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New York Consortium for Alzheimer Research and Education
The New York Consortium for Alzheimer Research and Education (N.Y.C.A.R.E.) is the joint effort of
the Alzheimer’s Association New York City Chapter and the Education and Information Cores of the
Alzheimer’s Centers — Columbia University, College of Physicians and Surgeons; Mount Sinai Medical Center; and New York University School of Medicine funded by the National Institute on Aging.
An Embarrassment of Riches,
or “Which Drug Study Do I Choose?”
When faced with a progressive neurodegenerative
disease such as Alzheimer’s
disease, many patients and their families
choose — for intellectual, emotional, personal and altruistic
reasons — to consider enrolling in an investigational
drug study, or “clinical trial.” One perceived advantage is
personal — as a participant in a drug study, a person with
Alzheimer’s disease may receive a treatment that is effective
against the disease, some years before the treatment
is proven and available on the market. Another advantage
is altruistic — the patient and their caregiver will be
part of an “experiment” to prove whether a drug is truly
effective or not. This is enormously important, because
if people didn’t volunteer for studies, medical progress
could not occur. Participating in a drug study requires
effort and time. Participants undergo many more medical
visits and many more procedures than would occur
with usual clinical care. This may include blood tests,
urine tests, spinal fluid tests, electrocardiograms, MRI
brain images, various types of PET brain images, cognitive
testing, questionnaires or other procedures. And of
course nearly all drug studies are “placebo controlled.”
This means that some of the participants receive an inactive
placebo (sugar pill) for all or at least part of the study.
Many drug studies are also “blinded, randomized” trials,
which means that participants are randomly selected to
receive either a placebo or the active drug (i.e., the drug
that is being investigated), and that no one — including
participants and the study team — knows who has been
selected to receive the placebo. Many placebo-controlled
studies are followed by an “open-label” extension
study in which ALL participants receive the active drug
for a specified period of time or until the active drug is
either approved or proven to be ineffective or unsafe.
Once a patient and caregiver have decided they are
interested in a drug-study trial, they are faced with the
decision of which drug study to choose. Thanks to recent
advances in our understanding of Alzheimer’s disease,
primarily the result of brain-tissue autopsies and
animal-research studies, there are now many Alzheimer’s
drug studies available. So, how does one decide which drug study to consider? Should one base this on rumors
from Wall Street? Internet-site buzz? Catchy radio advertisements?
Study doctors or location? Study participation
payments? There are many factors to consider,
which include issues of personal preference, convenience,
drug and procedures. What may seem easy to one person
may be out of the question to someone else.
Location of the drug study: A clinical trial may
involve many visits to a hospital, doctor’s office or clinical
research center. Typically a caregiver must accompany
the patient for most or all visits. Distance from home to
study site may be an important factor. This depends on
individual mobility, whether using car or public transportation,
and on caregiver time schedule and availability.
Some clinical trials offer home visits, which may prove
convenient for some participants.
Eligibility: Every study has its own specific entry
criteria which are used to determine if each individual is
eligible to participate in the study. These entry criteria
include an allowable age range, mental status examination
performance score, laboratory values, MRI findings, and
various criteria related to medical history and medications.
Sometimes (often??) a patient may not be eligible
for the drug in which they are interested. In many cases,
a patient may be eligible for multiple studies. However,
one can only be enrolled in one drug study during any
given time period.
Drug study benefits: For many people, the possibility
of personal benefit is a key issue. While an investigational
drug study cannot guarantee any direct personal
benefit to participants, many people view the possibility
of receiving the active drug as a potential benefit. As
mentioned earlier, most drug studies are placebo-controlled.
The likelihood of receiving the active drug is
different for each study but often ranges between 50%
and 75%. Some drug studies include a “delayed-start”
design in which all participants who complete the study
ultimately receive the active drug. And some placebocontrolled
studies are followed by open-label extensions
during which participants are offered the active drug.
Patients and caregivers often want to know which
of the available investigational drugs is likely to be effective.
It is important to understand that clinical trials
are designed to answer questions about the safety and effectiveness of new drugs. There are a variety of opinions
regarding any particular experimental drug. Drug
studies are not generally carried out on drugs that are
thought to be without any potential benefit, or on drugs
that are found to be unduly risky.
Drug development occurs in 3 stages prior to approval.
Phase 1 studies are preliminary safety studies
that are rather short in duration. Phase 2 studies may be
“exciting,” with study of the effectiveness of more novel,
untested agents. Phase 3 studies are based on Phase 2
studies suggestive that the drug development is worth
pursuing. Increasingly, Phase 2 studies are larger and
longer, and there is less distinction in Alzheimer’s studies
between more advanced Phase 2 and less advanced Phase
3 studies.
Drugs have different proposed mechanisms of action.
While scientists may argue that one or another therapy is
more promising, these drugs are investigational because
no one is certain which mechanism of action, or which
drug based on a given mechanism, is going to be proven
to be safe and beneficial. Many patients find the careful
clinical, laboratory, and neuroimaging monitoring — as
well as frequent access to experienced Alzheimer-care
study team members — a desired study benefit not specifically
related to a drug.
Drug safety: Each person has their own degree
of risk-aversion. No drug, experimental or marketed, is
without risk. In general, the side-effect profile is known
better in marketed drugs than on drugs in Phase 3 testing,
and usually known better for drugs in Phase 3 testing
than for those in Phase 2 trials. Some risks may be
more worrisome to particular patients. For example, if a
patient has a history of stomach problems, they may be
less inclined to consider a drug with potential gastrointestinal
side-effects, although fortunately side-effects are
not guaranteed.
Drug administration: Some studies involve oral
medications, usually taken either once or twice daily.
Other studies involve injections or intravenous infusions
that may be as frequent as weekly or biweekly, or only
once every 1 to 3 months. Often patients and caregivers
have a personal preference as to whether daily oral medications,
injections or infusions are more desirable.
Visit procedures: All studies involve interview,
examination, EKG, blood and urine tests, and cognitive
testing. Studies may involve various numbers of CT or
MRI brain imaging, PET brain imaging, and lumbar
puncture (spinal tap) to provide for analysis of cerebrospinal
fluid.
Visit frequency: The frequency of visits varies from
study to study, but most studies require more frequent
visits initially and less frequent visits as the study progresses.
Visits may occur weekly, biweekly, monthly or
once every 2 or 3 months. The amount of time required
for each visit also varies. Some studies may include visits
or treatments at home. Study duration varies from study
to study and may be as short as 12 weeks or as long as 2
years.
For patients and caregivers who are interested in participating
in clinical trials for Alzheimer’s disease, this is
an exciting time. There are a number of important clinical
drug studies ongoing for the treatment of Alzheimer’s
disease. This is promising and gives stronger hope that
one or more of these drugs may be proven safe and effective.
But it does also present patients and caregivers with
choices to be made as to which study is best for them,
each considering their own personal circumstances and
preferences.
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