Recently, a medication called tofersen or Qalsody was approved as a treatment for people with SOD1 ALS and on July 25, 2023, our team administered the first commercial dose of toferson. Make sure to read “We Need Your Help to Find the Cure” below for more information and to see how “at risk” family members can help with research. 

There is still time to register – and sponsor the Terance Mathis Celebrity Golf Tournament, benefiting Emory ALS Center. This two-day event is taking place at Harrah’s Casino in Cherokee, NC and at the Sequoyah National Golf Club. Information and links are below in this newsletter.

We have a new resource for our Emory ALS Center families. Love Not Lost is a nonprofit organization dedicated to ensuring everyone feels loved and supported through loss. You will want to make sure to read the article about this wonderful organization. 

We want to remind you also that the complete Emory ALS Center Resource Guide is available on our website. All sections have great information for living with ALS; the Patient Resources section is particularly full of many places of support for patients, caregivers, and families. 

Enjoy this issue; Celebrate Life and Imagine a Cure; and know that your Emory ALS Center team is working hard every day for you and with you. 

We Need Your Help to Find the Cure

How can “at risk” family members help?

Although ALS often does not run in families, approximately 10% of ALS cases are familial due to a genetic cause. Scientists and researchers can learn critical information about the underlying biology of ALS and potential treatments from asymptomatic relatives of people with genetic ALS, or “at risk” family members. There are several exciting projects that have the potential to move science forward to find and answers to ALS that have eluded us for so many years.

ATLAS: The ATLAS study is seeking healthy family members who are related by blood to someone with ALS due to a mutation in the superoxide dismutase (SOD1) gene. Recently, a medication called tofersen or Qalsody was approved as a treatment for people with SOD1 ALS. Now, the company Biogen is studying healthy people who have been tested and carry a mutation in the SOD1 gene. If one qualifies and wants to enter the study, they are followed by having blood drawn monthly, in their home, looking for an indicator that shows ALS may be developing. If and when we see this early warning sign, treatment with Qalsody or a placebo will be started. By comparing the group that gets active drug to the group that gets placebo, we hope to find out if the drug delays the onset of the disease, or even prevents it. We also could learn more about what happens to the body before ALS symptoms start, and this knowledge could help us develop diagnostic tools and treatments for the broader ALS community. Contact Meraida Polak to participate.

PiCALS: The PiCALS study is seeking healthy people who are related by blood to someone with ALS or frontotemporal dementia due to a mutation in the C9orf72 gene. Qualified participants will come to Emory for a clinical assessment, have blood drawn, and have a spinal tap. We do not have an effective treatment for C9ALS, but studies like this one could help scientists find one. When we do, the participants in PiCALS will have contributed information to the search for a drug that can delay or prevent C9ALS. Contact Jen Taylor to participate.

If you want to help but do not qualify for the ATLAS or PiCALS studies, then you can participate in Emory’s Clinical Research in ALS Study (CRiALS). CRiALS participants are asked for a one-time donation of blood and information about your health history and family history. Moving forward, if you give permission, we will let you know if there are additional opportunities for research. Contact Jane Bordeau to participate.

These three projects can help lay the groundwork for critically important treatment studies in the future.