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Singing for a Cure

Deborah Silver, an accomplished vocalist and performer, has been active in the fight against ALS since 2009, when her sister was diagnosed with the disease.

My sister, Marjie Block, was diagnosed with ALS in 2009. I was by her side visiting doctor after doctor hoping to get any diagnosis other than ALS. We even prayed for Lyme Disease….anything but ALS! Ultimately, this unfortunate diagnosis stayed the same and Marjie’s courageous battle began.

As our family rallied behind her, we became determined to win this seemingly unwinnable ordeal and have committed ourselves to fighting alongside all ALS patients. My children have launched several projects to raise funds for a cure including my daughter’s website, hopeheARTbyMadison.com, so far raising over $100,000. Having always felt there is a reason for everything, perhaps this disease ended up in our family because we simply will not rest until ALS is eradicated. This is why I am always trying to come up with ideas of how to raise even more funds for a cure.

Throughout this process, I have met some of the most impressive and special people in the world who also happen to be battling ALS. My heart goes out to everyone and their families struggling with this horrendous disease.

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FDA Accepts New Drug Application for Potential ALS Treatment Edaravone

Following up on our June 2016 blog post about edaravone, an intravenous drug therapy produced by Mitsubishi Tanabe Pharma Corporation, locally based in Jersey City, N.J., with a head office in Osaka, Japan, there has been recent news as to the status of the drug in the U.S.

At the end of August 2016, the U.S. Food and Drug Administration (FDA) accepted a New Drug Application (NDA) for edaravone, which has been used to treat stroke victims in Japan for the last twenty years. In a phase II clinical trial of edaravone, in a sub-group of people with ALS in Japan, researchers found a statistically significant improvement in the ALS Function Rating Scale-Revise. Edaravone works by getting rid of toxic by-products generated in cells, which is thought to protect against oxidative stress.

An “NDA” includes a comprehensive overview of a drug, including results and data analysis for the entire clinical development program and earlier preclinical testing, as well as the proposed labeling and manufacturing plans of the new drug.

This is extremely positive news for the ALS community, as acceptance of the NDA is the final stage of the development and approval process. Moreover, this is the first time that the FDA has ever accepted an NDA without first requiring an Investigational New Drug Application (IND), something that would have delayed the delivery of edaravone by many years. We appreciate the FDA for taking this step to help speed the approval of a potential ALS therapy. If approved, edaravone would be commercialized under the brand name Radicava™

While the company requested “priority review” for edaravone, which would have meant a six-month review time, the FDA accepted the application with a standard ten-month review. This means that a decision on the application is expected by June, 2017 based on timelines set by the Prescription Drug User Fee Act. This does not mean that the review will take the full ten months or preclude the FDA from acting sooner. We have been in contact with our colleagues at the FDA to reiterate the urgent need to move forward as quickly as possible.

The ALS Association joins the ALS community in urging a timely review of edaravone so that, if approved, it becomes available to people living with ALS as soon as possible. Every day makes a difference to people and their families fighting this disease. This truly is an exciting development for the ALS community, as it’s only the third potential ALS drug in history to advance this far in the drug development process (Riluzole and Myotrophin were the first two).

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“Not Alone” – A Daughter’s Perspective

Jordan Jhaveri is part of a family living with ALS. She was eleven when her dad, Akhil, was diagnosed in 2011. Here she gives her sixteen-year-old perspective that was recently posted on her dad’s blog recently.

As my dad’s health declines it’s gotten a lot harder to “keep on keepin’ on.” I’ve decided to share my perspective so that maybe other people can know they are not alone in their battles.

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Jordan Jhaveri

No one is truly “put together.” No one actually has everything “under control.” I am convinced that life is a big card game in which it is part chance, part strategy and planning, and we make up all the rules as we go. Even when someone seems to be completely untroubled, there is no way to know what happens behind closed doors.

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The ALS Association Care Services Survey Results

The ALS Association is pleased to share the results of the 2016 Care Services Community Survey. Below we offer some background on the survey and highlight a few significant responses. We also provide information on how you can give additional feedback on The Association’s programs in September.

Background

In July 2016, The ALS Association invited members of the ALS community (defined as people living with ALS, family members, and caregivers) to participate in a community care services survey. This survey included 15 questions and was shared via several of The Association’s communication channels. Nearly 800 members of the community responded.

Providing multiple opportunities for people living with ALS to comment on our work is vital, and the insights gained from this survey will be combined with the results of our ongoing series of Listening Tour meetings with the ALS community. We will use all of this information to inform strategic planning, help identify and prioritize program initiatives, focus on potential program improvements, and contribute data and information for advanced decision-making.

Survey Highlights

When asked what programs they have accessed through local ALS Association chapters, 52 percent responded that they have utilized an equipment loan closet. Forty percent visited a certified center or recognized clinic, 34 percent accessed a support program, and 31 percent received an education service from their chapter.

When asked which areas The Association should be devoting more care services resources to, more than half of respondents chose assistive technology (58 percent) and home health benefits (52 percent). Thirty-nine percent chose programs that support increased access to care and services, while 36 percent wanted more resources related to Medicare, Medicaid, and veterans insurance programs.

Thirteen percent of respondents also indicated that finances, costs, and/or insurances make up the greatest challenge that they and their family face. Other significant challenges include accessing home health benefits (10 percent) and equipment loans (nine percent).

For more information about the survey, including responses for all questions, read the full report.

Another Opportunity for Feedback

Did you miss the chance to take the Care Services survey? We’re providing another opportunity for the ALS community to give feedback during our second “virtual listening tour” on September 27, 2016 from 1-3 p.m. EDT. Register here!

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Every Bravery Adds Up

Jeanna Grammer lives in Tuscaloosa, Alabama. She is employed at a non-profit Hospice and feels so blessed in making a difference! Her true passion is being a voice for people with ALS by volunteering with her local ALS Association chapter and writing letters in her local newspaper.

My mom was diagnosed with ALS in May 2011. She literally lost her speech within 6 weeks of slurring her words. She was semi-retired but enjoyed her job and co-workers, so she still worked part time at a hospital as an insurance biller. Bravery!

Unfortunately, she had to retire just two months later because she was unable to talk to insurance companies on the phone. She retired with over 25 years and the retirement celebration was fit for a queen! She wasn’t sad that day; she loved seeing all her friends, co-workers and family. She never lost her loud Italian laugh and it was heard many times at the celebration. Bravery!

"Every Memory Adds Up"

Every Memory Adds Up

Megan Wilkerson lost her father, Dr. Walter Root, to ALS earlier this year. Her family’s story was recently profiled in a feature article in the San Antonio Express-News. In her #EveryDropAddsUp story below, Megan describes the value of the memories she was able to make with her father and family after his diagnosis.

My dad lost his battle to ALS on May 31, 2016. I had known this day would come since his diagnosis on January 5, 2015. We had our suspicions something “bad” was happening with Dad, but ALS was something that happened to ‘other people,’ not us. I had become aware of the disease due to the Ice Bucket Challenge in Summer ’14, but had NO idea what would lie ahead.

Our family decided to embrace the diagnosis and enjoy our lives together. Since there is no cure or effective treatment for ALS, we knew the likely hood of him being “cured” was dismal. My dad chose to live with his disease, even though we lost him a little each day. He traveled, he loved, he laughed, he lived. I can honestly say (with many tears) he lived 100 years in his 59.

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"Every Thought Counts"

Every Thought Counts

Caroline Tredway’s sister, Nell Hardy, is currently living with ALS. In response to her sister’s #EveryDropAddsUp story, Caroline submitted her own essay, titled  “Every Thought Counts.”

Every Thought Counts

Picture, for a moment, that you’re in a straitjacket. You can’t move any limb to free yourself. All day and night long.

That’s how I imagine my younger sister Nell has felt over the past seven years as she battles the biggest foe of her life: Amyotrophic Lateral Sclerosis, or ALS.

I think of Nell when I do the simplest of things that are out of reach for her.

"Every Person Adds Up"

Every Person Adds Up

Julie Fitzpatrick’s grandmother (above left) died of ALS and Julie (above right) has recently been diagnosed with Primary Lateral Sclerosis. Julie lives in Reston, Virginia and is a scientist, specializing in human health risk assessment, for the U.S. Environmental Protection Agency.

ALS changes countless lives. It is nearly impossible to count every person.  It is not just the people with ALS, but everyone they touch and everyone who touches them and on down the line.

From the time I was a small girl I knew that my grandmother, Mildred Anderson, died from complications resulting from ALS.  She died in February 1960, when my mother was preparing to give birth to my older brother.  One of the big regrets of my mother’s life is that her mother never got to meet her four children and see what wonderful people they became. It is not just every person that has ALS that adds up, it is all the people in their lives and those that come after them. It could be said that “Every Family Adds Up.”

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“Me, You, and Lou” – A Letter from Someone Newly Diagnosed with ALS

Adam Smith, 37, was an overly active person until a mysterious, undiagnosed illness turned his life upside down. He saw multiple doctors until one realized the problem was not in his head, but very real. After a trip to the Mayo Clinic, Adam was diagnosed with Lou Gehrig’s Disease in May 2016. After his diagnosis, Adam sent the following letter to his friends and family. He is supported and loved by his wife, family, friends, and sweet Akita.


Dear Friends,

To point at a day on the calendar as the best and worst day of your life is a rare feat. On May 27, 1985, my wife was born – best day of my life. On May 27, 2016, I was told I’m dying – worst day of my life.

“You have Lou Gehrig’s Disease. There is no treatment, there is no cure. I’m sorry, Adam.”

Mayo Clinic, Rochester, MN
May 27, 2016

As my doctor delicately explained that my body will slowly become paralyzed over the next 2-5 years until I can no longer take a breath, my thoughts drifted to my wife and our families. Turning 31 and being told your husband is dying is unimaginable. I could only think how every birthday for the rest of her life she will have a painful memory that will never fade over time. Thirty-one is too young to be told you’re going to be a widow. It’s too young for dreams to be crushed and vows to become impossible to achieve. Thirty-one is too young to be told you’re going to be taking care of your sick husband for the rest of his short life. Heartbreaking.

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Researchers Identify a New Therapeutic Target for C9orf72-Associated ALS

Researchers supported by The ALS Association announced that they had identified a new protein called SUPT4H1 that has potential to be used in therapy development for people whose ALS is caused by the C9orf72 gene. This research was published in the journal Science by Drs. Aaron Gitler at Stanford University in Stanford, California and Leonard Petrucelli at the Mayo Clinic in Jacksonville, Florida. Read on to learn more about this latest discovery.

Why SUPT4H1?

Drs. Gitler and Petrucelli targeted SUPT4H1 since it was shown that reducing its presence decreases the production of proteins containing long repeat expansions associated with another neurodegenerative disease, Huntington’s disease. However, reduction of SUPT4H1 did not affect expression of normal, non-expanded proteins.