Subject: (Editorial):  Punishing Alzheimer's Patients
Date: Published: 2/14/89 (180 lines)
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

    REVIEW & OUTLOOK (Editorial):  
    Punishing Alzheimer's Patients

    "Last August," says Dee Cash, "my father didn't know where
 he was."  Her father, Roy Gabbard, was diagnosed three years
 ago as having Alzheimer's disease.  "He'd sleep for an hour,"
 says Mrs. Cash, "and then be up for 12.  He'd tear the sheets
 off his bed.  He'd fall and hit his head.  His bedroom was
 stripped of everything."  This past October, a neurologist at
 Methodist Hospital in Indianapolis began treating her father
 with THA, an experimental Alzheimer's drug she obtained from
 a respected Canadian pharmaceutical company.  Around
 Christmas, Mrs. Cash's parents drove to her home in Georgia.
    "When they pulled in the driveway, he looked at me and
 smiled and hugged me," she says.  "He couldn't feed himself in
 August, and now he's feeding himself.  We put the fork in his
 hand.  He gets into the shower now and turns the water on.  He
 has not done this in a year's time.  He's shampooing his own
 hair and brushing his own teeth.  And now he sleeps."
    Mrs. Cash got her father's THA from Pharmascience Inc. of
 Montreal, which is currently supplying the drug to
 Alzheimer's researchers in Canada and Europe.  THA's positive
 effect on some Alzheimer's patients was first reported by Dr. 
 William Summers in the New England Journal of Medicine, and a
 large-scale federal trial of the drug is now under way.  Mrs. 
 Cash and about 50 other Alzheimer's families succeeded in
 obtaining shipments of the drug by invoking a Food and Drug
 Administration program to allow individuals to import
 unapproved drugs for their personal use.  Last July 23, FDA
 Commissioner Frank Young made a dramatic announcement that
 the agency wouldn't stop patients from importing
 "personal-use" amounts of drugs not available in the U. S. 
 Speaking that day to AIDS advocates, Dr. Young said, "There
 is such a degree of desperation, and people are going to die,
 that I'm not going to be the commissioner that robs them of
 hope."
    On December 6, 1988, the FDA robbed Roy Gabbard and other
 Alzheimer's patients of hope.  The agency put out an "import
 alert" specifically banning the importation of THA. Dee
 Cash's second order of THA for her father has never arrived.
 Other Alzheimer's patients and their families are also
 discovering that the FDA is seizing their shipments of THA
 from Pharmascience.
    ---
    Even without completion of the FDA trials, desperate
 Alzheimer's patients had ample reason for seeking THA. 
 Researchers from Montreal General Hospital and the Karolinska
 Institute of Sweden have reported significant benefit from
 using THA. Archer Martin, a British chemist and holder of the
 Nobel Prize, recently attributed improvement in his own
 Alzheimer's to treatment with THA during a clinical trial at
 London's Institute of Psychiatry.
    To all appearances Alzheimer's patients came within the
 purview of Dr. Young's July 23, 1988, announcement permitting
 imports for personal use.  But now, all these people have been
 cut off.  This FDA action against those who've imported THA
 for their personal use raises questions about the sincerity
 of the agency's commitment to carrying through on reforms
 intended to make promising drugs available faster.  Under
 intense pressure, the agency sometimes responds.  When the
 pressure is off, as in this case, the agency's bureaucrats
 seem willing to sacrifice patients such as Roy Gabbard.
    Naturally, the FDA's reasons are aimed at protecting Mr. 
 Gabbard.  The import alert says, "THA is known to be a potent
 hepatotoxin, and widespread exposure to the drug could result
 in many cases of serious liver injury."  But this side effect
 is by now well recognized.  Mr. Gabbard's doctor is sampling
 his blood for liver problems, and research experience
 suggests that if problems develop his liver would return to
 normal if the drug were stopped or the dosage reduced.
 Hepatotoxicity is a familiar side effect of drugs already on
 the market; this standard would reduce the FDA's import
 policy to almost nothing.  Rabbi Morton Rosenthal, who
 obtained one shipment of THA to treat his mother at a nursing
 home in New Jersey, says, "By their logic, chemotherapy
 should be banned."
    To make matters worse, it is far from clear how, or even
 when, the FDA came to its decision on THA. The December 6
 import alert cites a decision against THA by Commissioner
 Young at a meeting on July 28, though all Pharmascience
 imports came through in the interim.  We called the FDA to
 clear up this matter but did not hear back from the agency.
 Charles Greene of Tequesta, Fla., the son of an Alzheimer's
 patient, says:  "I wrote Dr. Young on September 27 and they
 sat on my letter while they made a decision and others were
 bringing it in.  I tried to go the proper route.  I'm very
 bitter."  FDA Associate Commissioner Hugh Cannon wrote to Mr. 
 Greene's Congressman, Tom Lewis, that the anti-import policy
 is intended to prevent "the widespread use of a dangerous and
 possibly ineffective drug."  He added, "While we sympathize
 with Mr. Greene, it clearly would be imprudent at this time
 to allow the use of THA outside the context of the
 multicenter trial currently being conducted."
    Which brings up the second reason, or maybe the real
 reason, for the FDA policy.  The import alert cites THA in the
 federal trial as an IND, or investigational new drug.  It
 states, "We have been advised by the Division of Neurological
 Drug Products that a problem exists in the recruitment of
 people for these active INDs because of the large amounts of
 THA currently being imported illegally."
    What this means is that the FDA is willing to abandon Mr. 
 Gabbard and other Alzheimer's victims to protect its
 multicenter THA trial.  That is, it wants to make sure that
 the only way patients can get access to the drug is by
 enrolling in the study and subjecting themselves to a regimen
 designed to settle scientific issues, not to help the
 patients being treated.  The study does not accept all
 patients who apply, only those who meet the criteria needed
 for scientific exactitude.  But to coerce those patients it
 wants, it seeks to deny the drug to everyone else.  Roy
 Gabbard can suffer.
    This is the dirty little secret of the Kafkaesque world
 into which the FDA forces sick Americans.  At least patients
 in the Alzheimer's study all get the drug, the scientific
 "control" being taking the same patients off it.  In trials of
 other drugs, though, patients are randomly assigned to
 treatment and control groups.  A patient may get the drug he
 or she is seeking, or may get a placebo.  Just last week this
 paper reported on the FDA's bizarre handling of the AIDS drug
 ganciclovir.
    ---
    Distributed free by its maker to thousands of patients,
 ganciclovir in the opinion of many doctors who've used it
 deters some AIDS sufferers from going blind.  The FDA,
 however, says Syntex Corp. can't get approval to sell it
 because they've never submitted results from a rigorous
 FDA-type trial.  Today, no knowledgeable AIDS patient will
 enter such a trial for fear of getting a placebo.  But that's
 the only way they can get access to the drug now.
 Ganciclovir's toxicity is also comparable to the very toxic,
 already approved AIDS drug AZT. 
    We are fast arriving at a point where the FDA system
 primarily ensures that science benefits, while the system's
 skewed values increasingly cause people to lose faith in it.
 What, for instance, would the public think if the FDA's big
 THA study said the drug's actions were ineffective or
 unclear, after it had already seen the agency simultaneously
 try to punish THA's discoverer, Dr. William Summers, and ban
 imports of the drug?  Would the public regard the FDA's study
 as the product of science or a bureaucratic turf warfare?  
    Dee Cash's immediate worry is her father, Roy Gabbard.
 "The thought of not being able to continue this is more than
 we can endure.  I will do anything, to be honest with you.  My
 father still has Alzheimer's.  We're not talking about a
 miracle.  He's still restless and agitated about two hours a
 day.  But he's dressing himself, buttoning his shorts, and
 zippering his pants.  All this may seem insignificant to the
 FDA people but it's a lot when you have someone like him.  And
 he feels better.  He used to cry a lot and say I want to go.
 That's all stopped."  When Mr. Gabbard's THA runs out, he
 likely will revert to his previous state.
    The grimmest irony in the THA story is that the FDA may
 drive Alzheimer's families toward the foreign promoters Frank
 Young mentioned in his big announcement last July.  At great
 expense, for example, Mrs. Cash and Messrs. Rosenthal and
 Greene could fly their parents to Parkstar Limited in Nassau.
 Parkstar sends letters from Post Office Box CB 10981 in
 Nassau to U. S. Alzheimer's patients inviting them down for
 treatment with THA. We called Parkstar, and a Mrs. Engram
 said the clinic has under way a "THA trial that's being done
 in connection with something in the U. S.  " Mrs. Engram said a
 doctor would call us back to supply medical details.  The
 doctor never called.  At a Southern Illinois University
 symposium on Alzheimer's last March, we were approached by a
 group that wanted to discuss setting up THA treatments in
 Costa Rica.
    There is something grievously wrong with an FDA system
 that could end up sending Roy Gabbard, who is 63, for THA
 treatment in some place like the Bahamas, rather than to
 Methodist Hospital near his home in Indianapolis.

[This article is made available here by Dow Jones Co. for the 
personal and non-commercial use of callers to this bbs, in the 
hope that it will be of some help to those who are suffering 
from the disease and others who are seeking to help them.]