ALS Drug KPT-350 Advances Towards Clinical Trial

Biogen has acquired KPT-350, a potential ALS therapeutic that has been investigated in preclinical trials to treat ALS by researchers funded by The ALS Association’s Lawrence and Isabel Barnett Drug Development Program and The Milton Safenowitz Postdoctoral Fellowship Program, Karyopharm Therapeutics recently announced. Biogen acquired KPT-350 in a $10 million up-front payment agreement with Karyopharm, a deal worth up to $217 million in future milestones, plus royalties, to treat neurodegenerative diseases.

KPT-350 is an oral drug that targets inflammation and neurotoxicity in the brain and spinal cord that demonstrates neuroprotective responses. This important partnership will advance KPT-350 research to forge its way to enter an ALS clinical trial in the near future.

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ALS Ice Bucket Challenge Year-End Update: Over $94 Million in Commitments Since 2014

In the summer of 2014, the ALS Ice Bucket Challenge inspired 17 million people to upload videos and raised $115 million for The ALS Association. Since then, The ALS Association has committed more than $94 million toward its mission, including over $82 million to fund research, and helped forge nine new global research collaborations.

“The Ice Bucket Challenge was a global phenomenon, thanks to the generosity of millions of people, resulting in a tremendous amount of awareness of the disease and spurring a huge increase in our research spending,” said Calaneet Balas, president and CEO of The ALS Association. “Since 2014, we’ve committed over $82 million to fund diverse, cutting-edge research, including over 150 projects in the last year alone. By its nature and design, research requires time, and people with ALS don’t have a lot of time. But we’ve seen exciting breakthroughs from the research we fund – including some that were considered high-risk.”

ALS Ice Bucket Challenge donations have helped lead to the discovery of four new ALS genes, including NEK1, one of the most common. In addition, scientists who are a part of the Neuro Collaborative, which was started with an initial investment of $5 million from ALS Ice Bucket Challenge donations, have developed antisense drugs targeting two of the most common ALS genes, C9orf72 and SOD1. Our investment in this innovative technology also translates to other neurodegenerative diseases, making the impact of ALS Ice Bucket Challenge donations even greater.

ALS Ice Bucket Challenge donations have also helped spur advancements in assistive technology to develop flexible, accessible devices to help people living with ALS better communicate to improve their quality of life. ALS Ice Bucket Challenge donations have also helped us increase the number of Certified and Recognized Treatment Centers of Excellence and the size of the grants to those centers.

Prior to the ALS Ice Bucket Challenge, we supported 34 Certified Treatment Centers of Excellence and had no immediate plans to increase that number. Today, we support 58 Certified centers, in addition to 20 Recognized Treatment Centers. (The primary difference between Certified and Recognized Treatment Centers is that Certified Centers conduct ALS research.)

To see our research accomplishments and highlights for the past year (2017), please click here. To see a specific list of all the projects and project descriptions we have funded since the ALS Ice Bucket Challenge, please click here.

“We fight for treatments and a cure for ALS every single day,” Balas said. “We are the largest private funder of ALS research and are currently funding research projects in eight countries. We are also helping ensure people with ALS receive multidisciplinary care that we know enhances and extends life. And we’re amplifying the voices of more than 16,000 ALS advocates to push for better public policies that can help us fight for treatments and a cure.”

The ALS Association is committed to transparency in how donor dollars are fueling efforts to find treatments and a cure for the disease. And we’re committed to maximizing all donations from the ALS Ice Bucket Challenge and beyond.

For more information about what’s happened since the ALS Ice Bucket Challenge, please visit ALSA.org/IBC. We will continue to update this site.

New ALS Resource Guide Delves into All Aspects of Multidisciplinary Care

A new educational resource from The ALS Association, Including the Multidisciplinary Team Approach in Your Care, is now available. The guide provides an overview of the multidisciplinary team approach to care, describes a typical clinic visit, and discusses additional options for care and support from providers outside of the core care team

“People living with ALS can maintain independence longer and enjoy improved quality of life when given options for symptom management, assistive technology, adaptive equipment, education, care services, and emotional support,” says Kimberly Maginnis, senior vice president for Care Services for The ALS Association. “Our certified centers and clinics follow a multidisciplinary care model, bringing together a team of health and social care professionals who are experts in addressing the needs of people living with ALS and their families.”

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Andrew Rea: The Celebrity Chef Behind the ALS Pepper Challenge

Andrew Rea, a New York-based chef, filmmaker, and host of the hugely popular YouTube cooking show “Binging with Babish,” was hit hard when his friend, Garmt van Soest, messaged him to say goodbye during his final days. On October 23, Garmt passed away from complications from ALS.

Andrew needed to honor Garmt in some way, and thought capitalizing on the trend of people recording themselves eating super spicy peppers might be the best place to start. On November 6, Andrew created the first ALS Pepper Challenge.

He says, “I thought, ‘Why don’t we put some purpose behind all this pain that we’re putting ourselves through?’ I hope I can raise funds and awareness for The ALS Association, a great organization doing important work researching the disease. More importantly, I want to help play a part in sharing Garmt’s message of strength, positivity, and love.”

Continue reading Andrew Rea: The Celebrity Chef Behind the ALS Pepper Challenge

ALS Registry Meeting Prioritizes Recruitment and Retainment Strategies

On December 13-14, 2017, 52 people gathered in Charlotte, N.C., to discuss how to increase awareness and participation in the National ALS Registry, the only population-based registry in the country collecting information to help scientists learn more about who gets ALS and its causes. Researchers use the Registry in their studies to find possible treatments and a cure.

The ALS Association believes collaboration with all partners will intensify the momentum needed to advance the Registry. Attendees in last month’s meeting — people living with ALS and their family members, numerous representatives from The ALS Association chapters, members of the National ALS Registry Taskforce, and representatives from the Agency for Toxic Substances and Disease Registry (ATSDR) at the Centers for Disease Control and Prevention (CDC) – are all critical to the Registry’s success.

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Researchers Uncover a Common Link Between ALS and CTE

Recent studies point to a possible connection between ALS and chronic traumatic encephalopathy (CTE), the type of traumatic brain injury associated with playing football and other contact sports. NFL veterans Steve Gleason, Tim Shaw, O.J. Brigance, Dwight Clark, Kevin Turner, and others were diagnosed with ALS after their football careers ended. CTE has also been found in military veterans, who are twice as likely to be diagnosed with ALS.

While 4 to 6 percent of CTE cases end up demonstrating either clinical or pathological characteristics of ALS, called CTE-ALS, the question of whether CTE and ALS are connected remains controversial. Much more needs to be learned to fully understand the connection.

Dr. Michael Strong and colleagues from the Schulich School of Medicine & Dentistry at Western University in Canada delved into the controversy. They compared molecular and pathological signatures of post mortem brain and spinal cord tissue obtained from people who lived with CTE and CTE with ALS (CTE-ALS) compared to controls.

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New Important Insights on How TDP-43 Impacts Intracellular Transport in ALS

New research published recently in the journal Nature Neuroscience provides a detailed analysis of TDP-43 aggregate composition and sheds important new insight on intracellular transport defects in ALS and other neurodegenerative diseases.

TDP-43 protein is a pathological hallmark of both ALS and other neurodegenerative diseases that is mislocalized in the cell’s cytoplasm, the area of the cell that surrounds the nucleus, and accumulates into aggregates — protein clumps. Through funding by The ALS Association and others, Dr. Wilfried Rossoll and colleagues at Mayo Clinic in Jacksonville and Emory University took a deep dive in understanding how TDP-43 is involved in disease.

Their analysis of TDP-43 aggregate composition showed that TDP-43 mislocalization and aggregation causes direct defects in the transport of proteins between the nucleus and cytoplasm though nuclear pores, called nucleocytoplasmic transport. Importantly, they found TDP-43 aggregates in postmortem brains from people who lived with ALS show nuclear pore defects that are comparable to their findings in cells, suggesting a similar disease pathway in humans. Read more to learn about these exciting, novel findings.

Continue reading New Important Insights on How TDP-43 Impacts Intracellular Transport in ALS