The Federal Drug Administration (FDA) recently approved a new drug to treat ALS, which uses intravenous (IV) infusion for administration. IV therapy can be administered in multiple sites of care: your doctor’s office, a clinic, a hospital, or your own home. Whether you receive treatment will be a decision based on clinical input from your physician, your insurance coverage, and personal preference. The following information focuses on home infusion therapy: the basics, the team members involved, the supplies required for home infusion, and where to get them; and finally, coverage options for home infusion therapy.
From donations raised through the ALS Ice Bucket Challenge, The ALS Association, in partnership with the Greater New York Chapter, made a $2.5 million commitment to the Center for Genomics of Neurodegenerative Disease (CGND) at the New York Genome Center (NYGC). This commitment, combined with a matching gift from the Tow Foundation, was one of the driving forces supporting the NYGC’s ALS research program in 2014. Three years later, the NYGC’s CGND has made enormous headway in the ALS genetics space and has become one of the major leaders in the field. Their accomplishments are broad in that they have sequenced and analyzed hundreds of ALS DNA samples, while pairing this information with patient clinical history and more. We are pleased to note that Tom Maniatis, PhD, one of the NYGC’s original founders and renowned ALS researcher, was recently appointed Scientific Director and Chief Executive Officer of the NYGC.
The Center for Genomics of Neurodegenerative Disease (CGND) at the New York Genome Center (NYGC) is a prominent player in ALS genetics, leading the way in ALS gene discovery. We are proud to report on its many successes in ALS genetics that were made possible by our major funding efforts. With donations raised by the ALS Ice Bucket Challenge, The ALS Association made a $2.5 million commitment to NYGC CGND in partnership with the Greater New York Chapter, which was matched by a gift from the Tow Foundation. They are proud to collaborate with many of the large, global strategic initiatives that we also support, including Project MinE and Genomic Translation for ALS Care (GTAC) Consortium, and partner with, including Answer ALS and Target ALS. In part I of the NYGC progress update, today’s guest blogger, Dr. Hemali Phatnani, Director of the CGND, reports on the many great achievements out of the NYGC CGND accomplished through their collaborative research approach.
In 2004, Paul Carey Jr. was 13 years old, and his brother Christian was 11. The boys were both active in all kinds of extracurriculars. Paul was a passionate hockey athlete and Christian a talented young actor, and both excelled at schoolwork.
One Friday in May 2004, their world turned inside-out. As they sat on their living room couch in their Cincinnati, Ohio home, their parents explained to them that their mother, Lorri, had been diagnosed with ALS. Lorri was 38 years old.
Meet Dr. Veronique Belzil, an Assistant Professor in the Department of Neuroscience at Mayo Clinic College of Medicine in Jacksonville, Fla., and a former ALS Association Milton Safenowitz Postdoctoral Fellow. Having been personally touched by ALS, she is extremely dedicated to finding treatments and ultimately a cure for this devastating disease.
Her work focuses on sporadic ALS, which is a type of ALS that is not genetically inherited and accounts for approximately 90% of all ALS cases. We are proud to report her recent successes of identifying new ALS genes and disease pathways that can be targeted for developing potential ALS therapeutics. This important work was published in August in the journal Acta Neuropathologica. Read more about Dr. Belzil’s extraordinary work, her vision for the future of ALS research, and what she is up to now.
In general, people with disabilities are traveling more than ever, including those living with ALS. In response, the travel industry is paying greater attention to their special needs by providing more services and accommodations. The amount of information is increasing and is more readily available from disability organizations and transportation company websites. In preparation for the upcoming fall travel season, we held a webinar “Traveling with ALS.” Read more for some great tips on how best travel with ALS and to help people with ALS and their companions anticipate some of the challenges associated with accessible travel.
The clinical trial to test RNS60, a new compound for the treatment of ALS, which was supported in part from a $1.0 million grant through the ALS ACT initiative funded by The ALS Association and ALS Finding A Cure®, is now actively enrolling. This randomized placebo-controlled phase II study is being run by the IRCCS Mario Negri Institute for Pharmacological Research in Milan and the ALS Center of the Maggiore University Hospital in Novara Italy. We are happy to report that the first patient was dosed in mid-July 2017.