This last year has seen incredible progress in the fight to create a world without ALS. The FDA approval of Relyvrio (AMX0035) in 2022 and Qalsody (tofersen) last month are significant steps in the effort to make ALS a livable disease. As we celebrate the success of these long-term investments, we continue to fund the next generation of possible treatments, as well as the researchers that are searching for them.

In March 2022, after several months of multiple doctor visits and many tests, Jacob Harper was diagnosed with P525L FUS ALS—a nano-rare version of ALS only found in people under the age of 25. Jacob is on the Jacifusen clinical trial trying to slow progression, as he shares his testimony with people from all over the world.

The FDA announced it has granted accelerated approval to tofersen, a treatment for people living with mutations of the SOD1 gene. This is a significant victory for the ALS community and our efforts to make ALS livable for everyone, everywhere, until we can cure it.  

While knowledge is power in many circumstances, knowing you have lost several members of your family to familial ALS, and understanding you may also carry the mutated gene, can be a heavy burden to live with. With technology continuously evolving, new potential treatments in the ALS research pipeline through clinical trials and the ability to get answers through a genetic test, there are more factors to consider today than ever before. Peter Hackett shares his family's ALS story and what led him to the decision of taking the genetic test.

The FDA advisory committee vote on tofersen Wednesday was an exciting and critical moment in the ALS community’s efforts to find new treatments and cures. The committee voted unanimously to recommend accelerated approval of the new treatment.

Larry Falivena, member of The ALS Association’s Board of Trustees who is living with ALS, shares his unique experience with the disease and his perspective on genetic testing.

With the emergence of gene-targeted therapies, knowing whether your ALS has an underlying genetic cause is becoming more important. Research has shown that about two-thirds of people with familial ALS and about 10% of people with sporadic ALS (no family history) have a mutation (or change) in at least one of the more than 40 genes that have been linked to the disease.

For people living with ALS, the enjoyment and escape video games may have once brought is far too often another thing the disease takes from them. As muscles weaken and fine motor functions decrease, handling video game controllers and keeping up with fast-paced game play can cause frustration and cause people to give up on gaming all together.

Dr. Anuradhika Puri is a postdoctoral research associate at Washington University in St. Louis. We talked with her to learn more about her ALS research, as well as her interests outside the lab.

The ALS Association has awarded more than $700,000 to support five promising early career scientists through its Milton Safenowitz Postdoctoral Fellowship Program. This program encourages a pipeline of ALS researchers and innovative ideas that can lead to better ALS treatments and care.

We spoke with Pam Knott, vice president of data and technology at The ALS Association, to learn more about how the Association is harnessing the power and potential of big data to speed up the process of empowering people to live longer lives, to access care, to bring new treatments to market, and to reduce the harmful impact associated with the disease.

We talked with Dr. Stephen Johnson, postdoctoral fellow from Massachusetts General Hospital, to learn more about his research focused on ways to accelerate discovery of efficacious therapeutics through identification and validation of novel outcome measures using readily available technology.

The ALS Association spent over $2 million helping fund the development and clinical trial of AMX0035. When the results of that trial showed it was safe and effective in treating ALS, the ALS Association led an advocacy campaign to push the FDA to approve the drug. After two years of advocacy, the FDA finally approved AMX0035. 

We talked with Dr. John Kalambogias, postdoctoral fellow from Columbia University, to learn more about his research focused on dysfunction and degeneration of corticospinal tract neurons in ALS mouse models.

We discussed the incredible strides being made in the science of preventing ALS with Dr. Stephen Goutman, Director of the Pranger ALS Clinic and Associate Professor of Neurology at the University of Michigan.

We spoke with Dr. Melinda Kavanaugh, clinical social worker and associate professor at the University of Wisconsin-Milwaukee, to understand more about young caregivers and the potential harms caused by the lack of quality of sleep they receive and what can we do about it.

We talked with Dr. Devesh Pant, postdoctoral fellow from Emory University, to learn more about his research focused on revealing the underlying molecular mechanisms in ALS caused by the SPTLC1 mutations.

We talked with Dr. Ahmad Al Khleifat, recent winner of the distinguished ENCALS Young Investigator Award and postdoctoral fellow from King’s College London, to learn more about his research focused on disease gene identification through next generation sequencing, coupled with advanced data analysis to deliver diagnostic tools for complex disease genetics.

The work to make ALS a livable disease and continuing to search for a cure rests in no small part on a robust drug discovery pipeline. That is why increasing interest in ALS research is critical. 

Expanded Access, or “compassionate use” as it is often referred, allows patients with a terminal diagnosis early access to new therapeutics that show promise – even if the patient is not involved in the ongoing clinical trial – or if the medication has not yet been approved by the FDA.