Author Archives: MOFF

2022 Hike x Brunch

The Martha Olson-Fernandez Foundation Annual Hike x Brunch

  Saturday, February 26, 2022 and Sunday, February 27, 2022

Thank you to all donors, MOFF hikers, random SLO hikers, silent auction donors and winners, and social media giveaway participants and winners!

Traditional Hike: Over 50 MOFF associated hikers completed either Felsman Loop or Bishops Peak in SLO and brought roses either to the peak or to Martha’s memorial bench. The MOFF team was present at the trailhead from 9:00 AM – 11:30 AM PT on Saturday and Sunday. Many people who were initially unfamiliar with MOFF also opted to take a rose with them on their hike to help us raise awareness of ALS. It was a beautiful weekend to do so!

Virtual Hike: Over 60 Instagram/Facebook users tagged @moff.cureals and hashtagged #cureals #nevergiveup in their hiking stories! It was a tough competition, but in the end, Nancy Walter and Courtney Crawford won the social media giveaway! Thank you to all of our virtual participants for raising awareness in your communities! MOFF supporters hiked in NY, CA, AZ, TX, HI, MX and the UK! Visit the MOFF instagram and view these stories in our highlights section.

Other Ways to Participate

Silent Auction: Congratulations to our Silent Auction winners! Participants were represented in 5 different states!

Donate: Together we raised over $15,000 to support MOFF’s ALS Patient Care and Research Programs! Way to go!

Why We Hike

This event honors the life of Martha Olson-Fernandez and every other individual who can no longer hike due to the effects of a neurodegenerative disease. Martha was a well-known SLO community member and an avid hiker when she was diagnosed with ALS in 2011. Her memory lives on through this MOFF community and her family and friends. It is through Hike x Brunch event that we are able to reflect on the lives of the incredible individuals the SLO community has lost to ALS and send strength to those who are living with the disease today.

Thank you to the SLO families that have participated in the MOFF Hike x Brunch since the 1st Annual in 2015 and to all of you who have joined us along the way!!

ThinkALS Tool

The ThinkALS tool was developed by the ALS Association to assist clinicians in the ALS differential diagnosis process. On average, ALS takes one year from time of onset to a definitive diagnosis. The obvious reason a rapid diagnosis is not possible is that a diagnostic biomarker for ALS does not yet exist. That is to say, you cannot run a lab test on a nasal swab and detect ALS, but rather every other condition must be ruled out first. Conditions commonly confused with ALS include spinal muscular atrophy, peripheral neuropathies, infections such as Lyme disease and hepatitis C that cause muscular weakness, Parkinson’s Disease, and behavioral variants of frontal temporal dementia. If you are interested in reading more about the ALS diagnostic delay and potential solutions, we encourage you to read the previous MOFF blogs listed at the bottom of this post.

MOFF will be distributing this ThinkALS tool to primary care practices in 2022 in an effort to identify people living with ALS earlier and refer them to an ALS specialist (neurologist) as soon as possible.

Call to Action: Print and share, or email this tool to your primary care physician’s office. Start a conversation about ALS with your health care provider. The more we talk about it, the more awareness we will raise, and the earlier ALS patients can get the medical team they need.

Click the image above to access the PDF version

For further information on the ThinkALS tool, visit this webpage.  

A Solution to the ALS Diagnostic Delay

1 in 300

The Don Woodward ALS Research Grant Sponsorship

Who is Don Woodward?

Don Woodward is a legendary member of the San Luis Obispo, CA community. He was an incredibly talented football player, golfer, and businessman. His name is well known in the California car industry where he started his business career at one of his grandfather’s dealerships, Lompoc Honda. Don’s name is one that brings a smile to everyone’s face that knew him and “f*#k ALS” energy to those who understand he was taken when he had much more life to live. Don’s wife at the time, Maggie Woodward, selflessly cared for Don throughout his ALS journey. She remains a pillar of strength and provided Larry Fernandez valuable guidance when Larry was going through his caretaking experience with Martha. The burden of ALS on families is palpable. The Woodward and the Fernandez families had everything you need to make the most out of the time you have left: love, humor, and Don and Martha.

Friends of Don golf in the Martha Olson-Fernandez Foundation (MOFF) Annual Golf Tournament and the “Woody” tournament at the San Luis Obispo Country Club each year. His memory truly is kept alive by his family, friends, and the golfing community. His friends, John Ronca and Scott Dierks, work closely with MOFF. John has served on the MOFF board for the past 2 years.

Why does the Don Woodward ALS Research Grant exist?

The grant exists to connect the golfers and sponsors that participate in the MOFF Annual Golf Tournament fundraiser directly to an ALS lab. When Martha outlined MOFF’s mission in 2012, she wrote on a piece of paper that “All funds go to research.” This grant is our way of making sure the MOFF community stays rooted in our mission and that our donors feel that connection to the research projects that are creating potential therapies for this brutal disease.

What labs have received this grant money ($10,000) in the past?

2019: USC Ichida Lab for ALS (Dr. Justin Ichida)

2020: Duke University ALS Lab (Dr. Richard Bedlack)

2021: The ALS Cure Project at Lawrence Livermore Labs (Mike Piscotty)

How do I apply to have my lab considered to be the recipient of the Don Woodward ALS Research Grant?

Email moffoundation@gmail.com and ask for our request for proposals (RFP) application. The deadline for proposal submissions is July 1st each year. Grants winners will be announced in September and grant funding will be distributed in February of the following year.

How do I become the Don Woodward ALS Research Grant Sponsor?

MOFF encourages any individual or company to become the Don Woodward ALS Research Grant Sponsor. Sponsors will have the opportunity to tour the ALS research lab that is selected and discuss the project with the researcher. Sign up to receive notifications from MOFF here. MOFF will announce the opening of registration for our annual golf tournament event in July. Once you receive notification of the golf tournament event, simply sign up via our event platform that will be listed in the email. Alternatively, email moffoundation@gmail.com and announce your intention to be the sponsor.

 

A HUGE special thanks goes out to Larry Fernandez who has been the Don Woodward ALS Research Grant Sponsor for the past 3 years. Thank you, Larry for your continued support!!

Art for ALS Healing: Ken Brenner

Ken Brenner is an incredibly talented artist living with amyotrophic lateral sclerosis (ALS). His talents hardly stop at art. An athlete, successful Silicon Valley banker, husband, and father of 3 children and grandfather of 2, Ken has lived a full life thus far, and has much more to live.

Ken was diagnosed with ALS in late 2020. The diagnosis and his disease progression sent shock waves through the Brenner family. It forced Ken to retire from a long banking career which he was not ready to be done with. His wife also retired, and their children moved back to CA from New York City to be with their father. Needless to say, all of their lives were uprooted and their trajectories changed. That’s just what ALS does.

Ken’s retirement plan was to focus on his art and to show the world his artistry. When ALS happened, he still found a way to make painting work.  “Art gives me something to focus on,” Brenner said. “I lose myself; I don’t think about my issues. So it’s sort of a mental break.” As time elapsed, he had trouble holding a brush, but continued to paint with his fingers. His paintings showcase his creativity, personal exploration, disease progression, and his will to face the joy and the struggle of each new day.

Ken’s oil paintings are well recognized and sought after by well-established private and corporate art collectors. In 2020, his path crossed with a younger latino artist, Octavio Molina, who had faced his own set of challenges growing up in Mexico before immigrating to the US. The two formed an incredibly special bond and have helped each other through their collaborating on paintings in 2020-2021. “He always tells me he’s good at framing my chaos,” Brenner said of Molina in a joint interview with ALS News Today.  When it comes to ALS, there is definitely a lot of chaos.

The Brenner Family and The Fernandez Family were serendipitously connected through their mutual friend, Alexis Schwartz. Together, they are fundraising for the Expanded Access Platform (EAP) Program at The Sean Healey Center for ALS. Their fundraising goal is $30,000.00. This is the cost to put 3 individuals living with ALS through the EAP program for 1 year.

Donate to the EAP at MGH today via the link below. Learn more about the expanded access protocol programs and why they are so important to ALS patients here.

01-Nov Update: We reached our goal! $30,000 will be donated to the MGH Expanded Access Protocol Program in Ken’s name in February of next year (2022).

  BUY KEN BRENNER ART:

View Ken’s art via the links below and contact Jordan Brenner at jordan@100collective.com.

Ken Brenner’s article in ALS News Today ALS News Today

MGH Expanded Access Protocol (EAP) Program

The Martha Olson-Fernandez Foundation will donate $30,000 from their upcoming 9th Annual Golf Tournament (02-Oct) to the Expanded Access Protocol (EAP) Program at the Healey Center for ALS at Massachusetts General Hospital. Here’s why:

Amyotrophic lateral sclerosis (ALS) is a fatal, neurodegenerative disease that affects roughly 9 out of a 100,000 people in the United States at any given time1. Put in more relatable terms, the average person has a 1 out of 300 chance to develop ALS in their lifetime 2.

There are currently only 3 FDA approved therapies indicated in the treatment of amyotrophic lateral sclerosis (ALS). These drugs, RADICAVA®, NEUDEXTA®, and Rilutek fall short of providing significant, disease modifying effects for those living with the disease. Those individuals that seek a more significant therapeutic benefit and a better chance at survival enroll in clinical trials. There are currently 86 global clinical trials that are currently recruiting patients3. The caveat is that nearly 60% of ALS patients fail to qualify for clinical trial enrollment due to the rigid inclusion/exclusion criteria written into each trial protocol.4  Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials.5

2020 statistics provided by the Golden West Chapter of the ALS Association revealed that there were just over 90 patients living with ALS on the Central Coast. These patients, right in our backyard, are dying. Their only chance is experimental therapies. The MGH Expanded Access Protocol (EAP) provides just that. Since the second quarter of 2018, The MGH EAP has supervised 133 ALS patients receiving access to therapies not yet approved by the FDA. These patients have seen therapeutic benefits and their stories of function recovery are inspiring.

Some of the drugs the EAP at MGH allows access to are currently being tested in the Healey Platform Trial: Verdiperstat, CNMAu8, and Pridopidine. All of these drugs are administered under the supervision of an interdisciplinary team of doctors and longitudinal data is collected for research purposes to assist with therapy validation and categorization.

This fundraising effort is a partnership between the Ken Brenner Family and the Martha Olson-Fernandez Foundation. To date, they have raised $6,900.00 and they need your help to reach their $30,000 goal. Since it costs $10,000 to provide 1 patient access to therapy for 1 year, this total will fund 3 patients.

NEVER GIVE UP

Donate to the MGH EAP Program today.

  1. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6567553/
  2. https://www.neurologylive.com/view/an-overview-of-causes-and-risk-factors-for-als
  3. https://clinicaltrials.gov/ct2/results?cond=ALS&Search=Apply&recrs=a&age_v=&gndr=&type=Intr&rslt=
  4. https://alsnewstoday.com/news-posts/2019/01/21/new-eligibility-criteria-needed-to-improve-als-trial-outcome/
  5. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5443564/

9th Annual MOFF Golf Tournament: October 2, 2021

 
 

Thank yous, tournament prizes, and silent auction prizes will be sent to the event participants throughout October. 

Click here to view event results

COVID-19 Notice: We are excited to see everyone out on the course! In order to keep everyone safe, we are encouraging that all of our golfers, volunteers and visitors have negative Covid-19 tests. If you are experiencing any symptoms we respectfully request you refrain from attending.

The 9th Annual Martha Olson-Fernandez Foundation Golf Tournament has ended.

Special thanks to all the golfers, sponsors, and volunteers that made this event possible. 

Golfer and Volunteer FAQs

Q: Where is the tournament?

 A: Cypress Ridge Golf Course in Arroyo Grande, CA

Q: When is the tournament?

 A: Saturday, October 2, 2021

Q: When does check-in open?

A: 9:00 AM

FYI There will be a putting competition happening during check-in.

Q: When does the tournament start? 

          A: At 10:00 AM. Please arrive no later than 9:45 AM if possible!

          Q: Will breakfast and lunch be provided?

A: Yes! Coffee and breakfast burritos will be available at check-in and DePalo and Sons will be providing lunch. Please let us know if you would like a vegetarian option!

Q: What course games will be available? 

A: Closest to the Pin, Beat the Pro, Longest Drive, Hole-in-One, and more!

Q: How will the winners be determined? 

A: The top 2 scoring teams out of Net scoring category and top 2 teams out of the Gross scoring category will be selected at winners! There is also a hackers prize.

Q: Will there be an awards ceremony this year? 

A: No. Winners will be announced via email, on this webpage, the MOFF instagram account, and the MOFF FB account.

Q: Can I golf next to my friend’s foursome? 

A: Yes! Call or text Victoria at 805 458 9673

Click the registration form image below for more tournament details.

Click to navigate to the gt event page

2021 Fiesta Dinner

Welcome to the Martha Olson-Fernandez Foundation Fiesta Dinner

This event is held each year to honor the winner of the Fiesta Raffle at the Martha Olson-Fernandez Foundation (MOFF) Golf Tournament. The winner of this year’s dinner was: Sean St. Dennis! All raffle earnings were donated to the ALS Association Golden West Chapter and were earmarked for patients living on the Central Coast of California. This funding is incredibly important. ALS can cost a single family up to $200,000 of out of pocket expenditures a year.

We are honored to host Sean and Patty St. Dennis and their guests. We hope everyone has a wonderful evening!

2021 Virtual Hike x Brunch Event

Thank you to all the participants of the 2021 MOFF Hike x Brunch! This year, we had hikers tuning in on their Facebook and Instagram accounts from San Luis Obispo (SLO), Danville, Marin, San Francisco, the OC, Lafayette, Cayucos, Morgan Hill, Irvine Hills, Santa Monica, Atascadero, Washington, and New York City. The winners of the instagram giveaway were: Caroline Chalmers and Rachel Dettmer! They won MOFF swag and a gift bag from Bronze Sun Spa in SLO.

We are incredibly grateful to the silent auction item donors and silent auction participants. The virtual silent auction took place on the MOFF Hike x Brunch event site and was a great success!

It was wonderful to see all the hikers who stopped by our MOFF table at the Patricia Street entrance of Felsman Loop in SLO. We had roses in place for hikers to take up to Martha’s memorial bench on the loop.

The Splash Cafe brunch boxes were a hit! Thank you to all the hikers who preordered brunch boxes and picked them up after their hike. $4 from each brunch box purchase went to MOFF.

Thank you to Cafe Andreini for giving away free coffee to raise awareness of ALS in our Central Coast community!

This event happens annually to raise awareness of ALS in our community and to raise money to fund ALS patient care and research. Martha was an avid hiker- we encourage everyone to go on an adventure and hike for all those with ALS who cannot hike anymore.

Meet the MOFF Event Planning Intern: Victoria Humphrey

Victoria Humphrey will be joining the MOFF team this month! We are very excited to welcome her into our event planning efforts as we work to raise money to fund ALS research and patient care initiatives! Read a brief interview with Victoria below.

Q: Where are you attending college and what is your major?

A: I am a Cal Poly student majoring in Experience Industry Management with a  concentration in Event Planning

Q: Why does event planning at MOFF interest you?

A: While planning events is an intriguing job, being able to plan events for MOFF that  contribute to finding a cure for ALS is both intriguing and fulfilling work that I am excited to take on!

Q: What is your favorite fitness activity?

A: My favorite fitness activity is cheerleading as I was a collegiate cheerleader for the past four years

Q: What is your favorite slocal hangout?

A: My favorite place to hangout with friends and family is Avila Beach.

Q: Where you see yourself in 5 years?

A: In 5 years I see myself starting my own business, planning and executing events for causes I find passion for.

Q: What are you surprisingly good at?

A:  I am surprisingly good at calligraphy, I love using this skill to make home decor.

PSA: The final question to this interview may seem rather dark because it deals with death. That being said, the fatal nature of ALS brings death to the forefront and causes people living with ALS and their caregivers to grapple with the concept on a daily basis. MOFF has been operating as a grant making nonprofit within this space for 8 years now. We have seen too many incredible people have their lives cut short because of ALS. It is important to bring the urgency associated with living life in the face of death into our fundraising efforts. 

Q: What is your relationship with death?

A: Death used to be my greatest fear, but after experiencing more deaths within my life I have grown to appreciate it. Death is still something I fear, but I have accepted that without it life is not as extraordinary. 

MOFF Response to Loss

The ALS community lost two incredible people this past Thanksgiving week. Leslie Sands (1951-2020) and Patrick Quinn (1983-2020). Our hearts go out to their families and friends. This is a difficult time for everyone during the economic and emotional hardship of COVID-19. Adding the painful loss of a loved one makes isolation that much more difficult. We will be sending prayers their way.

The statistics behind the fatality of ALS paint an image laden with loss:

Incidence:

Each year in the United States 5,000 people are diagnosed with ALS. This seems like a relatively small number until you start to look at the numbers in terms of the lifelong chances of an individual contracting the disease: 1 in 300.

Survival:

50% of those 5,000 individuals will live 3-5 years.

25% live 5 years or more and yet another 10% will live more than 10 years.

In the end, ALS is 100% fatal.

Thankfully, over the past 8 years, an increase in disease knowledge has led to slightly longer survival and higher quality of life. This is in part due to the now 4 FDA approved drugs for ALS: Riluzole, Radicava, Tiglutik, and Neudexta. These drugs are a step in the right direction, but they are not enough.

Emotional Rollercoaster: 

Every time we lose a member of this tight-knit community, everyone feels it. For people working to solve the ALS puzzle, it feels like the weight of a failure, the breathlessness of time running out, and the frustration of not being able to do enough to save that individual. Then, there is the subtle fear over how many people will die before a cure is found and the esoteric contemplation of why ALS exists and why it is so ruthless.

MOFF Response:

This roller coaster causes MOFF to operate like we are running out of time, because we are. We fund the most cutting edge research, and we grant money to patients to help improve their quality of life in whatever ways current care standards permit. We seek alliances within the ALS nonprofit community that allow us to connect ALS families to the services they need, and we work with ALS collaborations that aim to share ALS information and knowledge. When we lose a person living with ALS, we don’t stop, we keep going.

Clinical research is where we direct people living with ALS who are not satisfied with the current, minimally effective standards of care. Clinical research is what we emphasize will bring about the next effective treatment to ALS, because it will. There are currently 11 Phase 3 clinical trials that are recruiting in the U.S. We encourage all ALS patients that are interested in finding a cure, to participate in one of these trials. If you would like further information on navigating the clinical trial landscape or would like to speak with someone who has gone through the clinical trial experience, please email Natalie Fernandez at moffoundation@gmail.com

 

#GivingTuesday: December 1, 2020

#GivingTuesday is a nationally recognized day of philanthropic giving. According to the #GivingTuesday creators, it is a “global generosity movement.” Last year, the total amount that was donated to nonprofits in the U.S.A was $1,970,000,000.00! That is a pretty incredible number.

This year on #GivingTuesday, MOFF is emphasizing the urgency with which we need to invest in ALS research. Every 90 minutes someone dies of ALS. This has been happening since Lou Gehrig had the illness in 1939. Something needs to change, now. Every cent counts towards this fight.

Donate Today

In 2019, MOFF hosted a #GivingTuesday campaign that provided information on the below ALS FAQs.

Q: Does ALS have a genetic cause?

A: Yes. 10% of all ALS cases are caused by genetic mutations that are inherited from a family member. 90% of ALS cases are sporadic in origin which means the cause is unknown. That being said, there are some gene mutations that are implicated in sporadic cases, such as the C9orf72 gene. The plot thickens.

Q: How does “sporadic” ALS happen??

A: We wish we knew! There are many scientific theories surrounding sporadic ALS. Here are a few: Oxidative stress, mitochondrial dysfunction, glutamate toxicity and toxic exposure (occupational hazards, cyanobacteria etc.). Read more here.

Q: Has anyone survived ALS?

A: Yes! Dr. Richard Bedlack at Duke studies what he calls “ALS reversals.” There are 48 documented cases of ALS being reversed. MOFF has committed funding to Dr. Bedlack’s research on the microbiome of people who have “reversed” ALS for FY2021.

Q: Did the Ice Bucket Challenge help at all?

A: Yes. The ice bucket challenge raised $115M in the U.S. alone. Internationally it raised $220 M. $90 M of those funds have been distributed throughout the scientific community and have lead to identification of new pathological mechanisms and genetic causes of the disease. Here is a breakdown of the impact of those funds.

Q: What type of ALS did Martha have?

A: Martha had bulbar-onset ALS.

Q: Is ALS more common in men or women?

A: ALS is 20% more common in men than women. However, according to recent research, the incidence becomes more evenly distributed among gender with increasing age.

What Happened in Guam? (Halloween Edition)

Classic Halloween stories have a spooky setting (like a haunted house or an abandoned log cabin), a deranged animal, and a full moon. While the story you are about to read doesn’t contain any of these elements, the reader may argue that it’s spookier, because it’s real.

Let us begin. The setting is in present day, Umatac, Guam. To most, Guam is a beautiful island in the South Pacific that is utilized by the U.S. military for its strategic positioning. If you talk to any scientist that studies neurodegenerative diseases, they would not site Guam’s natural beauty, but rather, they would allude to Guam’s dark medical history. One that has puzzled the scientific community for years. According to a 2017 article written in Penn Medicine Magazine, 1904 was the date the first official reports documented a peculiar disease that was plaguing the Umatac community. It is said that folklore reaching back two centuries documented cases of the same symptoms: “tremors, paralysis of the arms and legs, missed memories, and bouts of dementia” (Penn Medicine Mag 2017).

The Chamorro people are the locals of Guam. They called the disease lytico-boding, which alluded to the paralysis and listlessness that characterized the disease. The more formal scientific name is amyotrophic lateral sclerosis-parkinsonism-dementia or ALS/PDC. Lytico-boding was a very fast progressing disease that affected the islanders from age 15 to age 50. In 1954, it was the leading cause of death among the Chamorro people. A 1954 article in the Neurology Journal reported the incidence rate of lytico-boding to be “50-100 times that of the global average for ALS during this time.” That’s insane!

It is truly spooky. Luckily, the rate of disease has decreased significantly in the past decade, but that doesn’t erase what caused the disease in the first place.

Scientists have studied the disease for nearly a century and still have no definitely answers. That is certainly not due to a lack of hypotheses or funding. Parties involved in trying to solve the Chamorro people’s medical mystery have included the National Institute of Health (NIH), the Department of Defense (DOD), multiple academic institutions, and scientists from various disciplines. Notable researchers who have studied lytico-boding include:

John Q. Trojanowski, Dr. Gerard D. Schellenberg, Dr. Leonard T. Kurland, Majorie Whiting (anthropologist), Dr. Peter Spencer, Dr. Paul Cox, and Dr. Oliver Saacks.

Scientific hypotheses have all pointed to environmental factors such as the cycad seeds that the villagers consumed, military waste, poor eating habits, or interfamilial marriages. The Chamorro people have their own theories, such as the disease is the aftermath of a curse from an angry Catholic priest. A popular, more recent theory, developed by Dr. Paul Cox was featured in the February 2019 edition of Fortune Magazine. His research points to the high concentration of ß-N-methylamino-L-alanine (BMAA) that remained in the fat tissue of the bats that the Chamorro people ate as a delicacy. Dr. Cox affirms that a small molecule amino acid known as L-serine can combat the affects of BMAA. He is currently  involved with two Phase II trials of L-serine, one for ALS, and one for Alzheimer’s disease (AD). He believes L-serine will prevent neurofibulary tangle formation in AD and increase ALSFRS-R scores in ALS patients. The video below explains his research in Guam and in the U.S.A. further.

While lytico-boding has been useful for developing questions and theories of other neurodegenerative diseases, the mystery remains unsolved and downright SPOOKY. For more information on the medical mysteries of Guam, click here to read Steve Graff’s complete article.

HAPPY HALLOWEEN

If you are interested in more cases of neurodegenerative disease clusters. Check out the link below.

ALS Clusters in Japan 

ALS Research Paper Review (ALS RPR)

Natalie Fernandez is honored to be co-chair of the newly formed ALS Research Paper Review (ALS RPR) Platform. Her co-chairs are Phil Green and John Russo. Both John and Phil are incredible ALS Advocates and ALS Research Ambassadors. Collectively, Phil and John have been involved in pretty much every major ALS nonprofit org and ALS initiative that has brought about patient-driven change. That list includes I AM ALS, Team Gleason, EverythingALS, The ALS Association, and numerous patient and caregiver advisory committees. John Russo is the mayor/networker/diplomat of the ALS community per se and Phil should receive his honorary PhD in the mail any day now. His knowledge of ALS research and clinical trials is invaluable.

What is the ALS RPR? This initiative was suggested by a couple members of I AM ALS and NEALS and is intended to bridge the gap between scientific research papers and lay people working to understand the disease. The co-chairs plan to host an hour long session once a month in which we review a paper with the researchers that authored the study. Supplementary materials will be provided to assist with scientific and research concept understanding. Our goal is to provide a platform for an engaging conversation that pays respect to and fosters understanding of research within the ALS disease state.

Dr. Bedlack from the Duke ALS Clinic has graciously agreed to be our scientific advisor to help get the project off the ground. Thank you Dr. Bedlack 🙂

Centaur Trial (AMX0035): Longterm Survival Data

MOFF attended the NEALS webinar hosted by Dr. Sabrina Paganoni and Dr. Merit Cudkowicz on Wednesday, Oct 21, 2020. Click here to gain full access to the webinar once it becomes available.

Background Information

  • AMX0035 is a combination of two small molecule drugs: Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA).
  • The Centaur Trial (NCT03127514) looked at the functional impact of AMX0035 on the ALSFRS-R score of people living with ALS vs. a placebo. The total trial enrollment was 137.
  • The statistically significant Centaur Trial results are documented here. In short, the drug causes significant slowing of ALSFRS-R decline.
  • The open label extension of the Centaur Trial was utilized to observe the impact of AMX0035 on the long-term survival of people living with ALS.
  • The readout contained 35 months of data (beginning from initial trial enrollment period in 2017/2018).
  • The readout came from a comparison of trial participants who took AMX0035 vs. those who were administered a placebo.
Key Findings
  • The following results were disclosed by the principal investigator of the Centaur Trial, Dr. Sabrina Paganoni, during the webinar:
    • There was a 6.5 month longer median survival in the group originally randomized to AMX0035 than the placebo group. The median survival durations were 25 months and 18.5 months respectively.
    • Please read the Oct 16th Amylyx Pharmaceuticals press release of their long-term survival data findings here.
Both the placebo and non placebo groups had the option to take riluzole and/or endaravone. That being said, there was a sensitivity analysis done in both the function and survival studies. The sensitivity analysis concluded that AMX0035 had affects on top of the standards of care aka riluzole or endaravone.
Q & A
Q: Could this work for variants of ALS, such as primary lateral sclerosis (PLS)? A: Given the MOA of AMX0035, it is reasonable to think that it may be effective. However, no PLS patients were enrolled in the Centaur Trial.
Q: What is the route of administration? A: The formulation used in the Centaur Trial was a powder form of both drugs that you mix with water.
Q: Will it be affordable? A: Not determined at this time.
Q: Will AMX0035 be available through Expanded Access? A: Not determined at this time.
Q: Were there notable side effects? A: Mild nausea and diarrhea was common and expected in first 3 weeks of the trial.

Prop 14 (CA)

What’s all the buzz about Proposition 14? Why is the scientific community encouraging everyone to vote “Yes” ?

Prop 14 plans to use debt to fund biomedical research: $5.5 billion in state general obligation bonds to be exact. The basic math is this: $5.5B granted today will accumulate $2.3B in interest. This interest will be payed back over 30 years in annual installments of $260 million by “Primarily General Fund tax revenue” aka, your taxes.

How is the $5.5B being distributed?

The grants will be distributed by the California Institute of Regenerative Medicine (CIRM) to “educational, nonprofit, and private entities for: stem cell and other medical research, including training; stem cell therapy development and delivery; research facility construction and associated administrative expenses”(Official Voter Information Guide).

This debt funding concept is not new. Prop 14 is preceded by Prop 71 which awarded $2.7B in stem cell grants since 2004. That money was utilized for important cancer research projects at a time when the federal government refused to fund stem cell research. The catch? The research was supposed to pay for itself with projected revenues from successful therapies. That has not yet happened.

Revenue projections aside, stem cell research is incredibly important to the ALS community. Please watch this webinar to hear experts discuss stem cell projects involving basic research and potential therapies for ALS that depend on Prop 14.

Healey Platform Trial Monthly Updates

The clinical operations team involved with the Healey Platform Trial is truly going above and beyond the typical level of transparency. They have promised to host informal weekly Q&A sessions and will also hold monthly updates on the platform trial. If you would like to register for any of the weekly Q&A sessions or monthly updates please visit the Massachusetts General Hospital (MGH) websiteTheir website also contains basic information about the trial, details on the 5 therapies being tested, and a library of previously recorded webinars.

Thank you to the individuals from the ALS patient and scientific communities that are involved in elevating the bar for ALS clinical trials. Thank you specifically to MGH, NEALS, and Berry Consultants. This is truly exemplary.