40 Time November 4, 2019
O
ur world has never wiTnessed a Time of
greater promise for improving human health.
Many of today’s health advances have stemmed
from a long arc of discovery that begins with
strong, steady support for basic science. In large
part because of fundamental research funded by the National
Institutes of Health (NIH), which traces its roots to 1887,
Americans are living longer, healthier lives. Life expectancy
for a baby born in the U.S. has risen from 47 years in 1900 to
more than 78 years today. Among the advances that have helped
to make this possible are a 70% decline in the U.S. death rate
from cardio vascular disease over the past 50 years, and a drop
of more than 1% annually in the cancer death rate over the past
couple of decades. As one more dramatic example, thanks to
remarkable advances in antiretroviral drugs, most Americans
with human immunodeficiency virus (HIV) can now look for-
ward to an almost normal life span.
Yet, despite this astounding progress, much more remains
to be done. Among the many efforts now poised to change the
future of health are those to harness the power of gene editing,
expand the reach of cancer immunotherapy, map the human
brain and build a solid foundation for a more individualized
approach to health care, often called precision medicine. And
along with the bright promise of preventing, treating and curing
some of humankind’s most feared diseases come some crucial
questions about how to ensure such breakthroughs are applied
both ethically and equitably.
One Of the great things about basic science is that it is
impossible to predict where it might lead. For example, no one
could have imagined that relatively routine efforts to sequence
bacterial genomes and to improve yogurt production would
lead to development of a revolutionary new gene- editing tool.
But it did! In the late 1980s, scientists found strange repetitive
DNA sequences called Clustered Regularly Interspaced Short
Palindromic Repeats (CRISPR) in bacteria, and a couple of de-
cades later, other researchers discovered that the CRISPR system
helped yogurt’s beneficial bacteria fend off viral invaders, by de-
tecting and snipping their DNA. After CRISPR’s exact mechanism
was figured out, this exquisitely precise gene- editing technology
was quickly put to work in a wide range of biomedical settings.
Researchers think CRISPR and related gene- editing tech-
nologies hold tremendous potential for treating or even cur-
ing the thousands of diseases for which we understand the mo-
lecular mechanism but treatments are limited or unavailable,
such as sickle-cell disease, muscular dystrophy, Huntington’s
disease and a long list of others. All of these exciting treatment
opportunities involve editing the DNA of spe-
cific cells that can help the intended patient
but are not passed on to future generations.
Here is where gene- editing technology en-
counters a critically important ethical bound-
ary. NIH and virtually all credible international
bodies remain opposed to clinical applications
of heritable gene editing, which involve using
gene editing on human embryos, sperm or
eggs. These interventions are difficult to justify
medically and would irreversibly alter the DNA
blueprint of future generations of humankind.
Another rapidly emerging field, cancer
immunotherapy, is also the fruit of decades of
basic research. In fact, one fascinating study
showed that a successful immunotherapy ap-
proach, called checkpoint inhibitors, arose
from a century of cumulative work by more
than 7,000 researchers, including 2018 Nobel
laureates James Allison and Dr. Tasuku Honjo.
Other pioneers in the effort to enlist a patient’s
own immune system in the fight against cancer
include Dr. Carl June and Dr. Steven Rosen-
berg, who are now looking to extend and fine-
tune their cell-based strategies so they benefit
more people with many more types of cancer.
While cancer immunotherapy is still in its
infancy, some impressive reports of its ability
to save lives are beginning to roll in. For exam-
ple, new survival data from one of the longest-
running immunotherapy trials—a combina-
tion approach using checkpoint inhibitors
for metastatic melanoma—showed that 52%
of patients were still alive after five years. Be-
fore the advent of immunotherapy, the five-
year survival rate for this deadly form of skin
cancer was only about 5%.
Perhaps no basic science endeavor has a
more ambitious goal than the NIH-led Brain
Research Through Advancing Innovative
Neuro technologies (BRAIN) Initiative: devel-
oping the tools needed to understand how the
human brain’s roughly 100 billion cells, each
with about 1,000 connections, interact in real
time. As a result, we will have a much better
grasp of how the brain works to produce our
motor activities, memory deposition and re-
trieval, cognition, emotions and behaviors.
Brain diseases still pose some of the great-
est mysteries in modern medicine. So the
aim of the upwards of 500 investigators at
more than 100 institutions supported by
the BRAIN Initiative is to spur progress in
neuroscience, much as the international
Human Genome Project did for genetic re-
search. Such understanding will open new
avenues to treat Alz heimer’s disease, autism,
depression, epilepsy, Parkinson’s disease,Medical
science’s age
of discovery
By DR. FRANCIS S. COLLINS
HEALTH CARE • E S S AY
Collins is the
director of
the National
Institutes of
Health (NIH)