Hope Now For ALS

What is ALS?

"Every 90 minutes someone is diagnosed with amyotrophic lateral sclerosis (ALS), a progressive>neurodegenerative disease that causes muscle weakness, paralysis, and ultimately, respiratory failure.ALS, also known as Lou Gehrig’s disease, Charcot's disease, and motor neuron disease (MND), attacks certain cells in the brain and spinal cord needed to keep our muscles moving. Early signs and symptoms

of ALS include:

  • muscle cramps and muscle twitching
  • weakness in hands, legs, feet or ankles
  • difficulty speaking or swallowing

The senses, including hearing, sight, smell, taste, and touch, are not affected by ALS.There is no single diagnostic test for ALS. However, experts in the disease, usually neurologists specializing in neuromuscular diseases, are very capable of diagnosing ALS. In some cases, they might order additional tests if the diagnosis is not clear. These include:

  • electromyography and nerve conduction
  • magnetic resonance imaging (MRI)
  • genetic tests
  • muscle biopsy
  • spinal tap
  • blood and urine tests

Most people with ALS live 2-5 years after their first signs of disease. About 10% of people with ALS survive at least 10 years. This variable rate of disease progression makes prognosis difficult to predict and therapies challenging to develop. Currently, there is only a single medicine for specifically treating ALS - riluzole. The drug, marketed by Sanofi-Aventis under the name Rilutek, extends survival by only about 2 to 3 months." (ALS Therapy Development Institute)

The Mission of Hope NOW for ALS
Time for 21st Century Clinical Trials!

We are PALS (people with ALS), their friends and family members, united by our belief that we must do everything in our power to accelerate the realization of a cure. Our mission is to expedite access to promising investigational drugs for the current generation of PALS through the FDA's Accelerated Approval Program (AAP) and other pathways, and to speed up ALS drug development by promoting improved alternative and humane designs for clinical trials.

Updates

Latest From HNFA

Dear supporters, We have some exciting news to report! As you may recall, Hope NOW (HNFA) met with the FDA for the fourth time in late August and proposed a clinical study concept that incorporated predictive algorithms, more-sensitive and faster “adaptive trials” as an alternative to the current slow and less-sensitive “population mean comparison” trials, and crossover of patients from the placebo control arm to the active drug. The FDA expressed support and suggested we might want to work directly with the researchers – and we have! So far, two biopharmaceutical companies are on board. We connected them with a leading data analytics firm so that impending trials can be designed far more efficiently with faster and more meaningful results. This could pave the way for a drug or treatment to be given faster consideration for Accelerated Approval (AAP) – or even full approval (registration) – as the evidence of efficacy will be stronger. All the components of… Read More

Latest Posts

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… Read More

Latest Posts

No Accelerated Approvals (AAP) for ALS!

1) Call to Action (details below) 2)  Hope NOW will be issuing a new CHANGE.ORG petition that focuses on our current goals. Please watch for it in your email: “Terminally Ill Patients Denied Treatment” and please sign it right away. We continue to make progress in our negotiations with the FDA for smarter, faster, and more humane clinical trials. The FDA has expressed support for the study design proposed by Hope NOW and we are now scheduling meetings with researchers and drug companies to expedite its implementation. However, we have also learned the FDA will not utilize the 1992 Accelerated Approval Program (AAP) due to a technicality called “surrogate endpoints”. This strict interpretation of AAP is closing the door to fatal diseases like ALS which do not have a verified “surrogate endpoint”. ALS treatments need AAP, a “conditional approval” that allows PALS access to promising treatments after a Phase 2… Read More

Latest Posts

Hope Now for ALS – 21st Century Trial Design original proposal to FDA

ALS is a progressive neurological disorder that has always resulted in progressive deterioration and death.  There is no cure and the only approved treatment extends life on average 2-3 months.  80% of ALS patients die within 5 years of diagnosis and 90% within ten years.  ALS is an extremely heterogeneous disease, where patients progress at vastly different rates and often in non-linear fashion.  More than 20 different genetic mutations are associated with it but only explain 25% of total cases.  These facts make it very hard for a drug to pass a series of clinical trials and be approved using a traditional randomized double blind placebo test protocol.  Differences in the underlying progression rates between randomly selected placebo and non-placebo groups reduce the signal to noise ratio of observed changes in the primary endpoint during the trial period, producing significant statistical noise and limiting the ability to reach a standard… Read More

Latest Posts

Hope Now for ALS in the news!

Taking page from early AIDS activists, ALS patients push for access to experimental drug Ron Leuty Reporter- San Francisco Business Times A one-time tech security specialist, “Mike” now calls himself an amateur chemist and neurologist, seeking out new drugs to treat the deadly muscle-wasting disease known as amyotrophic lateral sclerosis. It’s not a hobby; it’s his life. Diagnosed in October 2013 with ALS — or Lou Gehrig’s disease — the 55-year-old Massachusetts resident believes he’s found hope in an experimental drug under development by a small Palo Alto company. Lab tests showed that Mike has the same makeup of biomarkers, or disease characteristics, as ALS patients who responded in a 2012 study of Neuraltus Pharmaceuticals Inc.’s drug. If only Mike and other ALS patients could get “compassionate use” access to Neuraltus’ drug, they wouldn’t have to wait for a formal clinical trial to see if the compound slows ALS from progressively… Read More

Latest Posts

Hope Now for ALS update: Summary of July 6th call

Dear Hope Now for ALS (HNFA) supporters, We apologize for the delayed update. We have been trying to schedule the next meeting so we could report that in this update. After our July 6 meeting, we submitted a detailed proposal to the FDA and requested a timely response, but it appears the FDA is stalling. They suddenly want to invite other “stakeholders” to our meeting. We believe this is a tactic to bring people and organizations into the discussion that will defend the status quo and to convert the role of actual PALS and CALS into a minority voice. With much regret, we need to report that after three meetings we see little evidence that the FDA is willing to be flexible with respect to clinical study designs and data analysis approaches that could accelerate the development and delivery of safe and effective medicines for ALS. WE WILL NOT ACCEPT… Read More

Our Next Event

Watch this space for upcoming events.


Hope NOW for ALS will be participating in the June 16, 2016 rally in Washington DC in support of three legislative bills: Right to Try, REGROW (stem cell therapies), and RESULT (treatments approved in other countries). For more information and to RSVP go to http://www.myrighttotrynow.com/.

Promising Therapies: Treatments in the Pipeline

DONATIONS

We are a volunteer organization without any paid staff. If you would like to donate to Hope NOW for ALS to cover costs for promoting our mission, printed materials, and rally expenses, please click the Donate button below. We are a 501 (c)(3) nonprofit for ALS. Donations are tax deductible: EIN 46-5766950.

 

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The History of Hope NOW for ALS

What began with a petition on Change.Org quickly evolved into a series of petitions that generated over half a million signatures worldwide. The catalyst for what is now a global grassroots patient-led movement, was a drug called GM604. In October 2014 a press release from the drug maker, Genervon, reported promising results of a Phase II clinical trial and the ALS patient community took notice. We started with petitions calling on the FDA to grant accelerated approval to GM604 and quickly followed that with the creation of social media pages and a rally in Washington DC, on March 25, 2015. Through these efforts we have learned we need to do MUCH MORE to expedite drug development and approvals for ALS and formed the new organization, Hope NOW for ALS.

 

Hope Now for Als.org

 

DISCLAIMER: Hope NOW for ALS does not endorse any particular treatment. When considering paying for treatments please ask yourself the following:

Is the treatment legal?
Do I understand and accept the risks?
Is the cost of the treatment reasonable?

It is the position of Hope NOW for ALS that sponsors of an unapproved treatment should provide it at no cost, or at the true cost of manufacturing (as required in the U.S.). ALS is an expensive disease, to say the least. Everyone needs to give careful consideration to the best use of their personal finances.

The focus of Hope NOW continues to be Accelerated Approvals (AAP) for ALL promising treatments, a benefit to both patients and the developers of these treatments.