Author Archives: MOFF

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.

Novus Acquisition of Anelixis

The Martha Olson-Fernandez Foundation (MOFF) was very excited to hear that Novus Therapeutics had finalized its acquisition of of Anelixis Therapeutics this past September.  Anelixis Therapeutics was the for-profit drug development arm of ALS Therapy Development Institute (ALSTDI) that was created specifically to develop their anti-CD40L antibody therapy for ALS known as AT-1501. An article published in BioSpace earlier this fall announced that Novus plans to “finance the advancement of Phase II clinical trials for AT-1501.” This is great news!

Since 2016, MOFF has granted $105,000.00 to ALSTDI. In 2017, MOFF funding was earmarked to finance preclinical studies for AT-1501. The project was selected by MOFF due to the selective and specific nature of the AT-1501 antibody. These characteristics were a result of the years of development the potential therapy had undergone at the Biogen and later the ALSTDI labs. The MOFF board found the novel method of targeting the co-stimulatory pathway as an attractive investment.

AT-1501 not only holds promise in ALS, but also for Type I Diabetes, and other neurodegenerative and autoimmune diseases. The MOFF board is looking forward to following the development of this therapeutic option.

Learn more about AT-1501 in an ALSTDI Enpoints podcast episode here.

Raising Local ALS Awareness

The New Times SLO recently published an article on The Martha Olson-Fernandez Foundation. The reporter, Karen Garcia, does a good job of touching on the origins of MOFF, providing a glimpse into the annual events, and highlighting MOFF’s two critical programs: ALS patient care and research.

In the upcoming months, MOFF looks to do more to raise ALS awareness in San Luis Obispo (SLO). If you live in the SLO community and are interested in this initiative, please reach out to us via our “Get Involved” page.

CRISPR and ALS

Brief Overview

CRISPR is a highly specific and efficient genome editing technology. CRISPR facilitates the addition, deletion, or alteration of an organism’s DNA (NIH). If you would like a more specific explanation of CRISPR, feel free to watch the 1:39 video from the Mayo Clinic below.

As one can imagine, this is exciting and simultaneously, terrifying technology. It holds incredible potential for countless rare, genetic diseases. The concept of curing a disease by removing a faulty gene, is not new, but CRISPR is definitely one of the more cost-effective and reliable ways to do so. Thus far, CRISPR technology has been tested on many rare disease areas, including cancers, sickle cell anemia, Huntington’s Disease, Alzheimer’s Disease, and ALS. In 2018, Target ALS provided CRISPR Therapeutics with a 2-year grant to study ALS and Frontotemporal Dementia (FTD). We are excitedly awaiting those results.

Current CRISPR Challenges

The success of CRISPR technology depends on the structural validity of in vivo disease models, meaning how well the animal model of the disease actually portrays the disease in humans. The ALS field has historically struggled with this issue. CRISPR success also depends on the capabilities of high throughput genome mapping technology, meaning, how much knowledge we have about the genome we will be editing. As we observe these two key elements (animal model validity and genome mapping technology) becoming more efficient and reliable, we will begin to see very obvious therapeutic results from CRISPR.

Another roadblock to CRISPR’s usage in ALS is the fact that sporadic ALS is a messy disease. Messy meaning there are often a number of genes that contribute to the disease manifestation. Not only that, but these genes are not all ‘bad’ genes, but they are mutated in some way. For example, the protein C9orF72 is indicated in both familial and sporadic ALS. The normal function of this gene is to “influence the production of RNA from genes, the production of proteins from RNA, and the transport of RNA within the cell” (NIH). In ALS, C9orF72 is expressed as a hexanucleotide repeat expansion. If you program CRISPR technology to eliminate C9orF72, you may eliminate ALS, but you will also eliminate all the normal function of the protein, which could be deadly.

CRISPR Breakthroughs

Treatment of a Mouse Model of ALS by In Vivo Base Editing

In this study done at the University of Illinois, ALS disease progression was slowed in mice when CRISPR was applied. Researchers utilized cytidine base editors (CBEs) to remove SOD1 gene expression. “CBEs are a type of CRISPR single-base editors that change specific nucleotide bases in a DNA sequence” (ALSNewsToday). The result of using this type of CRISPR technology is that it does not delete the SOD1 gene, but rather it induces a ‘stop’ signal early in the gene expression so that it cannot progress to be a mutant SOD1 gene. Very cool.

The most common CRISPR headline you will see in relation to ALS is this one:

CRISPR reveals possible ALS drug target

In this particular study, a research team at Stanford used CRISPR to effectively ‘knockout’ or cut random genes out of the genome so that they observe “genes that protect neurons against the toxic effects of protein aggregates by being inactivated” (FierceBiotech). Their study revealed that “knocking out a gene called TMX2 prevented cell health in mouse neurons.” In the ALS world right now, finding possible therapeutic targets is a huge deal and most researchers are hard at work knocking out genes to do so.

The final, and arguably the most exciting, CRISPR breakthrough is highlighted in these two articles:

CRISPR-based method allows for reversible RNA editing

RNA-targeting CRISPR could yield treatment for Huntington’s and ALS. 

The methodology behind this concept is mind-blowing. This article is from 2017, but they are still fine-tuning the science today. Researchers at the Broad Institute have dubbed the technology: REPAIR (RNA Editing for Programmable A to I Replacement). How does it work? To start, one has to understand the basics of transcription and translation. Here is a crude summary: DNA is transcribed to RNA which is then translated into proteins. This technology does not cut DNA, but instead, it binds to RNA and effectively changes one nucleoside in the RNA helix. Therefore, when RNA goes to translate into a protein, it will encode for a properly functioning protein instead of a mutant protein involved in disease progression. Amazing.

Woof, that was a lot of science. Thank you reading if you made it to the end. To conclude: CRISPR Technology holds incredible potential for ALS and other rare diseases. Researchers just need to perfect it in a lab so that when it is applied to humans, no one comes out missing a limb :).

COVID-19 Impact on ALS Clinical Research

depict COVID-19

COVID-19 Impact on ALS Clinical Research as of July 23, 2020


Of the 95 ALS clinical trials that are currently active in the United States, only 5 were suspended due to the COVID-19 pandemic. Also, many trials that were starting their enrollment back in March, have paused their enrollment processes for the time being. Overall, the ALS research community has been incredibly adaptable. Telehealth, laboratory shift work, and continued perseverance define today’s ALS research atmosphere. The staff at MGH working on the HEALEY Platform Trial has reassured everyone that they are hard at word on the behind the scenes protocol establishment and site readiness preparations.

We are all reminded that ALS does not stop during a pandemic. It is still 100% fatal. Every 90 minutes someone dies from ALS and every 90 minutes someone is diagnosed with ALS.

How I AM ALS is Influencing Clinical Trials Design

medical environment

I. What is wrong with the current ALS clinical trial landscape?

I am going to answer this question with a vignette: An individual with ALS decides to enroll in a clinical trial that they found on ClinicalTrials.gov. They check to see if they meet the trial requirements and are pleased to learn that the trial includes their type of ALS. Not only that, but the trial site is located close to their home and only requires one visit every two weeks.

This story is invented. It rarely happens that way.

Information about enrolling in ALS clinical trials is not readily available to recently diagnosed ALS patients. The trial information that is available, is not easily comprehensible for those not in the clinical research field. Even if a patient knew what a clinical trial was and started looking on clinicaltrials.gov, they would spend months sifting through phases, mechanisms of action, enrollment criteria and maybe, just maybe, they would find a trial that does not exclude them. How can you be excluded from a trial? Below is the exclusion criteria for a ALS dietary supplement trial that started in 2017:

  • Forced vital capacity <40% of predicted
  • Dependence on mechanical ventilation for more than 12 hours per day
  • Exposure to any experimental agent within 30 days of entry or at any time during the trial
  • Women who are breastfeeding, who are pregnant or are planning to become pregnant
  • Women of childbearing potential not practicing a medically accepted form of contraception
  • Enrollment in another research study within 30 days of or during this trial
  • Mini-Mental State Exam (MMSE) score <20
  • Patients with symptomatic cardiac disease or hypercholesterolemia
  • Patients with myocardial infarction within 6 months of this trial
  • Renal dysfunction defined as BUN and creatinine >2xULN
  • Known mitochondrial disease
  • BMI<18.5
  • Prior use of a 4:1 ketogenic diet or Atkins diet within 1 month of this trial
  • Impaired liver function, defined as AST or ALT of 3xULN
  • Patients who have a pacemaker or other internal electronic medical device
  • Known allergy or hypersensitivity to milk or soy products.

Yes, if you are allergic to soy you are disqualified from this trial…you get the point.

Exclusion criteria is only one of the many hoops ALS patients must jump through before they find the trial that is right for them. The length of ALS trials usually prevents the majority of patients from being able to complete them due to their rapid disease progression. Not only that, but what if you spent 6 months in a clinical trial in which you were a part of a placebo group? At that point, you spend 6 months not receiving an active drug, and you are probably disqualified from joining other trials. Okay, what if you are receiving the active drug? There probably weren’t any biomarkers that tracked your disease progression, so you would never know if your disease stopped progressing due to the drug or if you just had a slow progressing form of the disease. The list goes on…

II. What is I Am ALS?

I AM ALS is a patient-led ALS organization that was co-founded by Brian Wallach and his wife, Sandra Abrevaya, in 2019. Brian was diagnosed with ALS when he was 37 years old. He has an incredible story that has been told on several platforms. This video is a does a great job of visualizing it. I AM ALS empowers and enables people living with ALS. They are inserting the patient voice into politics and drug development and making sh*t happen. What exactly are they doing? I AM ALS has 3 key focuses: Advocacy, Patient Services (Navigation Program) and Research. This blog post focuses on their advocacy work.

III. What is I AM ALS doing to fix ALS clinical trials?

I AM ALS is visiting drug developing companies and introducing their Patient Centric Trial Design (PaCTD). What is PaCTD? It is a five star rating system that demands the following (this list is not exhaustive):

  • Scientific justification for all exclusion criteria
  • Minimization or elimination of placebo controls (e.g shared placebo group)
  • Use of biomarkers
  • Open label extension available for all trial participants
  • Use of novel methods for data capture
  • Unblinded Independent Intern Efficacy Review Board” (IRB) to review trial results while in progress to identify subsets of patients who are benefitting
  • Significant consideration for access to Compassionate Use (e.g Expanded Access)

The companies’ trial designs are rated according to how well they address the requirements in the PACTD. It is important to note that this rating scale does not in any way comment on the efficacy of the ALS trial. It is simply a way for people living with ALS to select the trial that most humanely considers their trial participants.

The PACTD requirements have the potential to impose serious financial strain on drug developing companies. They also have implications in the IND-enabling studies which happen well before Phase I clinical trials. That is why the I AM ALS constituents are meeting with drug developers early in their development process.

This is a time of true clinical trial innovation. I AM ALS is making drug development companies uncomfortable, but in the best way. In a way that demands critical thinking and promotes improvement. Patient centricity is not the end to profitability.

8th Annual MOFF Golf Tournament Results

The 8th Annual MOFF Golf Tournament took place at Cypress Ridge Golf Course on Oct. 3, 2020. It was an incredible success! Thank you to all everyone who made the event possible: sponsors, golfers, volunteers, and silent auction participants. We could not have done it without each and every one of you.

Click to view tournament results

Interested in participating in the MOFF Golf Tournament next year? Make sure you are signed up for MOFF’s email communications here

Thank you to all our attendees for abiding by COVID-19 Safety Precautions!

  • Individual tee time format instead of a shotgun start.
  • 120 in-person golfer limit
  • Online silent auction
  • Guest speakers featured on the MOFF golf tournament event website.
  • CA mask guidelines and social distancing regulations were respected
  • Temperatures were taken
  • All golfer merchandise was handled with gloved hands
  • Hand washing and hand sanitizer usage was encouraged
  • Online awards ceremony
  • No Tapas Reception

ALSA Patient and Caregiver Advisory Committee (PCAC) Involvement

Natalie Fernandez recently became a member of the Patient and Caregiver Advisory Committee (PCAC) at the National Chapter of ALS Association (ALSA). The PCAC is co-chaired by John Russo and Ken Menkhaus who are both incredibly knowledgable and influential ALS advocates. If you are interested in learning more about their perspective, Ken has a blog called, Ken’s Caucus. The PCAC currently has 20 members and each member serves two year terms. The purpose of the committee is to provide informed guidance to ALSA and the ALS Focus Steering Committee. Responsibilities of the committee members include: Generating survey topics, reviewing survey questions, and ensuring patient and caregiver authenticity.

Natalie is honored to be a part of the PCAC. ALSA staff works hard to provide care and support for ALS families. They are also the largest funder of ALS research projects in the world, which is pretty incredible considering they are a nonprofit. While Natalie is aware of the tension that exists between ALSA and more grassroots ALS movements, she is excited for this opportunity to focus and inform initiatives that directly affect families living with ALS. In the words of Sandy Morris, “We are all fighting for the same thing. When we find that we are fighting each other, we are on the wrong path.” It through collaboration and focus on our shared goal that we are going to find a cure for ALS.