CHALLENGE A FEW YEARS BACK. IT
WAS A VIRAL FUNDRAISER FOR
RESEARCH ON A L S LOU GEHRIG'S
DISEASE FOR A WHILE. IT MAY
HAVE SEEMED LIKE EVERYONE KNEW
SOMEONE WHO WAS DUMPING ICE
WATER ON THEIR HEADS HERE YOU
SEE NEARLY 500 PEOPLE DOING IT
IN CENTRAL PARK BACK IN 2015.
NOW, AS IT TURNS OUT, THE ICE
BUCKET CHALLENGE HELP FUND A
POTENTIAL BREAKTHROUGH, AN
EXPERIMENTAL DRUG. A L S
STANDS FOR A MILD TROPHY
LATERAL SCLEROSIS. IT'S A RARE
NEUROLOGICAL DISEASE THAT
AFFECTS THE NERVE CELLS
RESPONSIBLE FOR VOLUNTARY
MUSCLE MOVEMENT SYMPTOMS GET
WORSE OVER TIME. SO WALKING OR
TALKING OR SWALLOWING IT MORE
DIFFICULT TO CONTROL AND
CURRENTLY A LESS HAS NO CURE.
NOW ADVOCATES ARE PUSHING THE
FDA TO APPROVE THIS
EXPERIMENTAL DRUG. BUT WE JUST
LEARNED TONIGHT. BUT AN FDA
ADVISORY PANEL HAS VOTED NOT
TO ENDORSE IT. THAT MEANS THEY
DID NOT CONSIDER THE DATA
STRONG ENOUGH TO SUPPORT
APPROVAL. BUT THIS WILL STILL
GO THROUGH TO THE FULL FDA FOR
A FINAL DECISION. JOINING US
NOW IS DOCTOR STEVEN SHELL,
SOME UNDER ALLAH. JUST WHO
LEADS THE A L S CENTER AT NEW
YORK'S MOUNT SINAI HOSPITAL.
HE'S BEEN INVOLVED IN THESE
DRUG TRIALS. DOCTOR SHELL. SO
WELCOME TO THE PROGRAM.
>> ALL RIGHT. THANKS. WHAT DO
YOU MAKE OF THE FINDING FROM
THIS ADVISORY PANEL. WELL, IT
WAS IT WAS GARY WAS SUGGESTING
NOT APPROVED THE DRUG. YOU SAY
THERE'S MIXED FEELINGS EVEN ON
THE PANEL. IT'S A IT'S
DIFFICULT BECAUSE THIS WAS A
PHASE 2 STUDY. A SMALLER DRUG
TRIAL. THAT WAS TO LOOK AT SAFETY AND TRENDS TOWARD
SAFETY AND TRENDS TOWARD
EFFICACY. IT. IT WAS WELL
DESIGNED AND IT IT DID SHOW
OUR TREATMENT AFFECTING THE
PRIMARY OUTCOME MEASURE. LESS
FRS, WHICH IS THAT OUR WHICH
IS FUNCTIONAL RATING SCALE
USED TO CHECK. THERE IS MOTOR
FUNCTIONS IN THE BODY AND. YOU
KNOW, WE SHOWED IN THE FACT
USUALLY THESE SMALLER STUDIES
NEED CONFIRMATION BY A BIGGER
LARGER TRUMP ORIGINALLY
ANALYTICS THE PHARMACEUTICAL
COMPANY WAS ASKED START OF
PHASE 3 TRIAL LARGER, MORE
DEFINITIVE, MORE STATISTICALLY
STRONG TRIAL AND THAT'S ON
BECAUSE A GREAT NEED OF OUR
PATIENTS TO HAVE EFFECTIVE
TREATMENTS THIS TO TREATMENTS.
WE HAVE. REALLY JUST VERY MODEST BENEFIT IN-STORE
MODEST BENEFIT IN-STORE
DISEASE. IT WAS IT YOU KNOW,
IS WE REALLY NEED MORE DATA.
>> BUT THE PATIENTS PUSH BUSH
FOR THIS. AND YOU KNOW, AND I
UNDERSTAND. BUT WE REALLY DO.
I REALLY LIKE TO SEE THE
LARGER TRIAL TO CONFIRM THE
SPINE.
>> WELL, I CAN I CAN
UNDERSTAND AND I CAN I CAN
TELL YOU'RE DISAPPOINTED BY
THE ADVISORY PANELS VOTE,
ALTHOUGH GRANTED THIS DOES
STILL GO TO A LARGER PANEL FOR
A FINAL DECISION. AND WE KNOW
THIS HAS BEEN A LONG TIME
COMING. I MEAN, PAT QUINN, WHO
IS THE CO-FOUNDER OF THE ICE
BUCKET CHALLENGE SADLY DID NOT
LIVE TO SEE HIS MOVEMENT FUND.
THIS POTENTIAL A NEW DRUG.
TALK ABOUT HOW PEOPLE WHO
DON'T UNDERSTAND A L S ABOUT
HOW WE TREAT A LESS TODAY AND
WHAT THE HOPE WAS FOR THIS
MEDICATION. HOW THAT WOULD
ADVANCE THE TREATMENT.
>> WELL.
>> YOU KNOW.
>> YEARS AGO, DECADES AGO WE
HAD A DRUG REALLY AND IMPROVED
SURVIVAL BY A COUPLE OF MONTHS
AND THAT'S STILL VERY ACTIVE
TREATMENT TODAY AND AND IN
2017 AND APPROVED. THIS IS AN
IV TREATMENT. THEY ALSO SHOWED
MODEST EFFECT OF SLOWING
PROGRESSION OF THESE
FUNCTIONAL SCALE BLS FRS SAW.
THAT LOOKS TO STRENGTHEN MEASURES. A FUNCTION OF ISSUES
MEASURES. A FUNCTION OF ISSUES
AND. IT'S
>> SORT OF A SWEET THAT'S
OKAY. YOU'RE DESCRIBING THAT
THEY'RE BEEN MET. THE BEEN
SOME TREATMENTS THAT HAVE BEEN
AVAILABLE FOR YEARS AND YEARS.
IT SOUNDS LIKE WHAT YOU'RE
SAYING IS THAT JUST NOTHING
REALLY.
>> RIGHT THIS. IS THAT TRUE
BASED ON A SINGLE JAPANESE
STARTED, BUT IT WAS IT WAS A
PHASE. 3 TRIAL. SO WE NEED
BETTER TREATMENTS TO SLOW THE
DISEASE AND MISS.
>> AND THIS STROME LOOKS VERY
PROMISING. AND IN THIS STUDY
WAS PUBLISHED IN THE NEW
ENGLAND JOURNAL OF MEDICINE
BECAUSE IT WAS VERY
SCIENTIFICALLY SOUND OUR
CENTER IS PART OF THIS REPORT
OF MEALS IN NORTHEASTERN A
LESS IS A CONSORTIUM. AND A
MASS GENERAL WHERE THE
PRINCIPAL INVESTIGATORS.
>> YEAH, I KNOW I HAVE TO LET
YOU GO IN A MINUTE, DOCTOR.
BUT I JUST WONDER WHAT IT'S
MEANT FOR YOU AS A RESEARCHER
AND AS A CLINICIAN TO HAVE
THIS KIND OF PUBLIC SUPPORT
BEHIND THAT. SOME OF THE ICE
BUCKET CHALLENGE IS KIND OF A
FAD WHEN IT HAPPENED. PEOPLE
GOT A LITTLE MORE AWARENESS OF
A LESSON THEN LIFE WENT ON.
BUT THIS IS KIND OF WHAT THE
INTERNET CAN DO. WHAT IS NOT
DECIDING TO SHARE TED VIDEOS
AND VIRAL MEMES AND QANON
DIS-INFORMATION, RIGHT. THIS
IS LIKE THE PART OF THE
INTERNET. EVERYONE KIND OF
DREAM. THE INTERNET COULD BE
VERY PRO SOCIAL WHAT IS IT
MEANT FOR YOU AS A RESEARCHER
TO HAVE THIS KIND OF PUBLIC
SUPPORT.
>> I THINK IT'S GREAT AT THAT.
THE FDA IS LISTENING TO
PATIENT ADVOCACY GROUPS AND
LISTENING TO PATIENTS NOW. AND
SO THEY REALIZE THE PATIENTS.
WE ORPHAN DISEASE, A RARE
DISEASE WITHOUT EFFECTIVE
TREATMENTS ARE WILLING TO TAKE
MORE RISKS AND THEY THEY'RE
WILLING TO EVEN TO TRY DRUGS
THAT MAY HAVE MARGINAL
BENEFIT. SO THAT'S WHY THEY
REALLY PUSHED FOR THIS. AND IT
WOULD BE NICE TO SEE THE FDA
APPROVED. THIS WOULD SOME SORT
CONDITIONAL APPROVAL. YOU KNOW
THAT IF THAT THE CREW WHOLE
DEPEND IN FINAL APPROVAL
DEPEND ON THAT RESULTS OF THE
LARGER PHASE 3 TRIAL.
>> AND BRIEFLY BEFORE I HAVE
TO LET YOU GO. WHAT'S THE NEXT
STEP IN THIS. IS THERE
ANYTHING BUT THE RESEARCH TEAM
CAN DO TO ADD MORE EVIDENCE
FOR THE FULL FDA OR IS IT KIND
OF FULLY IN THE FDA'S. AND
RIGHT NOW BEFORE WE GO.
>> YOU KNOW, I THINK THAT THE
COMMITTEE SUBMITTED ITS REPORT
AND NOW IT'S UP TO THE FDA
MEAN THAT THERE WAS, YOU KNOW
THAT IN THIS DRUG THAT WAS A
CLINICAL EFFECT AND WELL AND
PATIENTS WANT IT. AND WE'LL
HAVE TO SEE WHAT THEIR FINAL
DECISIONS.
>> DOCTOR STEVEN CHELSA OF
MOUNT SINAI HERE IN NEW YORK.
I APPRECIATE YOU BREAKING THIS
ALL DOWN FOR SIR. THANK YOU