Annual International Alliance of ALS/MND Associations and MND Association Events Provide Knowledge, Hope, and Inspiration

We always enjoy our time at the annual events hosted by the International Alliance of ALS/MND Associations and the MND Association. This year was no exception. We listened, learned, networked, got inspired, caught up with old friends and colleagues, and so much more.

Here’s a summary of some of the interesting ALS and MND (motor neuron disease) research and information from the events held in Boston earlier this month.

Annual Meeting of the International Alliance of ALS/MND Associations

On Wednesday, December 6, as part of the Annual Meeting of the International Alliance of ALS/MND Associations, we heard a panel of experts present about ALS research through a Facebook Live discussion. The Annual Meeting brings together ALS/MND associations from around the world to share initiatives and develop strategies for fighting ALS/MND and serving people who live with the disease.

This is just some of what was discussed during the Facebook Live Ask the Experts session:

  • Jonathan Glass from Emory University in Atlanta shared how he carried out ALS protein analyses and demonstrated biological features that separate the different cases of ALS versus ALS/FTD versus FTD. This is exciting work that we’re proud to support! Dr. Glass also beautifully explained ALS genetics and gave an understandable ALS research overview. As more ALS genes identified, more potential ALS therapeutic targets are developed!
  • James Berry from Massachusetts General Hospital in Boston explained the ALS drug development process. In basic terms, you start with basic science, go to animal studies, move to obtain a FDA Investigational New Drug approval, go through three phases of clinical trials, and then finally receive FDA approval. He also emphasized the benefits of enrolling specific ALS disease subgroups into clinical trials in the correct way could potentially reduce trial size and increase the likelihood of clinical trial successes.
  • Participating in ALS trials give people hope and, more importantly, tends to slow disease progression. Visit the website of our partner, Northeast Amyotrophic Lateral Sclerosis (NEALS) Consortium, to learn about ALS trials:
  • Steve Perrin with ALS Therapy Development Institute (TDI) encouraged ALS researchers to use telehealth to measure data at home. He said it helps boost compliance of clinical trials and he’s seen benefits in ALS TDI’s precision medicine program.

2017 Allied Professional Forum

On Thursday, December 7, we spent the day at the 2017 Allied Professionals Forum, which is organized by the International Alliance of ALS/MND Associations and focused on practice and experience for health professionals in the care and support of people living with ALS/MND. Here are a few highlights:

  • Leslie Ryan with The ALS Association Rocky Mountain Chapter is gathering information about how people with ALS are using cannabis to manage physical and emotional symptoms. Many people responded saying that they benefited from cannabis, but more information needs to be gathered to maximize benefits and care.
  • Krista Strait-Higgins with The ALS Association Iowa Chapter is working with our chapters to develop an online tool featuring web-based educational resources that takes information most requested by ALS families and puts it front and center in an organized way. Families can then access the information when they feel it is best for them.
  • Melinda Kavanaugh with The ALS Association Wisconsin Chapter led the charge to develop The ALS Association’s first national resource guide to help families support their children and youth and also assist professionals working with families affected by ALS. Read our blog article outlining all her exciting work with children impacted by ALS here: http://bit.ly/2C1WcWC.
  • Patricia Stanco is understanding the changing needs of people living with ALS by working to help The ALS Association Florida Chapter evolve to serve more people: broadening community reach, empowering people, and increasing human connections. Forming new partnerships within the community and giving one-on-one care is so important in this effort.

Our chapter Care Services staff are constantly adapting and changing to ensure they’re providing the best possible care, equipment, technology, support, and resources to people living with ALS. Basically, they’re amazing!

Annual International Symposium on ALS/MND

From Friday, December 8 through Sunday, December 10, we attended the Annual International Symposium on ALS/MND, a unique event organized by the MND Association that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields.

Here’s a condensed summary of some of the exciting research presented by our partners and researchers we fund:

  • Luc Dupuis with INSERM in France is exploring the mechanisms of FUS-mediated ALS, while Mr. Jonathan Grima is working in the Rothstein lab at Johns Hopkins to understand ALS disease pathways involving the nuclear pore complex. We’re so proud to help fund these groundbreaking research projects!
  • Merit Cudkowicz with Massachusetts General and our longtime partner received the ALS/MND Alliance Forbes Norris Award. It is well-deserved!
  • Marka van Blitterswijk, Assistant Professor of neuroscience at Mayo Clinic in Jacksonville, Fla., and a former Milton Safenowitz postdoctoral fellow, received the 9th Paulo Gontijo International Award in Medicine. We’re so proud to see her move ahead in her career with much success!
  • Pete Frates, Pat Quinn, and Anthony Senerchia received the ALS/MND Alliance Humanitarian Award for their remarkable work founding the ALS Ice Bucket Challenge. Nancy Frates, Pete Frates’ mom, accepted the award on their behalf with a passionate speech. We can’t thank them enough for everything they’ve done!
  • Bernard Muller was awarded a 2017 Shay Rishoni Patient Impact Award for his critical work founding Project MinE, whose goal is to sequence thousands of people living with ALS to identify new ALS genes. We are so happy to see him honored and fortunate to partner with Project MinE.
  • Rick Bedlack with Duke University and ALSUntangled presented facts and myths associated with patient self-experimentation of drugs.
  • Frank Bennett with Ionis Pharmaceuticals provided an update on antisense technology and how it progressed from a controversial idea to a FDA-approved drug for spinal muscular atrophy. He also gave details on the state of antisense technology for ALS and other neurodegenerative diseases. The SOD1 antisense trial is ongoing and the C9orf72 trial is slated to begin in the near future.
  • Mary-Louise Rogers with Flinders University and Dr. Michael Benatar with University of Miami presented their progress in ALS biomarkers. Dr. Rogers is moving forward with an ALS urinary biomarker to measure disease progression, disease prognosis, and a drug’s potential effect, and to improve diagnosis. Dr. Benatar is exploring a biomarker called neurofilament light chain to look at pre-symptomatic disease.
  • Adriano Chio, a past Sheila Essey awardee from University of Torino, provided details about ALS epidemiology, such as how genetics impact the number of steps needed to have neurodegeneration. The number steps in genetically-mediated ALS needed to cause neurodegeneration is less than the number of steps needed in non-genetic cases.
  • Kevin Horton with Agency for Toxic Substances and Disease Registry (ATSDR) at the Centers for Disease Control (CDC) discussed his analysis of clinic distance to patients across the country. This was the first study to examine the proximity of population-based ALS prevalence cases for the entire U.S. to multi-disciplinary clinics (MDCs). Of the cases studied in 2013, almost half live greater than 50 miles from a MDC, which emphasizes the need for better access to care for all people living with ALS.
  • Nicolas Maragakis highlighted the benefits of exercise for people living with ALS and MND. Read our blog article for more detail.
  • Bjorn Oskarsson with Mayo Clinic’s Jacksonville campus comprehensively discussed how cramps are treated for ALS/MND based on data systematically gathered from numerous studies and clinical trials. In general, he found that more and larger studies are needed to evaluate treatments for muscle cramps.
  • Our partners from Cytokinetics, Biogen, and Ionis Pharmaceuticals presented a poster outlining the results of the IMPACT ALS survey. With almost 1,534 participant responses, the survey gives a deep dive on how the burden of ALS impacts people living with ALS and caregivers.

The researchers and health and social care professionals we heard from at these events exuded so much passion, urgency, and dedication. It was tremendous to experience and gives us hope for finding more effective treatments and a cure!

In honor of this holiday season, do your part to have an impact on people living with ALS. Host a Facebook fundraiser to help us meet our goal of $100K raised through personal fundraisers this month. (We’re well on our way!) Donations will help us fund even more research and provide additional support for people living with ALS and their families.

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