Autism Clinical Trials for Suramin

Hi, everyone, my name is Noelle. And if you told me 10 years ago that I’d be asking you to help fund what has the potential to be one of the greatest medical breakthroughs of our time, I would have never imagined it.

 I’m the mom of three beautiful children, Dominic, Luke and Brooke. Dominic is my firstborn. He turned fourteen today and so many emotions come up for me every April.  Every year that he gets older, and he moves farther from his developmental age and closer to adulthood, different levels of grief come up for me. 

I relive the day he was diagnosed with autism when I felt  like someone physically punched me in the stomach and sucked all of the air and life out of me.  I naively asked the Developmental Pediatician who diagnosed him, "what is the cure?" She looked at me like I had two heads and said "there is no cure."  I will never forget that moment as long as I live, and as my husband and I walked out of her office down the hall, I told him that we will find a way and find a cure. Since that day I have searched, researched, experimented and done every therapy possible, only to find mild gains, but certainly not "recovery".

Every year I brace myself for new milestones missed, middle school dances not attended, no parties with friends, no peers to text or hang out with, no going to high school and having all the typical high school experiences, no girlfriend, no sports teams, and the list goes on and on. Every year there wil be more heartbreak for us and so many other families living with this condition. As I watch my other children grow up and hit their milestones such as, getting their driver's licencse, going to college, getting married and having children one day. I worry about Dominic and how he will feel when his siblings grow up and move on, how loneliness, isolation, boredom amd deprerssion is a major comcern for adults living with autism .  It can be heartbreaking and it takes deep soulful  work to waIk in gratitude each day for what I do have.  One thing I do have is a purpose driven life to do everything I can for my son and others like him.

In 2015, Dominic spent 16 days in the ICU in a New York hospital after enduring three brain surgeries to stop a lifetime’s worth of debilitating seizures. On top of autism, he also has intractable epilepsy, which means seizures that do not repsond to medication. We were incredibly lucky that Dominic successfully came through his surgeries and has been seizure free for a year and a half . One night, as I sat by his bed in that ICU, gazing at his sweet face framed by swaths of bandages, I had one of those awakenings – those spiritual “flashes” - where you realize you’re in this world to do something much bigger than you were ever prepared to do. I became clear about my life's mission: I would do everything I could to fund research designed to help people - not just children, but also teens and adults - living with autism.

 But aren’t there countless treatments for autism? Don’t most autistic people lead happy and productive lives if they just get enough therapy? For many that is just not true and only a small percentage of the very high functioning have this luxury.

You might think, by glimpsing at Dominic, that he’s a typical teen: tall, strong, handsome, curious, loves girls and his cell phone.

 But typical stops there.

Autism severely diminishes most areas of Dominic’s life. His language and communication skills are impaired, as are his motor planning, ability to learn, executive functioning, social skills, and his sensory system.  As a result of all of these stresses, his anxiety level is almost always sky-high and any change or deviation from his daily routine causes meltdowns. His emotional life is always on the brink of collapse. His nervous system is like a hurricane. He also has gastrointestinal difficulties. Though Dominic is able to speak (many people with autism are non-verbal), he spends so much of his time obsessing over his passion of photographing street signs and how he catalogs and sorts these photos. We would love to one day have a real conversation with him, to see him make a friend and to be interested in the world around him.

Now that he has entered adolescence, my husband and I worry about who will take care of him when we are gone.  Where will he live? Will he be able to have a life filled with meaning and purpose and loving relationships? Will he ever be safe?  Most importantly, who will love him?

 Autism is more prevalent than all other childhood disorders combined - yet it receives less research dollars than any other single disorder. I know that great change is brought about by people personally affected by issues, whose passions drive research forward. But too often, parents raising a child with autism are too overwhelmed with caring for their child. They just don’t have the energy or resources to make a difference. 

In searching for effective treatments and research that can truly make a difference for the symptoms which isolate and exhaust my beautiful boy, I came across some incredibly promising research coming out of the University of California, San Diego.  I found an extraordinary man by the name of Dr. Robert Naviaux, a physician and specialist in human genetics and metabolism, who has a career track record of making important scientific discoveries. The autism community is so fortunate to have this brilliant mind turn his thinking towards autism.

Dr. Naviaux, a Professor at UC San Diego, and his dedicated team of scientists have spent decades investigating the response of cells to various triggers – biological, environmental and genetic. As a result of this research, the team has discovered potentially the first effective treatment for the symptoms of autism: Suramin, a drug originally used in Africa to treat “sleeping sickness.”  Fortunately, with funding, gaining FDA approval for Suramin will be much quicker than for new compounds.    

Dr. Naviaux’s initial animal model work, testing Suramin to relieve the symptoms of autism, was ranked the most viewed paper in 2013 by the Simons Foundation Autism Research Initiative (SFARI).

Dr. Naviaux and his team at UC San Diego have now completed the first clinical trial of Suramin to treat children with autism spectrum disorders.

Dr. Naviaux has been doing clinical trials for over 20 years and says he has never seen anything like this before.  A full scientific report of the results likely will be published in the first half of 2017.  

Several more clinical trials, culminating with a multicenter clinical trial, will be necessary to collect all the data the FDA will need to review and approve Suramin for the treatment of autism. In order to continue the important momentum behind this work, Dr. Naviaux needs $1.5M to continue the clinical trials.  Because his work is innovative, philanthropy is playing a critical role in providing initial results that could lead to NIH funding for additional trials. 

Dr. Naviaux’s mission is to provide children and families with a safe and effective treatment for the symptoms of autism. There is no other medical treatment available which provides real hope for those living with autism – and time is of the essence. 

As of this writing, Dr. Naviaux’s work has been on hold since he finished the first clinical trial in March 2016 because funding is not yet in place. For those of us who have children, teens and adults living with autism, every day of living life in sensory crisis is another day of happiness and independence lost.  Can you imagine if you had a child with a disorder that impacted not just one facet, but EVERY facet of his life (sensory, motor, communucation, socialization, gastrointestinal, conginitve)? Wouldn't you do everything you could to find answers? How frustrated would you be knowing there is groundbreaking, gamechanging research that in five years could bring FDA approal for a drug that can reverse autism by turning on development, and NOTHING is happening??  Dr. Naviaux needs $1.5 million for the next phase of clinical trials. In the world of fundraising this is not a lot.  

The rates of autism are climbing every year with no end in sight, this is everyone's problem and it is my strong belief that we all need to worry about the future generations and our grandchildren down the road. An MIT researcher, Stephanie Seneff, Phd,  claimed that “At today’s rate, by 2025, one in two children will be autistic.”  We must do something about this epidemic that is claiming our best and brightest.

So please join me in supporting the next phase of Suramin trials, which will bring us closer to changing the lives of thousands of children like my brave Dominic.

I am donating proceeds from my GoFundMe page to support Dr. Naviaux's lab at UC San Diego. This much-needed support will help fund clinical trials to get Suramin approved for the treatment of autism.

 My family, along with so many families living with autism, will be so grateful for your support. This can mean the difference between living a life of “just getting by” and living the independent life all of us so richly deserve.

Please shaere this page and help bring this cause to the forefront!

 From our family to yours,

Thank you.