NICOLE: CLIMBING TO NEW HEIGHTS, SPENDING TIME IN NATURE.
IT IS SOMETHING THIS FAMILY LIVES FOR.
>> CHRISTINE AND I HAVE ALWAYS BEEN ANY OUTDOOR FAMILY.
WE ALWAYS WENT ON HIKES AND CAMPING TRIP.
-- CAMPING TRIPS.
NICOLE: THE LAST YEARS HAVE BEEN TOUGH.
WE SET DOWN OUTSIDE THEIR HOME TO TALK ABOUT THEIR JOURNEY.
IT BEGAN WITH A SUPPLE -- A SUBTLE SYMPTOM.
>> HER VOICE WENT NASAL.
ALL OF A SUDDEN EVERYTHING SHE WAS SAYING SATELLITE SHE WAS TALKING THROUGH HER NOISE -- THROUGH HER NOSE.
NICOLE: DOCTORS ASSURED HER IT WAS NOTHING TO BE CONCERNED ABOUT BUT SHE NOTICED SMALL CHANGES.
SHE HAD BEEN STUDYING SIGN LANGUAGE BUT SUDDENLY LOST THE ABILITY TO MANEUVER HER FINGERS.
MORE THAN A YEAR AFTER HER VOICE CHANGED, FINALLY DOCTORS NEW HER SYMPTOMS WERE CAUSED BY A NEUROLOGICAL DISORDER.
SIX WEEKS LATER, A DIAGNOSIS.
ALS.
>> WE KNEW STEPHEN HAWKING ONLY HAD -- HAD ALS BUT ONLY AFTER SHE WAS BIG NOSED DID WE REALIZE IT WAS A TERMINAL DISEASE.
IT IS A GRADUAL DECLINE SO EVERY TIME THERE IS A NEW THING SHE CAN’T DO YOU GET USED TO THAT.
THEN THERE IS SOMETHING ELSE SHE CAN’T DO.
NICOLE: THERE WOULD BE HOPE.
BORN IN A COLLEGE DORM ROOM FROM A GRADUATE WHO HAD AN APPETITE FOR READING.
>> I REALIZED THERE WERE INFINITY PAPERS OUT THERE AND YOU CAN KEEP GOING THROUGH THEM.
NICOLE: TWO WEEKS BEHIND CLOSED DOORS AND JOSHUA REAPPEARED WITH AN IDEA.
>> WE WERE FRIENDS IN COLLEGE.
JOSH CAME TO ME AND SAID I HAVE AN IDEA FOR TREATING ALZHEIMER’S AND ALS.
AND I SAID I HAVEN’T SEEN YOU IN A FEW WEEKS SO I GUESS THAT EXPLAINS IT.
NICOLE: JOSHUA FOUND SUPPORT IN JUSTIN.
>> I KNEW IT WAS CRAZY BUT THAT WAS WHAT WAS EXCITING ABOUT IT.
THE AMBITION OF WHAT IF WE HAVE A TREATMENT SOLUTION.
WE MET UP AND TALKED ABOUT IT AND DECIDED TO START A COMPANY.
>> WHAT I WAS FOCUSED ON WAS THIS QUESTION OF HOW DO NEURONS DIE, WHAT ARE THE CHOKEPOINTS?
THAT SPOT THE IDEA TO MAKE A DRUG THAT TARGETED THE KEY PATHWAYS THAT RESULT IN A NEURON DYING.
IF YOU CAN BLOCK THOSE, THEN MAYBE THEY CAN LIVE LONGER AND FUNCTION LONGER.
NICOLE: INVESTORS WERE TRUMPED BY WHAT THEY WERE PROPOSING, A COMBINATION OF TWO DRUGS THEY BELIEVED WOULD PROTECT NEURONS FROM DEGENERATION.
THEY WERE TURNED OFF BY THE HURDLES, ONLY TWO DRUGS FOR ALS HAVE BEEN APPROVED.
THOSE WERE DEVELOPED BY WELL-ESTABLISHED COMPANIES.
>> MANY INVESTORS TOLD US I HOPE YOU ARE SUCCESSFUL BUT THAT IS A TOUGH DISEASE AND I’M NOT SURE IF I WANT TO INVEST IN THAT.
NICOLE: AGAIN, TIMING.
THE FORMATION OF THEIR COMPANY COINCIDED WITH THE ICE BUCKET CHALLENGE BRINGING ATTENTION TO THIS DISEASE.
AND NEW SUPPORT FOR THESE COLLEGE FRIENDS, NOW ARE FINDERS -- NOW COFOUNDERS.
>> WE WERE LUCKY TO RECEIVE SUPPORT FROM THE ALS ASSOCIATION AND PART OF THAT CAME FROM THE ICE BUCKET CHALLENGE.
NICOLE: CLINICAL TRIALS IN PARTNERSHIP WITH RESEARCHERS AT MASS GENERAL HAVE SHOWN ENCOURAGING RESULTS.
>> WE TOOK IT THROUGH THE CLINICAL STUDIES THAT ONE NEEDS TO SEE IF THIS IS A DRUG CANDIDATE.
THE MOST IMPORTANT THING IS IS THIS SOMETHING THAT CAN HELP PEOPLE?
ALONG WITH OUR COLLEAGUES AT MASS GENERAL AND AROUND THE COUNTRY WE RAN A CLINICAL TRIAL.
WE WERE REALLY EXCITED THAT LAST DECEMBER WE UNBLINDED THE TRIAL AND THE DRUG WORKED.
PEOPLE RETAINED FUNCTION MUCH LONGER ON KEY AREAS LIKE RE-THINK, SWALLOWING, WALKING.
WE ARE TREMENDOUSLY EXCITED AND OPTIMISTIC THIS MIGHT BE A NEW TREATMENT FOR PEOPLE WHO NEEDED.
NICOLE: THREE YEARS AGO, CHRISTINA WOULD BE THE FIRST OF 137 PATIENTS TO RECEIVE THE DRUG.
>> IT HAS BEEN EVIDENT THAT BEEN WORKING.
THERE WAS A BRIEF PERIOD WE WERE NOT ON THE DRUG AND DURING THAT PERIOD THERE WAS A RAPID DECLINE.
WHEN WE WENT BACK ON, IT STABILIZED AGAIN.
NICOLE: IT HAS TURNED WHAT WAS JUST INSPIRATION INTO A MISSION.
>> REACHED A POINT WHERE THE DRUG WAS FROM ANY OPPORTUNITY TO A RESPONSIBILITY.
IF THIS CAN HELP THEM, WE NEED TO DO WHATEVER WE CAN TO GET IT TO THEM.
>> IT MEANS SO MUCH.
IT IS AMAZING.
ANTHONY: SINCE WE FIRST AIRED THIS STORYPROMISING NEW FINDINGS WERE PUBLISHED IN THE JOURNAL, "MUSCLE & NERVE." THE CLINICAL TRIAL FOUND PATIENTS WHO TOOK THE SAME
MEDICATION THAT CHRISTINE TOOK LIVED A MEDIAN SIX AND A HALF MONTHS LONGER THAN THOSE WHO RECEIVED A PLACEBO.
COMING UP, COLON CANCER PA