CLINICAL NEWS
Trials of Vadastuximab
Talirine to Resume
After FDA Clinical Hold
The FDA lifted its clinical hold on early-
stage studies of vadastuximab talirine for
AML after halting them in early January
following four patient deaths.
Seattle Genetics Inc., the manufacturer
of vadastuximab talirine, said the clinical
hold was resolved through a compre-
hensive study evaluating more than 300
patients, and amendments were made to
the study protocols to further enhance
safety. Two early-stage trials will resume,
with plans for a mid-stage trial to begin
this year.
Results from a phase Ib study were
presented at the 2016 ASH Annual
Meeting and suggested that combining
standard 7+3 chemotherapy with vadas-
tuximab talirine was safe in patients with
newly diagnosed AML. Seventy-six per-
cent of patients achieved a response, most
of which (60%) were complete remissions.
Patients showed no evidence of in-
creased toxicity; 30- and 60-day mortality
rates were 0 percent and 7 percent, respec-
tively; and the rates of hematologic AEs
were similar to what would be expected
with standard chemotherapy alone, ac-
cording to the researchers.
Source: Reuters, March 6, 2017.
Development of CAR
T-Cell Therapy for Acute
Lymphocytic Leukemia
Shut Down Following
Clinical Holds
Juno Therapeutics, the manufacturer of the
experimental chimeric antigen receptor
(CAR) therapy JCAR015 for patients with
relapsed acute lymphocytic leukemia,
announced that it will discontinue the
development of JCAR015.
Last year, the FDA placed a clinical
hold on phase II trials of JCAR015 follow-
ing patient deaths from cerebral edema,
but then allowed trials to resume. The
manufacturers originally reported that
the deaths were related to the addition
of fludarabine to the pre-conditioning
regimen, and they proposed that the trial
continue with cyclophosphamide as a
substitution.
However, after five patient deaths
were reported, the manufacturer said its
internal investigation identified multiple
factors that increased the risk of severe
toxic reactions, including “factors related
to the product.”
The promising new class of immu-
notherapy, which removes T cells from
ASHClinicalNews.org
a patient’s blood and re-engineers them
to more efficiently attack cancer before
returning them to the patient, was highly
anticipated by health-care professionals
and patients alike.
Source: Reuters, March 1, 2017.
FDA Grants Marketing
Authorization for
Personal Genetic
Risk Tests
The FDA has approved marketing for
23andMe Personal Genome Service
Genetic Health Risk (GHR) tests, the
first direct-to-consumer tests that ana-
lyze DNA from users’ saliva samples to
calculate their genetic predisposal for the
following 10 diseases and conditions:
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Parkinson’s disease
late-onset Alzheimer’s disease
celiac disease
alpha-1 antitrypsin deficiency
early-onset primary dystonia
factor XI deficiency
Gaucher disease type 1
glucose-6-Phosphate Dehydrogenase
deficiency
hereditary hemochromatosis
hereditary thrombophilia
the test. Data were reviewed through the
FDA’s device premarket review pathway,
which regulates low- to moderate-risk
devices while simultaneously establish-
ing criteria (“special controls”) to ensure
accuracy, reliability, and clinical relevance
in the testing.
In 2013, the FDA warned the manu-
facturers of the GHR test to discontinue
marketing the test kit. The marketing ma-
terials stated that the device was intended
for use in the diagnosis and prevention
of diseases and disorders, but doing so
before the FDA had the opportunity to
review the statements, the manufacturer
was in violation of the Federal Food,
Drug, and Cosmetic Act. In response, the
manufacturer reconfigured their testing
platform and worked with the FDA to
design trials that would assess its health-
related claims.
In a press release announcing the deci-
sion, Jeffrey Shuren, MD, director of the
FDA’s Center for Devices and Radiologi-
cal Health, noted that “consumers can
now have direct access to certain genetic
risk information, but it is important that
people understand that genetic risk is just
one piece of the bigger puzzle, it does not
mean they will or won’t ultimately develop
a disease.”
The FDA cautioned that results from
testing should still be monitored by a
health-care professional because of the
risk associated with the test, including
false-positive and false-negative findings.
Source: U.S. FDA press release, April 6, 2017.
“Consumers
can now have
direct access to
certain genetic
risk information,
but it is important
that people
understand that
genetic risk is just
one piece of the
bigger puzzle.”
—JEFFREY SHUREN, MD
The FDA’s decision was supported by an
analysis of peer-reviewed literature estab-
lishing a strong link between genetic vari-
ants and the 10 conditions included on
FDA Clears Device
That Touts “Fast and
Virtually Painless”
Blood Draw
The FDA granted 510(k) clearance to TAP,
a blood collection device that is placed
on the upper arm and collects 100 µL of
capillary blood with the press of a button,
lasting two to three minutes. Seventh
Sense Biosystems, Inc., the manufacturer
of TAP, claims that the device is “fast and
virtually painless.”
The single-use device is attached to
the upper arm through vacuum pressure
and draws blood through an array of fine
needles. The drawn blood is held inter-
nally within the device until laboratory
analysis of hemoglobin A1c levels. The
device uses lithium heparin as its antico-
agulant, which the manufacturers noted
may limit the range of potential diagnostic
testin