Latest & Greatest
Europe Poised to
Approve Gene Therapy
for Beta Thalassemia
Europe’s Committee for Medicinal Prod-
ucts for Human Use (CHMP) recom-
mended that the European Medicines
Agency (EMA) approve Bluebird Bio’s
LentiGlobin gene-therapy product for
patients with transfusion-dependent beta
thalassemia. CHMP is the drug-reviewing
arm of the EMA, which is expected to
issue a final decision on approval by
summer 2019.
The LentiGlobin therapy involves
obtaining mobilized autologous CD34-
positive cells from patients, then transduc-
ing the cells ex vivo with the LentiGlobin
BB305 vector, which encodes adult hemo-
globin (HbA) with a T87Q amino-acid
substitution (HbAT87Q). Patients undergo
conditioning with busulfan, then modified
cells are re-infused. In 2018, researchers
reported in the New England Journal of
Medicine that treatment with the Lenti-
Globin therapy reduced or eliminated the
need for long-term red blood cell transfu-
sions in most participants with severe,
transfusion-dependent beta thalassemia.
CHMP reviewed data from a clinical
trial of 10 patients with beta thalassemia
who required transfusions at baseline.
After approximately three years of follow-
up, eight patients were no longer receiving
transfusions. The agency also reviewed
data demonstrating that LentiGlobin im-
proved patients’ hemoglobin levels.
While exact pricing details are not yet
available, the gene therapy is expected to
carry a six-figure price tag. Bluebird Bio
proposed a new installment-based and
outcomes-based payment model earlier
this year to allay potential concerns about
the high cost of this one-time treatment.
Sources: STAT, March 29, 2019; Bluebird Bio press release, March 25, 2019.
Federal Trade
Commission Wins Case
Against Predatory
Publisher
OMICS International, which publishes
nearly 700 scientific journals, was ordered
to pay $50.1 million in damages for engag-
ing in “predatory publisher” tactics. The
ruling was handed down by a federal judge
in the U.S. District Court in Las Vegas, who
reviewed a case brought by the U.S. Federal
Trade Commission (FTC).
Acting in its capacity as a consumer
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ASH Clinical News
watchdog, the FTC filed a suit alleging
that OMICS deceptively advertised its
peer-review process, promising authors
that editorial boards conducted rigorous
review. According to an investigation
published in Science, OMICS journals
approved most articles within days of
submission and only half of the approxi-
mately 69,000 manuscripts published
between 2011 and 2017 were ever sent
out for peer review.
“The FTC is closely monitoring this
industry,” the agency’s attorney Gregory
Ashe said in an interview with Science,
“and we’re hoping that the decision sends
a warning shot across the bow of would-
be predatory or deceptive publishers to
tread carefully. Re-evaluate the claims
that you’re making [so] you’re not mak-
ing claims that are not true.”
Representatives from OMICS Inter-
national said it plans to appeal the ruling.
Source: Science, April 3, 2019.
“The FTC
is closely
monitoring this
industry, and
we’re hoping
that the decision
sends a warning
shot across the
bow of would-
be predatory
or deceptive
publishers.”
—GREGORY ASHE,
FTC Attorney
OMICS also claimed to have more
than 50,000 scientists as experts and
editorial reviewers on its journals, some
of whom never agreed to serve. The
publisher was accused of the same tactics
in organizing its scholarly conferences:
It advertised that prominent academics
would attend, but many had not agreed
to serve as speakers or chairs.
The FTC also accused the publisher
of deceiving authors about the fees it
charged to publish manuscripts in its
open-access journals.
This represents one of the first rulings
of its kind against so-called predatory
publishers, which “unprofessionally ex-
ploit” the open-access publishing model,
primarily for profit. However, because
the publisher is based in India and the
judgment was made in a U.S. court, it is
unclear how the fine will be collected or
whether any portion will be dispersed
among authors whose work was pub-
lished in OMICS’ journals.
FDA and State
Authorities Renew
Efforts Against
Dangerous Stem
Cell Clinics
The U.S. Food and Drug Administration
(FDA) and New York’s attorney general
have announced new policing strategies
against for-profit clinics that offer expen-
sive and unapproved stem cell treatments
for a variety of conditions.
The FDA sent 20 letters to stem cell
companies, asking them to cease selling
unapproved products, which is a viola-
tion of federal laws. The FDA also sent
a formal warning letter to Cord for Life,
a company based in Altamonte Springs,
Florida, that has sold umbilical cord
blood products to stem cell companies.
In the letter, the cord-banking company
was cited for lapses in safety measures,
including failing to follow specific sterility
procedures or properly document its tests
and equipment.
In New York, the attorney general
filed a lawsuit against Park Avenue Stem
Cell, a clinic in Manhattan that is ac-
cused of engaging in “fraudulent and
illegal advertising regarding its stem cell
procedures.”
The latest steps are part of an ongoing
effort on behalf of the FDA and state-level
regulators to rein in a quickly growing
industry that, according to the agencies,
has injured at least two dozen patients and
cheated countless others.
However, critics question whether
the warning letters will have a substantial
impact on these clinics’ operations. “It’s
not that these letters are inconsequential,”
said Leigh Turner, PhD, a bioethicist
from the University of Minnesota who
has studied the industry’s growth. “But if
you’re trying to tackle this one business at
a time, you’re not going to make a dent.”
U.S. CRISPR Study
in Humans Gets
Underway
The first patients enrolled in a trial of
CRISPR-based gene editing at the Uni-
versity of Pennsylvania have been treated
with the powerful – and controversial –
technique, according to NPR. CRISPR is
well established as a research tool, but this
represents the first tangible step toward
using CRISPR in the clinic to treat disease.
The study involves removing patients’
immune system cells, genetically modify-
ing them in the lab to target cancer cells,
then re-infusing the modified cells. Two
patients – one with multiple myeloma
and one with sarcoma – have undergone
infusion of CRISPR-edited immune
cells. Both patients had disease that had
relapsed following standard treatments.
Eventually, the researchers hope to treat
18 patients in this study.
Gene editing has received increasing
attention in the past year, following reports
that a Chinese scientist had used CRISPR
to edit a gene in the germline of human
embryos to confer resistance to HIV infec-
tion. Recently, a panel of the World Health
Organization requested a moratorium
on all germline gene-editing experiments
in humans, calling this type of research
“irresponsible” without a central regis-
try of research and other precautionary
measures.
Several other human trials of
CRISPR-based gene editing in a variety
of conditions are planned and enrolling
participants, including two studies of
gene editing to correct the genetic defects
that cause sickle cell disease and beta-
thalassemia. ●
Source: NPR, April 16, 2019.
Sources: The Wall Street Journal, April 3, 2019; The Scientist, April 8, 2019.
June 2019