Medical Innovation: On the Edge of Change

Of the 10 most-common deadly diseases,
dementia (including Alzheimer’s disease) is the only one that has no
treatment. But—finally—we are on the cusp of potential diagnoses and therapies,
said panelists at an “Advancing Medical Innovation” conference yesterday in Washington, DC.

“The great news is if we develop therapies
for Alzheimer’s disease, which is very common, we’ll be developing therapies
for fronto-temporal dementia (which is less common) and Lewy body disease,”
said Dennis Selkoe of Harvard Institute of Medicine. “Rare diseases can
piggyback on common diseases, and all of them can move forward,” said Selkoe,
also a member of the Dana Alliance for Brain Initiatives.

Even the bad news—recent Alzheimer’s
drug trials failing to meet their treatment goals
—has silver linings, the panelists
said. In addition to learning more about the mechanisms of the disease, we have
learned how to run such trials better, for example. Also, the endpoints the
researchers set may have been too ambitious, and the population (people with
mild to moderate dementia) may have been too far progressed in the disease for
the drug to have much effect.

As Francis Collins, head of the National
Institutes of Health, put it later in the program, testing moderate-stage
people was “sort of like trying to get statins to work on people with end-stage
heart disease.” To succeed, we need to find a way to intervene earlier in the
disease, he said. By administering screening tests, which are not yet ready for
wide-range use but have been approved for experimental targeting, we
might find the cadre of people who would be ideal to test these compounds. And
if we do find that one type of drug works better in people who have one genetic
variant and another drug works better in people with another variant, which
seems likely, we can use newly developed genome-wide assays to distinguish
these groups of people.

Look for
news to come out around Oct. 8, Selkoe
said, such as “a big display of data that suggests a pathway to FDA approval”
in perhaps 3-4 years. “The FDA has said one well-done, rigorous trial will be
sufficient for approval; more trials will continue after approval. And there
are many shots on goal; not just one antibody.”

People are most excited about the idea of
vaccinating the population against dementia, Selkoe said. Alzheimer’s is not a
virus, but vaccines ramp up the immune system against many things: If we could accelerate
it to dissolve the plaques or tangles seen in degenerative diseases we might
use a vaccine as a preventive.

“We will be developing in the next few years
types of vaccines against Alzheimer’s,” Selkoe said. It won’t be like
pertussis, given to everyone, but maybe to people at highest risk and/or those
over 50. “In the future, you’ll treat Alzheimer’s starting at 50 in the doctor’s
office,” completing tests that use computer algorithms to determine your future
risks and requiring doctors to learn how to manage patient expectations and
predict their questions, he said. He estimated such testing might occur within
a decade.

“There’s a great deal of cerebral power being
applied to difficult problems” such as diagnosing dementia and searching for
treatments, Selkoe said. But another problem is getting what we know already in
the lab down to the doctors, nurses, and other caregivers in the clinic.

“It actually takes a while for practitioners—scientists
and doctors—to adopt the new knowledge,” Selkoe said. For example, in the
recent Alzheimer’s trials that did not reach their targets, researchers did
learn how to do such trials better—and that they need to figure out the right
group to target. One way is to look for biomarkers, including signs in
cerebrospinal fluid that suggest the disease is on the way. Unfortunately, the
way to get the fluid is to do a lumbar tap, and “everybody knows” taps are
difficult and painful. Even willing volunteer test subjects think twice when
asked to have one.

But according to Selkoe, doctors in Europe
have shown a way to perform the lumbar tap that doesn’t cause headaches, is
less painful, and allows people to walk out of the office with no problems.
That knowledge needs to spread so more people will have the procedure, giving
researchers a bigger pool to test with.  “We need to convince ourselves
that it’s not dangerous to have a lumbar puncture, if you have a doctor” who
does it right.

The session, held Thursday at the Ronald
Reagan Building, was hosted by Washington Post Live and sponsored by Lilly;
other panelists included Robert Egge of Alzheimer’s Association and Margaret
Anderson of FasterCures/The Center for Accelerating Medical Solutions. Other
sessions included STEM and scientific literacy, fostering innovation and job
growth, and more. The event was streamed live; Twitter users used the hashtag #medinnovate. Washington Post Live has posted video highlights.

–Nicky Penttila

[update 9.21.12, adding link to video highlights]

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