Spring Newsletter 2023 The ALS Association Greater New York chapter

Table of Contents

1. Walk to Defeat ALS
2. Research News
3. Education & Resource Summit
4. Advocacy
5. Team ALS - Half Marathon
6. Upcoming Events
7. Clinic Spotlight
8. Importance of Monthly Giving

Research news

The Food and Drug Administration (FDA) convened a virtual meeting of the Peripheral and Central Nervous System Drugs Advisory Committee on March 22, 2023, to review data supporting tofersen’s New Drug Application. Biogen is seeking approval of tofersen through the FDA's Accelerated Approval Program.

Tofersen is an antisense oligonucleotide (ASO) being evaluated as a treatment for SOD1-ALS. SOD1-ALS is a rare genetic form of the disease affecting approximately 330 people in the United States. While there are medications approved for broad ALS, no available treatments target a genetic mutation associated with ALS. Approximately 5-10 percent of people with ALS are thought to have a genetic form of the disease, however, they may not have a known family history of the disease.

The Committee voted unanimously yes (9 yes to 0 no), for consideration of a potential accelerated approval and voted 3 (yes), 5 (no) and 1 (abstain), for consideration of a potential traditional approval.

The committee reached consensus that the benefit-risk profile was favorable based on the review of the totality of data for tofersen in people with SOD1-ALS.

A final decision by the FDA for accelerated approval is expected by April 25, 2023.

Advocacy

The Greater New York Chapter has been a leader in advancing critical policies that help find new treatments and cures, optimize current treatments and care, prevent or delay the harms associated with ALS and assist the health and well-being of those living with ALS and their families. We recognize that federal and state policymakers have the power to change the future of ALS by acting on our policy priorities.

State legislatures in New Jersey and New York have supported The Greater New York Chapter over the last several years by helping to fund our critical programs and ALS Centers. In 2023, we increased our request significantly over previous years’ allocated funding. New York’s budget is due April 1st and New Jersey’s budget is due July 1st.

The Greater New York Chapter is also identifying ways that the New York and New Jersey state governments can prevent, or delay harms associated with ALS by supporting policies that can impact their lives. To that end, we have identified several pieces of legislation that have been introduced in both states that could improve the lives of those living with ALS. To read more on the specific bills we will be advocating for, click the button below.

Clinic Spotlight

The Stony Brook Neuromuscular Disease and Christopher Pendergast ALS Center of Excellence - was certified as the 34th Center of Excellence in the country in 2009, by The ALS Association. This distinction recognizes their collaborative approach and ability to provide the highest quality care for people with ALS and their families. It provides comprehensive diagnosis and treatment for patients of all ages with neuromuscular disorders. Led by Director Dr. Rahman Pourmand, with Dr. Simona Treidler as the Director of the Neuromuscular Division of the Department of Neurology, along with Dr. Nurcan Gursoy, they are experts in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease), neuropathy, myopathy, myasthenia gravis, and more. They see patients for advice, second opinions, diagnosis, or ongoing care.

Being a patient at the ALS Certified Clinic of Excellence at Stony Brook allows one to see members of the multidisciplinary team, including occupational therapists, physical therapists, nurses, speech pathologists, respiratory therapists, social workers, and dieticians in addition to the physician, all within a span of a 2.5-hour visit. The multidisciplinary approach increases the outcome of successful therapy and ensures that patients receive support in all aspects of their conditions. The Center's location in Suffolk County allows people to receive exceptional care on Long Island without traveling to Manhattan. They are equipped to provide long-term care. Their advanced technology helps to make a definitive diagnosis and then create individualized treatment plans.

Since joining the ALS Center team at Stony Brook in 2018, I have had the pleasure of working with a compassionate group of professionals who work hard, together to put the needs of the ALS patient, first. It is easy for me to say, the patients that come to our Center are in good hands. - Dr. Treidler

Education is also part of their mission, and often their physicians speak at The Greater New York Chapter's support groups or Talk. Learn. Connect. Series. They understand that ALS affects people in a variety of ways and encourage open discussion and question-and-answer sessions that include the entire family. They have counselors and social workers available to talk about anything from financial concerns to sexual problems. The Center often collaborates with the Stony Brook University School of Technology to assist with the development of assistive technology for the everyday living of the ALS patient. The Greater New York Chapter is proud to continue its long-standing partnership with the Pendergast ALS Center of Excellence at Stony Brook.