ALS Association-Funded Researchers Raise Their Voices to Discuss Latest Findings as Part of ALS Awareness Month

Researchers around the world working together for treatments and a cure for ALS are a main reason why we’re on the verge of changing the nature of the disease forever. As part of National ALS Awareness Month, we sat down with two ALS researchers at Johns Hopkins University in Baltimore who are funded by The ALS Association.

Dr. Ke Zhang, a postdoctoral fellow who was awarded a Milton Safenowitz Postdoctoral Fellowship in 2014 and another, Dr. Thomas Lloyd, associate professor of neurology and neuroscience, who we have supported for many years and currently fund.

ALS is a complex disorder with numerous molecular and cellular pathways involved in disease. All the underlying mechanisms of these pathways and how they could potentially connect and interact are still under intense investigation.

Together, with their colleagues, Drs. Zhang and Lloyd recently published an important paper in the prestigious journal Cell that links together two fundamental cellular pathways – nucleocytoplasmic transport and stress granule assembly – in ALS disease, moving them closer than ever to discovering new therapies for ALS.

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Update on Neuroscience in the Clinic: Antisense Oligonucleotide (ASO) Therapy

The 70th Annual American Academy of Neurology meeting, held in Los Angeles last week, provided an opportunity to check in on antisense therapies and the continued dividends from The ALS Association’s early investment in the technology.

Antisense oligonucleotide therapies are designed to prevent the production of disease proteins, saving the body of toxicity. Despite initial skepticism about the concept, The ALS Association took the lead in developing antisense therapies in 2004. As we learned in Los Angeles, that decision has contributed significantly to the progress made in the fight against ALS.

In December 2016, the Food and Drug Administration approved the first ever antisense drug, Spinraza. Spinraza targets spinal muscular atrophy (SMA), a common neuromuscular disease, which is the leading cause of genetic death in infants and toddlers. This is the first approved treatment for this disorder. This success provides hope for the future of antisense therapies targeting ALS.

Currently, there are more than 20 antisense drugs in preclinical stages or in clinical trials. This includes an ongoing trial to test antisense oligonucleotides that target SOD1, the second most common cause of ALS. That trial is currently in phase I/II. Also, work is being done to prepare the upcoming C9orf72 antisense clinical trial, slated to start in the near future (see more below). The C9orf72 mutation is the most common mutation associated with inherited ALS.

During the American Academy of Neurology Meeting in Los Angeles, experts from multiple neurodegenerative diseases came together to give an overview of ASO therapy past and present. Here is a short summary of the antisense technology presentations.

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Sheila Essey Award Winner Profile: Dr. Timothy Miller

Dr. Timothy Miller, the David Clayson Professor of Neurology from the Washington University School of Medicine in St. Louis accepted the 2018 Sheila Essey Award for ALS Research April 23 at the American Academy of Neurology 70th Annual Meeting in Los Angeles.

The ALS Association, in partnership with the American Academy of Neurology and the American Brain Foundation announced the award in March.

Dr. Miller is a dedicated physician scientist that has worked hard for many years with the goal to uncover effective therapies for ALS. We recently sat down with Dr. Miller to learn more about his impressive career in ALS, his current interests, and how his work has impacted the ALS field.

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Ice Bucket Donations at Work: A Superhuman Way to Look at Cells Using Artificial Intelligence

To make cell characteristics visible to the human eye, even under a microscope, scientists normally use chemicals that can kill the very cells they want to observe.

Dr. Steven Finkbeiner, director and senior investigator at the Gladstone Institutes in San Francisco (pictured above), recently teamed with computer scientists at Google for a groundbreaking new study funded by The ALS Association Neuro Collaborative through ALS Ice Bucket Challenge donations. The research shows that computers can see details in images without using invasive methods.

For example, using artificial intelligence (AI), computers can examine cells that haven’t been treated with chemicals and find a wealth of data that saves researchers time and effort, and the number of features that can be obtained from images is extraordinary. Extensive hours of researchers sitting at the microscope manually analyzing cell images could eventually become a thing of the past.

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Dr. Peter Creigh Wants to Improve ALS Care Through Virtual Research Visits

Today, we are happy to be joined by ALS clinician scientist Dr. Peter Creigh from the University of Rochester in Rochester, N.Y., the recipient of the 2018 Clinician Research Training Fellowship in ALS Research. The fellowship is given by The ALS Association, in partnership with the American Academy of Neurology (AAN).

Dr. Creigh is dedicated to improving ALS care by using telemedicine in ALS clinical research. He hopes to make it easier for people living with ALS to participate in research studies.

He says, “Virtual research visits, conducted in individuals’ homes through telemedicine, could potentially enable every individual with ALS, from anywhere, to participate and have a voice in ALS research.”

We recently sat down with Dr. Creigh to learn about his important work and get to know the person behind the white coat.

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Local Assistive Technology Lab Improves Lives of People Living with ALS

People living with ALS eventually lose the ability to speak. That means that preserving channels of communication is an important component of enhancing quality of life. The ALS Association – DC/MD/VA Chapter took this to heart when they initiated The Esther Lerner Brenner ALS Assistive Technology Lab in Maryland, which is designed to help people living with ALS communicate effectively for as long as possible.

“Our goal is to make sure you have the ability to communicate with not only your family and friends, but also with your doctors and therapists who are just getting to know you,” said Regan Flores, assistive technology specialist at The ALS Association – DC/MD/VA Chapter.

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Organ-Chip Technology Can Be Applied to Understand ALS Disease Pathways

Researchers funded by The ALS Association using state-of-the-art technology called an Organ-Chip, which essentially recreates human biology on a microchip, found that the human brain’s tiniest blood vessels can initiate spinal motor neuron development. Studies using this technology to track living tissues on a chip provide ALS researchers a unique way to study the disease processes in ALS and other neurodegenerative disorders.

Drs. Clive Svendsen and Samuel Sances from Cedars-Sinai Board of Governors Regenerative Medicine Institute in Los Angeles published this important work in journal Stem Cell Reports.

 

The researchers took human skin cell samples and genetically reprogrammed them into induced pluripotent stem cells (iPSCs) that can be turned into many different cell types in the body. Here, they turned iPSCs into spinal motor neurons, the cells that connect to muscle to signal muscle movement and which die in ALS. After creating blood vessel cells as well, spinal motor neurons successfully connected to blood vessels on the Organ-Chip.

We sat down with Dr. Sances, first-author of the study, to learn more about this exciting technology and how it could be applied to understanding the mysteries behind ALS disease pathways.

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