While 2020 left the ALS research community adjusting to unprecedented circumstances due to the COVID-19 pandemic, 2021 is showing some early promise as an exciting year in the search for effective treatments for amyotrophic lateral sclerosis (ALS). Here are some of the most important research findings and advancements to look forward to as we move into the new year, selected by the ALS Therapy Development Institute’s (ALS TDI) Chief Scientific Officer Fernando Viera, M.D.
Emerging Consensus about COVID-19 Vaccines and People with ALS:
The potential safety and efficacy of the various COVID-19 vaccines becoming available this year for people living with ALS is perhaps the most pressing concern currently facing this community. As of the publishing date, we know that the Pfizer/BioNTech and Moderna vaccines currently approved in the US have been overwhelmingly well-tolerated among those who have received it, although there have been some rare cases of adverse allergic reactions.
Currently these vaccines are mainly available to groups like frontline healthcare workers and nursing home residents in the early phases of distribution, although rules vary from state to state and country to country and the situation is evolving rapidly. As these vaccines become more widely available to the general public and, especially, people with comorbidities like ALS and other neurodegenerative diseases, we are certain to continue to learn more. The most important thing for people with ALS is to stay in touch with their doctors and clinical teams for the latest news and advice about the COVID-19 vaccine.
Telemedicine for ALS Clinical Care:
Adapting to new ways of engaging in the various activities of daily life in a remote setting has been one of the biggest trends of this past year– everything from work to school to our social lives has moved online to some extent. Medical care has been no exception, with many healthcare providers moving to telemedicine appointments for services that did not require direct patient contact.
In 2021, it will be intriguing to see how much of these innovations produce long-term changes for ALS clinical care. While there are some aspects of care – like some drug infusions or physical examinations – that simply cannot be done remotely, new telemedicine infrastructure and technology presents an exciting opportunity to minimize the risk of exposure for people living with ALS for the duration of the pandemic. These new innovations could also alleviate some of the burden of getting to the clinic for those with limited mobility even when it’s over – especially for those who do not live close to their care providers or even clinical trial sites.
For those interested in telemedicine for ALS, ALS TDI’s Precision Medicine Program is currently accepting new remote participants for 2021.
Results from the Tofersen clinical trial:
Tofersen, also known as BIIB067, is an antisense oligonucleotide (ASO) treatment for SOD1-associated ALS in development by Biogen and Ionis Pharmaceuticals. It works by introducing an artificial piece of DNA capable of binding to SOD1 mRNA and stopping the production of the SOD1 protein. The treatment was tolerated in a phase 1 trial, and a phase 3 study is expected to conclude in July of 2021. The results from this trial may provide further insight into the promise of ASO therapies for familial ALS.
Results from Arimoclomol Clinical Trial:
Arimoclomol, developed by Orphazyme, is a small molecule agonist of HSF1a, a master regulator of the heat shock response. A common feature observed in brain and spinal cord of people with ALS is evidence that misfolded proteins aggregate, or clump together. Arimoclomol is intended to clear these protein clumps by increasing the production of heat shock proteins which help the cell to repair or eliminated misfolded proteins in cells. A phase 2B/3 trial is scheduled to finish in January of 2021.
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