Drug Halts Decline In Alzheimer's Patients

ljw1004 writes "Alzheimer's researchers are divided on whether the disease is caused by 'beta amyloid' (a peptide found in Alzheimer brains) or by 'tau protein' (normally used for cellular scaffolding, but can aggregate out of control and destroy neurons). Today in Chicago a new drug has been announced that stops tau aggregation and appears to have halted Alzheimer's-related decline in 300 clinical trial patients. The drug is known as 'rember.' Do you have friends or family who appear to be on the road to dementia? Here is an online questionnaire, part of one used in the clinical trial to diagnose dementia. (Disclosure: I made the online questionnaire, and my father is one of the scientists behind the drug.)"

Comments

Dementia != Alzheimer's

[morgan_greywolf]

It's important to remember that Dementia != Alzheimer's. Alzheimer's can cause a form of dementia (Alzheimer's-related dementia), but dementia has many other causes, some are age-related and some are not.

Rember is Methylene Blue

[Fallen Andy]

See here (a very well known biological stain) - so short term toxicity is probably well understood - long term? Who knows yet. At the very least it ought to open the way to a new class of drugs for this terrible condition

Andy

Re:tested?

[DrDNA]

This drug is in the second of three phases which are required prior to FDA approval.

Phase 1: safety at various dosages
Phase 2: small test of efficacy and determining proper dosage
Phase 3: larger test of efficacy

It is still years away from the market. There was a screw-up in the formulation of the highest dose in this study, and the lowest dose had no effect, so only the middle of three doses tried had any effect. I found that out here

Re:tested?

[MrMr]

Except that in this case you can skip all that and do a little experimentation on your grandparents. This is not a new drug: it's plain old methylene blue, which has been used for all kinds of purposes for a century (from anti-malaria drug to aquarium antifungal)
See this

Re:"Rember" is methylthioninium chloride?

[ljw1004]

I should say that Claude Wischik thinks he *does* know what causes Alzheimer's disease. He's sure that tau tangles cause it. He's spent the past twenty years accumulating evidence and trying to convince people of the fact, but it's been hard because of the entrenched scientific dogma that amyloid causes it. The success of this drug finally is a vindication.

You're absolutely right, though, it was a case of trying lots of chemicals. At least, the larger pharmeceutical companies have been trying hundreds of thousands of chemicals from their libraries. A smaller company like TauRx can only manage far fewer.

But what's needed is a test-tube test to judge whether your chemical works. Previous attempts have judged whether their test chemicals work to prevent Amyloid buildup, and so they skip right over the useful ones. Claude Wischik realised that the test-tube test should be judging whether a drug works on tau tangles. This test-tube assay was the first key invention.

After that, you need an animal test to judge whether the drug works in animals. The second key invention by TauRx is a transgenic mouse where you can make it selectively express tau aggregates. They created mice with alzheimers, watched them make their demented way around water-tanks looking for firm ground, and then showed that Rember improved their condition.

You're right to ask about the temporary remissions. The clinical trial lasted 19 months and had 321 patients -- not a short trial! The test results had a p-value of 0.2%, i.e. there's a 0.2% chance that the improvement was due to the common random fluctuation rather than the drug's effect.