Subject: New Drug May Help Chronic Fatigue Sufferers 
Date: Published: 10/2/91 (131 lines)
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

    Technology & Health:
    New Drug May Help Chronic Fatigue Sufferers
    ----
    By Ron Winslow
    Staff Reporter of The Wall Street Journal

    Researchers said a clinical trial showed that a
 controversial experimental drug called Ampligen significantly
 improved symptoms of people suffering from severe chronic
 fatigue syndrome.
    The study of 92 patients is the first major trial of any
 drug for the mysterious ailment and the results yield fresh
 evidence that it may be caused by some viral infection and
 immune system irregularities.  Researchers said that magnetic
 resonance imaging exams showed that about half the people in
 the study had unexplained brain abnormalities.
    The study was conducted by Hem Pharmaceuticals Corp.,
 which is developing Ampligen.  While the drug appeared to be
 effective for some people, it doesn't cure the ailment.  And
 it isn't clear what portion of those diagnosed with chronic
 fatigue may benefit from Ampligen or how long they would have
 to be treated.
    "You can't really judge yet whether it will prove to be a
 feasible therapy," says Anthony Komaroff, a Harvard Medical
 School researcher who is familiar with the findings.  "It's
 only a partial report.  But it surely looks promising."
    A spokesman at the Food and Drug Administration said the
 data are "very preliminary" and that "it's too early to claim
 that Ampligen is a dramatic treatment for chronic fatigue
 syndrome."  The spokesman added that "the product has
 significant side effects that would have to be considered
 against any claimed benefits."  The agency is constrained by
 law from commenting more specifically, the spokesman said.
    People familiar with the process say Hem filed an
 application with the FDA on Sept. 3 to try the drug on a
 limited number of additional people under a policy aimed at
 broadening access to promising experimental medicines for
 serious diseases before they are approved for marketing.  The
 agency has 30 days to respond to the application, known as a
 Treatment IND, and a decision is expected by later this week.
    A favorable decision isn't assured, but it would be a
 boost for Hem, a fledgling, closely held company based in
 Philadelphia that has struggled with credibility problems in
 an effort to prove that Ampligen is an effective treatment
 for a variety of illnesses, including AIDS and some cancers.
 In any event, any decision on whether to permit broad
 marketing of Ampligen is several months or years away.
    Chronic fatigue syndrome is characterized by severe,
 prolonged fatigue, muscle and joint pain and by neurological
 problems.  Symptoms mimic other diseases, making diagnosis
 difficult, and many doctors believe it results from
 psychiatric troubles.  Severity varies; some patients continue
 working while others are completely bedridden.
    It isn't known how many people are afflicted, but the U. S. 
 Centers for Disease Control in Atlanta, which is conducting a
 study to determine its prevalence, says it gets up to 3,000
 calls a month from people who think they have the illness.
 The Chronic Fatigue and Immune Dysfunction Syndrome
 Association Inc., a patient organization based in Charlotte,
 N. C., says it received 40,000 inquiries last summer alone.
    Patients in the study were severely stricken with the
 ailment for at least three years and all required assistance
 for such routine activities as getting dressed, going to the
 bathroom and balancing a checkbook.  All were being treated by
 one of four doctors who specialize in chronic fatigue
 syndrome with practices in Charlotte and in Incline Village,
 Nev., Portland, Ore., and Houston, and who are among
 co-authors of the study.
    Researchers said 45 of the patients received twice-weekly
 infusions of Ampligen while 47 got a saltwater placebo.
 Neither doctors nor patients knew who was getting the drug.
 Some patients experienced flu-like reactions early in the
 study, but the incidence of such "adverse events" wasn't
 statistically different between the drug and placebo groups,
 according to William Carter, principal investigator and a
 co-inventor of the drug.  He said he knew of no instances
 where a dose was reduced or treatment was stopped due to side
 effects.  Dr. Carter presented results of the study at a
 meeting of the American Society for Microbiology in Chicago.
    Dr. Carter, who is also president and a shareholder of
 Hem, said the study found that by two separate scoring
 systems, patients treated 24 weeks with Ampligen increased
 their daily living activity levels on average by about 20%,
 compared with little or no increase for those on placebo.
 Ampligen patients on average showed increased endurance on
 treadmill tests by the 24th week while the performance of
 those on placebo declined.  Seven Ampligen patients were
 hospitalized for their symptoms during the course of the
 study for a total of 19 days, Dr. Carter said, while seven
 placebo patients spent a total of 114 days in the hospital.
    The patients "advanced progressively in daily living
 activities," Dr. Carter said.  "They thought better, were less
 forgetful and had fewer headaches than patients in the
 placebo group."  While many Ampligen patients continued to
 have symptoms after 24 weeks, most could carry on their lives
 with more independence, he added.
    Dr. Carter said three factors were associated with
 patients who responded particularly well to Ampligen:  an
 acute flu-like onset of the illness; elevated levels of
 interleukin-1, an infection-fighting agent of the immune
 system, at the beginning of the study; and presence on an MRI
 exam of unidentified white spots in front portions of the
 brain associated with memory and certain intellectual
 activities.
    Scientists believe the drug works by regulating an immune
 system pathway that in normal people becomes activated when
 the body senses it is being invaded by a virus.  Elevated
 interleukin-1 levels are one indication that in chronic
 fatigue patients, the pathway is overactive.  Researchers also
 believe that Ampligen blocks virus production.
    At the end of the study, interleukin-1 levels had
 diminished in patients who responded to the drug, Dr. Carter
 said, an indication that the viral pathway activity was
 reduced.  It isn't yet known whether the white spots, which
 have been reported in chronic fatigue patients by other
 researchers, healed after Ampligen treatment, he said.
    Whether or not the drug ultimately is useful in treating
 chronic fatigue, the study's findings refute arguments that
 its symptoms are provoked by psychological problems, says
 Harvard's Dr. Komaroff, who is a member of Hem's scientific
 advisory board, but who hasn't any financial stake in the
 company.  "This isn't a medicine that acts on the psyche," he
 says.  "It acts on the body."

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