Dr. Marka Van Blitterswijk: ALS Researcher Focused on Discovering Biomarkers for the C9orf72 Gene Mutation

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of ALS forever.

Dr. Marka Van Blitterswijk from the Mayo Clinic Jacksonville is a scientist and one of our former Milton Safenowitz postdoctoral fellows. Since moving on from the program, she has established her own ALS lab as an assistant professor. We recently awarded her a prestigious multi-year grant surrounding her biomarker work.

Today, we sit down with Dr. Van Blitterswijk to hear about her latest work in ALS biomarkers, focused on the most common gene mutation in ALS, called C9orf72. We are thrilled to see her career and ALS research efforts thrive.

Continue reading Dr. Marka Van Blitterswijk: ALS Researcher Focused on Discovering Biomarkers for the C9orf72 Gene Mutation

Milton Safenowitz Postdoctoral Fellows Come Together to Share Innovative Ideas

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of ALS forever.

The ALS Association is proud to have supported the development of bright, young scientists through the Milton Safenowitz Postdoctoral Fellowship program since 2004. The Safenowitz family, through our Greater New York Chapter, founded the program in memory of Milton Safenowitz, who died of ALS in 1998.

On June 21, our newest class of Milton Safenowitz Postdoctoral Fellowship awardees came together for a bi-annual symposium in New York City. The fellows were joined by four established researchers –Dr. Sandrine DaCruz from University of California San Diego, Dr. Tania Gendron from the Mayo Clinic Jacksonville, Dr. Timothy Miller from Washington University St. Louis, and Dr. Hemali Phatnani from the Center for Genomics of Neurodegenerative Disease (CGND) at the New York Genome Center – who gave advice on each fellow’s work.

Dr. DaCruz and Dr. Gendron are former Milton Safenowitz fellows who are now highly regarded ALS researchers running their own ALS labs.

Dr. Timothy Miller was the keynote speaker at the luncheon. He gave an overview of his work in ALS genetics and biomarkers (a substance you can measure in the body that changes over time).

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2018 marks the 20th anniversary year of Milton’s passing, and we were honored to be joined by the Safenowitz family to mark the occasion. Marilyn Safenowitz, Milton’s wife and matriarch of the family, which now includes nine grandchildren and one great-grandchild, proudly handed out the fellowship awards to our new class during the luncheon.

And Ryan Levine, Milton’s grandson, gave a moving speech about his grandfather, describing him as a person who “people should emulate because of the empathy and love that he exuded and his dedication to help others.”

Milton Safenowitz Group Shot

The ALS Association provides the only postdoctoral fellowship program specifically tailored toward supporting first-year postdoctoral fellows in the ALS field, made possible by the generous support of the Safenowitz family and donors across the globe, including support from many of our chapters and the ALS Ride For Life.

The Association understands that attracting bright, young scientists to enter and then remain in ALS research is essential to moving the field forward. Our Milton Safenowitz Postdoctoral Fellowship awards were created specifically to encourage and facilitate promising new scientists to enter the ALS field.

The pathway for a postdoctoral fellow to attain their own laboratory at an academic institution is long and difficult. According to the journal Nature, people remain a postdoc for as many as five years with a relatively low salary, and many fellows become discouraged by the prospect of life in academia and, instead, take positions in industry or abandon careers in research altogether.

The ALS Association is committed to turning the tables on these statistics. Through mentorship and resources provided by our Milton Safenowitz Postdoctoral Fellowship program, over 90 percent of award recipients finish their fellowships and start their own ALS laboratories.

That means the program not only increases the pool of bright young researchers in the fight against ALS, but also balloons the number and quality of innovative ideas across the research pipeline.

Since its founding, the program has proudly sponsored 64 postdoctoral fellows have published over 167 publications in top journals. All of our fellows are and have been regular presenters at major scientific meetings.

Our Milton Safenowitz Postdoctoral Fellowship program gives young scientists the opportunity to obtain invaluable experience through the development of high-quality papers, collection of data, and support of applications for further funding from the Association, the National Institutes of Health, and other sources.

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Safenowitz fellows come into the laboratory with innovative ideas that give fresh perspectives on ongoing work. Funding from The ALS Association allows Safenowitz fellow to effectively test those ideas to produce brilliant work and publications in top scientific journals.

Safenowitz fellows also leverage funding from the Association to establish their own ALS research laboratories, where they continue to move their ideas toward potential treatments, as well as mentor other bright, young ALS researchers.

Our fellowships also provide fundamental mentorship to young scientists through relationships with their own mentors and partnerships with other ALS researchers.

Donate now to help us support more innovative, collaborative work to help advance research and care for people living with ALS and their families.

Don’t miss out on doubling your impact! This is a critical time to donate since our corporate partner, Biohaven Pharmaceuticals, is matching all gifts up to a total of $25,000! Biohaven is committed to supporting innovation to help improve the lives of all people impacted by ALS.

To learn more about Biohaven’s commitment to advances in ALS and other neurological diseases, click here.

Collaboration is at Our Core…and Spurring Innovation Along the Way

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of ALS forever.

Since our founding in 1985, we have put collaboration at the forefront of everything we do. The gift of the ALS Ice Bucket Challenge in August 2014 gave us the unique opportunity to up our game in establishing innovative research partnerships around the world.

We continue to foster those partnerships throughout the ALS community today. Our partnerships span to all areas, from academic institutions and researchers, to industry (biotech and pharmaceutical) partners, to government organizations, to other nonprofit organizations.

Continue reading Collaboration is at Our Core…and Spurring Innovation Along the Way

New Review Focuses on the Connection of Disturbances in RNA-Binding Proteins to ALS

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of ALS forever.

In the May 2018 issue of Frontiers in Neuroscience, current The ALS Association-funded researchers, Drs. J. Paul Taylor and Maria Purice of St. Jude Children’s Research Hospital in Memphis, published a comprehensive mini-review describing disease pathways that cause ALS, with a focus on mutations in RNA-binding proteins.

Many recent innovations in ALS animal and cells models that were instrumental in understanding the role of RNA-binding proteins in ALS are described.

Dr. Purice is a current Milton Safenowitz fellow who is currently studying how molecular pathways lead to both inherited (in which researchers have identified mutated ALS genes) and sporadic (non-inherited) forms of ALS, with a focus on RNA-binding protein, TDP-43.

In this review, Drs. Purice and Taylor do a deep dive into how disturbances in RNA-binding proteins, including TDP-43, cause ALS.

Continue reading New Review Focuses on the Connection of Disturbances in RNA-Binding Proteins to ALS

ALS Community Workshop: How to Attend the Live Stream, Ask Questions, and Make Comments

On Thursday, July 12, the ALS community will have the opportunity to meet with representatives from the FDA and the pharmaceutical industry during an ALS Community Workshop in order to discuss the latest FDA guidance document related to ALS drug development.

The Workshop will be streamed live on our ALS Community Workshop webpage, Facebook page, and YouTube channel from 9:00 a.m. EDT to 3:00 p.m. EDT.

Both in-person and online attendees will be able to ask questions and comment. Continue reading for instructions on submitting questions and comments that may be addressed during the Workshop.

If you plan on attending the (online) live stream event, please register here: https://thealsassociation.formstack.com/forms/signup.

Continue reading ALS Community Workshop: How to Attend the Live Stream, Ask Questions, and Make Comments

Pison Technology Ignites Innovation to Help People With ALS Communicate

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with the disease to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of ALS forever.

Frustrated with the limited availability of assistive technology devices for his mother, who was diagnosed with ALS, Dexter Ang quit his finance job, partnered with David Cipoletta, an underwater robotic engineer, and set to work developing technologies that could universally, massively, and quickly improve the quality of life for people living with ALS.

Dexter and David named their start-up Pison Technology after Ang’s mother’s cat, who provided her with comfort.

“It was an honor for us to participate in and win a prize at the Assistive Technology Challenge because it validated that our technology is at the forefront of innovation for people with ALS,” said Dexter. “The world needs technologies like ours to be able to help all patients worldwide. The ALS Association, along with NASA, will be major partners with us as we do our research and as we begin distribution and sales of our product worldwide.”

Continue reading Pison Technology Ignites Innovation to Help People With ALS Communicate

How the ALS Ice Bucket Challenge Has Helped Spur Gene Discoveries

Innovation plays a key role in The ALS Association’s fight to develop treatments and a cure for ALS and to empower people living with ALS to live their lives to the fullest. During June and July, we are celebrating some of the key innovations helping us change the nature of the disease forever.

Before the ALS Ice Bucket Challenge, The ALS Association allocated $6 million annually to funding ALS research. After the ALS IBC, the Association has budgeted about $18 million per year to research and has so far committed $84 million to ALS research. From this investment, there has been massive payoff in a significant increase in ALS gene discoveries.

SOD1 was the first ALS gene discovered, in 1993. Since then, over 30 genes have been reproducibly linked to ALS. And in the almost four years since the ALS Ice Bucket Challenge, five new ALS genes have been discovered and validated to contribute to the development of ALS – KIF5A, NEK1, C21orf2, TUBA4A, and TBK1. This rate of gene discovery is the highest in ALS history.

Every new ALS gene discovered represents a new ALS therapeutic target. The more ALS therapeutic targets identified, the more likely that a treatment will be found.

The ALS Association has been committed to gene discovery since its founding in 1985. It has supported research that contributed to all the major gene discoveries in the graph below.

Gene discovery graph.jpg

Continue reading How the ALS Ice Bucket Challenge Has Helped Spur Gene Discoveries