Evaluating the Potential of Intrathecal Gene Therapy

EP: 1.Describing SMA Progression Over Time

EP: 2.Unmet Needs of Older Patients Living With SMA

EP: 3.Examining the Importance of Early Treatment for SMA

EP: 4.Key Functional Milestones Affected by Disease Progression

EP: 5.The Importance of Supportive Care for Patients Living With SMA

EP: 6.Introduction to Therapies Used to Treat SMA

EP: 7.Splicing Modifiers and Gene Therapy for SMA

EP: 8.Key Clinical Considerations for the Management of SMA

EP: 9.Assessing Different Factors When Selecting a Treatment

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EP: 10.Evaluating the Potential of Intrathecal Gene Therapy

Clinical Brief: Emerging Therapies for Older Patients With SMA

Main Discussion Topics

• Development of intrathecal gene therapy for patients older than 2 years
• Current clinical trial data on intrathecal gene therapy
• Safety considerations and adverse events with gene therapy
• Considerations for sequencing or combining different treatment approaches

Key Points for Physicians

• Intrathecal gene therapy aims to deliver treatment directly to spinal motor neurons using lower doses.
• Clinical trials (STRONG, STEER, STRENGTH) show modest efficacy signals with intrathecal delivery.
• Safety concerns include potential dorsal root ganglia toxicity and hepatic inflammation.
• Patient preferences may favor continuing current effective therapies rather than switching to newer options.

Notable Insights

The panel emphasized the need for cautious interpretation of early clinical trial data, noting that modest functional improvements must be weighed against potential serious adverse events.

Clinical Significance

While intrathecal gene therapy shows promise for expanding treatment options to older patients with SMA, physicians should approach emerging therapies with measured expectations, carefully weighing demonstrated efficacy against safety concerns.