Terminally Ill Denied Treatment – that Healthy People are Paid to Try

In Phase 1 trials, healthy people, often students, are offered money to test new drugs for safety.  In a Phase 2 trial, people with the targeted disease are enrolled and may experience benefits – only to be denied access at the end of the trial.  For terminal diseases like ALS that is a death sentence!


Hope NOW has launched a new Change.org petition at www.change.org/hopenow

featuring Amanda Bernier, https://www.facebook.com/AmandasAngelsALS. Amanda is the young mother and former firefighter who learned she had ALS at age 29, just weeks after becoming pregnant. We are petitioning Congress and the FDA to grant terminally ill people the right to try investigational therapies by utilizing the Accelerated Approval Program (AAP) the way it was intended.  In 1992, in response to HIV/AIDS activism, the FDA adopted the Accelerated Approval Program (AAP) – a conditional approval to treat this fatal disease. Later AAP was applied to treatments for heart failure and cancers.  So why isn’t it being applied to ALS and other rapidly fatal diseases!

Hope NOW  is currently focused on two initiatives:
#1: “21st Century Patient Rights”  
We continue our political effort to pressure Congress and the FDA for the “right to try” investigational therapies and to apply the 1992 Accelerated Approval Program (AAP) as it was intended, so that all citizens, regardless of their socio-economic status, have genuine access through their health insurance.
The PROBLEM:  The FDA will not grant Accelerated Approval (AAP) to any ALS treatment because they claim the disease needs to have a validated “surrogate endpoint”.  FDASIA passed in 2012 by Congress clearly states that actual “clinical endpoints” can be used. The FDA’s position appears to be in violation of  FDASIA and willful resistance to using AAP which allows a conditional approval prior to Phase 3 trials.  Hope NOW is calling out the FDA for not following the will of Congress and the American people.  It is our position that patients, in consultation with their doctors, have the “right to try” investigational therapies.
#2:  21st Century Trials
We have been meeting with the FDA since May 12, 2015 to share the “state of the science” and urge the FDA to accept and adopt faster, smarter, and more humane clinical trials. Our 4th meeting with the FDA took place Aug 24, 2015.

The FDA still adheres to a 1962 clinical trial protocol that is fixated on an arbitrary statistical number.  This outdated protocol has created a multi-billion dollar clinical trial industry and approvals that takes an average of 12 years at a cost of up to $1 billion for a new treatment.  The FDA has not uniformly adopted current state-of-the-art science applications for trials.  Unlike 1962, today we have computers to analyze large databases, strategically populate trials, utilize complex predictive algorithms, and detect small changes and patterns in clinical data.

*A surrogate endpoint is typically a biomarker intended to substitute for a clinical endpoint to save time and money.  It is  most useful in slow-moving diseases where measuring the progression of clinical endpoints (e.g. patient function or survival) could take years. The ALS research community has not yet concluded there is a verifiable biomarker for ALS. However, forced vital capacity (FVC), can and should be regarded as both a clinical and a surrogate endpoint.

Amyotrophic Lateral Sclerosis (ALS) or Motor Neuron Disease (MND) is a degenerative neurological disease that affects neurons in both the brain and spinal cord, causing voluntary muscles throughout the body to wither. It is always fatal, on average within 3 years. It afflicts people of all races, genders and ages.  People with ALS (PALS) lose the ability to eat, drink, speak, walk, use their arms and hands, and ultimately breathe. Up to 6,000 U.S. citizens are diagnosed with ALS each year, and up to 35,000 have the disease at any given time.

There are experimental drugs showing promise that may improve quality of life, and slow or even stop the progression of this horrific disease – BUT WE ARE DENIED ACCESS TO THEM UNDER OUR CURRENT SYSTEM.

9 thoughts on “Terminally Ill Denied Treatment – that Healthy People are Paid to Try”

  1. My mom died of ALS. The only FDA APPROVED drug to “slow” progression did the contrary. As soon as my mom began taking it, she got worse. I’m not blaming them, but just pointing out it was nice to have hope and a chance. We would do it again even tho it made the disease progress faster. I just think that if you are terminal and rapidly declining from ALS, you should have a choice to TRY anything that has a little hope….Instead of waiting. We would’ve tried anything we COULD and we did. I hope they let others at least try and have a CHANCE

  2. Please Do Not let this opportunity for help be denied . We need a cure now ! Time is running out for a lot of people. Think about what you would do if your Husband, wife,father, mother,son, daughter or family member was effect by this…… I bet you would feel more empowered to approve it !!!! Thank You

  3. This mom and child touch my heart…please give her the medication she need…that she will be able to raise her beautiful baby daugther. Blessings!!!

  4. Have a very dear friend with end stage ALS and if there is anything that can be done I hope that can happen.

  5. Please help people, with ALS, so that they can live a longer and happier life. God Bless them and anyone else with a chronic disease, where no cure, has been found to work yet.

  6. My mother got this awfull dieses. With no hope patients wither and die. This needs to be changed. My mother passed in 2003. She got her diagnosis in July. There is no one test to get a diagnosed. A neurologist at the Mayo Clinic ask my mother to stick out her tounge. The outside of her tounge quivered. With that she was told she had ALS and passed Oct. 26.

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