Category Archives: ALS Research Events

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

 
 

The 9th Annual Martha Olson-Fernandez Foundation Golf Tournament is approaching quickly!

At this point in time, the tournament is sold out but sponsorship and volunteer opportunities are still available!

The silent auction is set to open at the end of September- BID TO WIN!

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, 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 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.

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.

CLICK HERE TO VOLUNTEER 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. 

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 

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

2020 Virtual SLO ALS Walk

MOFF ALS Walk Team

Thank you to all those who helped us raise $1,825.00! Your generosity during this time of COVID-19 is what keeps the ALS patient care community going. 

In case you missed it, the MOFF team this year  walked for people living with ALS, the families of those who passed away from ALS, and we walked in memory of Katcho Achadjian.

Because ALS is fatal, our community is no stranger to loss and each year we invite more angels into our midst. This year, the MOFF team was dedicated to one of our SLO community leaders, Katcho Achadjian. He did not die from ALS, but his support of the ALS community was heartfelt and strong. We know he and Martha are getting into trouble wherever they may be 🙂 TO KATCHO! TO MARTHA! TO EACH OF US LIVING EACH DAY TO THE FULLEST. Stay strong.

Please view the MOFF Virtual ALS Walk Team video below. Check it out:

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

FLYER

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.

http://www.worldbrainmapping.org/Annual-Congress/

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.

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.