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Sign to support treatment for ALS: Expanded Access Programs and Accelerated Approval (GM604, NurOwn, etc.)

This petition had 4,490 supporters


Show support for Cheryl and Other ALS Patients:

Step 1. Sign this petition with at least your name, city, state/country and zip/postal code.

Step 2. Forward this petition to your network via email, Facebook, Twitter or other social media.

Step 3. Visit “I Can’t Wait”-PALS First Campaign to read personal stories, information about ALS, and learn how you can help.

Step 4: Join the "I Can't Wait" -PALS First Campaign on Facebook to keep updated on the petition.

ALS (Motor Neuron Disease or Lou Gerig’s Disease): Cheryl’s Story

Cheryl (Duluth, Minnesota, USA) devoted mother, wife, first-rate paralegal, compassionate church and community leader and active outdoors enthusiast was diagnosed with Amyotrophic Lateral Sclerosis (ALS) in 2011 at the Mayo Clinic in Rochester, Minnesota (to read more about Cheryl's and other ALS stories, please follow our blog http://alsicantwait.blogspot.com/).

ALS, more commonly known as Lou Gehrig’s disease, is a type of motor neuron disease in which the body attacks motor neurons, or the nerve cells found in the brain, brain stem and spinal cord resulting in the loss of control of muscle movement.  Patients progressively worsen losing the ability to walk, move their arms, eat food or swallow, speak or breathe on their own, eventually resulting in death. 

ALS Patients Running Out of Time for Available Treatments

There is no cure for ALS.  Treatment focuses on symptom management.  There are a few clinical trials offered for medications, treatments, and procedures.  Unfortunately, due to costs they are limited to only a few patients and have strict research criteria that leave many not eligible to participate.   

Most ALS patients live on average 2 to 5 years after diagnosis.  It takes 8 to 20 years to bring medications, treatments, and procedures from research and clinical trial phases to market.  ALS patients need treatment now.  We simply Can’t Wait. 

Hope through Expanded Access Programs (EAPs) and Accelerated Approval

There is still hope for Cheryl and the estimated 8,000 U.S. citizens, 19,000 European citizens and countless others who are diagnosed with this incurable disease each year and their families.  The answer is Expanded Access Programs (EAPs) and Accelerated Approval. 

EAPs and Accelerated Approval allow dying ALS patients who do not qualify for clinical trials a chance to explore new, promising medicines, treatments, and procedures.  Examples of promising ALS treatments include:

·      NurOwn patient bone-marrow derived stem cells (BrainStorm Cell  Therapeutics, Ltd.)

·      GM604 (Genervon Biopharmaceuticals, LLC)

·      NeuralStem Cell Therapy (NeuralStem, Inc.)

·      Ozonezumab/Nogo-A inhibitor (GlaxoSmithKline)

·      Tirasemtiv (Cytokinetics, Inc.)

·      Arimoclomol (Orphazyme ApS)

·      NP001 (Neuraltus Pharmaceuticals, Inc.)

Access to EAPs and Accelerated Approval Possible with Cost-Recovery, Opens New Research Opportunity

A major concern for pharmaceutical and therapeutics companies and the treating hospitals with respect to offering expanded access to patients is recuperation of costs.  Working with a third party non-profit organization such as the ALS Emergency Treatment Fund, we believe will help resolve this issue by opening up grant and other funding opportunities as well as an option for patient cost-sharing in order to provide potentially life-saving treatment for those in ALS.

We can learn a lot from EAPs and Accelerated Approval that will help tailor treatments for ALS patients.  Due to costs, clinical trials only allow for small numbers of participants, which lowers a study’s statistical power needed to show safety and effectiveness with confidence.  Many clinical trials also have a wait-in period or cannot proceed until the study has reached its participant quota, or amount of people necessary for the trial.  By the time the trial starts, participants that were previously eligible may suddenly become ineligible because of their disease progression.  Including individuals with late-stage ALS through EAPs and Accelerated Approval would allow the research to be more representative of the actual ALS population.  

You have the power to save Cheryl and other ALS patients. 

Step 1. Sign the petition. 

Step 2. Forward the petition to your contacts. 

Step 3. Visit “I Can’t Wait”-PALS First Campaign.

Step 4: Join the "I Can't Wait" -PALS First Campaign Facebook Community Page.

(c) This petition is copyrighted.  Permission must be obtained from creator to use any of the wording.



Today: Elise is counting on you

Elise Lamont needs your help with “Expanded Access and Accelerated Approval of ALL ALS Treatments (GM604, NurOwn, etc.)”. Join Elise and 4,489 supporters today.