Author Archives: Brian Anderson

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.

On November 15 2015, Martha Olson-Fernandez Foundation officers, Larry and Natalie Fernandez, attended the ALS Leadership Summit in Boston. The summit, hosted by ALS Therapy Development Institute (TDI), offered a wealth of knowledge on recent advancements in ALS research projects. The summit and featured speakers from Biogen, ALS TDI, The Forbes Norris ALS/MDA Clinic in San Francisco, and the Harvard School of Public Health. The attendees included investment companies, top researchers in the NMD/ALS field, pharmaceutical company representatives, as well as ALS patients and their families. The energy in the conference room at the Hyatt was charged by powerful minds, and a frustrated search for answers to such a complex disease pathology. The element of time was what everyone felt short on.

The takeaway from the conference was that ALS is not an untreatable disease, but an underfunded one. Recent research has surfaced many new findings about the disease and the role of the immune system in the final stages of the disease, but due to the nuanced subjectivity and spectral nature of ALS, researchers are still racing time to find treatments.

Overall, the summit centered around several key topics:
1. The importance of utilizing biomarkers to track pathogenesis and drug efficacy in ALS patients.
2. The methodology behind initiating, and executing preclinical research projects at ALS TDI
3. The structure and function of the Precision Care Program at ALS TDI
4. The discovery of the role of the immune system in the later stages of ALS and how this immobilizing stage of ALS can potentially be slowed and prevented with AT-1501.
5. Biogen’s discussion of new ways to visualize how effective intrathecal drug delivery is at getting the drug molecules passed the blood brain barrier and into the Central Nervous System (CNS).

The ALS TDI Leadership Summit hosted an awards ceremony during which it recognized several courageous people dedicated to fighting ALS. The 2015 Leadership Award winners are listed below:
Award: Stephen Heywood Patients Today Award. Recipient: US Navy LT. Commander (RET.) Matt Bellina (Holland, PA)
Award: Stephen Milne Adventurous Spirit Award. Recipient: Sarah Coglianese (San Fransisco, CA)
Award: Mary Lou Krauseneck Courage & Love Award. Recipient: Bobby Forster (Newport, RI)
Award: Fran Delaney Challenge & Respect Award. Recipient: Beth Hebron (Maplewood, NJ)
Award: Stephen Mile Adventurous Spirit Award. Recipient: Maureen Ramirez and Team Godfather Charitable Foundation
Award: Stephen Heywood Patients Today Award. Recipient: Jay Smith (Austin, TX)

Thank you to ALS TDI for the informative and well executed leadership summit.