SPEECH OF HONORABLE JOHN E. FOGARTY, 2nd CONGE NAL DISTRICT OF RHODE
ISLAND, AT LUNCHEON HONORING DR. SIDNEY FARBER THE GOTHAM CHAPTER OF THE
NATIONAL FOUNDATION FOR MUSCULAR DYSTROPHY, INC., ON MAY 4, 1957, IN THE
EMPIRE ROOM OF THE WALDORF ASTORIA HOTEL, NEW YORK CITY.

Mrs Toastmaster, Mayor DeMuro, Dr, Farber, members and friends of the
National Foundation for Muscular Dystrophy. It gives me a feeling of beth
pleasure and pride to be here today. I am pleased at the opportunity to
address a group which is concerned with public health and medical research
problems and is doing something about them. And, I am proud of my aoquaintance-
ship with the man who is being honored here today.

You may wonder how it is that I have come to know Dre Sidney Farber. a Et
The fact is that--on mumerous oceasions--Dr. Farber has come before committees ce
of Congress to report on the problems, the needs, and the progress in medical,
science. He bar found the tine to do this deapite his buy career An researty
teaching and medical service, Advice such as his, freely given in the public .
interest, has been of tremendous value to the Congress which mst have the best
possible counsel if it is to legislate with wisdom ws . ie :

I do not believe that the National Foundation for Muscular Dystrophy” a a
could have selected a more deserving individual for the award being bestowed
teday. His contribution to the furtherance of medical research has been a |
magnificent one, His status in the field of cancer research is that of a
leader as well as of an innovator, And what he has done for the well-being ~~
of children is not susceptible to statistical measurement, m

_ But perhaps most important of all, Dr. Farter is that precius «sss
intermingling of scientist devoted to precision research and humanitarian
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devoted to the well-being of his fellow man. It seems to me that the truly
great scientific achievements of our era have been the product of precisely>
trained minds which were dedicated to humanity and the general welfare.

I should like, in the next few minutes, to talk with you about some
of these achievements in the medical research field, And I should like,
if possible, to put those achievements in perspective. In short, I think
it is as important to know how far we have yet to go as it is to know how
far we have come. ,

And the distance in both directions is considerable!

As you may know, I have spent some 17 years in Congress. For more
than half of that time, I have been directly concerned with health and welfare
matters. For most of the past nine years, I have served as chairman of the
House subcommittee dealing with appropriations for the Department of Health,
Education, and Welfare and its predecessor, the Federal Security Agency.

So you might say that I have been able to observe the medical progress
of the postwar decade from an excellent vantage point. We have, indeed, taken
some giant strides in thet decade.

Let me cite just a few examples.

A whole new field of chemistry--better known as chemotherapy--has
opened up for the treatment of that terrible killer, cancer. Chemicals are
now making it possible to control and alleviate some forms of cancer which
formerly did not respond or did not respond as readily to the surgeon's knife
or to radiation.

The infectious diseases are lending themselves more and more to control
in the laboratory and in the clinic, Polio is but one case in point.
3.
: The progress in the heart field has been little short of phenomenal.
In a few short years, it has become possible to operate on heart eondiclens
involving the arteries leading to the vital organ as well as the heart itself--
conditions which previously would have doomed the individual to death, or, at
best, to that tragic twilight of existence in which the least expenditure of
energy was considered too great.

Why, only the other day, I read that some scientists had even gone to
work to perfect a “substitute”heart which would replace the original when it
faltered. I am sure this 1s. speculative and very experimental work--but it does
demonstrate how rapidly the frontiers in science are being pushed back.

I could go on and cite dozens of other examples of progress against
the cripplers and killers of our time. Yet, it is well for us to bear in
mind that the terms “progress” and "conquest" are not synonymous.

We have made advances against cancer. True enough. Yet, about
250,000 Americans died ef cancer last year. ;

The Salk vaccine appears to have opened the way to the ultimate
defeat of polio. But, last year, there were still more than 15,000 polio
cases reported in this country.

Wonders oes been done with heart therapy. But in 1956, no fewer
than 800,000 Americans died of heart disease.

ear do I after you thece clenente of stexh esntrest? dn I ay the
less pleased with the progress we have made? Should any of us be any the
less pleased?

Of course not!

I am merely suggesting that--hard though it may be for some of us--

we must temper our desires and expectations with the soothing balm of
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patience and understanding. We must, perhaps, ind a bit and take stock of
our national medical research effort. We must distinguish between medical
miracles--which seldom occur--and solid advances, which often do. We must,
in short, kook at medical research in perspective.

I am pleased to report that our most recent action in the House,
relative to appropriations for the National Institutes of Health, reflected
that attitude in full. I might add, parenthetically, that I think these
Public Health Service, tax-supported research programs have certainly fulfilied
the expectations which brought them into being. The research at Bethesda and
the research and training supported in hundreds of institutions throughout
the country through needed hte accomplished « great deal and certainly &
paved the way for many conquests to come.

Particularly pertinent to the progress that has been made in the fight
against dieease is the very fine cooperation that has deve loped between public
and private organizations--a cooperation which has grown particularly close
in the post-World War II period. Public and private research efforts have
complemented each other in nourishing the research effort. And the role played
by voluntary organizations such as the National Foundation for Muscular Dystrophy
both in fostering research and in educating the public! as to its importance
has been of immeasurable benefit to the nation.

In carrying out their broad educational efforts, I think the voluntary
groups have been particularly careful to distingéish between conquest of disease
on one Rees and progress on the other. And in no disease area is this distinction
more important than that into which miscular dystrophy falls. I speak, of course,
of the neurological and sensory disorders and--with your indulgence--I should like
to concentrate on the problems presented by these diseases and on what is being
‘done about them at the National Institute of Neurological Diseases and Blindness.
in Bethesda.
+e.

The National Institute of Neurological Diseases and Blindness, ws many
of you know, is the "baby" of the seven Institutes which make up the National
Institutes of Health. However, it is a lusty baby.

And, though the U. 8. Public Health Service has been doing medical
research since 1887, the National Institutes of Health as such are just out
of their swaddling clothes. If I remember correctly, only one of the seven
Institutes--the Cancer Institute--was organized prior to 1948.

So, in effect, the progress in medical research made possible through
the programs of the National Institutes has been achieved within less than ea
decade.

But, I have digressed. For it is withihe National Institute of
Neurological Diseases and Blindness, and with its research attack against the
neurological and sensory disorders, that this group is most directly corcerned,

In the five years since its establishment as an operative organization,
the Institute has been concerned with the crippling disorders which attack the
nervous system and which afflict an estimated 20,000,000 Americans today.
Muscular dystrophy, cerebral palsy, mental retardation, epilepsy, multiple
sclerosis, blinding diseases like glaucoma and uveitis--these are among the
many disorders which the Institute and its many cooperating research
organizations are attacking.

When you stop to think that the diseases I have just named are among
the “unknowns” of medicine--that up to a few years ago we had almost no
information as to the specific factors which bring them on~~you can get
some ides of the scope of the problem they pose, What I am saying is that
6.
eything Yaieh science has been able to discover about de totus and causes
of these neurological and sensory disorders represents very substantial progress.

And--in those cases where effective treatment has dateatty been developed--
progress has bordered on the miraculous.

I can perhaps illustrate my point best by directing your attention to
one disorder which today afflicts an estimated 500, 000 American men, women
and children. It is disease vhich I om sure Br. Farber here hes more then
@ passing acquaintance with inasmuch as it makes its presence felt very early
im life. I refer to cerebral palsy-er--as some doctors put it--the cerebral palsies.

A ftiend of mine has referred to cerebral palsy o "a blank spot on
the map of disease.” He is, I hth, winy close to correct. For I understand
that there is still disagreement emong the doctors as to exactly what can-
stitutes the disease. |

Qn these facts, however, thers is pretty general agreement: Cerebral palsy
is brought on by factors active during the ~~ perinatal period--the tims
span from about one month after conception to about one month after birth. It
may, however, not manifest itself for several years and--in the past--it has
not always been correctly diagnosed when it has manifested itself. The
disease, which results from damage done to the brain in its developmental
stages, is generally characterized by loss or impairment of control over
voluntary muscles.

I don't think that I have to tell this group anything sbout the econcate,
social and psychological cmatquenees of the disorder either for the individual
afflicted or for his immediate family. The cost in these tragic terms is
immeasurable.
7

What then, is the Institute at Bethesda doing about cerebral palsy?

For one thing, the Institute has launched a nation-wide collaborative
investigation directed to determining exactly what goes on during the perinatal
period to bring on cerebral palsy. The investigation is also designed to
develop crucial information pertinent to the research attack against such
related diseases as mental retardation and epilepsy and various forms of
blindness and deafness.

When this broad investigation is in full ps it is expected that
15 research organizations throughout the country will have joined the Institute
in carrying it out. The study may go on for as long as a decade before all
the sought-for data are collected. Eventually, as many as 15,000 cases may
be under investigation each year.

It is the hopeof the Institute that, once the data have been collected
and analyzed, medicine will have a relatively precise idea as to exactly how
cerebral palsy and related disorders are caused. And, given such information,
it is felt that effective preventives and therapy would not be too far away.

I have been told that there is one type of cerebral palsy for which
research has already developed a treatment effective in many cases. I refer 7
to kernicterus, which is better known as the “yellow jaundice” of infancy
and which generally attacks within the first 48 hours after birth. Kernicterus
is traceable to the so-called Rh factor--blood incompatibility between mother
and child.

An Institute grantee discovered that a substance known as bilirubin
was excessive in the blood of those exposed to the disease. He developed a

specific test for the bilirubin level in the blood as well as a miltiple

‘
~*

+. Sacknique
transfusion fér reducing that level.
8.

When the blood transfusions are made immediately after birth,
kernicterus is often prevented, :

I.should like, very briefly, to review some of the other very promising
developments in the nsurological snd sensory disease field over the past
several years.

Toxoplasmosis infection is a virulent form of the blinding disease,
uveitis. The infection is brought on by a parasite. Last year, a new
diagnostic technique was developed which permits the early detection of this
infection and--in so doing--will make possible the prevention of many cases
of blindness.

Or take brain tumors.

One of the major problems confronting the surgeon who suspects a
brain tumor is to locate it in specific terms without having to resort to
exploratory surgery. Another, perhaps even more basic problem is to locate
the tumor in time since many tumors do not readily lend themselves to early
detection by existing methods.

During the past year, investigators at the National Institute of
Neurological Diseases and Blindness told me that they have developed a brain
tumor detection technique which is more than 80 percent effective. The device
precisely defines the size and location of growths, many of them Jeunteeeenell’
in the brain. Efforts are already underway to increase the technique's
effectiveness.

Still another development of interest has to do with a blinding disease
known as retrolental fibroplasia. Over the past decade, this disease has blinded
thousands of premature infants. Today, as the result of a coordinated nation-

wide research attack involving 18 medical institutions, retrolental fibroplasia

is rapidly disappearing.
How did it happen? The explanation is almost too simple. A
researcher working with animals found a clue indicating that retrolental
fibroplasia might be due to excess oxygen. The nation-wide investigation
I have just mentioned was launched to check the finding, The finding was
confirmed and now--through the judicious use and control of oxygen in
prematurity cases--the disease is on the verge of extinction.

A report which came to me only a few weeks ago shows that--here in
New York City--the incidence of retrolental fibroplasia has dropped
seventy-eight percent in one year.

Now, the developments I have just described are most encouraging.
And there are others of equal importance that I could point to in the
neurological field.

But--and I feel I must be absolutely frank about this--there are many
diseases for which the outlook is not nearly as encouraging. I regret to
report that the neurowuscular diseases and muscular dystrophy are among these v
diseases,

That is not to say that a cure or effective treatment for muscular
dystrophy will not be achieved. Mor is it to say thet gush cure or treatment
is necessarily still many years away,

It is only to say that the current st@tus of our knowledge of this
disease is such as to indicate that cure and preventionare not Just around
the corner. |

As you know, scientists are not even sure of the tustitie causes of
muscular dystrophy. They do know that it is a progressive, non~contagious
wasting disease of the muscles. And they suepect that faulty metaboliem
Ww,
may have something to do with bringing it on. Some scientists think that
the inability of the body to utilize Vitamin E properly may play «a oan: |

But it is not really known for certain. | |

As for effective treatment, a wide variety of substances and diets
have been used in an effort to control or halt the disease. But, to date,
there is no substantiated evidence that any of the methods used are truly
effective over the long run. ,

Mow, does this mean that we are making i casia ah es premnes eanbets
muscular dystmphy. .. . that little is being done about this and related
. disease? 3

The naswer is a clear-cut “no,”

At the National Institutes of Health in Bethesda, hundreds of thousands
of dollars are being spent on basic research into ei eee and function of
the nervous system. Additional hundreds of thousands are being expended
through cooperating research projects directed toward the same ends. This,
in addition to the funds made available from private sources.

Por if there is to be a cure or effective treatment for muscular
dystrophy and related neuromiscular diseases such as myasthenia gravis, our
most direct approach is to learn the specific causes. And we cannot normally
expect to know those until we know exactly how nerve impulses are transmitted
from nerve to nerve and from nerve to muscle.

We are handicapped in our search for cures and preventives if we are
unable to explain exactly what happens when nerve impulses are blocked, as is
the ease in neuromuscular diseases. The muscles just do not get the “message”

from the nervous system.
7 i *

Today--at Bethesda and elsewhere--scientists are hard at work trying
to find out what blocks.that message and the specifics of why and how the
nervous system operates in its relationships with other parte of the body.

Experiments are underway to deteruine the impact of temperature, pressure
and usage upon nerve fiber.

A new electronic device has been developed which detects and accurately
measures even small amounts of a chemical called acetylcholine in the nerve
fibers. This chemical plays an essential role in the nerve impulse trans-
mission,

An ingenious micro-electrodse recording device has been devised which
makes it possible to measure electrical impulses of millionths of a volt
lasting for only millionths of a second in the so-called synapse--the space
between nerve endings.

Now, these developments are in what is generally called the basic
research field. They are perhaps not as dramatic as some which have been
widely publicized, But they are every bit as important. They are the
stepping stones we must have if we are to span the sea of knowledge that lies
between us and the effective treatment and cure of muscular dystrophy.

I think, my friends, that you can see that there is hope--much hope,
There is aleo much work to do. Much has been achieved. Much remains to be
achieved, Much more will be achieved. 7

There igs no question in my mind but that the National Foundation for
Muscular Dystrophy--like the other voluntary groups concerned with the
conquest of disease--will have much to do with writing the record of

this achievement.
'e

” a alae ean a ee
Se ee Se en

a 2 &

As those of us concerned with medical research look ahead, we can
see the broad outlines of the work which has been cut out for us. But we
can also take heart from the perceptive words written by Samuel Taylor
Coleridge: eck without hope draws nectar in a sieve, and. hope without
an object cannot live.” | 3 |

Poet Coleridge's little verse is, I think, s most appropriate theme
on which to finish. I thank you very much. 7