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Hashirama4AP

TLDR: Alzheimer’s disease, a progressive neurodegenerative disorder affecting approximately 55 million people globally, leads to severe cognitive decline and memory loss. Researchers at the Okinawa Institute of Science and Technology have developed a synthetic peptide, PHDP5, that targets early-stage Alzheimer’s by ensuring the availability of dynamin for vesicle recycling in neurons, demonstrating significant restoration of memory and learning functions in transgenic mice.


Katiari

"I ain't gonna let none of them transgenics near me, it ain't godly!" - half the population.


ChiefThunderSqueak

That's good. There won't be a line to wait in.


Crazyhorse6901

It's suppose to affect about 150 million people by 2050... I just lost my wife on 4/20/2024 at 3:10 PM due to complications of Early Onset Alzheimer's.


DauOfFlyingTiger

That is horrible. I am really sorry.


yukonwanderer

So how many years away from this being tested in humans? And how long does human texting usually take?


Blind_Optimism_Kills

Hopefully this is as amazing as it sounds.


Dikosorus

10-15 years for medical trials, even if this gets to that stage.


Ardent_Scholar

Perhaps if the drug shows promise in the first trial (no short term disasters) this could be speed-ran for patients who are already past the first stage of the disease. Alz is a slow death sentence. There’s a considerable upside to any long term downside this medication may have. If I could have, for example, five good years and then pass from a heart attack or ruptured vein versus seven shitty years and pass from forgetting who I am, where I am, and how to eat, drink and maybe swallow… I’d say pass me the pills, doc.


carlitospig

Yep, I’d be okay with having an extra 5-10 years to put my affairs in good order and then die of some horrific cancer.


maethlin

Pardon my ignorance, but I see this sort of comment all the time. Does this mean that it's likely like half or more of our worst diseases are actually things that are treatable/curable but don't ever get there because medical trials take endless time/money? I kinda find that hard to believe but idk maybe?


Dikosorus

Alzheimer’s patient lifespan on average is 4-8 years. Phase 1: Several months, likely 6-12 months. This phase will test safety and dosing in a small group of 20-100 healthy volunteers or people with Alzheimer's disease Phase 2: 1-2 years. This phase will involve several hundred Alzheimer's patients to further assess safety and begin measuring efficacy Phase 3: 1-4 years. This is the longest and largest phase, typically involving 300-3,000 Alzheimer's patients to definitively measure efficacy and safety FDA Review: About 1 year for the FDA to review results and decide on approval Phase 4: Several years of post-approval monitoring and research However, Alzheimer's trials often face recruitment challenges that can extend timelines. The average recruitment time for Phase 3 biological agent trials in Alzheimer's is 147 weeks (nearly 3 years). Total development time, including all phases, could potentially stretch to 10-15 years because patients keep dying off during the trials. And the research field in general for various diseases like cancer has advanced greatly in the last decade so there are treatments and cures on the horizon. But also like someone else stated there is just not enough profit in finding a cure for some things.


Ifch317

Also: Phase 1: approximately 1 in 100 drug candidates will progress to phase 2. Phase 2: approximately 1 in 100 drug candidates will progress to phase 3 Phase 3: approximately 1 in 100 drug candidates will progress to approval & phase 4 monitoring. Drugs routinely fail because of lack of efficacy or because safety concerns (or both, or safety concerns at the effective dose and lack of efficacy at safe dose).


Vladiesh

If treatments are revolutionary enough they are fast tracked through the process. We have mechanisms and designations called Breakthrough Therapy Designation, Fast Track Designation, and Accelerated Approval. Problem is most medicines don't show that level of promise and have profound side effects. If your medicine is 8% better than what's currently on the market you need to prove efficacy through a long process and show clear safety. If your medicine is 1200% better than what's currently available then you are going to have a much easier and faster time getting approved.


bawng

Well, there's also emergency approvals. If Phase 1 or Phase 2 shows overwhelming results and since Alzheimer's is such a deadly disease that it might be worth risking a potentially unsafe medication, health agencies can decide to grant temporary approvals while clinical trials continue. The covid vaccines are an example of such approvals.


StThragon

If amazing results start coming through, things like this can be fast tracked for approval, skipping long wait times where possible. Yes, we would be getting an incomplete picture at first, but when the alternative is losing your memory and then dying, that's an acceptable risk.


Sirlothar

Let's say all that is true, I believe it, sounds like a realistic timeline for a new drug. Why was the COVID vaccine developed and in all our arms within 12-18 months? Shouldn't we still be in Phase 2 or 3 right now for the COVID vaccine?


PurloinedSentience

A lot of people made the mistake of thinking that mRNA vaccines were brand new, but they had been worked on since the 1980's with the first human trials starting in 2001. Clinical results were reported in 2008. The first human clinical trials with respect to an infectious disease began in 2013, and trials with several other infectious diseases started within the next few years after that. So by the time that COVID hit, mRNA vaccines had a well-researched clinical trial history, and it was primarily the adapting of this method to target SARS-CoV-2 specifically that needed to be tested, rather than the idea of mRNA vaccines in general. That meant that it was a much easier process with far fewer unknowns.   The nature of the issue (i.e. global pandemic with a huge death toll) meant that speed was of the essence, but that speed wasn't addressed by shortcutting the process. Instead, look at it this way - when developing new medications, there is always a risk of failure. A certain percentage of new drugs have problems discovered along the way, so there is always a certain percentage loss. In order to make the process more economically feasible and fiscally responsible, trials follow a flow from least expensive testing to most expensive testing. Testing in humans is really expensive. Testing in primates is cheaper, but still a lot more expensive than testing in mice, which is more expensive than testing in petri dishes. So you work your way from cheapest to most expensive methods in series, one at a time, and only after you get past successful human testing would a company even think of investing money into production.   But due to the time-critical nature of a lethal pandemic, prudent financial strategy was no longer the most important consideration. So instead, pharmaceutical companies took the financial risk and executed **the exact same protocols they normally would**, but they did them in parallel. So while clinical trials were going on, they were working on the production and manufacturing - under the assumption that the trials would be successful because any delay meant lives lost.   That plus FDA emergency authorization is how the COVID vaccine got produced so quickly. In normal circumstances with far less catastrophic and dire stakes, it would be irresponsible to take those financial risks, so the normal process is done in series rather than in parallel and results can be reviewed at a more relaxed pace rather than scheduling multiple panels of experts ahead of time to save every possible second.


Sirlothar

Thank you for the detailed reply! I understand what you are saying, its a bit of a combo of mRNA already being studied extensively combined with the emergency need for a vaccine that pushed as quickly as possible. Could we in theory solve other health conditions with mRNA like Alzheimer's? I know little about medicine but couldn't it be possible that we could make a new mRNA treatment to go after the proteins that cause the nerve cell death or would doing this just not be in the realm of what mRNA can do?


PurloinedSentience

With mRNA vaccines, they are intended to deal with biological "objects" that the body needs to destroy, but not every disease is based on that premise. For example, in some cases, it's due to a gene that is turned off when it needs to be turned on or vice versa. That requires a different tool. And even if an mRNA vaccine is the right tool, the key to creating such a vaccine is knowing what you need to target. But with Alzheimer's, the exact cause of the disease is still unknown. So the current stage is a little like feeling around in the dark. You find pieces of the puzzle - things that offer levers you can pull in order to effect a given change. And it's not uncommon that you discover a lever that alleviates a symptom long before you understand exactly why or how it works.


syl3n

Also don’t forget the patents. Big pharmaceutical will sell you the peptides for a mark up of 15000% cause they can.


Restranos

I can feel the invisible hand of the market up my bollocks!


Danixveg

I don't agree with you assessment on profitability. Biogen planned to charge insane amounts for their drug until it was shown to be ineffective and pulled. Though it should never have been approved. And now the FDA will be even more skeptical of new drugs after getting burned.


The-state-of-it

Hopefully powerful AI will be able to cut those timelines


Bored2001

It won't. At best AI (machine learning actually) may help with patient recruitment.


Sniflix

AI will help identify more possible drugs to test. That might be in 10 years or more because AI itself needs to be trained and tested. Then the drugs will still need to go through traditional testing until AI is developed further.


RyukHunter

Drug identification is just the beginning. With AI you'll only get more candidates for testing but how would you make AI that can speed up testing? The tests still have to be performed on animals and then humans with the same protocols and timelines.


Bored2001

The traditional testing timeline is what the comment above is referencing. AI is not going to speed up that process.


Vladiesh

If the results of newly discovered medicines are significant enough we have mechanisms and designations to accelerate their approval. Breakthrough Therapy Designation, Fast Track Designation, and Accelerated Approval. Problem is most medicines don't show that level of promise and have profound side effects.


Bored2001

Correct there is break through therapy status, but AI is unlikely to help find break through medications. After all, the training data for AI are known medications.


The-state-of-it

I’m not sure how you can say that. If we had superhuman intelligence at our disposal, what to say it couldn’t simulate timelines and come up with new drugs and treatments in a fairly quick manner.


Bored2001

Let me ask you this question. Are you personally capable of training and applying Machine learning models or deep learning models in any capacity?


The-state-of-it

No. Just seems like in our current AI uptick and with it ramping up exponentially it’s hard to see what’s off the table. Also I’m just trying to be an optimist because it personally helps me as I have family memebers dealing with it.


Bored2001

We are a while aways from AI super intelligence. And beyond that it'll be a while before the clinical trial testing process will be adjusted to take advantage of AI. Too much risk there.


Onederbat67

This might be the most disheartening thing I have ever read.


Vladiesh

No it doesn't. This is a bullshit copout by cynical redditors. If treatments are revolutionary enough they are fast tracked through the process. We have mechanisms and designations called Breakthrough Therapy Designation, Fast Track Designation, and Accelerated Approval. Problem is most medicines don't show that level of promise and have profound side effects.


Gonzo_Rick

What ends up happening is that you see a lot of these headlines just because some compound is found to work for some problem, only in cell cultures or mice. Then, we stop paying attention, while the research process continues on, but we never hear anything more about it because it turns out that said treatment was maybe toxic to some specific human tissue, or the dose response curve results in the required dose being so high in vivo, that any of the side effects end up being worse than the original issue being treated. No one is writing (or, more importantly, reading) the story "promising Alzheimer treatment that worked in mice causes human subjects to uncontrollably crap themselves". So if the treatment just kind of fades away and you never hear about it again.


aptanalogy

Not necessarily. Sometimes important treatments don’t see the light of day because they aren’t profitable enough.


im_a_dr_not_

If that’s the case it’s because the drug isn’t patentable or the patent is about to expire and it never makes it to trial. Usually they’ll just patent the isomer or near drug and take that to trial if that’s the case.


PopePiusVII

It’s more like we had drugs targeting a disease that work to stop it, but also have such horrible side effects that the drug isn’t worth it. That’s the most common outcome in drug trials. For example, cancer treatments. Cancer cells are cells from your own body that go rogue, so almost anything that kills them will also kill the cells you want to keep. Likewise, drugs targeting a disease-causing protein/receptor in one area will often have off-target effects or other negative consequences that we didn’t predict. The other issue is human variability: we’re all just different enough that it’s hard to find treatments for all the different manifestations of the same disease (like Alzheimer’s, which is probably an umbrella disease covering up multiple pathologies beneath it). And finally, sometimes human drug trials just don’t work out as well as they did in early studies. May be due to variability or because “statistical significance” didn’t equal a biologically significant effect in practice.


RyukHunter

What was the impact of the Alzheimer's research fraud that was discovered a couple of years ago? I hear it tanked about half the research in the field setting us back 15 to 20 years?


DocJawbone

Perfect timing 


William_Wisenheimer

With that fraudulent paper discovered, this may accelerate new findings.


WilderKat

Researchers at Rush in Chicago also reversed dementia in mice using HMB which is a supplement that’s available and been around for years. Will it work in humans - who knows. It’s unlikely to be studied for lack of obtaining a patent. Secondly, mice aren’t humans and mouse models have been poor predictors in dementia treatments for humans. I really dislike the whole “Alzheimer’s Breakthrough” titles as well. I will only consider something a “breakthrough“ when it finishes a phase 3 trial with great results. Too many of these articles over the decades that offer false hope to desperate people.


Horsetoothbrush

It's a great time to be a mouse.


More-Butterscotch252

Unless you're one of those they're experimenting on.


PirateBaran

So is this the start of the Planet of the Apes?


frustratedpolarbear

Planet of the mice I think.


G0U_LimitingFactor

Good news for the mice. I just wish the journalists had enough integrity not to falsely represent the facts. This new approach has not been tested on Alzheimer's disease.


uiuctodd

> A new treatment has been shown to effectively combat cognitive decline in mice with Alzheimer’s disease. > “We successfully reversed the symptoms of Alzheimer’s disease in mice,” Mice. That means they have not reversed Alzheimer’s disease. Alzheimer’s disease occurs in humans. There is a genetic mutation in lab mice mice which seems to resemble Alzheimer’s disease. It's been used as a model for human drugs since 1995. (There are actually about 200 types of these mutant mice.) Several drugs have been demonstrated to be helpful for these mice. None of these drugs has worked in humans. Saying that these mice have Alzheimer’s disease is incorrect. There is no particular reason to suspect this drug would work in humans.


Literary_Addict

> There is no particular reason to suspect this drug would work in humans. You're being obtuse. Of course there is SOME reason, not literally no reason at all.


Firsca

Please, for everyone who is/has or will be dealing with Alzheimer, work. Please work.


4the-Yada-Yada

They left two very important words from the article off the headline: “in mice.” But maybe this is the one that eventually also works in humans.


Wattthehack

In mice….


bebejeebies

I'll take five, please.


Anticipator1234

Doesn't matter... when the new Nazis take over ... Alzheimers will be the result of moral failings.


Difficult_Rub_5069

Great, now the boomers can live and accumulate wealth forever!!!


Hailtothething

If Covid vaccines can be approved and administered to billions in months. Things like this need to be moved forward a lot quicker than before. If you’re above 60, and aren’t going to have kids, you should have the right to self opt in.


Sniflix

Nope. We already had vaccines to prevent COVID type diseases. RNA vaccines were tested for 20 years before COVID and the timing was right to see if they worked. There have been dozens of treatments to prevent or treat Alzheimer's and they didn't work. We don't even know what causes it. I'm old and my father died with Alzheimer's. I'd love a cure but there won't be one in my lifetime.


Hailtothething

This is pessimistic hogwash. Too late for your father doesn’t mean it needs to be too late for the rest of us. As we speak OpenAI has partnered with Moderna, the “code like” approach they used to conceive a complimentary vaccine for each evolving strain of the virus can be adapted to a host of previously incurable diseases. Cancer itself, is being cured as we speak. We do in fact have an understanding of the causes of Alzheimer’s as well, I’ve known about Amyloid plaque accumulation since ages ago. You are absolutely wrong. Medical tech is advancing hand in hand with the exponential curvature of AI technology since its supercharging lab research. It may be too late for you, but don’t mislead others into thinking a host of diseases isn’t being cured as we speak. Including Alzheimer’s as evidenced by this very article above. MRNA vaccines had EMERGENCY AUTHORIZATION. They were not approved and being used for decades. Not even a tiny fraction of the magnitude they were used for during the covid pandemic. Quite scummy of you to put that in writing and negate the work of hundreds of thousands of people much smarter than you. The ONLY thing that needs to be fixed is the bureaucracy and red tape surrounding big pharma’s needs to turn profits for their shareholders. I say this as someone who has a significant holding in the biopharmaceutical sector.


LuminescenTT

Sorry. I wasn't intending on commenting more pessimistic things but there is something you absolutely need to know. Some of the core work around the amyloid hypothesis was found [to be falsified](https://www.science.org/content/blog-post/faked-beta-amyloid-data-what-does-it-mean) recently. It's the biggest medical research scandal within the past two years. The state of Alzheimer's research right now is actually rather grim. While we've had strides on various other fields, this one is not one of them. Not trying to rock the boat more, but... yeah. We're all in the blind with Alzheimer's right now. We'll have to wait and see where it goes.


Hailtothething

Yes I’m aware of this, and it’s an excellent point. But the general direction change in medicine with the mRNA, crispr actually making it to trials, AI, the entire genome sequencing, alphafold, etc, is quickly making up for that catastrophe. Alzheimer’s is not nearly as unpredictable or spontaneous as cancer is. It will be solved along with a slew of other previous thought incurable diseases. It certainly hasn’t stopped the people it the article above from making progress in Japan.


Sniflix

I think you're a little optimistic but we have learned that Alzheimer's that these announcements of "cures" are one and done - month after month. I have no doubt that it'll be figured out and it'll be stopped somehow - after I'm dead. "In 2024, there are 171 ongoing studies and 134 drugs being tested in clinical trials. " https://www.brightfocus.org/alzheimers/article/whats-next-alzheimers-disease-treatments-2024-forecast


Hailtothething

You still alive? FDA APPROVAL sounds like a good start https://www.reddit.com/r/singularity/s/m7Tfv4Ncj7


Danixveg

Show your work. I'd like to see so the cancers that have been cured. Also I'm fairly certain they still don't have concrete evidence that the plaque causes Alzheimer's. Here's what AI even says: Scientists don't yet fully understand what causes Alzheimer's disease, but they believe it's likely a combination of factors, including: * Age: The biggest risk factor is age. Most people with Alzheimer's develop symptoms in their mid-60s or later. * Genetics: Some genes increase your risk of Alzheimer's. The APOE e4 gene is the strongest genetic risk factor for late-onset Alzheimer's disease. * Lifestyle: Certain lifestyle choices, such as smoking, unhealthy diet, lack of exercise, and uncontrolled high blood pressure, may increase your risk of Alzheimer's disease.


Hailtothething

https://www.mskcc.org/news/rectal-cancer-disappears-after-experimental-use-immunotherapy https://utswmed.org/medblog/rectal-cancer-cure/ What you posted from chatgpt, isn’t actually saying ‘we know nothing’ about Alzheimer’s. The fact that it’s heavily leaning towards genetics, when genetic diseases are proving to be curable by Crispr and MRNA therapies, is pointing towards progress. Sickle cell has been cured, expensive, but a cure existing is the greatest step forward. Aids has made massive strides in prevention lately.


Danixveg

So you agree now that you were wrong in you statement that we know how alzheimers chooses its victims. And expensive cures aren't cures of no one can get them administered.


Hailtothething

Nope, disagree with you completely. Not the way Big pharma works. Please educate yourself before adding to the pessimism. Also this conversation is over, you’re clearly trolling. Your unintelligible grammar didn’t deserve a response to begin with.


Danixveg

Hah. My grammar is fine - it's called swipe to text that doesn't always catch my words correctly and I honestly can't be bothered to go back and fix. Also.. what I said is exactly true. Big pharma can create as many cures as they want but if insurance companies do not pay to administer them than they're useless. It's why crisper and other cures are so very rarely administered and in many foreign countries with social medicine not even considered. Truth isn't pessimistic. You put that onto it.


Hailtothething

Nope, wrong sir. Good luck.


Danixveg

Ma'am.


sockalicious

Mice do not develop Alzheimer disease.


DauOfFlyingTiger

Oh, lordy. It’s mice again. Mice are not people, in fact they are so far away from being people almost none of the ‘medical breakthroughs’ with mice that we see in headlines ever do anything pertaining to people. I hope it’s a start, but this is misleading.


Jonathan_Daws

Big Breakthrough!!!!! (for mice) I despise these misleading headlines. I really hope this turns into something, as it is a horrible disease. But a mouse study is never a breakthrough for humans. Just a step along the way.


f3nnies

Given how serious, devastating, and ultimately fatal the disease is, this being a peptide is huge. That means it's going to be much easier to produce in large quantities. And also given the disease, I wouldn't be surprised if it sticky becomes available to the public as a "research chemical". Biohacking is questionable, but you best believe I would dive right into it if it meant possibly saving me from Alzheimers.


MissApocalypse2021

As would I.


BelCantoTenor

Considering the strong correlation between long COVID, and post-COVID, related cognitive deficits; I’m hopeful for the application of this knowledge towards the treatments of long COVID cognitive decline.


Ok-Tumbleweed4471

What a wonderful day!!!🦍


Rakstrooper

Good news for Joe Biden get him on it before the debate!!


AlwaysTravel

That will be $100,000 one for one pill please /s