Future
of Health and Healthcare
·
The current market for stem cell therapies is growing at 36% per
year and will rapidly expand when a breakthrough treatment for non-communicable
disease or a lifestyle factor occurs.
·
Funding is flowing into the sector from venture capitalists and
large companies.
·
With greater resources and an increasingly experienced talent
pool, scientists and physicians may be able to develop stem cell science to
cure the world's most challenging diseases.
Right
now, your body’s stem cells are working hard replacing your skin every two
weeks, creating new red and white blood cells and completing thousands of other
tasks essential to life. They are your own personalized fountain of youth.
Scientists
generally agree that a stem cell should be able to do both of the following:
·
Self-renew: divide into another stem cell, which means making an
identical copy of itself; and
·
Differentiate: change into a variety of other cell types.
One
theory of ageing suggests that between the ages of 30 and 50, our stem cells
reach a turning point and start to decline in number and function. This results
in the typical features associated with ageing.
A brief
history of stem cells
There
does not seem to be a single discoverer of stem cells. Accounts date back to
the 1800s and even further, but the first successful medical procedure
was a bone marrow transfusion in 1939.
Advances in immunology led to donor matching, initially via siblings and close
relatives. Unrelated donor matching flourished in the 1970s, alongside donor
registries.
In the
1980s, scientists identified embryonic stem cells in mice, leading to the 1997
cloning of Dolly the Sheep. This created immense interest for human and medical
applications and a backlash in the US as federal R&D funding was
essentially halted in 2001.
In
2012, a Nobel Prize was
awarded for the earlier discovery of induced pluripotent stem cells (iPS).
Essentially, they return potency and self-renewal properties to mature non-stem
cells, essentially making them act like stem cells again.
In the
decade between 2010 and 2019, the first wave of stem cell start-ups emerged,
alongside R&D programmes at many large pharmaceutical companies, leading to
innovation and the first human clinical trials for iPS and other related therapies.
Regenerative
medicine in 2019
According
to Q3 2019 data from the Alliance for
Regenerative Medicine, there are 959 regenerative medicine companies
worldwide sponsoring 1,052 active clinical trials; 525 of these companies are
in North America, 233 in Europe and Israel, and 166 in Asia. In aggregate, $7.4
billion has been invested in regenerative medicine companies in 2019; $5.6
billion of which has been dedicated to gene and gene-modified cell therapy,
$3.3 billion in cell therapy, and $114 million in tissue engineering.
Overview
of the cancer stem cells market
Perhaps
most excitingly, curative therapies are hitting the market and the results are
astonishing: 60% of Acute Lymphoblastic Leukemia patients taking Novartis’
Kymirah showed a complete response (no traces of cancer) and were declared in
full remission. Meanwhile, 75% of patients with Transfusion-Dependent
β-Thalassaemia treated with bluebird bio’s Zynteglo achieved independence from
transfusions. Perhaps most astonishingly, 93% of spinal muscular atrophy
patients treated with Novartis’ Zolgensma were alive without permanent
ventilation 24 months after treatment. We should expect more medical
breakthroughs in the coming years.
What is
next?
New
science, new start-ups: several companies in the sector have
gone public or been acquired. These exits led to the recycling of talent and
capital into new companies. Because the science and commercial systems have
also advanced, the companies in the next wave are pursuing bigger challenges,
driving innovation, with even greater resources.
Patients
are eager: the current market for stem cell
therapies is growing at 36% per year, though it will rapidly expand
when a breakthrough occurs toward the treatment of a non-communicable disease
(such as cancer, diabetes, heart disease) or a lifestyle factor (for example,
growing hair in the correct places, expanding cognitive abilities or increasing
healthy lifespan).
New
R&D models: funding is flowing into the sector from large companies,
VC funds, and institutions such as the California Institute for Regenerative
Medicine (CIRM) and New York State Stem Cell Science programme (NYSTEM). Some
of the leading university R&D platforms include the Center
for the Commercialization of Regenerative Medicine in Toronto,
the Stanford Institute for Stem Cell Biology and Regenerative
Medicine, the Oxford Stem Cell Institute, and most notably,
the Harvard Stem Cell Institute (HSCI).
Founded
in 2004, HSCI has established a phenomenal track record. It provided the first
$200,000 in funding to Derrick Rossi’s lab, which inspired the largest biotech IPO to date.
HSCI scientists were also co-founders or principals in the three most prominent
gene-editing companies (CRISPR Tx, Intellia and Editas), the combined
$1.55-billion True North/iPierian acquisitions and the recent $950-million
acquisition of Semma Tx, Frequency Tx, Fate Tx, Epizyme Inc., and Magenta Tx.
Investment
opportunities
For the
casual investor, Evercore ISI is building a Regenerative Medicine Index, which
may be the simplest way to build a portfolio. For institutions and those with
deeper pockets, regenerative medicine funds are forming, including the
Boston-centric Hexagon Regenerative Medicine Fund, which aims to create
companies out of the Harvard Stem Cell Institute.
Where
can I get some stem cells?
Caveat
emptor. Though patients’ needs are immediate, those seeking treatments should
think very carefully about the risks. There are many dubious clinics touting
expensive stem cell treatments and some patients have experienced horrifying
complications. Dr. Paul Knoepfler of UC-Davis has
written a practical and scientifically accurate guide, a strongly
recommended read if you or a family member are considering treatment or a
clinical trial.
Why
stem cells? Why now?
The leading causes of death in 1900 were
mostly infectious/communicable diseases. While the prevalence of most causes
has diminished, the largest increases include heart disease (+40%) and cancer
(+300%). Granted, this is partly due to doubling life expectancy and a lack of
death from other causes. However, given time and resources, scientists and
physicians may cure these challenging diseases.
Total
disease burden by disease or injury
Today,
six of the seven leading causes of death are non-communicable diseases (heart
disease, stroke, lung diseases, cancer, Alzheimer’s disease and diabetes).
Based on the early promise mentioned above, regenerative medicine may be our
best hope to solve the great non-communicable diseases of
our time, and perhaps the single most transformative medical
innovation in a century.
No comments:
Post a Comment