Caught this from Dateline tonight. RE Myotrophin, sounded like it has a third chance. Sorry about the caps, one little unpleasant thing from the capture board. FWIW.
Channel 4 Tue Jul 08 21:54:19 1997
THAT THIS MIGHT BE WRONG
WITH ME.
>> Reporter: FOR MONTHS,
SHELBIE OPPENHEIMER HAD KNOWN
SOMETHING WAS WRONG.
SHE JUST DIDN'T KNOW WHAT.
SHE WAS ONLY 28 AT THE TIME
AND WORRIED ABOUT THE GROWING
WEAKNESS IN HER LEFT HAND
AND THE MYSTIFYING TWITCHES THAT
FOLLOWED.
FINALLY AFTER MONTHS
OF UNCERTAINTY, A DIAGNOSIS THAT
TURNED OUT TO BE MUCH WORSE THAN
ANY OF THE SYMPTOMS.
WHAT DID YOUR DOCTOR SAY TO YOU?
>> HE SAID, I'M VERY SORRY
TO TELL YOU BUT YOU HAVE
AMYOTROPHIC LATERAL SCLEROSIS
WHICH IS LOU GEHRIG'S DISEASE.
>> Reporter: AMYOTROPHIC LATERAL
SCLEROSIS OR ALS IS A CONDITION
THAT MAKES NERVES DIE,
PARALYZING MUSCLES, LEAVING
PEOPLE PHYSICALLY HELPLESS.
LOU GEHRIG DIED OF THE DISEASE
IN 1941, AND TO THIS DAY DOCTORS
STILL DO NOT KNOW WHY IT
STRIKES.
A CURE IS NOWHERE IN SIGHT.
THERE WILL COME A TIME IF THIS
PROGRESSES AS IT WILL
WITHOUT SOME BREAKTHROUGH, WHEN
YOU WILL NOT BE ABLE TO BREATHE
FOR YOURSELF?
>> RIGHT.
>> Reporter: WHEN YOU WILL NOT
BE ABLE TO EAT?
WHEN YOU WON'T BE ABLE TO MOVE?
>> UH-HUH.
>> Reporter: AND YOU WON'T BE
ABLE TO SPEAK?
>> RIGHT.
>> Reporter: BUT YOU'LL BE ABLE
TO THINK EVERY BIT AS CLEARLY
AS YOU DO NOW.
>> YES.
THAT WILL HAPPEN.
>> Reporter: AND TRAGICALLY,
WHEN THE DISEASE IS FINALLY DONE
PARALYZING, IT KILLS.
TYPICALLY WITHIN THREE TO FIVE
YEARS OF DIAGNOSIS.
>> IT'S EXTREMELY FRIGHTENING.
I WANT MY NORMAL LIFE BACK.
I WANT A FAMILY.
I WANT A FUTURE TO LOOK FORWARD
TO.
>> Reporter: SO YOU CAN IMAGINE
HOW SHE FELT TO HEAR ABOUT A NEW
EXPERIMENTAL DRUG CALLED
MYOTROPHIN.
DEVELOPED OVER THE PAST TEN
YEARS, ITS MANUFACTURERS CLAIM
MYOTROPHIN CAN DO SOMETHING
NO OTHER DRUG CAN -- IMPROVE
THE QUALITY OF LIFE FOR ALS
SUFFERERS, BY SLOWING
THE PROGRESSION OF THE DISEASE.
THE ONLY OTHER ALS MEDICATION
BRIEFLY EXTENDS LIFE BUT DOES
NOTHING TO RELIEVE SUFFERING.
SO WORD OF MYOTROPHIN WAS BIG
NEWS.
>> WE LEARNED THAT THEORETICALLY
IT CAN GROW YOUR NERVES
AND COUNTERACT THE DEATH
OF YOUR NERVES.
AND THAT SOUNDED VERY EXCITING
TO US.
>> Reporter: BUT THE QUESTION
IS, WILL SHELBIE OPPENHEIMER
EVER BE ABLE TO GET MYOTROPHIN?
MAYBE NOT, BECAUSE THE DRUG
CAN'T GO ON THE MARKET UNTIL
THE GOVERNMENT DETERMINES IT'S
BOTH SAFE AND EFFECTIVE.
WHILE THE FOOD AND DRUG
ADMINISTRATION HAS ACKNOWLEDGED
THAT THE MEDICATION IS SAFE, IT
HAS YET TO DECIDE IF IT'S
EFFECTIVE.
BUT THIS DOCTOR THINKS IT WORKS.
>> IT SLOWED DOWN THE RATE
OF DISEASE, IT INCREASED QUALITY
OF LIFE!
ARE YOU HAVING ANY FALLS?
>> WELL, NOT LATELY, BUT I DID
FALL.
>> Reporter: DR. DEBORAH GELINAS
IS ONE OF THE FEW PHYSICIANS WHO
HAVE SEEN MYOTROPHIN IN ACTION.
SHE GAVE THE DRUG TO 45 PATIENTS
AS PART OF A STUDY AND IS
CONVINCED IT HELPS A GOOD
PORTION OF ALS SUFFERERS DO
EVERYDAY TASKS, LIKE FEED
AND DRESS THEMSELVES OR BRUSH
THEIR TEETH, A FEW MONTHS LONGER
THAN PATIENTS WHO DON'T TAKE
THE DRUG.
>> YOU AND I WHO ARE HEALTHY
THINK NOTHING OF THREE MORE
MONTHS.
SOMEONE WHO IS LOSING FUNCTION
AND WHO HAS THREE MORE MONTHS
OF WALKING THINKS OF ALL
THE TRIPS THEY ARE GOING
TO TAKE.
IT'S A DIFFERENT OUTLOOK.
>> Reporter: ALL OF WHICH MEANS
THE WORLD TO SOMEONE LIKE
KYLE HAHN.
HE'S THE GUITARIST YOU SEE
IN THIS VIDEO TAKEN NOT LONG
BEFORE HE WAS DIAGNOSED
WITH LOU GEHRIG'S DISEASE.
THAT WAS LESS THAN TWO YEARS
AGO.
THIS IS KYLE HAHN TODAY, HOW
SHELBIE OPPENHEIMER IMAGINES
SHE WILL BE IN JUST A FEW YEARS.
HIS MIND UNTOUCHED, HIS BODY
RAVAGED BY ALS.
>> DO YOU KNOW IF ANYBODY'S GOT
ANY BRAINSTORMS?
>> Reporter: THOUGH HE'S GREATLY
PHYSICALLY IMPAIRED, KYLE'S
FRIEND, TERRY FRANK, SAYS HE IS
ACTUALLY MUCH IMPROVED.
FIVE MONTHS AGO HE WON A LOTTERY
ENABLING HIM TO TAKE MYOTROPHIN
ON AN EXPERIMENTAL BASIS.
BUT ALL THAT COULD END
IF MYOTROPHIN IS NOT APPROVED
BY THE FDA.
>> HE'S ABLE TO TURN HIS HEAD
SIDE-TO-SIDE.
HE'S GOT REAL STRENGTH
IN HIS NECK.
>>eporter: KYLE, IS THERE ANY
QUESTION IN YOUR MIND THAT
THE FACT THAT YOU CAN TURN
OVER IN BED AND MAKE YOURSELF
COMFORTABLE WITH NO ONE'S HELP,
THE FACT THAT YOU CAN EAT
SOMETHING THAT AT ONE POINT
YOU COULDN'T, ARE DUE
TO MYOTROPHIN?
>> IT HAS TO BE.
>> Reporter: THERE'S NO QUESTION
IN YOUR MIND AT ALL?
>> NO QUESTION.
>> Reporter: BUT THE U.S. FOOD
AND DRUG ADMINISTRATION IS NOT
NEARLY AS CONVINCED THAT
MYOTROPHIN REALLY WORKS.
SO NOW THE FDA FACES
A DILEMMA --
ON THE ONE HAND APPROVING A DRUG
IT DOESN'T TRULY BELIEVE IN.
ON THE OTHER DENYING THAT DRUG
TO DESPERATE PATIENTS WHO SEE
MYOTROPHIN AS THEIR ONLY HOPE.
>> PLEASE KEEP YOUR MARKS AS
BRIEF AS YOU POSSIBLY CAN.
>> Reporter: AT A RECENT
HEARING, RECORDED ON HOME VIDEO,
AN ADVISORY PANEL FOR THE FOOD
AND DRUG ADMINISTRATION MADE UP
PRIMARILY OF NEUROLOGISTS
EXPRESSED SKEPTICISM
ABOUT THE DRUG'S EFFECTIVENESS.
>> WE HAVE SEEN COSMETIC CHANGES
IN THE DATA, NOTHING
SUBSTANTIAL.
AND THAT'S WHAT'S SO UPSETTING.
>> IF THIS DRUG DOESN'T WORK,
AND WE SAY THAT IT DOES WORK
THAN THAT IS A DISSERVICE.
AND MY PROBLEM IS I AM NOT
CONVINCED FROM THIS DATA REALLY
THAT IT WORKS.
>> Reporter: BUT EVEN
IF THE SCIENTIFIC STUDIES ARE
NOT COMPLETELY CLEAR IS IT
COLDHEARTED TO DENY PATIENTS
LIKE SHELBIE OPPENHEIMER A DRUG
THEY WANT?
THAT THEY'RE CONVINCED DOES WORK
WHEN NOTHING ELSE CAN HELP?
BRUCE JENNINGS DOESN'T THINK SO.
HE'S A MEDICAL ETHICIST
AT THE HASTINGS CENTER,
A RESEARCH INSTITUTE
IN NEW YORK.
>> I THINK THE FDA DOES HAVE
A RESPONSIBILITY TO PROTECT
PEOPLE FROM THEIR OWN
DESPERATION.
IT MAY SEEM THAT IN A TERRIBLE
FATAL DISEASE YOU OUGHT TO BE
COMPLETELY HANDS OFF
BECAUSE THERE IS NO OTHER HOPE.
>> Reporter: WHAT IS THERE
TO LOSE?
>> RIGHT.
BUT IT IS PRECISELY
IN THE CONTEXT OF THOSE TERRIBLE
DISEASES THAT PEOPLE BECOME MOST
DESPERATE AND VULNERABLE AS WELL
AND THEY WILL GRASP AT STRAWS.
>> Reporter: EXCEPT THESE PEOPLE
HAVE SAID TO US WITH THIS
DISEASE, "IF WE, IF WE ARE GOING
TO BE EXPLOITED, WELL IT'S NOT
GOING TO BE FOR VERY LONG.
BECAUSE THE PROGNOSIS IS
SO AWFUL.
AND IF, IF WE'RE GRASPING
AT STRAWS, WELL THAT'S ALL
WE HAVE TO GRASP AT."
>> THAT'S RIGHT.
WELL, WE ALSO HAVE TO MAKE
A DISTINCTION BETWEEN A DRUG
THAT MIGHT POTENTIALLY OFFER
A CURE OR AT LEAST A SIGNIFICANT
REMISSION.
AND A DRUG THAT MAY OFFER ONLY
A VERY LIMITED AMOUNT OF EXTRA
TIME.
>> Reporter: BUT AGAIN,
THE PATIENTS ARE SAYING EVEN TWO
TO THREE MONTHS OF BEING ABLE
TO BSH YOUR TEETH AND BUTTON
YOUR SHIRT IS A HUGE, HUGE
IMPROVEMENT OVER THE
ALTERNATIVE.
>> THAT WOULD BE A HUGE
IMPROVEMENT.
I WISH THAT THERE WERE GOOD
EVIDENCE THAT ANY DRUG WOULD
OFFER PEOPLE THAT KIND
OF BENEFIT.
>> Reporter: THE COMPANY THAT
MAKES MYOTROPHIN WON'T SAY HOW
MUCH THE MEDICATION WOULD COST
PATIENTS.
BUT EVEN IF ALS SUFFERERS ARE
WILLING TO PAY FOR IT
THEMSELVES, JENNINGS SAYS ANY
NEW DRUG ALSO REQUIRES
CONSIDERABLE PUBLIC FUNDING.
>> SHOULD WE SPEND X MILLIONS OF
TLARS ON A DRUG THAT MAY ONLY
HAVE A PLACEBO EFFECT,
REMEMBERING THAT, UNFORTUNATELY,
THE WAY THE HEALTH CARE SYSTEM
IS TODAY EVERY DOLLAR WE SPEND
ON ALS AND ONE DRUG IS A DOLLAR
WE DON'T SPEND ON SOME OTHER
DISEASE, SOME OTHER PATIENT,
SOME OTHER FAMILY WHO IS ALSO
AT GREAT NEED.
>> Reporter: BUT TRY TELLING
THAT TO SHELBIE OPPENHEIMER WHO
LIVES WITHOUT HOPE
AND WITHOUT MYOTROPHIN
OR TO KYLE HAHN WHO LIVES
IN FEAR OF BEING CUT OFF
FROM THE DRUG AT THE FDA
HEARING, TERRY FRANK SPOKE
FOR HAHN.
>> BEFORE I BEGAN RECEIVING
MYOTROPHIN THREE MONTHS AGO,
I WAS IN A DOWNWARD SPIRAL.
>> Reporter: SHE REREAD HIS
SPEECH FOR US.
>> I COUGHED OVER EVERY BITE
OF FOOD.
SWALLOWING HAD BECOME VERY
DIFFICULT.
JUST A COUPLE OF SUNDAYS AGO,
I WAS ABLE TO EAT AN ENTIRE
PLATE OF FRIED OYSTERS
WITHOUT A SINGLE PROBLEM.
I LOVE FRIED OYSTERS!
>> Reporter: SHELBIE OPPENHEIMER
ALSO SPOKE TO THE FDA PANEL.
>> AT AGE 28 I WAS DIAGNOSED
WITH A TERMINAL ILLNESS.
BECAUSE OF ALS I MAY NEVER HAVE
CHILDREN OF MY OWN.
WHAT I FEAR MOST IS NEVER
HEARING THE WORDS, "MOMMY,
I LOVE YOU."
ALL I ASK FOR IS THE CHOICE,
THE OPTION TO SEE IF MYOTROPHIN
WILL SLOW MY PROGRESSION AND BUY
ME VALUABLE TIME.
>> Reporter: AFTER TEN HOURS
OF SCIENTIFIC PRESENTATIONS
AND TESTIMONY FROM DOCTORS
AND PATIENTS, THE COMMITTEE WAS
READY TO DECIDE WHETHER OR NOT
TO RECOMMEND MYOTROPHIN BE
APPROVED BY THE FDA.
>> MY HUSBAND AND I, AND MOST
OF THE PATIENTS ALREADY HAD
TEARS RUNNING DOWN OUR FACE,
BECAUSE WE KNEW WHAT IT WOULD
BE.
>> WE HAVE TO SAY THAT THIS IS
A DRUG THAT WE AS A COMMITTEE
CAN SAY IN GOOD CONSCIENCE IS
CLEARLY GOING TO BE BENEFICIAL
TO PEOPLE.
I'D LOVE TO BE ABLE TO DO THAT.
>> Reporter: DESPITE APPEALS
BY PATIENTS FACING THE BLEAKEST
FUTURES COMPLETE PARALYSIS,
PREMATURE DEATH -- DESPITE
PATIENTS WHO SAID THEY COULD NOT
SEE THE DOWNSIDE TO APPROVING
A SAFE DRUG, THE PANEL VOTED SIX
TO THREE NOT TO RECOMMEND
APPROVAL.
>> THE PANEL WAS
ASKED TO MAKE A DIFFICULT
DECISION AND I THINK THAT
THEY MADE THE WRONG DECISION.
THEY CAME ACROSS AS
STATISTICIANS AND SCIENTISTS.
THEY DID NOT COME ACROSS
AS MEMBERS OF OUR COMMUNITY.
>> MOST OF US HAVE BETWEEN THREE
AND FIVE YEARS.
WE DON'T HAVE TIME TO WAIT
FOR DATA TO FIT PERFECTLY.
AND AS LONG AS MEDICATION IS
SAFE, WE WANT THE OPPORTUNITY
TO TAKE IT.
EVEN IT IT'S PROVEN JUST
A LITTLE EFFECTIVE, THAT'S
SO MUCH MORE THAN WE HAVE NOW.
>> Reporter: KNOWING WHAT
YOU KNOW ABOUT THIISSUE,
IF YOU WERE A MEMBER
OF THE PANEL, HOW WOULD
YOU VOTE?
>> I PROBABLY WOULD SAY, NO.
I WOULD PREFER TO HAVE BETTER
EVIDENCE THAT THIS IS ACTUALLY
GOING TO HAVE SOME BENEFIT
TO PEOPLE.
>> Reporter: EVEN WITH THOSE
PEOPLE IN TEARS IN FRONT OF YOU,
IT'D BE NO?
>> YES.
YES, I'M AFRAID SO.
BECAUSE IN THIS PARTICULAR CASE
THE TEARS ARE I BELIEVE BASED
UPON FALSE INFORMATION, FALSE
HOPES.
>> THAT'S ABSURD TO ME.
BECAUSE IF IT DIDN'T WORK,
WE WOULDN'T TAKE IT.
AND FALSE HOPE FOR SOMEONE
WITH ALS IS STILL HOPE.
WE DON'T HAVE THAT AT ALL.
>> WE ASKED THE SIX PANELISTS
WHO VOTED "NO" TO PARTICIPATE
IN OUR STORY BUT THEY DECLINED
TO SPEAK WITH US ON CAMERA.
THE FOOD AND DRUG ADMINISTRATION
IS EXPECTED TO MAKE ITS FINAL
DECISION ON MYOTROPHIN
IN THE NEXT FEW WEEKS.
ALTHOUGH THE FDA IS NOT BOUND
BY THE DECISION OF THE ADVISORY
COMMITTEE,IT RARELY GOES AGAINST
THE PANEL'S ADVICE.
>>> COMING UP NEXT -- |
