New ALS drug gains FDA approval

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For the first time in 22 years, a new drug was approved by the FDA to treat ALS, otherwise known as Lou Gehrig’s Disease. Meanwhile, the yearly cost of living with ALS can reach $200,000, and the ALS Association Arizona Chapter is planning a fundraiser to help sufferers of the disease with costs. We’ll hear from Dr. Jeremy Shefner, professor and chair of neurology and senior vice president at Barrow Neurological Institute, and Taryn Norley, executive director for the ALS Association Arizona Chapter.

TED SIMONS: FOR THE FIRST TIME IN TWENTY-TWO YEARS, THERE IS A NEW DRUG THAT'S BEEN APPROVED BY THE FDA TO TREAT ALS, WHICH IS ALSO KNOWN AS LOU GEHRIG'S DISEASE (ALS). THE NEW TREATMENT PROVIDES SOME ENCOURAGEMENT FOR PATIENTS AND THEIR FAMILIES. THE DISEASE IS STILL ALARMINGLY EXPENSIVE TO LIVE WITH, THE YEARLY COST WITH LIVING WITH ALS CAN REACH 200,000. HERE TO TALK ABOUT THE NEW TREATMENT AND FUNDRAISING EFFORST TO HELP PEOPLE SUFFERING WITH ALS DR. JEREMY SHEFNER, PROFESSOR AND CHAIR OF NEUROLOGY AND SENIOR VICE PRESIDENT AT BARROW NEUROLOGICAL INSTITUTE AND TARYN NORLEY, EXECUTIVE DIRECTOR FOR THE ALS ASSOCIATION ARIZONA CHAPTER.

TED SIMONS: GLAD TO HAVE YOU. LOTS TO TALK ABOUT HERE. LET'S GET A DEFINITION OF TERMS, WHAT IS ALS?

JEREMY SHEFNER: ALS IS ONE OF A GROUP OF DISEASES CALLED NUERODEGENERATIVE DISEASES. SO IT'S IN THE CATEGORY OF DISEASES INCLUDING, ALZHEIMERS DISEASE, PARKINSON'S DISEASE. SO FOR WHATEVER REASON, WE DON'T KNOW THE REASON MOST OF THE TIME, A CERTAIN PART OF THE BRAIN BEGINS TO DEGENERATE AND IN ALS IT'S THE PART OF THE BRAIN THAT DEALS WITH MOVEMENT. SO PEOPLE START WITH A WEAKNESS IN THE HAND, LEG, THE FACE AND THE WEAKNESS JUST SLOWLY SPREADS FROM WHERE EVER IT STARTS.

TED SIMONS: SO IT ATTACKS NERVES CELLS AND PATHWAYS TO THE SPINAL CHORD? OR THAT KIND OF THING?

JEREMY SHEFNER: IT EFFECTS BOTH. THE NERVES THAT GO FROM THE BRAIN TO THE SPINAL CHORD, AND THE NERVES THAT GO DIRECTLY TO THE MUSCLE.

TED SIMONS: WHAT'S YOUR EXPERIENCE WITH ALS, IS IT HEREDITARY? SO WE SEE THIS RUNNING IN FAMILIES?

TARYN NORLEY: SO 10% OF IT IS HEREDITARY AND THEN 90% SPORADIC, WE DON'T KNOW.

TED SIMONS: SO WE REALLY DON'T HAVE ANY RISK FACTORS?

TARYN NORLEY: NO NOT THAT WE KNOW OF.

TED SIMONS: ANYTHING AT ALL?

JEREMY SHEFNER: WE'LL A COUPLE OF THINGS, STRANGLY, IT BENEFITS PEOPLE WHO HAVE THIS, IS THAT VETERANS HAVE A HIGHER INSIDENCE OF GETTING ALS THAN PEOPLE WHO HAVEN'T SERVED IN COMBAT. AND SO IT'S A SERVCE CONNECTED DISEASE FROM THE VA. SO PATIENTS WITH ALS WHO ARE VETERANS CAN GET VETERAN'S SERVICES. WHICH IS A SIGNIFICANT BENEFIT FOR THEM. THE ONLY OTHER RISK FACTOR THAT HAS BEEN AGREED ON, IS SMOKING. SMOKING DOES INCREASE YOUR RISK A LITTLE BIT.

TED SIMONS: INTERESTING. SO YOU NOW KNOW WHAT IT IS, HOW IS IT TREATED?

JEREMY SHEFNER: WE'LL I'VE BEEN TAKING CARE OF ALS PATIENTS SINCE 1990. AND WHEN I STARTED THERE WAS NOTHING TO DO, BUT TO MAKE THEM COMFORTABLE. IN 1993 THE FIRST DRUG IS STILL BEING USED TO TREAT ALS, CALLED RILUZOLE WAS APPROVED BY THE FDA. AND RILUZOLE, HAS A DEFINITE BUT VERY MODEST EFFECT. IT PROLONGS SURVIVAL FOR 3-6 MONTHS, PROBABLY IMPROVES FUNCTION A LITTLE BIT. THE HALLMARK OF CLINICAL CARE, HAS BEEN AND STILL IS, A MULTI-DICIPLINARY APPROACH TO MAKE SURE NUTRITION IS OPTIMIZED AND BREATHING HAS HELPED AS MUCH AS POSSIBLE. AND REHABILITATION IS PRESENT TO KEEP PEOPLE WALKING. BUT RECENTLY, AS YOU MENTIONED, A SECOND DRUG HAS BEEN APPROVED THAT CLEARLY IMPACTS ALS, IN A MORE SIGNIFICANT WAY THAN WE HAVE BEEN ABLE TO BEFORE.

TED SIMONS: EXTENDS LIFESPAN?

JEREMY SHEFNER: WE'LL WE DON'T KNOW THAT, BECAUSE THE STUDY THAT LED TO IT'S APPROVAL WAS ONLY A 6 MONTHS STUDY. SO WE DON'T KNOW THAT IT EXTENDS LIFESPAN AND ALTHOUGH IT SHOULD, IT SEEMS TO PRESERVE FUNCTION. AND THE LONGER YOU TAKE IT, THE BIGGER THE DIFFERENCE IS, BETWEEN THE PEOPLE WHO TAKE THE DRUG AND WHO DON'T. SO WE WOULD EXSPECT IT WOULD PROLONG LIFE.

TED SIMONS: THIS HAS TO BE ENCOURAGING NEWS FOR THE ALS COMMUNITY?

TARYN NORLEY: IT IS. THE HOPE THAT THIS DRUG IS BRINGING IS REALLY EXCITING.

TED SIMONS: AND IT ALSO MEANS THAT THERE'S A NEW DRUG OUT THERE THAT'S GOING TO HAVE TO BE PAID FOR. TALK ABOUT THE EXPENSE. I MEAN, THIS, YOU CAN REALLY RACK UP THE BILLS HERE.

TARYN NORLEY: YEAH, IT GETS VERY EXPENSIVE. AND FOR THE DRUG WE DON'T KNOW YET, AS FAR AS WHAT THE INSURANCE COMPANIES ARE GOING TO BE ABLE TO DO UNLESS DR. SHEFNER YOU HAVE ANYTHING TO SPEAK TO THAT?

JEREMY SHEFNER: WHAT THE COMPANY SAID IT IS GOING TO CHARGE, IT'S ABOUT 140,000 DOLLARS JUST FOR ONE YEAR FOR THAT DRUG.

TED SIMONS: JUST FOR THAT ONE DRUG?

JEREMY SHEFNER: IT'S AN INTRAVENOUS DRUG AND SO THE DRUG HAS TO BE INFUSED 10 TIMES A MONTH FOR IT TO BE DELIVERED TO PATIENTS.

TED SIMONS: OH MY GOSH.

JEREMY SHEFNER: SO IT'S COSTS AND EFFORTS ASSOCIATED WITH THAT AS WELL.

TED SIMONS: ALRIGHT, SO I KNOW THAT YOU HAVE AN EVENT COMING UP CALLED ‘BITE NIGHT'. TO RAISE AND HELP SOME OF THESE FOLKS. TALK ABOUT THAT.

TARYN NORLEY: SO ‘BITE NIGHT' IS A FUNDRAISING EVENT AND WE'LL HAVE 14 RESTAURANTS THAT COMPETE AGAINST EACH OTHER TO PROVIDE THE BEST BITE OF THE NIGHT. BUT THE REAL EXCITEMENT COMES FROM THIS YEAR, WE'LL HAVE OVRE 800 PEOPLE THERE ATTENDING AT THE WESTIN KIERLAND. AND WE HOPE TO RAISE A LOT OF FUNDS TO SUPPORT OUR PATIENTS. AND THAT'S REALLY WHAT THE ALS ASSOCIATION DOES HERE IN ARIZONA.

TED SIMONS: THE PATIENTS, ARE YOU SEEING.. IT TOOK 22 YEAR.. IT SOUNDS LIKE IT'S BEEN KIND OF QUIET AND KIND OF DARK FOR A WHILE. ARE YOU SEEING A LITTLE BIT OF LIGHT HERE?

TARYN NORLEY: WE ARE, YOU KNOW.. I THINK WHEN THE FDA MADE THE ANNOUNCEMENT, IT WAS A FRIDAY AFTERNOON, THE RIPPLE EFFECT. I STARTED RECIEVEING TEXT MESSAGES AND EMAILS FROM OUT PATIENTS, AND THE EXCITEMENT AND I'M SURE THE DOCTORS OFFICES WERE THE SAME.

TED SIMONS: YEAH, IT'S INTERESTING YOU SAY, TEXTS AND EMAILS FROM PATIENTS. PATIENTS, THE COGNITIVE FUNCTION OF PATIENTS, NOT AFFECTED?

JEREMY SHEFNER: IT CAN BE, SO ABOUT 20% OF PATIENTS HAVE PROBLEMS WITH THINKING, SOME OF THE ACTUALLY BECOME FRANKLY DEMENTED. BUT THE MAJORITY OF PEOPLE, GO THROUGH THIS DISEASE WITH THE SAME MENTAL STATE AND THE SAME PERSONALITIES THEY HAD WHEN THEY STARTED. IN SOME WAYS THAT'S A BLESSING AND OTHER WAYS IT'S A CURSE. BECAUSE YOU REMAIN REALLY AWARE, FOR THE ENTIRE LIFESPAN OF THE DISEASE.

TED SIMONS: AND AGAIN AS FAR AS, THE DECLINE OF THE PHYSICAL FUNCTION I WOULD IMAGINE FOR SOME IT'S QUICK AND FOR SOME IT'S SLOW. DO WE KNOW WHY?

JEREMY SHEFNER: YOU'RE EXACTLY RIGHT ABOUT THIS, THE VARIABILITY, SOME PEOPLE DECLINE FROM DIAGNOSIS TO DEATH IN ABOUT A YEAR. AND SOME PEOPLE LIVE DECADES OR LONGER. WE REALLY DON'T KNOW WHY THAT IS. AND THAT WOULD BE AN INCREDIBLY IMPORTANT CLUE TO HAVE. AND WE DON'T HAVE IT YET.

TED SIMONS: THE ALS ASSOCIATION, HOW DO YOU HELP FOLKS. IF SOME JUST FINDS OUT IF THEY OR A FAMILY MEMBER, OR A LOVED ONE HAS JUST BEEN DIAGNOSED? WHERE DO YOU COME IN?

TARYN NORLEY: SO WE PROVE, OUR BIGGEST PROGRAM IS OUR MEDICAL EQUIPMENT LOAN PROGRAM. SO WE OFFER MEDICAL EQUIPMENT TO OUR PATIENTS. IT CAN BE A POWERED WHEELCHAIR, IT CAN BE BATHROOM EQUIPMENT, WHICH CAN BE VERY COSTLY. AND WE ARE ABLE TO PROVIDE THAT TO THEM, FREE OF COST. WE ALSO HAVE SUPPORT GROUPS, YOU KNOW WHEN YOU'RE FACED WITH A TERMINAL DISEASE YOU WANT PEOPLE AROUND YOU WHO KNOW WHAT YOU ARE GOING THROUGH. SO OUR SUPPORT GROUPS HAVE BEEN VERY POSITIVE.

TED SIMONS: THE SUPPORT GROUPS I IMAGINE WOULD BE VERY POSITIVE FOR THE FAMILIES AS WELL. ARE THERE SEPARATE SUPPORT GROUPS ONLY FOR FAMILY MEMEBERS?

TARYN NORLEY: THERE ARE. SO WE HAVE OUR GROUPS THAT PROVIDES SERVICE FOR BOTH THE PATIENT AND THE FAMILY. AND WE HAVE A SEPARATE CAREGIVERS SUPPORT GROUP. AND THIS PAST YEAR WE RECENTLY STARTED OUR CHILDREN'S SUPPORT GROUPS. CHILDREN DO NOT GET ALS, BUT THEIR AFFECTED BY IT. WE SAY ALS AFFECTS THE ENTIRE FAMILY.

TED SIMONS: CHILDREN DON'T GET ALS, IS THIS AN AGE RELATED DISEASE?

JEREMY SHEFNER: IT'S AN AGE DEPENDENT DISEASE. SO THE AVERAGE AGE WHEN PEOPLE GET IT ARE ABOUT 57 TO 60. BUT I'VE HAD A PATIENT AS YOUNG AS 18. LOU GEHRIG WAS 36 WHEN HE GOT THE DISEASE AND I'VE HAD PATIENTS I'VE DIAGNOSED IN THEIR 90S. SO IT'S A HUGE SPREAD.

TED SIMONS: YOU KNOW I REMEMBER READING NOT TOO LONG AGO. LAST QUESTION HERE. THAT LOU GEHRIG MIGHT NOT HAVE HAD ALS. WHAT'S THAT ALL ABOUT?

JEREMY SHEFNER: WE'LL I THINK THAT WAS A MISREPRESENTATION. SO THAT TIES INTO THE ENTIRE NFL CHRONIC TRAUMATIC ENCEPHALOPATHY.

TED SIMONS: OH THE CTE THING.

JEREMY SHEFNER: A PATHOLOGIST IN BOSTON, LOOKED AT 10 ALS AUTOPSIES AND COMPARED THEM TO WITH PEOPLE WITH CTE AND SHOWED SOME SIMILARITIES. AND THEN MADE THE CASE THAT LOU GEHRIG, WAS A FOOTBALL PLAYER, WHO PLAYED, CORNELL WHEN THEY DIDN'T HAVE HELMETS AND MADE THE CASE IT MIGHT HAVE BEEN ALS. BUT CTE DOESN'T LOOK LIKE ALS. AND LOU GEHRIG HAD ALL IS MEDICAL FACULTIES WHEN HE DIED, AND HAD ALL THE SIGNS OF ALS AND NONE OF THE SIGNS OF CTE. SO I THINK LOU GHERIG, HAD LOU GHERIG DISEASE.

TED SIMONS: ALRIGHT, GOOD INFORMATION. GOOD TO HAVE YOU BOTH HERE, THANKS FOR JOINING US. AND GOOD LUCK AT ‘BITE NIGHT.'

Jeremy Shefner, M.D., PhD: Professor, Chair of Neurology, Barrow Neurological Institute

Taryn Norley: Exec. Director, ALS Association AZ Chapter

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