(Corrects first name of physician in 12th paragraph to Amesh)
By Julie Steenhuysen
CHICAGO Aug 1 The worst Ebola outbreak in
history is heaping new pressure on U.S. regulators to speed the
development of treatments for the deadly virus, which has killed
more than 700 people since February.
The U.S. Food and Drug Administration on Friday said in an
emailed statement the agency "stands ready" to work with
companies and investigators working with patients "in dire need
A senior official within FDA told Reuters the agency would
consider proposals for providing treatments under special
emergency new drug applications, if the benefits of the
treatment outweighed the potential safety risks.
"We take this very, very seriously," the source said.
FDA's statement follow calls by doctors fed up by the lack
of progress on Ebola treatments, a market deemed too small to
gain much attention by large pharmaceutical companies.
Earlier this month, the agency put a hold on a Tekmira
Pharmaceuticals Corp clinical trial of TKM-Ebola, one
of the few Ebola treatments advanced enough to be tested in
The hold prompted a North Carolina physician with family
members in West Africa to say enough.
"This should be the last Ebola epidemic without a cure,"
said Dr. Ahmed Tejan-Sie, an internist from Burlington.
Tejan-Sie started a petition on Change.org to urge FDA to lift
its hold on the drug. It now has 15,000 signatures.
Shares of Tekmira rose sharply on Friday on news that the
Ebola outbreak in West Africa has intensified, as investors
expect the drug trial will be considered again by
"I'm not advocating that they take it out of the lab and
start using it in West Africa. What I'm advocating is that the
trials be accelerated," said Tejan-Sie, who spent much of his
childhood in Ebola-ravaged Sierra Leone.
The director of the global charity Wellcome Trust last month
took it a step further, arguing in favor of offering
experimental treatments to people at high risk of dying from
Ebola, saying the normal drug development process takes too long
and should not apply in rapidly spreading outbreaks of diseases.
Giving experimental drugs to people in an epidemic is not
without precedent. "Usually when this happens, there are drugs
that are further along in development," said Dr. Amesh Adalja,
an infectious disease expert at the University of Pittsburgh
In the 2009 H1N1 flu pandemic, for example, BioCryst
Pharmaceuticals' experimental antiviral drug peramivir
was made available through emergency use authorization. But that
drug had been tested in widely in people by that time.
"With Ebola drugs, there hasn't been much work with them
outside of animal models," Adalja said. "That makes it very hard
to safely say we should use this in a compassionate use
situation, although there is definitely a case to be made in
these large outbreaks."
Tekmira's drug has only been tested in a few dozen healthy
people. The FDA stopped its study in July because of safety
concerns among people taking the highest doses of the drug who
experienced problematic immune responses.
The hold means that particular study cannot proceed, but it
does not prevent the company from submitting a new study
proposal, say in people already infected with Ebola, for whom
any safety risks from the treatment would be mitigated by the
risk of dying.
In that case, "the benefit-risk ratio changes completely,"
the FDA source said. "Anything that would shift the risk-benefit
to a more favorable outcome could potentially allow the
authorization of that study."
What is not clear is whether Tekmira or any developers of
possible Ebola treatments would choose to test their drugs in
patients infected with Ebola, particularly in the midst of a
raging epidemic in which emotions and expectations run high.
Tekmira officials did not return calls or emails on Friday
seeking comment. In a July 21 press release, the company said it
is "mindful of the need for this important therapeutic in
situations such as the ongoing Ebola outbreak in West Africa.
"TKM-Ebola is currently an unapproved agent and the
regulatory framework to support its use in Africa has not been
established at this time," the company added.
Dr. Thomas Geisbert of the University of Texas Medical
Branch has done animal studies on the Tekmira drug and said
there are few companies willing to develop Ebola treatments.
There is "little financial incentive," given the small market
potential for a drug that treats a rare disease afflicting
developing countries, he said.
Geisbert said the drug "works great in monkeys in the lab,"
but that is largely because it is given relatively early in the
course of infection.
"What if you start giving it to people who are almost dead
and they die, but it's not the drug's fault? Then you blame the
Geisbert said given the widespread mistrust of doctors in
West Africa, which has driven dozens of victims to evade
treatment, such an event could jeopardize the drug's
"It's a very delicate situation," he said.
(Reporting by Julie Steenhuysen; Editing by Michele Gershberg,
Martin Howell, Lisa Shumaker and Dan Grebler)