DEPARTMENT OF HEALTH, EDUCATION, AND WELFARE

PUBLIC HEALTH SERVICE . BETHESDA 14, MD.

 

NATIONAL INSTITUTES OF HEALTH
Tel: 656-4000

March 22, 1962

In reply refer to:
NBI-D

Dr. Joshua Lederberg
Department of Genetics
Stanford University
Medical Center

Palo Alto, California

Dear Dr. Lederberg:

Your letter of March 6 raises some very interesting questions
regarding the evaluation of drugs prior to their release for general
use, As you may have noted, a tranquilizing agent in common use in
Germany has recently been demonstrated to be responsible for an
"epidemic" of an unusual form of congenital malformation.

It is quite evident that the use of anticholesterol agents has serious
hazards. I understand that triparanol has been withdrawn from public
use because of untoward side effects--particularly cataract.

One of the most serious concerns to which I have recently become
sensitive has to do with the use of drugs during early pregnancy. It
would seem that any proposed new therapeutic agent should be tested
for its effect on the fetus before being released for general use.

iO / puss ayy

Dr. Roscoe Brady of our Institute is particularly concerned with fatty
acid synthesis in the brain and actually has a manuscript in prepara~
tion dealing with investigations on the inhibition of fatty acid
synthesis by brain enzymes, This is not quite the identical problem
to which you refer in your letter, but is in a closely related field.

The whole problem of evaluation of new drugs--not only for toxicity
but also for effectiveness--is a source of serious concern to this
Institute. There is a constant stream of proposed new therapies for

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mental deficiency. Some of these obviously are "quackery"--for
others, there is a sufficient kernel of possible usefulness to cause
us to feel some obligation to conduct a therapeutic trial. However,
few scientists have the enthusiasm to involve themselves in the
expenditure of time and energy which is required to conduct a good
field trial of a proposed new therapy--certainly unless there is
strong presumptive evidence that such a therapy should be useful.
Meanwhile, the public is spending millions of dollars for useless
treatments. It has thus developed that one of our major worries has
to do with the evaluation of newly recommended therapies, and we are
laying the groundwork for clinical trials in certain limited areas,

Thank you very much for bringing to my attention this particular
specific problem which certainly relates very closely to our area of
interest.

Sincerely yours,

Richard L. Masland, M. D.

Director, National Institute of
Neurological Diseases and Blindness