Translations New Scripps Research drug progressing through clinical trial, seeking to reverse underlying causes of osteoarthritis As we get older, the aging of our bodies
When given to animals, the compound
The trial is focused on assessing the
may often seem like a one-way street,
was effective at regenerating cartilage.
safety of the drug, as well as its initial
what with the steady arrival of blurry vision,
The researchers reported their findings in a
effectiveness in combatting osteoarthritis.
gray hair, wrinkled skin and other signs of
groundbreaking paper in the journal Science.
Together with Calibr, the clinical head of the
time. But there is reason to take heart—
KA34 program, Martin Lotz, MD, a professor
at least for people suffering from one of
The promise of regenerating cartilage
at Scripps Research, has tested the drug
the most common scourges of aging,
spurred Schultz and his team to pursue a
on 16 osteoarthritis patients. Enrollment is
osteoarthritis.
version of the compound that could reverse
accelerating as no drug-related adverse
osteoarthritis in humans. To do this, they
effects have been seen.
Osteoarthritis is the prevalent form of
turned to the drug discovery capabilities
arthritis, affecting tens of millions of
of Calibr, a separate nonprofit founded by
The Phase 1 trial is slated to wrap up by
people worldwide. The disease stems
Schultz that recently merged with Scripps
September 2019, after the drug is tested
from the wearing down of the cartilage
Research as its drug discovery division.
in all 60 participants. Assuming KA34
that protects the ends of bones. Currently,
proves safe and shows initial signs of
the only non-surgical therapies for the
“The story of KA34 is proof positive that
effectiveness, the next step would be
disease are drugs that treat the pain
integrating academic science and drug
a Phase 2 clinical trial to robustly test
and inflammation—the symptoms—while
discovery capabilities offers incredible
its ability to stop or even reverse the
having no impact on the loss of cartilage,
potential for speeding translation of
progression of osteoarthritis. In anticipation
the underlying cause of the disease.
foundational discoveries into real-world
of the next trial, Calibr scientists are
medicines,” says Schultz. “From the time
developing an extended release version
A new drug, developed by scientists at
we had a preclinical candidate identified
of KA34.
Scripps Research and being tested in an
to giving the drug to patients was roughly
early-stage clinical trial, could change
two years.”
that. The idea for the compound, called
38
“This is the first program of our new benchto-bedside initiative to enter the clinic,”
KA34, emerged from the laboratory of
In May 2018, Calibr scientists launched
Schultz says. “Over the next two years we
Peter Schultz, PhD, a chemistry professor
a Phase 1 clinical trial to test KA34 in 60
expect to move additional programs into
and the president of Scripps Research.
patients between the ages of 40 and
development including therapies for cancer,
Schultz and his team discovered a
75 with osteoarthritis. The California Institute
malaria, and lung and neurodegenerative
compound, called kartogenin, that
for Regenerative Medicine (CIRM), which
diseases.”
generates chondrocytes from stem cells,
financially supported the KA34 preclinical
the early-stage, highly flexible cells from
research effort from inception, awarded Calibr
which all specialized cell types develop.
an additional $8.4 million for the clinical trial.
SCRIPPS RESEARCH MAGAZINE
WINTER 2019
