Attention all patients at risk for memory loss, Medicare recipients, health care providers and families for the elderly.
A new drug has been FDA-approved for treating “Amyloid plaques” in patients with mild cognitive impairment (MCI) and mild Alzheimer’s disease (AD).
This is a major announcement for several reasons:
1. If you have been following the approval for this drug, you will know the following:
a. We have not found a successful treatment for an insidious disease (AD) that impacts so many Americans. It impacts everyone. There are only 2 approved medications that are at best, used to “slow down” the inevitable and tragic process that occurs in AD.
b. R&D in this area has been extremely disappointing as a result of the tremendously high cost of running clinical studies and the poor results and non-approval for many potential therapies.
2. What we do know about AD (in a very simplistic model) is that amyloid plaques develop in the brain and these plaques have a direct correlation clinically. Meaning, more plaques, worsened memory and overall outcomes. So the hypothesis goes: we find an “anti-plaque” therapy and hope it helps with memory.
3. Enter BIOGEN:
Major pharmaceutical company in New England that has partnered with a Japanese group, Eisai.
They have been hitting the “anti-plaque”industry since 2016. They initially released data in 2019 using a monoclonal antibody called Aducanumab. Monoclonal antibodies have revolutionized medicine in the last 20 years. If you are a cancer or rheumatologic patient, chances are, you’ve been treated with one of these agents. The dead give away, is cost, usually $20,000-30,000 for treatment courses. BIOGEN initially released data showing how effective Aducanumab was at reducing amyloid plaques first in non-humans and then in subjects with memory loss due to AD. They originally applied for FDA-approval back in 2019 and did not get the thumbs up. The problem was that the FDA needed clinical outcomes, ie improving someone’s memory. The data was not impressive when it came to taking someone with early AD and improving their cognitive skills using tests like the Mini Mental Statis Exam (MMSE). You know, “spell WORLD backwards.”
Like so many big pharm groups, they then “reanalyzed” the data (looking at subgroups that had specific characteristics such as age, or in this case, dose of the drug). They finally got the “signal” that they were looking for: at a higher dose and more treatment time, the patient clinical scores seems to get a “little” better.
They kept working on anti-plaque (high-risk/high reward) therapy despite other companies that were striking out and losing their shirts. They performed 2 additional clinical studies (EMERGE and ENGAGE) at an enormous cost to show that these new treatments would do more than just reduce brain plaques. The ENGAGE trial did not show the improvements they were looking for even at the high dose. However, the EMERGE study (after some adjustments) reported a 22% improvement in memory testing at 18 months on the drug in addition to expected plaque reduction on MRI scans.
In June this year, the FDA almost unanimously approved the drug, now called ADUHELM for treatment of MCI in patients with AD. That’s like most of my elderly patients in my clinical practice…!
And the price tag: $56,000 per year. You need 18 months by the way, so it’s close to 100K per patient. In this weeks New England Journal of Medicine, a commentary (Robinson, JC. 385. No22), estimates that if only 10% of the US population gets this new drug, spending for Medicare part B would increase from $37 billion to $69 billion per year. That’s a lot of revenues for an agent that showed small improvements in memory after a lot of manipulation.
I must come to a close.
My thoughts?
We need something for this disease called Alzheimer’s. It is our primary target for insuring our future and the future of our children.
We have failed thus far in providing even a trickle of a treatment to impact the outcomes of millions of Americans, many that are not quite there yet.
Is ADUHELM and anti-plaque therapies the long awaited answer?
Perhaps…but we need a fundamental change in how medicine advances occur: privatization of big pharma has provided many amazing breakthroughs in our world today.
Just 10 years ago, advanced skin melanoma was a death sentence. Monoclonal antibody therapies have transformed treatments…the rub?
The costs are astronomical.
We need real solutions for real problems treating real people like you, like me.
Please provide your own experiences, and personal opinions (without getting offensive politically, please).
This community will be impacted by the approval of this and other therapies that are in the pipeline to provide you and I a healthier and happier lifetime.
This is Dr Robert Dubin for Endocrine for You.
Thank you!