Category Archives: MOFF Blog

ALS Podcasts

Stay Connected

The individuals and companies within the ALS Community are innovative and inspiring. Their collective actions illustrate how love and the fight for life motive real change. The following podcasts cover stories from people living with ALS, the science behind the disease, treatment options, caregiving experiences, and several other pertinent topics. These podcasts are a great way to stay informed while on the move. Enjoy! 

Many of these podcasts are available on Spotify, iTunes, YouTube and Soundcloud. The options for each podcast will become apparent once you visit the webpage by clicking on the above icons. 

The Political Front: National ALS Advocacy Conference

The National ALS Advocacy Conference

(May 26, 2020 – June 3, 2020)

The National ALS Advocacy Conference was virtual this year! The Martha Olson-Fernandez Foundation (MOFF) attended two days of the virtual event. Continue reading to get the inside scoop on the political interests of the ALS community.

What is The National ALS Advocacy Conference?

The National ALS Advocacy Conference is a week-long event in which patient advocacy groups lobby to raise disease awareness, present funding requests, and push policy initiatives that will support the ALS patient community. The event has a similar structure each year:

  • Daily conferences are held that discuss ALS research updates, ALS community needs, specific policy initiatives, and congressionally appointed fund requests for the year.
  • ALS patients and their families visit the offices of members of Congress to personally tell their stories and request support for the funding or policy initiatives that will benefit the ALS community.
Who were the main players in the “ALS community” during the 2020 ALS Advocacy Conference?
  • Muscular Dystrophy Association
  • I AM ALS
  • Team Gleason
  • ALS Association
  • Les Turner Foundation
  • American Academy of Neurology
  • National Health Council
  • National Organization for Rare Disorders
What were the main “asks” of the ALS community for FY 2021? (The requests that are placed in May 2020 pertain to the fiscal year (FY) 2021).

The ALS community is advocating for the following public policy priorities in FY 2021:

  • ALS Disability Insurance Access Act (S. 578/ H.R.1407): Request to waive 5-month waiting period for Social Security Disability Insurance (SSDI). This Act builds off the previous motion passed in 2000 that waived the 24-month Medicare waiting period. This is an urgent agenda item and currently has support from 61 senators and 238 representatives.
  • Expand Access to Home Infusion for Medicare Beneficiaries
  • Promising Pathway Act: Proposal to amendment to the Federal Food, Drug, and Cosmetic Act to establish a time-limited provisional approval pathway for certain drugs and regulated medical products.
  • Accelerating Access to Critical Therapies for ALS Act: Creates a pilot program to support expanded access programs. Will dedicate $75 million available in FY 2021 and FY 2022 toward this program.
  • Justice for ALS Veterans Act (S. 3091/H.R. 4748): This bill ensures that surviving spouses of veterans with ALS receive fair dependency and indemnity compensation.
  • Request for $40 million congressionally appointed dollars for the Department of Defense ALS Research Program.
  • Request for $10 million congressionally appointed dollars for the National ALS Registry.
  • Request for $44.7 billion congressionally appointed dollars for the National Institutes of Health (NIH).
What politicians are supporting the ALS cause?

The below list and commentaries provide a snapshot into the wide-ranging political support the ALS community has garnered.

  • Senator Mike Braun (Indiana) is championing the ALS Disability Insurance Access Act. In the virtual meeting on May 26th he stated: “I don’t think there is a good reason why this should not pass.”
  • Representative Peter Welch (Vermont) is supporting the ALS Disability Insurance Access Act. His commentary during the virtual meeting was, “Congress is a tough institution to move,” but the ALS community’s “long term advocacy is working.” He also stated that we currently have 238 supporters in the House, if we obtain 290, it “can be put on a consent calendar.” In lay terms, this would deem the Act as a topic that is not controversial and does not need to be discussed and can be accepted without a vote.
  • Representative Jason Crow (Colorado) affirmed that the ALS community “has a friend and an ally in Congress.” He is a prior Army Ranger whose wife’s cousin passed away from ALS.
  • Congressman Jeff Fortenberry (New England) introduced the Accelerating Access to Critical Therapies for ALS Act.
  • Congressman Mike Quigley (Illinois) co-sponsored the Accelerating Access to Critical Therapies for ALS Act.
How do the 2020 ALS community funding requests compare to historical asks?
  • To give you an idea of how much the ALS community’s political involvement has grown, in 1998 the government allotted $15 million dollars total to ALS research initiatives.
  • In FY 2020, the ALS community requested $20 million congressionally appointed dollars for the Department of Defense ALS Research Program. This was request was granted.
  • In FY 2020, the ALS community requested $10 million congressionally appointed dollars for the National ALS Registry/Biorepository development. This request was granted.
  • In FY 2020, the ALS community requested $105 congressionally appointed dollars for the NIH. The request was granted.

The 2020 National ALS Advocacy Conference was marked by monumental support and engagement from members of Congress. Despite the virtual platform, the ALS patient voices and actions could not be ignored. One key takeaway from the conference was the importance of forming coalitions with other patient groups. It is by unifying the patient voices, and combining them with the urges of doctors in their respective fields, that disease-community demands will be answered. This year, the American Heart Association and the American Lung Association formed key alliances with the ALS advocacy community on specific interests.

Thank you to all the parties involved in this monumental conference. Your efforts are saving lives. More information can be found on the ALSA, MDA and I AM ALS websites.


The main sponsors of the National ALS Advocacy Conference were the following pharmaceutical companies: Alexion, Amylyx, Biogen, biohaven pharmaceuticals, Genentech, Mitsubishi Tanabe Pharma America, and Soleo Health.

Healey ALS Platform Trial

What is the Healey Platform Trial?

  • An ALS clinical trial that tests 5 different therapies at 54 different trial sites across the United States. You can learn more about the structure and originality of  the trial here.

How many patients will be enrolled in the Healey Trial?

  • 160 patients will be enrolled per each of the five treatment arms.

What is the duration of the trial?

  • 24 weeks

When does the Healey Trial start?

  • There is not an exact date set yet. That being said, best estimates have the trial starting in May or June. People living with ALS are encouraged to contact trial sites near their residence as soon as possible. The enrollment will be competitive, meaning on a first come, first serve basis.

What phase clinical trial is this considered?

  • The stages of the therapies in the Healey Trial fall under the phase 2/3 trial or what the FDA considers a pivotal trial.

Please explain the expanded access element of the  trial.

  • Since the duration of the trial is not very long compared to other pivotal trials, the FDA encourages patients who would like to continue therapy usage after the trial is completed, to do so. This will allow researchers to monitor the long term safety of the therapy.
Curious about patient eligibility criteria? Click here and scroll to the bottom of the webpage.

Click on any of the company logos below to learn about the therapy they are contributing to the platform trial.

For more Healey Platform Trial info please click here for the MGH website

COVID-19 (SARS-CoV-2) Resources

Massachusetts General Hospital (MGH) has recently released incredibly useful webinars and Q&A sessions regarding living with ALS during this time of SARS-CoV-2. The link to their resource page is here. 

Follow this link to view SARS-CoV-2 resources synthesized by the ALS/MND Alliance. 

Click here to learn the latest ALS community updates on SARS-CoV-2 from I AM ALS.

We pray everyone stays safe and healthy during this time.

-The Board of the Martha Olson-Fernandez Foundation

ALS Focus Survey

Survey 1 is closed. Click here for results

MOFF encouraged ALS families to participate in Survey 1 of the ALS Focus Survey Program. Survey 1 was developed by the ALS Association and targets ALS patients and their caregivers. The purpose was to collect information to help ALS advocates fight for access to care and financial security for those affected by ALS. 

Background: The ALS Focus Survey Program is a patient and caregiver led program that asks people with ALS and current and past caregivers about their needs and burdens. The goal is to learn as much as possible about individual experiences throughout the disease journey so that the whole ALS community can benefit.

All data collected is de-identified and shared free of charge to the entire ALS community. 

Click here to register for future ALS Focus Surveys

ClinWiki x ALSA GW x MOFF

Let’s face it, the clinical trial world is overwhelming, especially if you have recently been diagnosed with a disease. is a nonprofit foundation whose mission is to make clinical trials more transparent and approachable. ClinWiki works with various stakeholders across many therapeutic areas in order to invite collaboration into a space that was previously siloed. Their efforts allow clinical research teams, sponsor companies, and patients recognize that we are all working towards the same end: effective treatments and cures for the diseases that plague our population.

The ClinWiki ALS project began in December 2019 and is projected to continue through October 2020. Natalie Fernandez, CFO and Program Director at MOFF, is the ALS Team Project Leader. She will be working in collaboration with Amy Westermann, the Executive Director at ClinWiki, and Sheri Strahl, COO of ALSA GW to complete the project. The project goal is to orient ClinWiki to the ALS community and emphasize the priorities of ALS patients as they search for clinical trials.

ALS is a unique disease when it comes to clinical trials for many reasons. For starters, it is debilitating and fatal. These two facts alone make concepts such as traveling to trial sites, placebo controls, and the extremely long duration of clinical trials incredibly burdensome to people living with the disease. The role of ALSA GW and MOFF is to help format the ALS clinical trial search in such away that highlights the important aspects of the trial so that people living with ALS do not spend months sifting through the data on Enrollment criteria such as ALS onset, Forced Vital Capacity, and disease duration are just some of the tags being created in order to make the trials easier to sift through. Some key questions that will be highlighted are listed below.

  • Does this trial offer Expanded Access?
  • Does this trial offer Open Label Extension (OLE)?
  • Is travel required?
  • Does this trial offer remote monitoring?

Natalie is very excited to be working on this project. Stay tuned for the official launch in Fall 2020!

ALS Biomarker Panel at the Society for Brain Mapping and Therapeutics Conference

15–17 March 2019

SBMT 16th Annual Congress

Los Angeles Convention Center in LA


The 16th Annual World Congress of SBMT brought together physicians, scientists, policy makers, funding agencies and industry to further the advances and applications in brain and spinal cord mapping and image guided therapies (operative and non-operative). The conference took place at the Los Angeles Convention Center.

The goal of the conference was to create a critical mass by introducing synergy amongst inter-disciplinary researchers to further understand the brain function and nervous system. It also served as a platform from which to develop interactions between many of the stakeholders who had extensive collaborations at national and international levels.

The conference provided the opportunity to be at the forefront of brain sciences, therapeutics in general and neural stem cells interventions in particular. It served as a strong platform for industry and biotech companies to interact with academia in frontiers of science in this field for translational initiatives involving diverse patient’s interest groups.

16th Annual Congress of Society for Brain Mapping promotional video – 2019 from Brain Mapping Foundation on Vimeo.

Topics & Speakers

Moderator: Mike Piscotty, ALS Cure

Biomarkers to aid diagnosis and monitor drug efficacy in clinical trials for ALS
Robert Bowser, Ph.D. The Barrow Neurological Institute

Biomarkers for CNS injury and disease
Kendall Van Keuren-Jensen , Ph.D. TGen

A data-driven approach links microglia to pathology and prognosis in ALS
Johnathan Cooper-Knock Ph.D. University of Sheffield, UK

Allosteric assembly machine modulators for ALS therapeutics
Vishwanath R Lingappa, M.D., Ph.D. Prosetta Sciences

Financing ALS biomarker discovery
Natalie Fernandez, 2019 MBA Candidate, The Martha Olson-Fernandez Foundation

The interdisciplinary nature of the SBMT conference allowed the ALS biomarker panel members to learn and collaborate with fellow industry members. The biomarker research topics were promising. Stay tuned for research article postings on the studies discussed.

Director Bobak Kalhor interviewed each of the panelist for an ALS media project. Bobak is director of “A Dying King- The Shah of Iran.” He recently lost his mother to ALS.

MOFF x ALS Cure Project

The Martha Olson-Fernandez Foundation is proud to be in collaboration with the ALS Cure Project. The ALS Cure Project is run by Mike Piscotty who lost his wife, Gretchen Piscotty, to ALS in 2018. The Project is comprised of an executive council, a research counsel, and a business council. Natalie Fernandez, CFO at MOFF, sits on the business council. Read more about the ALS Cure Project’s vision and their $1 Billion grand prize here.

Mike Piscotty’s son, Stephen Piscotty plays for the Oakland A’s. The Piscotty family was featured in an ESPN Sports Center video when Gretchen passed away.

2019 California ALS Research Summit

Day 1 Takeaways: 

1. Whole genome sequencing (WGS) analysis projects are being undertaken by the following collaborative bodies: Track ALS, NeuroLincs, CABB, ALS Natural History, Answer ALS, NYGC ALS Consortium, and Target ALS. The data will be available to the public via the database ALSoD in early 2019.

2. There are several active stem cell trials being conducted in the U.S. by Braincell Therapeutics and also Avexis Pharma

3. Dr. Steve Finkbeiner from UCSF discussed machine learning in the context of ALS research. More specifically, he discussed how efficient and accurate the technology will be when it comes to exploring therapeutic targets and genetic patterns in neurodegenerative diseases.

4. There is currently a lot of research being done on the role of microglia, astrocytes, and macrophages in ALS. These research efforts attempt to understand the underlying pathology of the disease.

5. TDP43 was discovered to be present in all ALS models. It is now being looked at as a target that can be corrected with gene alteration.

Most of the top researchers in the ALS research world on the west coast were represented at the conference. The list below contains 5 ALS research pioneers. If you are interested in their research, each of them has many publications under their names.

* Leslie Thompson, PhD, UC Irvine
* Steve Finkbeiner, MD, PhD, UCSF
* Aaron Gitler MD, PhD, Stanford University
* Clive Svendsen MD, PhD, Cedars-Sinai Medical Center
* Dr. Richard Smith (CNS)

Day 2 Takeaways: 

1. The Lazarus Project was presented by Dr. Ranjan Gupta, MD from University of California Irvine. His research debunked the theory that motor function cannot be recovered after 6 months of de-innervation. His surgical procedures are truly miraculous. In some cases, he was able to execute nerve transfer surgeries to bring function back to limbs that had not been utilized for 6 years. Learn more about his research here.

2. Dr. Steve Finkbeiner’s lab revealed results from his high throughput microscopy project which targeted autophagy in ALS cells. Read more about his research here.

3. Mitsubishi Tanabe Pharma America, the company that makes Radicava, was represented by Dr. Stephen Apple, the senior medical director at the company. Dr. Apple discussed post market surveillance and their Phase 4 trial that they laid the foundations for. The Phase 4 trial plans to utilize biomarkers to detect the efficacy of the drug within ALS patients. Results from the study should be release in 2021.

4. New ALS targets were identified by Dr. Aileen Anderson from University of California Irvine. She studies spinal cord injuries and encouraged researchers to look into the autocrine signaling pathways in neuronal cells. More specifically, Dr. Anderson’s research explored neuronal stem cells to see if they had specific receptors that could be blocked. Her research found 5 novel receptors in neuronal stem cells that could be potential targets.

5. Routes of ALS drug administration were discussed in the context of antisense oligonucleotide therapies (ASOs) in order to answer the questions: How much virus is necessary, and where do you need to inject it in order to adequately cover the CNS? A new method of subpial drug administration (injection of the drug under the pia mater in the CNS) is being explored. Previously, intrathecal administration was the standard for ASO drug administration such as AVXS-10.

At the end of the day Ask the Experts took place in a separate building. At this panel, ALS patients and their families were able to discuss the most current scientific findings with the researchers themselves. Even though much progress has been made, the disconnect between the research world and the ALS patients was still noticeable.