HP Innovation Journal Issue 10: Fall 2018 | Page 62

Another novel approach to regenerative medicine is gene
therapy, which either inactivates or replaces improperly
functioning genes to cure or prevent a medical condition
or introduces a new gene to help fight against a disease.
Since August 2017, the FDA has approved three gene therapy
products, the first of their kind. Two of them reprogram a
patient’s own cells to attack deadly cancer cells in B-cell acute
lymphoblastic leukemia and non-Hodgkin’s lymphoma , while
the most recent treats a rare form of inherited vision loss
caused by mutations in a specific gene.
At the same time, new gene editing techniques like CRISPR
are accelerating the pipeline of gene therapies. In fact, human
trials have begun for the first gene therapy that modifies
DNA in living patients. If it is successful, it will open a new
pathway to treating diseases ranging from HIV to high
cholesterol.
Gene therapy research is also proving critical to dealing
with neurodegenerative diseases and memory loss. Forty
percent of the U.S. population over 65 suffers from memory
impairment. 4 Globally, an estimated 50 million people live
with dementia—a number that is expected to double every 20
years. 5 To help combat this, last year the National Institutes
of Health announced a $400 million surge in research
funding for Alzheimer’s and dementia research.
Promising therapeutic approaches that directly modify
Alzheimer’s risk-associated protein levels or slow down
the transcription of their corresponding gene could have a
profound impact by blocking the neurodegeneration that
leads to cognitive decline.
1 United Nations, 2017 World Population Ageing Report
2 United States Department of Health and Human Services
3 Frost & Sullivan, in partnership with A4M.com
4 Gary Small, “What we need to know about age related
memory loss,” BMJ, 2002 June 22; 324(7352): 1502–1505
5 Deloitte UK Centre for Health Solutions and Alzheimer’s
Disease International
Innovation Journal Issue Ten
» Innovation is dramatically
reshaping health care and
redefining what it means
to be human. We are at the
dawn of the bio-physical-
digital convergence, and we
don’t know where science
and technology will take us.
It raises concern, for some,
that our human curiosity
and ingenuity may help us
realize some of our wildest
dreams. The social, ethical,
physical and environmental
impacts of a bionic age
will challenge us, even as
they offer amazing gains
in health and longevity.
But as we weigh the risks
and benefits of these
technological age-defying
advancements, one thing
is for certain: we are only
getting started.