FDA to rule on ‘Lazarus’ drug | comment by Llewellyn King | 15 December 2012

December 17, 2012

From StamfordAdvocate.com, 15 December 2012. Words by Llewellyn King, executive producer and host of ‘White House Chronicle' on the US-based Public Broadcast Service.

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.

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