After being dosed with a drug made from the blood of young people, Alzheimer’s patients in a clinical trial retained memory and mental function for six months – when they would normally be expected to deteriorate.
Alkahest, a California-based startup, is one of a spate of companies that hope to harness young blood proteins to stop or reverse Alzheimer’s and age-related diseases.
Experiments in mice and petri dishes have suggested that these proteins can fix neuron coating – and now scientists are trying it in humans.
They’ve drawn wary comparisons to vampires, but Alzheimer’s patients in the latest trial are apparently unfazed by the treatments’ origin – and were able to remember better for it, preliminary data suggest.
So far, Alzheimer’s patients in the earlier stages of dementia showed no or far less cognitive decline than expected in the six months following treatment, according to a press release from Alkahest.
Alzheimer’s researchers have been eyeing proteins in young blood for treating the devastating disease. One startup claims its plasma-based drug paused declines for six months
The company reports that by three measures of cognitive function, patients in its trial showed no or less decline than expected during the six months following their two treatments.
Memory and functionality were tested by asking the patients to do things like recall words, draw shapes, carry on conversation, follow instructions, and remember information.
On the whole, they showed no changes by some measures and negligible changes for the worse by others.
Tantalizing though the report is, it doesn’t reveal any specifics, and the controversial nature of the drug used might stand in the way of its eventual Food and Drug Administration (FDA) approval.
Alkahest’s drug – GRF6019 – is made by distilling proteins from blood plasma taken from young donors.
‘Young blood’ transfusions actually have a long history, some of which is garish and a far cry from what such a treatment would look like today.
To study how the blood of a young organism might affect the body of an older one and vice versa, scientists have been stitching two animals together for 150 years.
It’s an important stage of research, still used and credited for revelations in how we treat everything from cancer to hormonal issues – but not reflective of what human therapies would look like.
THE WAR ON ALZHEIMER’S: MORE THAN 150 TRIALS HAVE FAILED IN 20 YEARS
Scientists have for years been scrambling to find a way to treat or prevent Alzheimer’s disease, which accounts for around two thirds of the 50million dementia patients worldwide.
But attempts to tackle the brain-destroying disease have been beset with failures.
In March this year the pharmaceutical company Biogen abandoned two late-stage trials of a promising Alzheimer’s drug, aducanumab, which it hoped would work by clearing the brain of sticky build-ups.
After years of research and testing the company decided its prospects looked poor in the end stages of a human trial and pulled the plug, wiping $18billion (£13.8bn) off its own market value.
In January, the firm Roche announced it was discontinuing two trials which were in their third phase of human testing.
It was trying to develop crenezumab, which worked by preventing build-up of plaques in the brain and had already been proven safe, but wasn’t having the desired results.
Between 1998 and 2017 there were around 146 failed attempts to develop Alzheimer’s drugs, according to science news website, BioSpace.
Billions of dollars have been invested in the industry and a successful, marketable treatment would likely make a fortune for the company which gets there first.
Experts have said a difficulty in testing drugs on the right people may be partly to blame – Alzheimer’s is rarely diagnosed before it has taken hold and, by that time, it is often too late or studying people becomes too difficult.
For drugs which try to modify the course of a disease, trials often have to be longer and more in-depth, making them more difficult and costly, researchers wrote in the journal Expert Opinion on Investigational Drugs.
The same researchers added scientists may be struggling to find the correct dose of drugs which could work, and that they may be recruiting the wrong types of people to test them on.
Scientists at Alkahest as well as other companies and research institutions have honed in on proteins found in the plasma. The populations of some of these proteins decline with age.
So, researchers hypothesize that as fewer of these proteins circulate throughout the body, via the bloodstream, aging and cell damage may accelerate.
Swapping in blood plasma still rich in these plasma proteins, then, might fight aging the the disease that tend to come with it – including Alzheimer’s.
To glean these proteins, scientists need young, healthy donor blood, which is then separated into its component parts.
Alkahest then uses plasma to make GRF6019, an injectable drug that its begun delivering to clinical trial patients with mild to moderate Alzheimer’s.
The company’s aim is to develop a drug to be used in broader Alzheimer’s treatment – so you won’t be seeing clinics, like those from controversial ‘young blood’ transfusion company, Ambrosia, popping up any time soon.
Alkahest won’t be revealing the specifics of their findings until December at a conference.
So far, dozens of Alzheimer’s drug trials have failed – disheartening results that have forced scientists to question everything they think they’ve learned about the disease’s causes and markers.
But some have suggested that it’s not the treatments nor the targets that have been wrong in those trials, but the timing.
‘The problem is that a lot of these trials started giving the medication late in the course of the disease when you already have a lot of damage and other processes going that are pretty impossible to top,’ Dr Suzanne Schindler, told DailyMail.com in a recent interview about her research on a blood test for Alzheimer’s.
Dr Schindler, a neurologist at Washington University, St Louis, says that if Alzheimer’s could be detected earlier, then new experimental drugs might actually have a shot at treating the disease before it’s too late.
Encouragingly, Alkahest’s trial was conducted in people in the earlier stages of dementia, when it might be possible to undo the damage.
GRF6019 is a long way off from being proven safe and effective enough to market to the broader population.
But if it does reach that stage, it will face a new challenge: a supply of young blood.
An estimated 5.8 million Americans have Alzheimer’s.
By 2050, that number is expected to bloom to 13.8 million, as the population ages.
About 6.8 million people donate blood in the US each year.
That still leaves many hospitals short on blood they need to treat people in emergencies, sustain surgical patients, keep those born with sickle cell anemia alive, treat other blood disorders, support cancer patients and much more.
For now, blood and blood plasma cannot be recreated in the lab, so if young blood-based transfusions do turn out to be the best treatment for Alzheimer’s demand for donations would surge, perhaps beyond the available supply.