♪ >> CLIMBING TO NEW HEIGHTS, SPENDING TIME IN NATURE, SOMETHING THIS FAMILY LIVES FOR.
>> CHRISTINA AND I HAVE ALWAYS BEEN AN OUTDOOR FAMILY.
WE ALWAYS WENT ON HIKES AND CAMPING TRIPS.
>> BUT THE LAST FEW YEARS HAVE BEEN TOUGH.
WE SAT DOWN WITH THEM OUTSIDE THEIR GRAFTON HOME TO TALK ABOUT THEIR JOURNEY.
IT BEGAN WITH A SUBTLE SYMPTOM.
>> HER VOICE WENT NASAL.
EVERYTHING SHE WAS SAYING SOUNDED LIKE SHE WAS TALKING THROUGH HER NOSE.
>> DOCTORS ASSURED CHRISTINA IT WAS NOTHING TO BE CONCERNED ABOUT, BUT AS THE DAYS PASSED, SHE NOTICED SMALL CHANGES AND BIG ONES.
SHE HAD BEEN STUDYING SIGN LANGUAGE, BUT SUDDENLY LOST THE ABILITY TO MANEUVER HER FINGERS.
MORE THAN A YEAR AFTER HER VOICE CHANGED, DOCTORS KNEW HER PROBLEM WAS CAUSED BY A NEUROLOGICAL DISORDER.
SIX WEEKS LATER, A DIAGNOSIS, ALS.
WE KNEW STEPHEN HAWKING’S HAD ALS.
IT WAS ONLY AFTER GETTING THE DIAGNOSIS CHRISTINA AND I REALIZE IT IS A TERMINAL DISEASE.
>> ONE THAT HAS RAPIDLY CHANGED THE LIFE OF THIS FAMILY OF FIVE.
>> IT IS A CONTINUOUS, GRADUAL DECLINE.
ANYTIME THERE IS A NEW THING SHE CANNOT DO, YOU GET USED TO THAT.
THEN THERE IS SOMETHING ELSE SHE CANNOT DO.
REPORTER: BUT THERE WOULD BE HOPE BORN IN A COLLEGE DORM ROOM BY A STUDENT AT BROWN UNIVERSITY WITH A VORACIOUS APPETITE FOR READING.
>> I GOT INTERESTED AND CURIOUS IN NEURODEGENERATIVE DISEASE AND REALIZED THERE ARE INFINITY PAPERS OUT THERE AND YOU CAN KEEP GOING THROUGH THEM.
>> REPORTER: TWO WEEKS, JUST TWO, BEHIND CLOSED DOORS AND JOSHUA COHEN APPEARED WITH AN IDEA.
>> JOSH CAME TO ME ONE EVENING AND SAID, HEY, I HAVE AN IDEA FOR TREATING ALZHEIMER’S AND ALS.
I SAID, I HAVE NOT SEEN YOU IN A FEW WEEKS SO I GUESS THAT EXPLAINS IT.
REPORTER: JOSHUA FOUND SUPPORT.
>> I KNEW HE WAS CRAZY, BUT THAT IS WHAT WAS SO 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 THE QUESTION OF, HOW DO NEURONS DIE?
WHAT ARE THE DECISION POINTS, THE CHOKEPOINTS, HOW DOES IT ULTIMATELY HAPPEN?
DESPOND THE IDEA TO FORM A DRUG THAT TARGETS THE PATHWAYS THAT RESULT IN A NEURON DYING.
IF YOU BLOCK THOSE, MAYBE YOU CAN MAKE NEURONS LIVE STRONGER AND FUNCTION LONGER.
REPORTER: INVESTORS WERE CHARMED BY WHAT THEY WERE PROPOSING, A COMBINATION OF TWO DRUGS THEY BELIEVED WOULD STOP NEURONS FROM DEGENERATION, BUT THEY WERE TURNED OFF FROM THE HURDLES.
ONLY TWO DRUGS FOR THE TREATMENT OF ALS HAVE BEEN APPROVED BY THE FDA, AND OF THOSE WERE DEVELOPED BY WELL-ESTABLISHED COMPANIES.
>> MANY INVESTORS TOLD US, WE HOPE YOU ARE SUCCESSFUL, BUT THAT IS A TOP DISEASE.
I AM NOT SURE I WANT TO INVEST IN THAT.
REPORTER: AGAIN, TIMING.
THE FORMATION OF THEIR COMPANY, AMYLAX PHARMACEUTICALS, COINCIDED WITH THE ICE BUCKET CHALLENGE, BRINGING ATTENTION TO THIS DISEASE AND NEW SUPPORT FOR COLLEGE FRIENDS, NOW COFOUNDERS.
>> EVERYONE KNEW WHAT A BIG DEAL IT WAS.
WE WERE LUCKY TO RECEIVE SUPPORT FROM THE ALS FOUNDATION.
PART OF THE FUNDING CAME FROM THE ICE BUCKET CHALLENGE.
REPORTER: CLINICAL TRIALS OF THE TREATMENT DEVELOPED BY THE COMPANY IN PARTNERSHIP WITH RESEARCHERS AT MASS GENERAL HAVE SHOWN ENCOURAGING RESULTS >> WE TOOK IT THROUGH THE
VARIOUS PRECLINICAL STUDIES ONE NEEDS TO SEE IF THIS IS A DRUG CANDIDATE.
ULTIMATELY, THE MOST IMPORTANT THING, CAN THIS HELP PEOPLE?
ALONG WITH OUR COLLEAGUES AT MASS GENERAL AND AROUND THE COUNTRY, RAN A CLINICAL TRIAL PEOPLE WITH ALS.
WE ARE REALLY EXCITED THAT LAST DECEMBER WE UNBLINDED THE TRIAL AND THE DRUG WORK.
PEOPLE RETAINED FUNCTION MUCH LONGER ON KEY AREAS LIKE READING, SWALLOWING, WALKING.
WE ARE TREMENDOUSLY EXCITED AND OPTIMISTIC THIS MIGHT BE A NEW TREATMENT FOR PEOPLE WHO REALLY NEED IT.
REPORTER: ABOUT THREE YEARS AGO CHRISTINA WOULD BE THE FIRST OF 137 PATIENTS TO RECEIVE THE DRUG.
>> IT HAS BEEN VERY EVIDENT IT WAS WORKING.
THERE WAS A BRIEF PERIOD WE WERE NOT ON THE DRUG AND DURING THAT PERIOD, THERE WAS A RAPID DIG RUSSIAN.
ONCE ON THE DRUG AGAIN IT STABILIZED AND SLOWED THINGS DOWN AGAIN.
REPORTER: WHAT WAS ONCE INSPIRATION INTO A MISSION.
>> YOU REACH A POINT WHERE THE DRUG GOES FROM AN OPPORTUNITY TO A RESPONSIBILITY.
IF THIS IS A TREATMENT THAT CAN HELP THEM, WE NEED TO DO EVERYTHING WE CAN TO GET THIS TO PEOPLE AS SOON AS POSSIBLE.
>> [INAUDIBLE] SHAYNA: SINCE WE FILMED THIS STORY, PROMISING NEW FINDINGS WERE PUBLISHED ON IN THE 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 PATIENTS ARE GETTING YOUNGER AND YO