Chances are that someone you know or a loved one has has Alzheimer’s disease and its slow progressing dementia. An estimated 6.5 million Americans—and 44 million people worldwide—suffer from it. Scientists are still uncertain about its causes: amyloid proteins, tau tangles, inflammation, fat protein complexes, high cholesterol and low bile acids, and even gum disease have all been hypothesized. We see headlines that proclaim “treatment success” and “reversing dementia in rats,” but even then, if you do get sick, there’s no cure. Why?
A glimmer of hope appeared in June 2021, when the Food and Drug Administration approved
Drug, Eduhelm. But studies have been mixed since then, and Medicare announced that it would pay only a $28,200 annual cost for patients enrolled in clinical trials, of which there are only a few. This month we learned about disappointing results from a trial in Colombia for Roche’s new amyloid-protein-targeted Alzheimer’s drug, crenezumab.
Still, the people I talked to who are funding Alzheimer’s research to one person are surprisingly hopeful. They see a pipeline full of potential, though they point to some flaws in the system.
My friend Elizabeth Gelfand Stearns, whose mother lived with Alzheimer’s for eight years, has raised nearly $10 million for the Judy Fund, which provides research funding and supports advocacy. When President Obama signed the National Alzheimer’s Project Act in January 2011, government spending on Alzheimer’s and related dementia research was $448 million, with the aim of “preventing and effectively treating Alzheimer’s disease and related dementia by 2025.” ” Ms. Stearns tells me, “Our pitch has changed from ‘this is a terrible disease’ to ’90 million baby boomers are entering the prime era for Alzheimer’s and will swamp Medicare and Medicaid.’ “It Worked: This Year the National Institutes of Health Received $3.5 billion In research funding for the disease. Sadly, NAPA now requires reauthorization by 2035. patience is a virtue.
So why is there no cure even after spending so much money on research? I could spend a dozen columns examining compelling theories and drugs. But approval of a drug by the FDA through trials funded by drug companies could take eight to 10 years and cost more than $2 billion.
It turns out that the drug-discovery process has a major flaw—namely, a wide gap between government research funding and private pharmaceutical company spending on drug tests. Another friend, Mickey Hogg, says this is known as “the valley of death,” where drugs often kill the dead. He is involved because his father showed signs of Alzheimer’s at the age of 65 and lived with it until the age of 79. A few months after his death, his mother showed signs of Alzheimer’s. She lived to 88.
What is Death Valley? The catch-22 is that Big Pharma will not fund human trials for promising new drugs without “proof of concept.” But you can’t show a proof of concept without human trials.
Ms. Hogg set out to bridge that gap by setting up the Part the Cloud Fund to direct funding to promising human trials. In 2012, the group funded seven projects. Over the past 18 months, it has raised $35 million, including $10 million from Bill Gates. Ms. Hogg is the world’s largest private fundraiser for Alzheimer’s. Today 60 trials are going on in the nine countries of this fund. Even better, Part the Cloud’s cumulative spending of $60 million has led to $1 billion in follow-on funding from pharma and venture capitalists. That is leverage.
How do they shortlist candidates? A peer-reviewed panel of 30 or more independent researchers studies 50 or so proposals each cycle, explains Heather Snyder, the Alzheimer’s Association’s head of medical and scientific relations. Five or six of them get funding.
Half the money goes to study previously approved drugs, with the hope of reusing them for Alzheimer’s. Most venture capitalists will not fund repurchased drugs because the returns are limited. “Our goal is to find effective drugs that work, period,” says Ms. Hogg. One candidate is leucine, a bone-marrow-cancer drug that reduces inflammation. Worth the effort. The more the better, “like shots on goal in hockey,” she says. “We are 20 years behind cancer.” I asked him how he had the patience for six to 10 years to get the drug approved. “It’s a slogan.”
The Alzheimer’s Drug Discovery Foundation, founded by Leonard and Ron Lauder, is also providing funding for human trials. It’s safe to say that many Big Pharma trials would not have been initiated without these groups bridging the “proof of concept” gap.
Ms Hogg says none of the cloud-funded trials have failed in the past 10 years. Some of the recent optimism relates to the drugs in the pipeline behind EduHelm—many more Phase 3 trials involving beta amyloid protein should report results in the next 12 months. Some parts are in cloud candidate phase 2, which means years away. I’m hopeful too, but I get what he means by a slogan.
Write to kessler@wsj.com.
Journal Editorial Report: The best and worst of the week from Kim Straussell, Kyle Peterson and Dan Henninger. Images: Composite: Mark Kelly
opinion | Alzheimer’s cure puzzle
Chances are that someone you know or a loved one has has Alzheimer’s disease and its slow progressing dementia. An estimated 6.5 million Americans—and 44 million people worldwide—suffer from it. Scientists are still uncertain about its causes: amyloid proteins, tau tangles, inflammation, fat protein complexes, high cholesterol and low bile acids, and even gum disease have all been hypothesized. We see headlines that proclaim “treatment success” and “reversing dementia in rats,” but even then, if you do get sick, there’s no cure. Why?
A glimmer of hope appeared in June 2021, when the Food and Drug Administration approved
biogen‘s
Drug, Eduhelm. But studies have been mixed since then, and Medicare announced that it would pay only a $28,200 annual cost for patients enrolled in clinical trials, of which there are only a few. This month we learned about disappointing results from a trial in Colombia for Roche’s new amyloid-protein-targeted Alzheimer’s drug, crenezumab.
Still, the people I talked to who are funding Alzheimer’s research to one person are surprisingly hopeful. They see a pipeline full of potential, though they point to some flaws in the system.
My friend Elizabeth Gelfand Stearns, whose mother lived with Alzheimer’s for eight years, has raised nearly $10 million for the Judy Fund, which provides research funding and supports advocacy. When President Obama signed the National Alzheimer’s Project Act in January 2011, government spending on Alzheimer’s and related dementia research was $448 million, with the aim of “preventing and effectively treating Alzheimer’s disease and related dementia by 2025.” ” Ms. Stearns tells me, “Our pitch has changed from ‘this is a terrible disease’ to ’90 million baby boomers are entering the prime era for Alzheimer’s and will swamp Medicare and Medicaid.’ “It Worked: This Year the National Institutes of Health Received $3.5 billion In research funding for the disease. Sadly, NAPA now requires reauthorization by 2035. patience is a virtue.
So why is there no cure even after spending so much money on research? I could spend a dozen columns examining compelling theories and drugs. But approval of a drug by the FDA through trials funded by drug companies could take eight to 10 years and cost more than $2 billion.
It turns out that the drug-discovery process has a major flaw—namely, a wide gap between government research funding and private pharmaceutical company spending on drug tests. Another friend, Mickey Hogg, says this is known as “the valley of death,” where drugs often kill the dead. He is involved because his father showed signs of Alzheimer’s at the age of 65 and lived with it until the age of 79. A few months after his death, his mother showed signs of Alzheimer’s. She lived to 88.
What is Death Valley? The catch-22 is that Big Pharma will not fund human trials for promising new drugs without “proof of concept.” But you can’t show a proof of concept without human trials.
Ms. Hogg set out to bridge that gap by setting up the Part the Cloud Fund to direct funding to promising human trials. In 2012, the group funded seven projects. Over the past 18 months, it has raised $35 million, including $10 million from Bill Gates. Ms. Hogg is the world’s largest private fundraiser for Alzheimer’s. Today 60 trials are going on in the nine countries of this fund. Even better, Part the Cloud’s cumulative spending of $60 million has led to $1 billion in follow-on funding from pharma and venture capitalists. That is leverage.
How do they shortlist candidates? A peer-reviewed panel of 30 or more independent researchers studies 50 or so proposals each cycle, explains Heather Snyder, the Alzheimer’s Association’s head of medical and scientific relations. Five or six of them get funding.
Half the money goes to study previously approved drugs, with the hope of reusing them for Alzheimer’s. Most venture capitalists will not fund repurchased drugs because the returns are limited. “Our goal is to find effective drugs that work, period,” says Ms. Hogg. One candidate is leucine, a bone-marrow-cancer drug that reduces inflammation. Worth the effort. The more the better, “like shots on goal in hockey,” she says. “We are 20 years behind cancer.” I asked him how he had the patience for six to 10 years to get the drug approved. “It’s a slogan.”
The Alzheimer’s Drug Discovery Foundation, founded by Leonard and Ron Lauder, is also providing funding for human trials. It’s safe to say that many Big Pharma trials would not have been initiated without these groups bridging the “proof of concept” gap.
Ms Hogg says none of the cloud-funded trials have failed in the past 10 years. Some of the recent optimism relates to the drugs in the pipeline behind EduHelm—many more Phase 3 trials involving beta amyloid protein should report results in the next 12 months. Some parts are in cloud candidate phase 2, which means years away. I’m hopeful too, but I get what he means by a slogan.
Write to kessler@wsj.com.
Journal Editorial Report: The best and worst of the week from Kim Straussell, Kyle Peterson and Dan Henninger. Images: Composite: Mark Kelly
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