An Open Letter Regarding STX209 to Dr. Severin Schwan, Roche Pharmaceuticals

To see Dr. Schwan’s response to this letter please see here:  Roche Response

Dr. Severin Schwan
Chief Executive Officer
Roche Pharmaceuticals
F. Hoffmann-La Roche AG
Grenzacherstrasse 124
CH-4070 Basel

June 10, 2013

Dear Dr. Schwan:

Behind every drug Roche Pharmaceuticals makes are the personal stories of patients and their families. You must hear these stories everyday, and we cannot imagine the pressures you face determining where to put your resources and whose lives – whose children’s lives – you will try to save. We understand you cannot support every drug and every trial, but you are a father, so surely you will understand our desperation and need to reach out to you directly to help our children. Like most parents, we are certain you would go to any lengths – contact any person – if your children needed help; so you’ll understand why we are writing you this letter.

Recently, Roche Pharmaceuticals was involved with a Cambridge, Massachusetts-based pharmaceutical company called Seaside Therapeutics, Inc., which has been developing a drug called STX209 (Arbaclofen).  This compound has been in Phase III trials for children with fragile X syndrome.

When Roche pulled back on its R&D, it ended its involvement and financial support for STX209. We understand there have been issues with the therapy’s trial results, but it had statistically relevant improvements to secondary outcome measures. Luca Santarelli, head of neuroscience research at Roche has been quoted as saying “We concluded that arbaclofen wasn’t going to provide that real difference for patients.” Dr. Schwan, we respectfully disagree, the improvements we have seen in our children have changed the lives of our families.

Without Roche’s backing, on May 15, 2013, Seaside Therapeutics was forced to issue the following statement to the clinics involved in the administration of Arbaclofen:

We regret to inform you that Study 209FX303 [An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of STX209 (Arbaclofen) in Subjects with Fragile X Syndrome] is being terminated immediately.  The closure of the study is due to resource limitations at Seaside Therapeutics, Inc., and is not related to any known safety issues in patients dosed with STX209.

As of July 1, 2013, none of our children will have access to STX209. The abrupt end of the study have left some of our children to suffer withdrawal symptoms. But that is hardly a consideration compared to what is about to happen to us all.  We’d like to share with you the thoughts of just a few of the families in the trial.

From Cortney AbouElSeoud, mother of Ayden, age 5:

“A little background first.  Ayden had a 5-word vocabulary, but seldom spoke.  Within one week of treatment with Arbaclofen, his tantrums diminished noticeably.  Within a month, he started using words spontaneously.  Now seven months into treatment, he has surpassed development goals in every area.  He speaks in small sentences, can isolate the sounds of the alphabet and can trace all his upper and lowercase letters.  On Arbaclofen, he could enter first grade on par with his peers.”

“I’ve often heard fragile X described as having the sound of a vacuum in your head.  Consider going through life daily with that sound and attempting to speak or to walk into a large room full of people.  Imagine how difficult it would be and how easily you could become frustrated.  Imagine then that there was something you could do to shut that noise off.  That is how I look at Arbaclofen…. Now we have to turn that switch back on.  Devastated does not even begin to describe the emotions I am feeling.  Terrified.  Worried.  Overwhelmed…. And the tears.   Because they can’t find enough money to finish the trial, I am facing a reality where I may never again hear my son say “mommy.”

From Deanna Acree, mother to Kolton Acree, age 5:

“Imagine your child is unable to tell you their tummy hurts; unable to make friends at school even though they love other kids; unable to run a quick errand with you because the store may over-stimulate them; unable to look at you at night while you tuck them into bed.  Just imagine!  Your child is locked in a world that no one can understand; they try and try but everyday life is a struggle. Now imagine your child is sick, he tells you what hurts; he goes to school, and has friends high fiving him in the hallway; he hugs you before bedtime and whispers ‘I love you.’  How do you look at your child and tell them their time in this world is over?  How can you possibly explain that they have to go back to that frightening, isolating world of their own?”

From Aimee & Josh Freedman, parents to Nathan & Zakira Freedman, age 7 & 12:

“Parents of children with special needs are often told that their feelings of grief are similar to the feelings of a death of a loved one.  You need to mourn the life that you had envisioned for your child and your family and start a whole new dream or reality.  We had already said our goodbyes to the life we thought we were going to have.  After 20 months of watching Nathan progress incredibly, we are now told it will all be taken away… sorry folks, no money for your child to keep taking a medication that has been absolutely life changing.  Now we must mourn again, once simply wasn’t heartbreaking enough.  Now that we have seen our child happy; learning to read; talking so much; having haircuts that we don’t have to hold him down for; waking up smiling instead of screaming… now that we have seen it, we will have to watch it all taken away.  How can anyone watch their child regress into a world that is so hard and uncomfortable for them that all they can do is yell?”

From Tiffany Pace, mother to David & Jonathan Wright, ages 13 &6:

“I’ve seen so many amazing things in the time since David started Arbaclofen.  He started reading! Not just sight words he’d memorized, but really reading! We never thought he’d learn to read at all.  He started doing math problems on his own. His handwriting became legible.  He began participating in class.  A year ago, we were planning his very first, EVER, birthday party, with friends! He was able to tell me who he wanted to invite.  For the past two years, he let us sing “happy birthday” to him – that was never possible before Arbaclofen.  He hugs and kisses me… he hugs and kisses his grandparents!  When he was little, I couldn’t even get him to hold my hand when crossing a street – that kind of small touch caused a complete meltdown.  He even hated being held as a baby.  Now he chooses to hug me!  I am completely terrified at the thought of losing Arbaclofen.  What if his rages come back? What if all these new skills go away?  I don’t know how to explain to David what is about to happen.  To either of my sons.  What will happen to my children when Arbaclofen is taken away?

From Melissa Welin, mother to Caleb Welin, age 9:

“I woke up the morning after I learned that the trial for STX209 was over and stood crying at the counter while making Caleb’s breakfast – which is always accompanied by a spoon of applesauce and his morning meds – knowing that we were one dose closer to the end.  I know nothing in this business is guaranteed; there’s a lot of money in pharmaceuticals because it’s expensive work.  Changing the world takes lots and lots of money.  I refuse to believe that this is as far as the story goes.  I’ve never expected a fairy tale ending, but this defies logic.  We are almost there.  In the face of incredible odds, Seaside has gotten us to the point where we can see the goal line.  Now we need that one person to stand up and clear the way.  Please help us.”

Stop for a moment and think about what these parents consider amazing – a hug from their child; hearing the words ‘I love you;’ watching their children learn to read, make a friend, have a life. Imagine your baby is born and doesn’t want you to touch them. They grow up and can’t speak to you. They scream and yell and you can’t make them feel better. Then… there is what seems like a miracle drug. Suddenly, you are able to get to know your child for the first time. You are able to share affection and celebrate amazing milestones. Milestones parents ought to be able to take for granted.

We know that as CEO, you are responsible to your Board and to shareholders; you have to make decisions based on cost/benefit analysis and put your resources behind the most promising therapies. But please, on behalf of our children, please find a way to reinstate some funding for the study of STX209. Ideally, we’d ask that you continue the trials and explore expanded use. But at a minimum, we ask that you help Seaside provide enough supply of Arbaclofen for the patients in this study who have benefited so dramatically.

We are sharing our stories as widely as possible in the hope of finding a way of turning this nightmare into a dream-come-true. On May 30, 2013, The Boston Globe published a story and on June 6, 2013, the New York Times did as well.  We have also reached out to our supporters in Congress. But we realize, ultimately, it will likely be your decision.

Please help us keep the children we have loved for so long, but only just recently had the chance to meet. Please, read the stories of the many families who are crying out for your help at Fragile X and Autism Families for STX209. We appreciate all the support and guidance you can provide.

We look forward to hearing from you with hope in our hearts.

Very sincerely,

If you would like to sign on to the letter and support our families, please enter your information below and click Submit.

Kimberly Dexter
Lynn, MA
Katy Long
Peoria, AZ
Laura McNall
Tucson, AZ
Ashley Materniak
Glendale, AZ
Matthew Shubeck
Linden, NJ
Zoe Kors
El Segundo, CA
Nancy Adalian
Visalia, CA
Nikki Norvell
Colorado Springs, CO
Elizabeth Christiani
Linden, NJ
Lloyd Pace
Visalia, CA
Holly Hogan
Hornell, NY
Doug Friedman
Flagstaff, AZ
Marsha Stickling
Dunlap, IL
Elona Swinehart
Goodyear, AZ
Meegan Croskey
Prescott, AZ
Caryn Peksa
Trinity, NC
Patrick Loader
Ventura, CA
Amanda Mahoney
Tulsa, OK
Derek Montgomery
Pekin, IL
Carla Parker
Des Moines, IA
Boise, ID
Kristin Sukhu
Laveen, AZ
Kathleen Baldukas
stoughton, WI
Bridget Foster
Litchfield Park, AZ
Nanette Mulvey
Pekin, IL
Cassie Anchors
Atlanta, GA
Stephanie Bradshaw
Tempe, AZ
Kaley Maley
Lawrence, KS
Cori Dawson
Canton, IL
Dianne Kixmiller
Kansas City, MO
Jayne Duggan
Melbourne, Australia
Kathleen Eck
Hutto, TX
sherril phillips
canton, IL
Jani Willis
Gardner, KS
Heidie Hornstra O’Neill
Flagstaff, AZ
Jani Willis
Jennifer Weitz
Southbury, CT
Allison Faulkner
Raytown, MO
Brooke Mabry
Marion, NC
Melissa Cutlip-Conley
Charleston, WV
Sharon Spencer
lakewood, CA
Melissa Perrine
Canton, IL
Denise Parker
Indianola, IA
Amanda Holton
Kansas City, KS
Ellen Gumbleton
Perth, Australia
Calloway Deegan
Lawrenceville, GA
Carrie Gravitt
Cuba, IL
Erin CrapserErin
Overland Park, KS
Christina Johnson
Canton, IL
Faye Kuperman
Lexington, SC
Shae Duggan
Ballarat, Australia
Diane Reed
Woodhaven, MI
Louise Melotte
Ballarat, Australia
Shae Duggan
Ballarat, Australia
Samantha Dudac
Southington, CT
Gerry Kraaijenbrink
Longmont, CO
Lia Codreanu
Niles, IL
Heather Kraaijenbrink
Longmont, CO
Pamela Christian
LaCygne, KS
Sarah Munt
B, Australia
Leah Geisert
Rancho Santa Margarita, CA
Molly Brown
Kingsport, TN
Mary Huelsmann
Wildwood, MO
David Lungarini
Ansonia, CT
Marcia Wentzel
Mesa, AZ
Jo Egitto
Hutto, TX
james pagdon
elizabeth, NJ
Ronit Sanders
Seattle, WA
Rachelle Zaborac
canton, IL
Dan Brughelli
San Jose, CA
Marie Murphy Hall
Hornell, NY
Chalsey Neva
Marion, IA
Shaylan Zanecki
Salt Lake City, UT
Rebecca Ellis
Mandeville, LA
Patrick Holton
Kansas City, KS
Mary Edwards
Tempe, AZ
Patricia Martin
Glendale, AZ
Tammie Anderson
marksville, LA
Ericka Fannon
Macomb, IL
Darla Parker
Altoona, IA
Angie meyers
canton, IL
Kris Miller
Cincinnati, OH
amber appello
slatington, PA
Bridget Holton
Kansas City, KS
Rose Rivers
Chicago, IL
Sajini Yogasundram
Leawood, KS
Linda Morton Hunter
Brookings, OR
Jeanine Gaumer
Canton, IL
Gary West
Kingsport, TN
Carolyn Siebert
Albuquerque, NM
Jennifer Mindell
Litchfield park, AZ
Holly blue
canton, IL
Mary Crippen
Chanhassen, MN
Beth Jacks
Wexford, PA
Bridget Dixon
Lawrence, KS
Lorie Sims
Brierfiled, AL
L Parker
Titusville, FL
Eric Cruse
Lawrence, KS
Melissa Hall
Friendswood, TX
Angie Taylor
Canto., IL
Eric Parker
Chandler, AZ
Angie Taylor
Donald Dancak
San Antonio, TX
BIlly Murphy
Hornell, NY
Denise Coleman
Canton, IL
Patricia Davis
kingsport, TN
Dominic Serrano
Kansas City, KS
Kelly Hilgefort
Fort Loramie, OH
Maureen Stein
Canton, IL
Amanda Hill
London, Canada
Megan Powell
Kansas City, MO
Kristen Freeman
San Jose, CA
Chrystal Redding
Flagstaff, AZ
Treena Roads
Jacksonville, IL
Lindy Rubio
Shepherd, MI
Jodi Hawkswell
Great Meadows, NJ
Debbie Planas
Miami, FL
Sue Wilson
Canton, IL
Pam Shoemaker
Rockledge, FL
Shari Peters
Normal, IL
Bridget Gordon
Redondo Beach, CA
Tamika Scott
Syracuse, NY
Jason Waldman waldman
Emmaus, PA
Tracy Hannam
Peoria, IL
Annette Pequignot
Mahomet, IL
Tamika Scott
Syracuse, NY
Leiver Garcia
El mirage, AZ
Katie Hencak
Melbourne, FL
Dom Fin
Fairfield, CT
Michelle Lee
Melbourne, FL
Elaina Aponte
west end, NC
Schirlla Lopes
Vila Velha, Brazil
Amy OConnor
Elkhorn, NE
Jessica Kooiker
Boyden, IA
Maria Smith
Baton Rouge, LA
Tara Pc
Chandler, AZ
Toni Wilson
Stanley, NC
Yvonne Key
Susan Furlow
Mulkeytown, IL
Viviene McNamara
Lawrenceville, NJ
Jay Adams
Wheeler, OR
Michele Jump
Canton, IL
Jennifer Libling
Southlake, TX
Catherine Gynn
Columbus, OH
Chad Green
Canton, IL
Lucas La Vista
Roselle park, NJ
Tom Turon
Linden, NJ
Cathleen Snyder
Pontiac, IL
Cyndi Hornbeck
Hornell, NY
Angela Gargano
Linden, NJ
Kathryn Mendoza
Titusville, FL
Kira Shmuler
Florham Park, NJ
Stephanie Hulshof
The Woodlands, TX
gina brenotn
union, NJ
cathy woerly
canton, IL
Dr. Marry Peterson-Suri
Southern Pines, NC
Monique Sofronski
Voorhees, NJ
Gwen Miller
Hamilton, Canada
Dawn Gipson
Melbourne, FL
Kelly Luthman
Russia, OH
LeAnn Feeman
Canton, IL
Carrie Funk
Surprise, AZ
Jeff Marshall
Crestview, FL
Elizabeth Overstreet
Laurel Hill, FL
Naomi Marshall
Rockledge, FL
Trae Buchanan
Houston, TX
Kristie Meyer
Fort Loramie, OH
Mary Belk
Pekin, IL
Sherri Johnston
Frankfort, IN
Michelle Pariseau
Vernon, Canada
Tina Nation
Flagstaff, AZ
Kim Garren
Canton, IL
paul & verlyn oedewaldt
peoria, IL
Melissa Donahue
East Peoria, IL
Heidi Yerbic
Canton, IL
Jason Dunham
Hornell, NY
Terryl Garrett
Visalia, CA
Roy Cremeans
Maywood, IL
Lisa Schwartz
Pamela Von Kaenel
Glendale Heights, IL
karen malmgren
canton, IL
Biliana Kaneva
Redwood city, CA
Amy Allard
Canton, IL
Delina Howard
Glen Arm, MD
Ginger Hammachi
Indialantic, FL
kelly taylor
canton, IL
Jillian Widger
Eddyville, IL
Faviola Schilling
Coquitlam, Canada
Melissa Edwards
Canton, IL
Emily Witte
Traverse City, MI
dorothy kissel
saginaw, MI
Brenda Rose
Canton, IL
Pamela Montez
Kansas City, MO
Sandi Rouse
Encinitas, CA
Mary Morosetti
Melbourne, FL
Sandra & Dick Lusink
Alpine, CA
Michelle Lew
Spring Valley, NY
Sue Rothert
canton, IL
Nicole Trowbridge
Kansas City, MO
Rachel Taylor
Canton, IL
Ted Rumler
Canton, IL
Karen Goger Karen
Kenilworth, NJ
Sandra Kennedy
Canton, IL
Melissa Saner
Omaha, NE
Susan Ellicott
Titusville, FL
Jeanine Gaumer
Canton, IL
Angelea Karr
Bloomington, Il, IL
Gary Beall
Deer Creek, IL
Rhonda Phillips
Litchfield Park, AZ
Kelly Wojtkowiak
Almond, NY
Ruth Weyers
Canton, IL
Sally Fiala-Farhang
Lakeside, MT
Eileen Parker
Tempe, AZ
Shelley Schiesswohl
Freeland, MI
Paula Fasciano
Morganville, NJ
Sherri Burgess
Hoschton, GA
Jennifer Pawlik
Chandler, AZ
Amy Fyle
Mesa, AZ
kathryn hartwell
Hornell, NY
Joanne Pignatore
Hillsborough, NJ
Jennifer Kirshner
Phoenix, AZ
Dannielle O’Hare
Melbourne, FL
Jennifer Rinaldi
Hornell, NY
Eric Welin
Cambridge, MA
Patricia Larkin
Le Roy, IL
Paige Edwards
Canton, IL
Marie-Luise Faber
Lansdowne, PA
Ginny Walker
Goodyear, AZ
Michael DiSarno
Gillette, NJ
Kari Schwartz
Hornell, NY
Adam Stewart
Buckeye, AZ
Michele Beeman
Allegan, MI
Cathryne Chatmon
Dallas, TX
Kristen Stewart
Buckeye, AZ
Jamie Rice
Gardner, KS
Michele Dixon
Linden, NJ
Angie Evans
Canton, IL
Kathy Morley
Nutley, NJ
Leslie Willis
Goodyear, AZ
nicole pierro
kenilworth, NJ
Christie Clark Friedman
Flagstaff, AZ
Nikki Gray
Douglas, MA
Laurie escobar Escobar
Miami, FL
julie mcmahon
purcellville, VA
Janice Riesterer
Sandusky, OH
Margaret Buchwald
Yorktown, VA
Denise Hall
Newport News, VA
Charity Diehl
Norton, OH
Lori Fuller
Elwood, UT
Jenniffer Swank
Canton, IL
Elaine Forman
Hayden, ID
orlando, FL
Jackie Skwira
Parlin, NJ
Patrick Gargano
Linden, NJ
Amy Neader
Powell, OH
Jennifer Hartmann
Kenilworth, NJ
Susan Kahan
Hinsdale, IL
Cindi Logue
Palm Bay,
Carrie Robertson
Fairfield, CT
Jeanette Crane
Visalia, CA
Jeffrey Butler
Shillington, PA
Barbara Voris
Shillington, PA
Catharine Campbell
Albuquerque, NM
Los Luna’s, NM
Josephine Gargano
Linden, NJ
Lance Kisamore
Baton Rouge, LA
Barb Long
Canton, IL
Gail Strommer
Royal Palm Beach, FL
Allan & Marilyn Kelly
Baton Rouge, LA
Hilary Downing
Whitehouse Station, NJ
Margarita DiSalvo
Tampa, FL
Michael and Elizabeth Ramey
Los Lunas, NM
Robin Nichols
Bethesda, MD
Brooke Stack
Olathe, KS
Melissa Rittenhouse
Fort Loramie, OH
Cindee Phillips
Indianapolis, IN
Jennifer Sims
Noblesville, IN
Maureen Hirscsh
Seven Lakes, NC
Sule Oztaskin
Staten Island, NY
Leslie Barger
Martinsville, VA
Shana Erenberg
Chicago, IL
Alex Planas
Miami, FL
Lisa Harrison
Canton, IL
Chris Sims
Noblesville, IN
Robbin Legg
Burlington, Canada
Angela Spoto
Kenilworth, NJ
Melissa Villalobos
Goodyear, AZ
Nathan Nuesse
Phoenix, AZ
Julio Cajaraville
Miami, FL
Sheri Mainprize
Ancaster, Canada
Ginat Gilad
Henderson, NV
Patrick E S Fancher
Hollister, CA
Tonya Clenney
colona, IL
Stacey McCune
Sudbury, MA
Kellie Lucas
Barnegat, NJ
Kristie Jackson
Kennebunk, ME
Susan Taylor
Lewistown, PA
Orna Camargo
Manchester, CT
Kathleen R. Goulding
Visalia, CA
Charlene Jackson
Kennebunk, ME

canadian web hosting