The U.S. Food and Drug administration (FDA) gave full approval today for Japanese drugmaker Eisai, and its American partner, Biogen, to market its latest medication, lecanemab (to be known as Leqembi) for those with mild Alzheimer’s disease (AD). According to Teresa Buracchio, acting director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research, “Today's action is the first verification that a drug targeting the underlying disease process of Alzheimer's disease has shown clinical benefit in this devastating disease."
Six months ago the FDA gave conditional approval for this
new medication, but there was much debate in the medical community about the
effectiveness demonstrated by its data. Medicare
said it would not pay for routine use of Leqembi until the FDA gave its full
approval. Today the FDA has given its
full approval. The
FDA acknowledged that after an 18-month clinical trial with 1800 participants,
results showed that those taking the new medication showed a significant difference
on an 18-point scale measuring cognitive functioning and memory. According to FDA, this study showed that
Leqembi “slowed memory and cognitive decline by about 5 months for those
receiving the medication as opposed to those receiving the placebo.”
A doctor quoted this
afternoon in a USA Today article said, “This drug is not a cure. It
doesn't stop people from getting worse, but it does measurably slow the
progression of the disease. That might mean someone could have an extra six
months to a year of being able to drive."
A doctor quoted this afternoon in The New York Times
stated, “The risks are very
vivid. Within the first few months, you
may have small bleeds or swelling in your brain, which may or may not be
symptomatic and if not detected in time can cause disability.”
The
FDA indicated that Leqembi will carry a” black-box warning” about possible side
effects such as brain swelling and bleeding. The warning does not mention patients who are
taking blood thinners, but Leqembi’s label says that “additional caution should
be exercised” when considering whether to give blood thinners to Leqembi
patients. As also noted in The New
York Times article, “Concerns about safety have been stoked by
reports of deaths of three clinical trial participants who
experienced brain swelling and brain bleeding, two of whom were being treated
with blood thinners. Eisai has said it is unclear if Leqembi contributed to
their deaths because the patients had complex medical issues.“
In my Personal Blog #28, I noted that ”17% of the patients taking this medication suffered cerebral micro and macro hemorrhages and superficial siderosis compared to 8.7% in the placebo group.” I also shared my feelings in PB #28 about medications yielding “statistical differences,” noting that although statistically significant those differences might not result in “real world” differences.
This
new IV medication is priced at about $26,500 for a year’s supply of injections
that are to be given every 2 weeks and Medicare will now cover about 80% of its
costs. This still leaves patients with 20%
of the cost, plus other related expenses for doctor visits, required scans,
etc. Personally, I’d prefer that Medicare
spend its money more wisely than subsidizing patients taking this new
medication. And I think patients can
better spend their money on support groups, saving for the future when home
health aides or assisted living or nursing home facilities may be needed, or
for other present expenses or future costs.
So what is my take on this FDA approval of Leqembi? Whereas I am truly pleased that we now have a
medication that has shown it may be able to slow cognitive decline and memory
loss by up to 5 months in an 18-month period of real time, I don’t think my
late wife would have considered taking such a medication. Why?
Three reasons:
First, getting IV injections every 2 weeks for a year would
have been a very stressful experience.
Just getting ready to go and driving to and from the doctor’s office to
receive IV injections twice a month would have been stressful. In addition, my wife always felt pain with
injections, bled easily, and her skin turned all pretty colors whenever
receiving a needle for any reason … possibly due to all the heavy-duty heart
medications she took each day, including 325 mg of daily aspirin. Even had my wife started this medication, I don’t think she would
have continued for too long. Those IV needles would have been painful as well
as stressful.
A second reason is the side effects. AD is serious enough as it is, a death
sentence with no cure and the 100% certitude of worsening conditions. Gaining a few additional months of time to
continue leading a more “normal” life before cognitive and memory issues worsen
even more would be wonderful … but what a heavy price if those side effects
occurred. Dealing with AD and heart
disease was enough of a double whammy for my wife. Possibly compounding that with brain swelling,
bleeding, headaches, etc. due to this medication could have made her situation
even worse than it already was
The final reason is the real world “effectiveness” of this
new medication. Study participants
taking the medication showed a gain of less than one half of one point on
testing … a statistically significant outcome, perhaps, but I question how much
of a clinical difference that difference would have really made in my wife’s
life or in mine. As that doctor quoted in
The New York Times noted with respect to the slowing of decline with
Leqembi, “The benefits of slowing are
subtle. You’re not going to experience the perception of changes in your
cognition or function in the same amount of time.” As I noted in PB #28, not all studies demonstrating
results showing “statistical differences” in testing translate into meaningful
and/or observable differences in the real world.
By the time my wife was in early stages of AD, our lives had
already changed dramatically. A few more
months of slower decline would have been welcome, but would probably not have
changed much about our daily lives. Especially
when one considers that the trade-off to perhaps gaining a few months of slower
decline might have led to losing the quality of life we still had. Slowing decline doesn’t mean ending it and we
may not have even noticed that slowing. The
added stress of two more doctor visits each month, the pain and after-effects
of IV needles, and possible additional pain or hospitalization due to severe
side effects of this new medication could have easily lessened whatever quality
of life we still had.
So what is my bottom line reaction to this FDA approval of
Leqembi? It is this: Would I have loved my wife to have declined
at a slower rate for a few more months in those early stages? Of course.
Would I have loved to have taken more trips with her? Absolutely. But worrying about the possibility of her
having a serious brain hemorrhage or other serious side-effect while traveling away
from home would have worried me too much to have taken that chance. In fact, I
would have been worried every single day, whether at home or not, had she been
taking this new medication.
I haven’t even mentioned another real word effect of this
new medication … the demands place upon the care partner of having to bring the
AD patient to and from doctor appointments.
Even in early stages, AD patients may need assistance in dressing, mobility,
may object to going out in bad weather or going out at a certain time of day,
etc. In addition, many care partners of
AD patients are still working when their loved ones are in early stages, and time
demands of taking loved ones to two visits each month for IV injections could
be an additional burden for them as well.
So whereas I am truly pleased to welcome a new medication to
try to help those with AD, I would not have found Leqembi worth the risk.
