An Interview with Wise Young, MD
Part 2
In August 1998 we interviewed internationally-renowned SCI research
scientist Wise Young, MD, PhD, director of the
Neuroscience Center and the Spinal Cord Injury Project at Rutgers
University. In Part 1 of the interview, published in the
Summer 1998 issue (Vol. 8, No. 2) of this newsletter, Young described
current avenues of nerve cell regeneration research
taking place around the world. Following is the second, and final,
installment.
Q: You have predicted that effective treatment for SCI will be available
around the year 2003. How
did you arrive at this time frame?
A: Christopher Reeve asked me two years ago how long it would take for
the first treatments for chronic spinal cord injury to
be available for people. At that time, I told him that it would take a
minimum of seven years. Christopher said that we should
make seven years the goal. It is important to understand that the
seven-year time is a goal and not a prediction.
Many obstacles lie in our path to a cure. The foremost obstacle is the
limited funds that are available to develop and test
therapies in clinical trial. At present, there is limited industry
interest in finding a cure for SCI. The NIH (National Institutes of
Health) is funding about $60 million of SCI research a year; most of
this research emphasizes basic mechanisms of cell injury
and growth. Clinical trials are not guaranteed to succeed and each may
take 3-4 years to complete. Every trial is like a throw of
dice; ideally, a clinical trial has a 50% chance of yielding a positive
result. To ensure that we have a "hit," we must do many
parallel trials. For example, the probability that we get an even number
on a dice throw is 50%. We may have to throw the dice
several times to get an even number. However, if 5 people were to throw
the dice at the same time, the probability that we will
have one or more even numbers is virtually certain.
We therefore have to push hard for many groups all over the world to
throw the dice. This will require at least a doubling of the
current funding level for SCI research from government, private
foundations, and industry. If we are able to get five clinical trials
going by 2000, I believe that we have a good chance that one or more
will be successful. That is the basis of the goal.
Q: How long before treatment will be widely available for people with
new injuries as well as those
with long-standing SCI?
A: The first therapy that improves function in chronic SCI will
probably be a drug called 4-aminopyridine (4-AP). A clinical
trial is currently being carried out to determine whether 4-AP improves
function. If the results are positive, this trial may lead to
limited FDA approval. If so, the drug may become available before the
year 2000.
If the current studies of IN-1 and L1 (see Part 1 of this interview,
Summer issue) show that these are safe therapies, they may
go into clinical trial by 2000; if so, it will take three years for the
trials to be completed. If either of those turns out to improve
function in humans, the first regenerative therapies may be available by
2003. We currently don't have an answer to the question
of whether any of these therapies work for people who have had their
injuries for many years. However, I believe that we have
the tools to kick start cells to grow again in the spinal cord. After
all, we know that cells will begin growing after an injury and
therefore have been systematically studying the spinal cord to find out
what triggers the growth of these cells.
Q: One hears claims of successful procedures and treatments, such as
shark fetal cell transplants
and omental transplants, that are only available outside the United
States. Why aren't these
procedures being stuied in this country? How can a lay person know which
new SCI treatments are
legitimage and which are "bogus"?
A: While we should not dismiss these therapies, most of the claims for
them are not based on convincing scientific data.
Therapies must be tested in rigorously controlled clinical trials in
which subjects are randomly assigned to receive either the
experimental therapy or standard therapy for comparison. Both the shark
embryo and omentum transplant therapies involve
surgery. Unfortunately, it is difficult to do controlled clinical trials
with surgical therapies because it is hard to operate on
somebody and not do anything. The only approach is to compare two
surgical therapies. Unfortunately, none of the groups
studying these therapies has attempted a rigorous comparison between
decompressive surgery with shark embryo or omentum
transplants and surgery that simply decompresses without transplants. Of
course, all treatments must be judged by the risks and
benefits that they offer. Because both shark embryo and omentum
transplants involve invasive surgery, the risks are not trivial.
To my knowledge, the benefits have been modest and not yet convincing.
Doctors who push unproved therapies are not
necessarily evil or bad doctors; they may truly believe that their
therapies work and that they are helping their patients.
There are several telltale signs of unproved therapies. If a person or
family is considering an experimental therapy, the following
are simple and common-sense things to do:
1.Ask whether the claimed treatment results have been published in a
peer-reviewed medical or scientific journal. Most
journals require rigorous proof of efficacy before they allow a
claim to be published.
2.Be wary of people who ask for a lot of money for an experimental
therapy. A common asking price for unproved
therapies is $50,000-$100,000. Most legitimate clinical trials will
not ask for large payments for unproved therapy.
Generally, if a therapy has a great deal of promise, the therapy
trial would be supported by a company or an NIH
(National Institutes of Health) grant.
3.Investigate the risks. Most therapies have risks. Carefully weigh
the risks and benefits of the therapy. Many people
believe that they have nothing to lose. Everybody has something to
lose.
The Spinal Cord Injury Project can be reached at Neuroscience Center,
Rutgers University, 604 Allison Rd., D413,
Piscataway, NJ 08854-8082 (732-445-6573,445-2061;
SCIProj(AT)biology.rutgers.edu).
=A9 1998 Northwest Regional Spinal Cord
Injury System