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Advocating for Change on Capitol Hill: 7 Years and Counting

Janet Palkewick Advocates for ALS on Capital Hill
Janet Palkewick Advocates for ALS on Capital Hill

Janet Palkewick is defying the odds, living with ALS for nearly 27 years. She was diagnosed in 1989 while pregnant with her fourth child. When she hit the 20 year mark, she decided it was time to join the larger fight to end ALS.

“I realized I was still living for a purpose. I decided it was time to get more involved with finding a cure,” Janet said.

Which is why Janet decided to become an ALS advocate. She will be heading to Capitol Hill with The ALS Association for the 8th time this May for our Public Policy and Advocacy Conference along with her children and grandchildren. This important conference includes a day of lobbying elected officials on Capitol Hill.

“Having 7 years of experience, I’m very comfortable speaking about ALS and asking for the needed funding. Is it worth the time, expense, being drained both physically and emotionally? A great big YES,” Janet said. “I feel accomplished, I was heard. I made a difference.”

Janet admits sharing her story with lawmakers can be difficult but says the lawmakers have always been receptive.

“I have never had a bad meeting. The lawmakers or their aides have listened with genuine interest and concern. It is very important for lawmakers to understand that ALS does in fact affect the whole family. We need to show the personal side of ALS, put a face on the disease. Sometimes it gets very emotional; there are lots of tears. But that’s okay, it’s making ALS real,” Janet said.

Staying motivated and positive isn’t always easy, but Janet says the progress she has seen since her diagnosis keeps her motivated.

“More has been accomplished toward finding an ALS cure in the last 10 years than the previous 70 years. Actually, since my diagnosis 27 years ago, the most headway has been made in the last 5 years. I am hopeful! I must remain hopeful that there will be a cure in my lifetime,” Janet said.

One of the bills our advocates will be talking to lawmakers about in May, would eliminate the 5 month waiting period for disability insurance for people diagnosed with ALS. Janet says it’s important for everyone to speak up on this issue, even if you are not directly affected by ALS.

“When there is an issue to be addressed, I not only send my letter, I also ask others to do likewise. Advocacy is not limited to PALS and family, anyone can be an ALS Advocate. I am a firm believer in fundraising and advocating all year long. After all, PALS and their families need help all through the year,” Janet said.

 Advocacy week will be held from May 14th – May 16th. To join the Greater New York Chapter delegation or to find out more about how you can get involved head to our website at als-ny.org or call Wendy DuBoff at 212-720-3054.

San Francisco 49ers Legend Dwight Clark Announces He Has ALS

Dwight Clark makes the Game Winning Touchdown catch to Defeat the Cowboys in the 1982 Superbowl.
Dwight Clark makes the touchdown catch that clinches the 1982 Superbowl against the Cowboys.

Former San Francisco 49ers receiver Dwight Clark announced Sunday that he was diagnosed with ALS. Clark is well known for his epic catch that led to his team winning the 1982 NFC Championship game. Clark earned two Super Bowl Rings while playing with the 49ers, and later became the team’s General Manager.

Clark wrote an open letter sharing his story. You can read his letter here. (http://debartoloholdings.com/dwightclark.html)

Clark wrote in his letter: I’ve got to devote all my energy preparing for this battle and I would hope you can respect my family’s privacy as I begin this challenge. My ultimate hope is that eventually I can assist in finding a cure for ALS, which disrupts the lives of so many and their loved ones.”

Our thoughts and prayers are with Dwight Clark and his family. The ALS Association Greater New York Chapter is fighting alongside Clark and all our PALS to help find that cure.

FALL Walks To Defeat ALS on October 9th & October 16th

Including the Launch of the Fall Walk in Central New Jersey

Matthew Ackerman – Co-captain and Founder of Team Tiger Lily’s

Joining hundreds of walkers is brand new team the Tiger Lily’s, who signed up after finding out there was a Fall walk close to home!

“This walk ended up working out great for us! This walk is close to home and the perfect time, and we were able to get other people to join our team,” Tiger Lily’s Team Captain, Dan Ackerman said.

Tiger Lily’s was started by 13-year-old Matthew Ackerman. Matthew wanted to form a team in honor of his Grandmother Lily, who passed away from ALS last year.

“My son Matthew had his Bar Mitzvah this year, and for his Mitzvah Project he wanted to do this Walk to raise money for ALS, because his grandmother passed away last summer from ALS,” Dan said.

Team Tiger Lily’s will have 10 walkers. Dan and his family are hoping to inspire others to get involved. He hopes with enough walks, dollars, and research, in the future other families won’t have to watch loved ones suffer with this fatal disease.

“Given what we know about this disease, raising money for additional research and a cure is very important. Seeing firsthand how debilitating it is, is just heartbreaking for both the family and the patient,” Dan said.

There is still time to get involved in both the Central New Jersey and Hudson Valley Walks to Defeat ALS. The Hudson Valley walk is Sunday October 16th at The Walkway over the Hudson, in Highland, New York. For more information, Click Here.

Every Act

By Nancy Frates

My son Pete was diagnosed with ALS four years ago. He was only 27.

Being a baseball family, we knew of Lou Gehrig’s Disease, but that’s all we knew.

As the former captain of his college baseball team, Pete has always been a leader and an agent for change. So, when we went back to the doctor a month later, he looked at her and asked what it would take to cure this disease. She said without blinking:

“One billion dollars.”

A billion dollars. I thought that sounded impossible, but Pete was up to the challenge.

Continue reading Every Act

Every Ally

By Andre Williams

Military veterans are twice as likely to be diagnosed with ALS as our civilian counterparts. Regardless of branch of service, duration of service, or era of service, veterans are confronted with ALS at an alarming rate.

I am a United States Disabled Veteran, diagnosed with ALS in 2009. I will never forget the day my doctor told me that on my final battle ground, my opponent would invariably take my life – I was given only a few years to live. Seven years later, I continue to defy ALS by bringing attention on Capitol Hill to this 100% fatal disease. In many ways, I have dedicated the rest of my life to ALS advocacy and to attracting more allies in this fight.

Continue reading Every Ally

Milton Safenowitz Postdoctoral Fellows Gather in NYC for ALS Association Workshop

Safenowitz ALS Research

Milton Safenowitz Postdoctoral Fellows; Marilyn Safenowitz and family; Dorine Gordon, President & CEO of The Greater New York Chapter; Lucie Bruijn, Ph.D., M.B.A., Chief Scientist; and Barb Newhouse, President & CEO of The ALS Association

On June 10, 2016, The ALS Association brought together the current and past awardees of its Milton Safenowitz Postdoctoral Fellowship to participate in a day-long research symposium and awards ceremony. The workshop was funded by The Greater New York Chapter of The ALS Association.

About the Award

The award was founded by the Safenowitz family through The Greater New York Chapter of The ALS Association. It is in memory of Mr. Safenowitz, who died of ALS in 1998. These awards are to encourage and facilitate promising young scientists to enter the ALS field. Fellows work with a senior mentor and receive extensive exposure to the ALS research community through meetings and presentations, like this workshop. After completing this fellowship, approximately 90 percent of the awardees stay in ALS research. They go on to establish their own laboratories to continue studying ALS and mentoring more ALS researchers along the way.

Collaboration Encouraged

This bi-annual workshop is designed to bring the latest postdoc awardees together to share research ideas and collaboration is highly encouraged. During the awards luncheon, the grandchildren of Mr. Safenowitz, Bailey del Valle, Samantha Levine and Jeff Safenowitz, expressed how important this program is to the memory of their grandfather. Marilyn Safenowitz presented the awards to all the current fellows. In addition, Ride for Life is currently supporting Dr. Dejun Yang and Christopher Pendergast was in attendance to present his award.

David Goldstein, Ph.D., Director of the Institute for Genomic Medicine at Columbia University, globally renowned for his contributions in infectious diseases and epilepsy, gave the key note presentation: “Precision Genetics and Precision Medicine in ALS: the Fruits of Collaborative Science.” He presented a comprehensive overview of how discovery of new genes can lead to the identification of new disease pathways that drive the development of ALS therapeutic targets. For example, whole genomic sequencing efforts recently identified a new ALS gene, TBK1, which will hopefully be targeted for therapy. Significant improvements have been accomplished in gene sequencing, to read DNA faster and at a decreased cost.

Dr. Goldstein pointed out that it would take 35 years to read a person’s entire genomic sequence, reading at an average rate of 162 characters per minute. He also spoke about the power of bringing genomic data closer to the clinic by correlating clinical data obtained from people living with ALS. Together, this information will empower stratified clinical trials to improve clinical trial outcomes. Lastly, Goldstein emphasized a collaborative view of gene discovery that sharing all genomic sequencing data gathered around the world will allow us to find ALS treatments and cures faster than ever before.

Fellow Research Summaries

During the workshop, each fellow presented a short talk representing the work they have completed that was made possible by their fellowship through The ALS Association. Below is the list of fellows with the titles of their talks. For the full listing of the research presentation summaries, click here.

• Marka Van Blitterswijk, M.D., Mayo Clinic Florida, Jacksonville, Fla.

Title: Predicting the phenotype in C9orf72 expansion carriers using machine learning

• Antonia Dominguez, Ph.D., Stanford University, Stanford, Calif.

Title: Functional analysis of C9orf72 mutations in ALS via targeted genome engineering

• Fernande Freyermuth, Ph.D., Massachusetts General Hospital, Charlestown, Mass.

Title: Targeting FUS misfolding to mitigate RNA processing alterations linked to ALS

• Veronique Belzil, Ph.D., Mayo Clinic College of Medicine, Jacksonville, Fla.

Title: Characterizing the contributions of epigenetic changes in C9FTD/ALS

• Claudia Fallini, Ph.D., University of Massachusetts Medical School, Worcester, Mass.

Title: Characterizing the pathogenic role of TDP-43 in PFN1-linked ALS.

• Florent Laferriere, Ph.D., University of Zurich, Zurich, Switzerland.

Title: Contribution of protein aggregation in the pathogenesis of ALS: seeding, spreading and toxicity

• Qiang Zhu, Ph.D., Ludwig Institute at University of California, San Diego, La Jolla, Calif.

Title: Gain of toxicity from C9orf72 ALS/FTD mutation is therapeutically alleviated with antisense oligonucleotides

• Ke Zhang, Ph.D., Johns Hopkins University School of Medicine, Baltimore, Md.

Title: The role of nuclear transport defects in the pathogenesis of ALS/FTD

• Philip McGoldrick, Ph.D., University of Toronto, Toronto, Ontario

Title: Pathomechanisms of C9orf72 in real time

• Dejun Yang, M.D., Ph.D., University of Massachusetts Medical School, Worcester, Mass.

Title: Determine the mechanisms of ALS-associated poly (GR) toxicity and screening for therapeutic drugs

• Marian Hruska-Plochan, Ph.D., University of Zurich, Zurich, Switzerland

Title: Deciphering the pathogenic mechanisms of C9orf72 dipeptide proteins

• Tao Sun, Ph.D., Stanford University, Stanford, Calif.

Title: Genome wide identification of RNA editing dysregulation and upstream regulators in ALS

• Kaspar Roet, Ph.D., Boston Children’s Hospital/Harvard Medical School/Harvard Stem Cell Institute, Boston, Mass.

Title: Which potassium channel drives ALS motor neuron hyperexcitability?

• David Medina, Ph.D., Barrow Neurological lnstitute, Phoenix, Ariz.

Title: Modulating retinoid signaling as a therapeutic approach for ALS

• Jone Lopez-Erauskin, Ph.D., Ludwig Institute for Cancer Research, University of California San Diego, La Jolla, Calif.

Title: Identify key molecular targets to counteract muscle denervation in ALS.

• Russell McLaughlin, Ph.D., Trinity College Dublin, Dublin, Republic of Ireland

Title: Molecular rapprochement of neurology and psychiatry: are ALS and schizophrenia biologically convergent?

The ALS Genetics and Clinic Event at the New York Genome Center

Screen Shot 2016-07-02 at 7.58.12 AM

Speakers from the ALSA event on June 11th (left to right): David Goldstein (Columbia University Medical Center/NYGC), Hemali Phatnani (NYGC/Columbia University Medical Center), Dorine Gordon (The ALS Association), Neil Shneider (Columbia University Medical Center), Lucie Bruijn (The ALS Association), Bob Darnell (NYGC/Rockefeller University), Matt Harms (Columbia University Medical Center), Tom Maniatis (Columbia University Medical Center).

NYGC hosted an event for The ALS Association (ALSA) on Saturday, June 11, 2016. The forum, “ALS Genetics and the Clinic” was attended by members of renowned academic and medical institutions and ALSA clinics in the greater New York area and Connecticut. It featured presentations by leading ALS researchers on ALSA-funded genomics programs, and updates on the fundamental advances in our understanding of ALS through genetic research.

The presentations were followed by a panel discussion on how to involve clinics in the greater New York area and CT in the research and biobanking efforts described during the talks. Among the topics discussed were how NYGC and Columbia University Medical Center could coordinate to meet the infrastructure needs of the various ALSA clinics, e.g., by helping to draft Institutional Review Board protocols, providing standardized protocols for biospecimen collections, and providing common data elements for clinical phenotyping.

In the evening, a presentation was given to a general audience, followed by a cocktail reception and tour of NYGC’s facilities.

This article was originally published on the NYGC website.

About the New York Genome Center

The New York Genome Center (NYGC) is an independent nonprofit at the forefront of transforming biomedical research and clinical care with the mission of saving lives. Comprised of renowned academic, medical and industry leaders across the globe, NYGC focuses on translating genomic research into clinical solutions for serious diseases. Our member organizations and partners are united in this unprecedented collaboration of technology, science and medicine. We harness the power of innovation and discoveries to improve people’s lives — ethically, equitably, and urgently. Member institutions include: Albert Einstein College of Medicine, American Museum of Natural History, Cold Spring Harbor Laboratory, Columbia University, Cornell University/Weill Cornell Medicine, Hospital for Special Surgery, The Jackson Laboratory, Memorial Sloan Kettering Cancer Center, Icahn School of Medicine at Mount Sinai, NewYork-Presbyterian Hospital, The New York Stem Cell Foundation, New York University, Northwell Health (formerly North Shore-LIJ), The Rockefeller University, Roswell Park Cancer Institute, Stony Brook University and IBM.

Greater New York Chapter Retains Charity Navigator 4-Star Rating

The ALS Association Greater New York Chapter has once again received a 4-star rating from Charity Navigator. The ALS Association Greater New York Chapter retained its 4-star rating under a new rating system with an enhanced financial methodology introduced by Charity Navigator in 2016.

We are proud to have achieved this honor as we continue our mission to support people with ALS and their families and to lead the fight to treat and cure ALS.

Mitshubishi Tanabe Pharma Submits New Drug Application for Edaravone to Treat ALS in the US

Mitsubishi Tanabe Pharma Corporation (MTPC), which is locally based in Jersey City, N.J. with a head office in Osaka, Japan, announced yesterday that a New Drug Application has been submitted to the U.S. Food and Drug Administration (FDA) for edaravone (MCI-186) for the treatment of amyotrophic lateral sclerosis (ALS).

Those in the ALS community may have questions about what this means for them. Below we provide some background information on edaravone.

What is edaravone?

Edaravone is believed to act as a free radical scavenger, a compound that works by getting rid of toxic waste generated as a normal by-product of cell function. In ALS it is thought that these by-products are not as effectively removed and the compound may be neuroprotective by relieving the effects of this oxidative stress. The increase in oxidative stress is thought to damage motor neurons (cells that die in ALS).

History of edaravone

Originally, edaravone was approved in Japan to treat stroke. A phase III double-blind placebo-controlled study (MCI186-J16) was conducted in people living with ALS to confirm safety and efficacy of edaravone. This study showed that edaravone did not significantly differ from placebo using the ALS Functional Rating Scale Revised (ALS-FRS-R). Investigators next conducted another phase III trial with a sub-group of people living with ALS that had greater baseline functionality. Results demonstrated that people living with ALS that had a forced vital capacity of >80% and had disease less than 2 years showed the greatest benefit with a statistically significant improvement in the (ALS-FRS-R). It was also established in an exploratory study that edaravone has no benefit from treatment in more advanced ALS cases.

In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea using intravenous injections of the drug. In the same year, the FDA and European Commission granted Orphan Drug Designation for edaravone. The Orphan Drug Act gives special status to a drug that treats a rare disease like ALS and gives incentives for pharmaceutical companies to develop said drug. Mitsubishi Tanabe Pharma is continuing to monitor edaravone’s effects in a post-market survey now that it has been approved. This should shed light on whether or not the drug improves survival.

Treeway BV in the Netherlands under the leadership of co-founders Bernard Muller and Robbert Jan Stuit is about to start a phase II/III clinical trial to test an oral version of edaravone (TW001). Taking the drug orally would be less invasive and more convenient to treat chronic ALS compared to the intravenous infusions of the drug. The TW001 efficacy study is a European study, so only people living in proximity to participating sites can participate.

A New Drug Application for edaravone was submitted in the U.S.

A new drug application (NDA) is a means for pharmaceutical companies to formally propose that the FDA approves a new drug for sale and marketing in the U.S. The NDA gives a full picture of the new drug, such as data from the clinic trials and animal studies, how the drug behaves in the body and how it is manufactured, processed and packaged. Mitsubishi Tanabe Pharma submitted edaravone to the FDA to request its approval in the U.S.

What’s next?

Edaravone is currently not approved by the FDA for use in the United States. It is not known yet whether the FDA will automatically approve edaravone in the U.S. or whether the FDA will require a phase III study in the U.S.

Questions?

Additional questions about edaravone can be sent totsandyali@als-ny.org.

Read full press release from Mitsubishi Tanabe Pharma here.

Senate Passes Durbin Amendment to Protect ALS Research at the DOD!

Advocacy Update
Advocacy Update: The Senate voted overwhelmingly to pass Senator Dick Durbin’s (D-IL) amendment (#4369) to the National Defense Authorization Act (NDAA) to protect crucial medical research programs at the Department of Defense (DOD).
 
Senator Durbin’s amendment strikes dangerous provisions from the NDAA that would have devastated medical research at the DOD, including the ALS Research Program, which to date has discovered four potential treatments for ALS.
 
Thank you to all our advocates for their outreach on this issue. Together, we are making a difference!