Sunday, December 16, 2012

FDA to rule on Lazarus Drug

http://www.stamfordadvocate.com/local/article/FDA-to-rule-on-Lazarus-drug-4121250.php

FDA to rule on 'Lazarus' drug
Llewellyn King
Published 4:41 pm, Saturday, December 15, 2012


WASHINGTON --For about a million Americans, Thursday will be a seminal
day. That's when some of them come before the Food and Drug
Administration to petition for approval of a potent and controversial
drug, a so-called Lazarus drug.

The drug, first synthesized by Hemispherx Biopharma, Inc., of
Philadelphia, in the 1970s, is Rintatolimod (tradename Ampligen),
which is used to treat chronic fatigue sSyndrome, also known as
myalgic encephalomyelitis. It is a grim but little-understood disease
of the immune system, resulting in collapse, pain, confusion and
sensitivity to light and noise.

Patients and their doctors want the drug, but there is concern that
the FDA will fault -- as it has in the past -- the scope of the
clinical trials and the documentation of collateral effects.

The FDA is expected to rule early next year.

The sickest of the sufferers, mostly bedridden and some so sick they
have to lie in dark rooms for 18 hours a day, are pinning their hopes
on a drug that will allow them to rise from their sick beds, thus the
Lazarus appellation.

Dr. Andreas Kogelnik, who runs the Open Medicine Institute in Mountain
View, Calif., puts the chances of FDA approval for Ampligen at just 50
percent. Although he is rooting for the drug to be approved, he says
the FDA may require more data on collateral effects. This has happened
in the past and the FDA has not been satisfied with previous
applications.

Dr. Daniel Peterson, who has been treating CFS since 1984 in Incline
Village, Nev., said recently that he thinks fewer than 100 people at
any one time, either through trials or compassionate waivers, are on
Ampligen. The drug is only available in a few states, most prominently
New York and Nevada. It is very expensive (about $25,000 for a course
of treatment) and has to be administered through intravenous infusion
-- a long, slow process, at regular intervals.

According to Kogelnik, some patients react poorly right off the bat,
while others show substantial improvement almost immediately.

Mary Schweitzer, a CFS sufferer, said that she can only walk when she
is getting Ampligen. She travels regularly from her home in Delaware
to New York, where Dr. Derek Enlander, who specializes in CFS, is a
major proponent of Ampligen therapy.

Even devout proponents of Ampligen do not tout it as a cure but as a
therapy that helps them move about and approach a kind of normalcy.

Anita Patton, who lives in Nevada, said in prepared testimony for the
FDA: "Ampligen increased my ability to eradicate viruses. I previously
had not been able to walk up the stairs to then being able to exercise
for 19 minutes on the treadmill.

"The joy that even a small improvement can give a person, to be able
to do household tasks or get out of the house and use my body to take
walks, is something that many patients do not have. The quality of
life of a patient with this horrible illness is so difficult not to
care for yourself and have to endure severe pain in muscles and
nerves, being too exhausted to even take a shower or lift my arms to
fix my hair.

"The suffering is immense. Many patients have to lie in bed for 18
hours a day, as I did before Ampligen, needing care and not having a
life of their own. Many patients have lost all friends and family."

When patient activists face the government in various hearings, it is
painfully asymmetrical, it seems to me. The sick tell sad stories of
suffering, loss of love as well as health, while the government people
talk abstractly about patient loads, international disease
definitions, allocation of resources and appear self-important rather
than appalled at the suffering that passes before them.

The patients turn to the government for recognition, but the
government turns them into a statistic.

--

(Llewellyn King is executive producer and host of "White House
Chronicle" on PBS. Email: lking(at)kingpublishing.com.)

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