Ch.4 — The Pharmaceutical Industry • 67 
It remains to be seen liow many patients will 
ibe bettei’ oft \\ itb human insulin. The |)root that 
lit improv es tlu*ra[n w ill take years. F’rogress on 
|the etiologv ot the ilisease— espeeially in itlenti- 
living it in those at risk or in improving th(> 
iiiosage form and administration of insulin— may 
have far more significant effects than new de- 
\el()[)ments in insulin {)roduction. Nevertheless, 
as long as priv ate enterprise sc?es fit to inv fst in 
|such develo[)ments, and as long as the cost of 
{treating diabetics w ho rtvspond pi’operly to ani- 
linal insulin is not inci'eased. biological produc- 
'tion of human insulin mav hcH ome a kind of in- 
Isurance for diahc'tics within the next few 
idcH'ades. 
ciHow I II iicmMovt: 
The second polvpc*ptide hormone currently a 
(candidate for KI).\ apfirov al is gi'ow th hormone 
|(CiHI. It is one of a family of closely l elated, rel- 
tatively large pituitary peptide hormones— sin- 
gle-chain polypeptides 191- to 19«-,\.\ in length. 
It is best known for the growth it induces in 
I many soft tissues, cartilage, and hone, and it is a 
Ireciuirement for jxistnatal grow th in man. 
^ rhe grow th of an organism is a highlv com- 
jple.x process that depends on the correct hal- 
lance of many variables: I he action of C'lH in the 
I body for example, depends on the presence of 
j insulin, whose secretion is stimulated by GH. 
! Under some circumstances, one or more inter- 
Imediarv polypeptides produced under the in- 
[tluence of GH by the liver (and possibly the 
I kidneys) may actually be the proximate causes 
I of some of the effects attributed to GH. In any 
'case, the biological significance of GH is most 
'clearly illustrated by the growth retardation 
> that characterizes its absence before puberty, 
! and bv the benefits of replacement therapy. 
In the United States, most of the demand for 
I human growth hormone (hGH) is met by the Na- 
tional Pituitary Agency, which was created in 
I the early 1960’s by the College of Pathologists 
I and the National Institute of Arthritis, Metab- 
: olism, and Digestive Diseases (NIAMDD) to col- 
} lect pituitary glands from coroners and private 
I donors. Under the programs of the NIAMDD, 
I hGH is prov ided without charge to treat chil- 
' dren with hypopituitarism, or dwarfism (about 
( 
t 
i 
l.tiOO patients, each of whom receives therapy 
for several years), and for research. 
While the National Pituitary Agency feels that 
it can satisfy the current demand for hCiH (see 
Tech. Note 3, p. 80.), it welcomes the promise of 
ailditional hGH at relatively low cost to satisfy 
ai'eas of research that are handicapped more by 
a scarcity of funds than by a scarcity of the hor- 
mone. However, if hGH is shown to he thera- 
peutically valuable in these areas, widespread 
use could severely strain the present supply. At 
present, the potential seems greatest for pa- 
tients with: 
• senile osteo[)orosis (hone decalcification); 
• other nonpituitary growth deficiences such 
as lurner’s syndi'ome (1 in 3,000 live 
female births); 
• intrauterine growth retardation; 
• bleeding ulcers that cannot be controlled 
by other means; and 
• burn, wound, and hone-fracture healing 
Two groups have already announced the 
preparation of micro-organisms with the capaci- 
ty for synthesizing GH. (See Tech. Note 4, p. 80.) 
In December 1979, one of these groups— Genen- 
tech —requested and received permission from 
the National Institutes of Health (NIH), on the 
recommendation of the Recombinant DNA Ad- 
visory Committee (RAC), to scale-up its process. 
Its formation of a joint-venture with Kabi Gen 
,AB is typical of the kind of alliance that develops 
as a result of the different expertise of groups in 
the multidisciplinary biomedical field. Kabi has 
been granted a New Drug Application (NDA) 
under which to market pituitary GH imported 
from abroad. 
OTHER HORMONES 
Additional polypeptide hormones targeted 
for molecular cloning (rDNA production) in- 
clude: 
• Parathyroid hormone (84 AA), which may 
be useful alone or in combination with cal- 
citonin for bone disorders such as osteo- 
porosis. 
• Nerve growth factor (118 AA), which influ- 
ences the development, maintenance, and 
