A Promising New Drug Combo Could Improve Spinal Muscular Atrophy Treatment


Spinraza® changed the game for people with spinal muscular atrophy (SMA) in 2016. It was the first medication for the neurodegenerative condition that is the leading genetic cause of infant mortality to get FDA approval. Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer and colleagues conceptualized and developed the medication.

A Promising New Drug Combo Could Improve Spinal Muscular Atrophy Treatment, “Sometimes you don’t want to use a ton of a drug. If you have a condition that allows you to use less of the drug, then you may have fewer toxicities. So the idea is to combine these two drugs to get maximal effects.” People with SMA don’t have enough of a protein called SMN. Spinraza is a type of molecule called an antisense oligonucleotide (ASO) that helps cells make more SMN protein from a gene called SMN2. New Drug Combo, The team discovered that there were roadblocks on the SMN2 gene when using Spinraza. This slowed down the cellular machine producing SMN protein. The drug VPA helps remove the roadblocks, allowing Spinraza to further increase the SMN protein output. When mice with SMA were treated with both VPA and a Spinraza-like ASO used for research, the mice survived longer and had improved muscle function. Over 11,000 SMA patients have been treated with Spinraza in more than 50 countries. Krainer’s latest research shows that there’s always room for improvement. He hopes the team’s findings will help optimize the efficacy of Spinraza treatments. He also hopes their work will help researchers who are trying to develop therapies for other neurodegenerative diseases.

Source: This news is originally published by scitechdaily



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