ALS Unfiltered is a series of expert-led presentations and candid conversations on ALS, current drug research, clinical trials and ALS resources. Hosted by experts and scientists from the ALS Therapy Development Institute, these sessions take place across different cities in the US. Unfiltered events have already taken place in San Francisco Bay Area, Seattle, and Raleigh this year. The fourth seminar of 2017 was held on August 23rd right here in the labs in Cambridge, MA. Over 20 attendees came to listen to a presentation by Robert Goldstein, Vice President at ALS TDI. The audience included people living with ALS, their friends and relatives, doctors and researchers, a science writer and staff from ALS TDI.
At ALS Unfiltered events, guests are encouraged to interact with the speaker and each other, asking questions or offering comments throughout the presentation. Sessions can differ depending on timing and attendees, but each one aims to cover topics such as genes associated with ALS, why it is so difficult to discover and develop effective treatments for ALS, what researchers at ALS TDI are doing right now to combat this disease, promising treatments in the pipeline from other biotech companies like Cytokinetics’s Tirasemtiv, Amylyx’s AMX00035 and Brainstorm’s NurOwn and finally, how is Precision Medicine impacting ALS research.
One topic which sparked a few questions was edaravone. Mitsubishi Tanabe Pharma America received FDA approval earlier this year to market the drug as Radicava in the USA and prescriptions started getting filled earlier this month. Participants voiced frustrations at many insurance providers not covering the drug right away. Some insurers may have felt the turnover time from approval to market was too short, perhaps needing more time to make a decision.
Other questions centered on ALS TDI’s current lead candidate, AT-1501. At this point, AT-1501 preclinical work is finishing up and we hope that, with enough funding, we can move AT-1501 into a phase 1 trial early next year. Participants also asked about Cytokinetic’s drug, Tirasemtiv, in particular, when it might be approved and available. There are a lot things to consider when answering this, first and foremost the results from the current pivotal phase three trial “VITALITY-ALS” that is likely to read out this December at the International Symposium on ALS/MND Research that ALS TDI is honored to be the local co-host of. If the trial reads out positively, the company would likely consider seeking marketing approval from the FDA and perhaps other regulators in other countries depending on the data. The timeline of preparing a new drug application and the review process varies greatly, but in all likelihood, it would take about a full year for a marketing decision to be made by the FDA following the trial reading out. However, the company, Cytokinetics, has a long and solid track record in the ALS community and should approval be sought and won, the timeline for availability will likely be quick, relatively speaking, in comparison to other drug launches. It’s important to also note that Cytokinetics is offering a unique open-label extension trial for those participants that completed the phase 3 study called FORTITUDE-ALS, in which all individuals are able to access tirasemtiv regardless of if they received placebo or not in the previous trial.
On a more general level about ALS, one attendee asked how one determines the date of disease onset. This came up, because onset of disease is frequently used as a criteria for eligibility in clinical trial enrollment, yet there is almost no way to precisely pinpoint an exact date of onset. A person with an ALS diagnosis may not notice subtle symptoms until months after they have begun, so it is a very subjective piece of data. It is best for a patient and their doctor to determine through a best estimation of when the patient began to experience symptoms.
Following the presentation, guests were welcomed to ask questions directly to Rob and other ALS TDI staff. Many took the opportunity to tour the lab for a first-hand experience of what the scientists at ALS TDI do. If you are in the Boston/Cambridge area, and would like to visit the ALS Therapy Development Institute to get your own up-close-and-personal look at the lab, just fill out this form to schedule a tour.
If you are interested in attending an ALS Unfiltered session there are three more scheduled for this year; Indianapolis on September 11th, Tampa on December 12th and Fort Lauderdale on December 13th. If you would like us to host an ALS Unfiltered in your area, please email info@als.net.