Research Update - Drug Repurposing - May 2026

Unravel Drug Repurposing

While doing the drug screening, it was discovered that there’s two groups of patients and they respond to completely different medications. However, it’s not SPATA5 vs. SPATA5L1.

This is important because it suggests that SPATA-related disorders may not be “one-size-fits-all” when it comes to treatment approaches. Different subgroups of patients may ultimately need different therapies or drug strategies based on how their biology responds.

In Progress: Testing top drug candidates on each subgroup. The larger subgroup has already gone through two rounds of testing with a third upcoming. The smaller subgroup will begin testing soon.

Up Next: Determine what the differentiating factor is so we can determine what group a patient would fall into for treatment. This is VERY important as each child will need a tailored treatment. This is going to be a challenge, but we are working diligently with Unravel to figure this out.

Once all screenings are done they will provide us with a report where we can then release information to families about what they've found.

How will we take this information into our kids? It's very important that we work with Unravel to come up with a plan to treat out children. Depending on the outcomes, we may be able to request off-label prescriptions. However, some drugs may require additional testing before treating our children.