The next twelve months are going to be exciting in ALS research. The approval decision from the FDA pending, at least two pivotal clinical trial results will read out, and several long anticipated studies will start to enroll or achieve full enrollment. Beyond that, significant advancements in communication innovations are underway across the world while those in the U.S. await the appointment of a new commissioner to the FDA. This is a summary of a webinar recording on February 9, 2017 outlining the top ten things that people should be on the lookout for this year in ALS. That recording is available online by clicking here.

Could We Be Soon Living in a World with Multiple Treatments for ALS?

Over the next 12 months, three outcomes could shape the future of ALS research. In June, the FDA (the regulator of drug marketing in the USA) is scheduled to rule whether or not edaravone (MT Pharma America) can be marketed as a treatment for ALS. Edaravone received approval in Japan in 2015 following a Phase 3 clinical trial in that country. Should marketing approval be granted by the FDA, it would be the first such newly minted “ALS Treatment” in in the U.S. in 30 years. 

There are two pivotal phase 3 clinical trials scheduled to “Read out” in 2017 as well, and depending on those announcements, there may be two more potential treatments for ALS going to regulators for marketing approval soon thereafter. Results from the phase 3 study of masitinib (AB Science) are scheduled to read out sometime in April or May of 2017 following final review of data from an EU based trial.  Cytokinetics is expected to announce the results of their phase 3 study “VITALITY-ALS” on tirasemtiv sometime in the fall. In most cases, patients in trials are no longer allowed to continue taking the experimental medication when the trial completes. Cytokinetics opened an open-label extension arm so that those in VITALITY-ALS allowing those who would like to continue to take tirasemtiv to do so. 

Should everything go as the investigators behind these medications hope, we could be living in a world where there are multiple treatments available to people with ALS. In the webinar we discuss in detail the proposed mechanisms of action and preclinical science behind these medications, and the potential impact they were suggested to cause on disease course according to earlier published clinical trial results.  

A Rich Pipeline of Diverse Clinical Trials in ALS

ALS is a complex, heterogeneous disorder against which patients will need multiple different therapeutic approaches. It is highly likely that the biggest impact in the battle against ALS will come in the form of a cocktail of several different treatments, each tackling different aspects of the biology behind the destruction of the central nervous system that we call ALS. 

Thankfully, there is a larger and a more target diverse pipeline of therapeutics today than ever before. These include a much anticipated Phase 2 clinical trial on NP001 (Neuraltus), expected to be fully enrolled by the end of 2017 with results at the end of 2018. Brainstorm Cell Therapeutics’ NurOwn program continues to advance back into clinical trial in the U.S., with a planned enrollment of a 150 person pivotal clinical trial to open by the end of the year.  NP001 targets immune system activation whereas NurOwn aims to provide neuroprotection by replacing dead or dying support cells inside the spinal cord. 

There are other exciting projects in the pipeline including an important gene therapy trial targeting SOD1 mediated ALS by Biogen & Ionis, a first in ALS patient trial of Copper ATSM taking place in Australia.  In the webinar we discuss in detail the proposed mechanisms of action and preclinical science behind these medications. We also  provide information on AT-1501, a compound that the ALS Therapy Development Institute calls the most exciting one they have worked on yet.  

Staying in Communication 

Often, people with ALS cite the loss of their ability to speak as the scariest part of the disease. To date, communication devices available have ranged from the simple letter boards or robotic sounding type-to-speech technologies to complete eye tracking technologies which, according to many users, don’t work well outside of very controlled environments. 

Today, there are several advancements occurring to bring new communication strategies forward for people with disorders like ALS. One of these is called message banking. One of the major differences between message banking and voice banking is that in message banking the user recorded the phrases that they commonly say, in their normal cadence, rather than recording individual words as they would in normal voice banking. This approach, being led in the ALS field by John Costello, Ph.D. Director of Boston Children’s Hospital’s Augmentative Communication Program, enables a user to “speak” in a voice closer to their own later on in their disease progression.

A person with ALS still needs to be able to control a computer or other device on their own and with ease. Today, the options are limited to mostly eye gaze technologies. However, Pison Technology, co-founded by Dexter Ang whose mother passed from ALS, has developed a device which is easily worn by a patient and leverages the residual signals coming from the patient’s muscles. This allows them to control the communication device, or other devices connected to it, using BlueTooth technology. Yet another group of researchers centered at the Donders Institute in Belgium have developed a highly sensitive wearable “helmet” that translates the brain activity (generally speaking) of the person, into text on a screen. Utilizing something called “NoiseTags” the user can control technology in their environment such as remotes, doors, etc. Neither of these highly innovative products are available on the market yet, but they are expected to move closer to the market over the next year, which is why they made the top 10 list now.

ALS TDI to co-host Biggest ALS/MND Meeting of the Year

This recap and webinar attempt to predict the top 10 things a person ought to be on the lookout for in ALS in 2017.  We will find out what happened will all the items on this list and more in December of this year. People with ALS, caregivers of people with ALS, neurologists, nurses, allied health professionals, drug companies, medical device companies, and nonprofit leaders from all over the world will join together in Boston for the biggest week in ALS research all year long.  Listed below are the meetings scheduled. Anyone can register (for a fee set by the organizers, not by the hosts!) and attend.

December 6 & 7: General Meeting of the International Alliance

  • Annual two day meeting of the leadership of 30-40 nonprofit organizations from dozens of different countries sharing best practices, local challenges, and ideas to increase ALS awareness. 

December 8: Allied Health Professionals Forum

  • A full day program designed with the health professional in mind. Topics range from technology and therapy to palliative care and other models of care. 

December 9, 10, 11: International Symposium on ALS/MND Research

  • Three days with dozens of talks, posters and opportunities to interact one-on-one with scientists working on ALS from around the globe.

The ALS Therapy Development Institute (ALS TDI) is proud to co-host this week long series of meetings in Boston with the ALS Hope Foundation at Temple University. The General Meeting and Allied Professionals Forum are organized by the International Alliance of ALS/MND Organizations, whereas the International Symposium on ALS/MND Research is organized by the MND Association.