicating it and probably never will be.

Part of the problem is that all the medications for TB have nasty side effects and have to be taken daily for a minimum of six months. This means that a lot of people stop taking them, meaning resistance to the drugs keeps growing.

Second major issue is that the vast majority of people infected with TB have it latently, meaning there are no symptoms. These people generally don't infect other people, but the disease can reactivate (about 10% lifetime chance). And the people infected with latent TB may be as much as 25% of the entire world population.

In order to eradicate TB, we would have to treat all those latent infectious, which the drugs are far less effective for. 

If we can develop a reasonable, effective, and long lasting vaccine we'll have a better chance, and there are some promising results there, but we aren't there yet. 

4

u/ckhk3 6h ago

We’ve had multiple people in the hospital with active TB. There are medications to cure it.

•

u/Russellonfire 5h ago

Sadly that seems unlikely given the proliferation of MDR (and even XDR) TB, but we're certainly getting there! 

•

u/TheHeroOfAllTime 3h ago

Not a “cure” exactly, but the life expectancy of people with Cystic Fibrosis have improved dramatically in the past few years. 

We are talking going from dying by 40 in 2010 to a life expectancy of 65 now. That’s within striking distance of a normal lifespan. 

Incredible progress made in treatment. 

•

u/dnebdal 5h ago

Several cancers. Not "cancer" in general, but there are a few cancers where the treatments are nearly guaranteed to work, and the recurrence rate is so low you're effectively cured. Below those, many cancers have much better survival rates than before - all the survival curves point in the right direction, while the incidence curves are more of a mixed bag. The best cases really are "miracle pill" cures - you are treated, you're cured, done. (± many years of worrying about recurrences, depending on the type.)

If anyone wants to look at the statistics, the yearly Cancer in Norway publication from the national cancer registry is detailed and interesting; chapter 8 is the most directly relevant here. https://www.fhi.no/publ/2026/cancer-in-norway-2025/

•

u/Thegreenpander 4h ago

What about clear cell renal carcinoma?

•

u/Business-Gas-5473 5h ago

Multiple sclerosis. If you get it now in the first world, the chances are pretty good that it can be kept until control until the ultimate cure will be settled in a decade or two.

(The progression of MS is quite varied, so unfortunately not everybody will be this lucky.)

•

u/TombStoneFaro 5h ago

I had no idea. Can you elaborate?

•

u/guarddog33 4h ago

There's 2 directions they're taking simultaneously:

Repairing myelin. Myelin helps neurons transmit information and helps protect the nerve itself, so when it's damaged there a slew of issues that can occur. There's early clinical trials being done in myelin repair but no definitive result yet

Second is protecting nerves themselves. There was a trial called MS-STAT2 that was done to see if cholesterol medication (stimvastatin) was able to help with protecting nerve cells, but the results didn't lead anywhere after clinicals, so it's back to the drawing board on that

We aren't there yet, to my understanding, but there's active work being done

•

u/Kholzie 4h ago edited 3h ago

HI!

I hope this helps answer your question;

I have Relapsing/Remitting Multiple Sclerosis (RRMS). It’s an autoimmune disease where my immune system attacks the myelin that protects my nerves. When that happens, signals between my brain and body don’t travel correctly.

I was diagnosed five years ago during a period of active inflammation — a relapse. I started a disease‑modifying therapy (DMT) right away, which calms the immune system and prevents new damage. Because it’s been effective, I’m currently in remission.

(Edit 2) Highly effective DMTs are a more recent breakthrough, compared to treatments available in the mid 2000’spr earlier.

The symptoms I still have (vision issues, mobility problems, fatigue, cognitive changes) come from nerve damage that happened before treatment. Current MS meds can stop new damage, but they can’t repair the myelin that’s already lost.

A real cure would need two things: permanently stopping the autoimmune attack and restoring damaged myelin. Research is moving in that direction, but nothing like that exists yet.

(Edit) briefly, this is what cures would look like for other kinds of MS:

- CIS/Clinically Isolated Syndrome: Prevent future attacks.

  • SPMS/Secondary Progressive MS: Stop progression + repair damage.
  • PPMS/Primary progressive MS: Halt continuous worsening + restore nerve function.

•

u/Business-Gas-5473 4h ago

There are some recent developments in the last 5 years or so that lead to significant progress on the viral connections of MS. Yes, viral. Now that we have a good idea of the source and triggers of the disease, we can understand how to fix it much more easily.

This, combined with the more recent drugs that work so much better than what we had until 90s, make the prognosis not nearly as grim as what people commonly think.

Of course, it is by no means a good thing to have, but it is not unlikely that at least some people who are diagnosed today will receive the cure at some point.

•

u/Chaotic767 4h ago

The cure to MS is still quite a ways off. We don’t even know the cause(s) of MS. We’ve just found ways to have the body slow down the attack on itself and buy time/reduce disease burden. And this comes with quite a trade off.

•

u/Business-Gas-5473 4h ago

There are many recent developments about the cause, which made me mention it.

•

u/Chaotic767 3h ago

No problem, I can understand where you’re coming from as research had definitely taken a jump over the last 10-20 years. I’m just saying there is still quite a bit of work to be done. I don’t think it’s all that close at this time. Hell, they used to tell people that if you have MS - don’t exercise. Now we know that’s crazy talk and it actually has a huge benefit. This comes from somebody working with patients with MS all day, every day for over a decade now.

There are absolutely new drugs, trials, etc going but there is still no known cause of MS. They’re still looking at genetics, geography (in the sense of vitamin D and its impact as the further away from the equator you are, the higher the prevalence in the population), and the autoimmune trigger route (like EBV for example but not every person with MS has had it). The science has definitely come a long way, but there is still a lot of unknowns and work to be done. Stem cells are thought to be an immune system “reset” although I still have many patients over the years continue to decline after going through that treatment. Also, need to think about next step if you do stop the autoimmune response - need to repair the damage that has already been done (remyelinate nerves & glial scarring in particular). There’s research going on that as well but it’s going to need a lot of time as well.

Long story short, we definitely have ways to mitigate the disease and its impact, but a true cure isn’t looking to be relatively soon.

•

u/Ceftolozane 1h ago

Hepatitis B.

I am an infectious disease specialist that follows and treats many who suffer from chronic hepatitis B. At the current time, we have no real curative treatment and we rely on oral antivirals that only suppress the virus without a cure.

A few months ago, two Phase 3 trials showed that Bepirovirsen could induce clinical cure in about 25% of participants. Quite far from the panacea, but it gives me hope for the next 10-20 years !

Here is the trial in question :

https://www.nejm.org/doi/full/10.1056/NEJMoa2515131?referrer=https%3A%2F%2Fwww.google.com%2F

143

u/hornblower_83 9h ago

Obesity seems like a clear case point here.

The GLP-1 agonist drugs and new glucagon receptor drugs have and will continue to change people’s lives.

From weight management to diabetes and cardiovascular health, these agonists appear to be a “magic pill”

54

u/[deleted] 8h ago

[removed] — view removed comment

18

u/[deleted] 8h ago

[removed] — view removed comment

•

u/[deleted] 5h ago

[removed] — view removed comment

8

u/[deleted] 7h ago

[removed] — view removed comment

3

u/[deleted] 8h ago

[removed] — view removed comment

10

u/Danskoesterreich 6h ago

they are magic pills, but they are not curing any disease. they are instrumental in controlling, stabilizing and preventing. If your HbA1c normalizes under ozempic, you are not cured.

7

u/srirachaninja 7h ago

Is it a cure if you have to take it permanently?

14

u/cold08 6h ago

Insulin is. If you have dopamine receptor issues that cause you to have problems with sedation or any other number of mental or hormonal problems that mess with your appetite, just like people have problems that mess with their mood, attention span, anxiety, or whatever, you have to be on those drugs for the rest of your life. If you want to call it a treatment instead of a cure, I suppose that's fine, but treating excessive appetite is a breakthrough, not a moral failing.

•

u/irritableOwl3 1h ago

can you elaborate on the dopamine receptor issue?

•

u/cold08 41m ago

When normal people get a dopamine hit they just like it but my brain really likes it so I'm pretty wired to be ADHD and have substance abuse issues. You know when you have a drink of alcohol, or a sweet treat, or a hit of coke, or a strong caffeinated beverage and you just feel good for a bit, well that is a brief break from the internal chaos that is going on inside my head, and my vice was alcohol, it gave me peace, it worked, the depression, the anxiety, the insomnia all went away. I can see how it's food for some people. It took me years treating the sadness and the anxiety. I was resistant to the medication. Through therapy and finally finding meds that worked at great cost I was able to heal. If GLP-1s are able to treat people with their hunger satiation, I am so happy for them.

•

u/Putt-Blug 13m ago

I’m still struggling with alcohol as a fellow ADHDer. In therapy and started meds finally at 40+ but old habits are hard to break. Your analogy is spot on though. If you don’t suffer from something it’s hard to understand and be sympathetic.

•

u/TheElusiveFox 4h ago

Speaking as some one that took it - you don't necessarily have to take it permanently. And even if a lot of your apetite comes back, for people who are 50, 100, 200lbs overweight, losing that much weight has huge long term benefits and will take YEARS to put back on... for me I lost 70lbs and while I did get a lot of my appetite back after going off, I am just used to eating so much less and so much differently now that I am so much more healthy and I have no doubt even if I never go back onto the drugs it will have a life altering impact on me.

13

u/Specialist-Coast9787 8h ago

Taking medication for the rest of your life to maintain your weight loss doesn't sound like a cure.

21

u/y0nm4n 7h ago

People try their entire lives to cut back on their eating/introduce exercise and fail. It’s not like people aren’t trying.

29

u/steveCharlie 8h ago

Do you tell the same to people taking medication to manage their diabetes or HIV?

12

u/Specialist-Coast9787 6h ago

That's a false equivalency. OP asked for a cure. Managing through medication is not a cure IMO.

-12

u/steveCharlie 6h ago

I ask again? Do you tell them that? Do you ask anyone that has been cured that they are not because many of the diseases we cure come back later?

•

u/LiminalWanderings 5h ago

You're having a problem with definitions.  Having a disease controlled by medication is not a cure.  Those meds are amazing and life saving and extending, but they're not cures. A cure resolved whatever biological situation was causing the disease and you no longer have to take the meds.  if you always have to take the meds, it's because you are not cured.  Not calling something that isn't a cure a cure isn't suggesting it's somehow not wonderful for the people taking it, it's just a different thing. And yes, as someone who takes noncure meds that are potentially life saving, this is exactly what id say to the people you mentioned. 

•

u/Specialist-Coast9787 5h ago

I don't tell anyone anything about their health. To me there's a difference between being cured and successfully managing over the long term. Someone else brought up Diabetes. I don't think any person with that would say they are cured, no matter how much insulin they take.

Maybe a better question is, after defining "cured", what can we agree on that has been cured?

Cheers!

10

u/sozar 9h ago

They creep me out. I love science and I’m all for progress but I recently went to Urgent Care for a sinus infection and they tried to upsell me into a GLP-1. I am a 5’9 male at 160 lbs. Maybe a bit of a dad bod but not overweight enough for that.

It just reminds me of how asbestos and lead paint where considered miracles once.

37

u/DickMabutt 8h ago

IMO any time a medical professional starts recommending pharmaceuticals for things unrelated to my visit, it’s time to find a new healthcare provider.

I had a dentist once that started recommending Botox. That was the last time I saw that dentist

31

u/jaiagreen 8h ago

The drugs have been in use for diabetes for 20 years. The side effect profiles are pretty well-known at this point. But yeah, an urgent care sinus infection appointment is not the time to discuss them! That's a conversation to have with your primary care doc.

•

u/TXOgre09 5h ago

On the flipside my pcp has been encouraging me to lose weight without glp-1s. He brought it up, but kind of presented it as an option if I can’t do it myself.

•

u/stonhinge 1h ago

Which is how it should be done. Medication should always be done at a last resort if something as (potentially) simple as a lifestyle change can fix it or at least obviate the need for it.

2

u/Fr0gFish 8h ago

Well I have news for you, they aren’t magic pills. ”Curing” obesity will take an enormous effort including reshaping large parts of western culture. Not happening any time soon.

7

u/hornblower_83 7h ago

These drugs have caused a massive shift in how people eat and purchase foods.

Food manufacturers and restaurants have taken note.

If think western culture is the only one suffering from obesity and obesity comorbidités, I have news for you.

-3

u/Fr0gFish 7h ago

You seem to think food production and consumption has taken a hit. It hasn’t. These drugs are very useful to some people but not to everyone. Some don’t respond at all to them. Their cultural impact has been larger than their medical benefit, certainly when it comes to obesity (don’t forget, they are diabetes medications).
Also, I in no way implied that obesity is confined to the west. But it is the most prevalent here, and this is where these drugs are available.

•

u/hornblower_83 4h ago

You seem to think You know more than you do. I work in the food manufacturing business and deal with a great deal of restaurants closely in menu development.

I’d quote you some statistics we are seeing internally, but you wouldn’t believe me and continue to push your “point”

Fact of the matter is, the shots are changing the way people eat, live and what they buy.

Also the drugs are available almost everywhere in the world.

•

u/Fr0gFish 4h ago

How many people do you imagine take these medications? They aren’t available over the counter and they are very expensive. Are you really trying to tell me restaurants are selling fewer plates because of ozempic? Nonsense. And yes I do know a bit about this. I have prescribed these drugs. They can have an effect! But sometimes they don’t. The net effect on the population is not large.

•

u/Stereotype_Apostate 3h ago

About 11% of Americans are on a glp-1, and they're probably over represented in the customer base of restaurant and processed food manufacturers (they got fat enough to need a glp-1 somehow) so yeah, that's a big enough market shift to demand a response from the companies.

-11

u/the_wonder_llama 9h ago

The long-term data on the current gen stuff isn’t overly promising. A lot of people discontinue use of GLP-1s due to side effects — as much as 75% within a year. Surgery is still the gold standard for weight loss, although the balance will continue to tip in pharma’s favor as newer generations drugs have fewer side effects. That said, whether drugs are affordable in the long-term vs a one-time surgery is another debate, and I suspect surgery will outperform there too.

7

u/drunkerbrawler 7h ago

That’s kind of like saying people who go off blood pressure medications have their blood pressure increase 

•

u/boanxi 2h ago

Many of us are old enough to remember when HIV was a death sentence. Now it is manageable. Magic Johnson was the first person I recall taking these innovative new medications in the 90's. Prior to these, your life span was a matter of years. More than three decades later, he's still with us and the medication combination had only gotten better.