Last month's webinar was a follow up of a list that Rob presented on in February. As research progresses, so do the lists, which is why you’ll notice that a number of topics have moved up or down the list while some have been added and others removed. 2017 has been a busy year for ALS research news with the approval of a new drug and a number of exciting potential treatments in late phase clinical trials.

Below you will find a short summary of the topics that we feel are most important in ALS research. Some trials that didn’t make the Top 10 but deserve a close eye are from Biogen, Denali, Q Therapeutics, and trials investigating biomarkers for ALS. For a full list of clinical trials happening around the globe right now in ALS, you can visit our clinical trials database.

  1. Radicava (edaravone): This list is ordered with topics that are closer to making a difference in the lives of people with ALS appearing at the top which is why we began our list with Radicava. Radicava was approved by the FDA in May of this year and is now available to patients with a prescription from their doctor. Hundreds of people with ALS are reported to have started taking Radicava in the US since it became available in the middle of August. This is the first drug that has been approved in the US in over 20 years.
  1. Tirasemtiv: Cytokinetics recently completed a phase 3 study measuring the efficacy of Tirasemtiv in people living with ALS. The company launched a follow-on open label extension trial (VITALITY-ALS) that allows all those that completed the phase 3 trial (called BENEFIT-ALS) to access tirasemtiv, and hundreds of people with ALS are currently taking advantage of that unique opportunity. We should know results at the end of this year from the pivotal phase 3 trial of tirasemtiv and have clearer understanding of the potential path to patient from there. Building on their ALS treatment pipeline, Cytokinetics is currently seeking people with ALS to enroll in a phase 2 trial on a similar, next generation drug known as CK-107.
  1. Precision Medicine: The ALS Therapy Development Institute’s Precision Medicine Program(PMP) has now enrolled over 450 people in the study. Participants in our program receive access to their data through an online portal that they can use to track disease progression through ALSFRS-R, accelerometers and voice recordings while in trial or while using treatments such as Radicava. Scientists at ALS TDI use participant data to try to identify subtypes of the disease ultimately making clinical trials more effective and efficient.
  1. NurOwn®: Brainstorm’s phase 3 trial is now enrolling at MGH and UCI with four other sites to open soon. The study lasts nearly a full year, including an initial observation period, bone marrow aspiration, three doses of treatment, and a final observation period. This study aims to gain greater understanding of the efficacy and safety of multiple doses of NurOwn. Biomarker research will also be conducted throughout the study.
  1. CNS1-NPC-GDNF: This is a novel stem cell research study. It differs from Brainstorm’s NurOwn treatment in two main ways, (1) this drug has never been used in people before this trial and (2) they are using stem cells that are not from the patients themselves but from donor samples. Previous research has shown that it is possible in rats, mice and monkeys. Investigators have been honest that this study will take several years to fully enroll and determine results regarding safety of this unique combination approach of gene therapy and cell based treatments. It is currently enrolling in southern California.
  1. AMX0035: AMX0035 is a combination of two compounds, sodium phenylbutyrate (NaPB) and tauroursodeoxycholic acid (TUDCA). We are still waiting to see preclinical data but a phase 2 study is currently enrolling at UMass (Worcester) and MGH (Boston) with a total of 25 sites due to enroll in the coming months. Those who are taking edaravone (Radicava) are eligible to enroll in this trial.
  1. Lunasin: This is a unique and interesting approach to clinical trials. All patient data is self-reported online in this open label study. The trial enrolled rapidly, as there was no exclusion criteria and drug was provided to all free of charge. Overall, no statistically significant effect on disease progression was found. There could be an issue with patients self-reporting ALSFRS-R, however previous papers from PLM and others have suggested that self-reporting is almost as good as clinical exams.
  1. Masitinib: AB Science will launch a confirmatory phase 3 in US following a phase 2 and 3 trial in Europe. The new trial design is yet to be made public and the timeline is unknown. We will post information about trial when it’s available and report on results as they are reported in Boston this December.
  1. AT-1501: This drug needs to get into people. It was specifically built by scientists at ALS TDI for those living with ALS and has promising and comprehensive preclinical data. It is currently in GMP manufacturing to ensure we have enough of the drug and will then be stabilized as a powder for easier delivery in trials to patients. FDA discussions on trial design are underway with a clinical trial expected to kick off in the first half of 2018.
  1. ALS/MND Symposium in Boston: Hosted by the ALS Hope Foundation, ALS TDI and MNDA (UK), the 28th International Symposium on ALS/MND Research will take place in Boston December 8-10. Many of the promising drugs currently in clinical trial will be discussed such as Cytokinetics’ Tirasemtiv and Brainstorm’s Nurown along with many more presentations and poster sessions. The event will also feature a unique “Ask the Experts” lunch session exclusively for people living with ALS/MND and their caregivers. The topic of our next webinar will be a preview of this event where we will talk about the program and take questions from participants. You can register for that webinar here.

If you would like more information on any of the above topics, be sure to watch the full webinar at https://www.als.net/als-webinars/archive/09262017/ or email us at letstalk@als.net. To join in the conversation about ALS research with the ALS community, check out our forum at www.als.net/forum .