17
‘This is the very
first time in the
field that we have
had the type
of positive news
that could be
transformational.’
MARIA CARRILLO,
chief science officer,
Alzheimer’s Associationplacebo. The only distinction was that
one study began earlier than the other.
That made a difference, Haeberlein
believes, since Biogen changed
protocols of the study after patients
began enrolling. Importantly, the very
first people to volunteer were given
much lower doses of the drug if they
had a mutation of the ApoE gene that
raises a person’s risk for Alzheimer’s—
and also seemed to raise the risk of
experiencing brain inflammation as a
side effect of aducanumab. About two-
thirds of the people who participated in
the studies carried this high-risk gene.
New data that became available after
these patients began receiving their
infusions, however, revealed that slightly
higher doses did not actually result in
a marked increase in side effects. So
these patients were then given higher
doses. But many had either not yet or
just begun their new doses when the
company analyzed its data on the first
half of patients last spring and decided to
end the study.
Over the summer, after seeing
the positive results from all the
participants, Haeberlein grew skeptical.
She dug deeper, breaking out how the
patients receiving the lower doses fared
on their cognitive tests. Those people
showed some improvements—more
than the placebo group but not as great
as those at the higher doses. “That was
when we as a team said, ‘Holy cow!’ ”
She walked across the Biogen campus
“on wobbly knees” and pulled Sandrock
out of a meeting, saying, “We’ve got
to talk.”
Other data supported the positive
effect of aducanumab. For example,
brain scans measuring changes in the
amount of amyloid buildup showed
declines in the plaques among people
getting the highest doses of the drug.
“Patients can’t influence the proteins
in their brain,” says Haeberlein. “These
wonderful readouts gave us further
confidence that what we were looking at
was very strong.”Biogen is noW Working with its study
sites and the FDA to launch a new trial,
in which the original study participants
would be invited back to receive adu-
canumab (with no placebo group). The
news is providing much-needed hope
for the Alz heimer’s field, which has been
battered by a series of failed drug tri-
als over the past decade. “There are lots
of implications for these results,” says
Maria Carrillo, chief science officer of
the Alz heimer’s Association. “This is the
very first time in the field that we have
had the type of positive news that could
be transformational.”
After his study was stopped in the
spring, Peter joined another trial testing
a different type of experimental treat-
ment. To join Biogen’s new study, he
will have to wait for it to be launched
and then for the drug he is currently
testing to wash out of his system, which
could take months. Even with those
hurdles, would he start taking adu-
canumab again? “I would, I would,” he
says. “Hopefully we can make a differ-
ence with this disease. We have to stick
with it and make a touchdown.” □