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Potential for Genetically Targeted Treatment in ALS


Medical experts discuss genetically targeted treatments for patients diagnosed with ALS.

Jennifer Roggenbuck, MS, LGC: Understanding the underlying genetic etiology of ALS [amyotrophic lateral sclerosis] can enable us to develop new treatments, whether those are specific gene-targeted treatments or treatments that address the metabolic pathways or whatever biological process is impacted by that gene. We see a lot of diversity in terms of the functional pathways that ALS genes can come into play. The genetic forms of ALS are the only times where we can put our finger on the exact cause. That’s powerful because it tells us the first step in the multidisease process, and it gives us the potential to intervene. That gives us a guidepost in our research to say, “Let’s look at this gene, this protein, this pathway to see what we can do to intervene.” The ways we might intervene could be impactful in people who don’t have a genetic form of ALS. We don’t have an FDA-approved therapy. The FDA has 1 under consideration, and we’re expecting a decision about that in April 2023. In the meantime, and with the other ALS genes, it’s powerful to know about these genetic variances because it gives a target for our research and an opportunity to intervene with respect to that gene, protein, or metabolic pathway.

Laynie Dratch, ScM, CGC: Although there aren’t any FDA-approved therapies for ALS, we’re hopeful that they’ll come soon. It is incredibly important that individuals are able to access genetic testing so that we’ll know if they’ll be eligible for these therapies when they’re approved. I fear that we’ll rejoice over the approval of a therapy, but we’ll have a large population of patients with ALS who’ve never been offered genetic testing. There’s going to be a delay in accessing that care, that approved therapy, because people need to meet with the genetic counselor and neurologist, have the genetic testing process started, wait a month or 2 to get results, and then initiate treatment. As we said before, the earlier you can start treatment, the better. It’s important that we start this process of offering genetic testing to all patients with ALS, so that if they do have a genetic etiology identified, they can know that as soon as this therapy is approved, it’s something they might want to consider. The same is true for clinical trials. We don’t want to wait until a trial becomes available to offer testing for the gene associated with the trial. We want to make sure we have a population that’s ready for the trials or the treatments that might come their way if they want to access them.

Transcript edited for clarity.

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