In 2016, Spinraza® became a game-changer for spinal muscular atrophy (SMA) patients. It was the first FDA-approved treatment for the neurodegenerative disease, which is the leading genetic cause of infant death.
One way to improve the effect of a drug is by increasing its dosage. But as with any drug, increasing the amount of Spinraza® also increases the risk of toxic side effects. The authors took a different approach. They discovered that pairing Spinraza® with valproic acid (VPA) may be an alternate way to improve its clinical effect without using more of the drug.
The senior author explains: “Sometimes you don’t want to use a ton of a drug. If you have a condition that allows you to use less 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. 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.
https://www.cell.com/cell/fulltext/S0092-8674(22)00529-3
Improving the therapeutic efficacy of spinal muscular atrophy drug!
- 574 views
- Added
Edited
Latest News
Personalized brain modeling…
By newseditor
Posted 29 Jan
Afternoon chemotherapy impr…
By newseditor
Posted 29 Jan
Propionic acid protects ner…
By newseditor
Posted 28 Jan
How cells prevent harmful e…
By newseditor
Posted 28 Jan
Structured cerebellar conne…
By newseditor
Posted 28 Jan
Other Top Stories
Brain cells that suppress cravings identified!
Read more
Dual and opposing role for a single protein in the brain
Read more
Receptor Protein in Brain Promotes Resilience to Stress
Read more
The molecular mechanisms behind addiction and relapse
Read more
AI neural network detects heart failure from single heartbeat
Read more
Protocols
Machine learning prediction…
By newseditor
Posted 09 Jan
Differentiating PC12 cells…
By newseditor
Posted 09 Jan
Ultrasensitive sensors reve…
By newseditor
Posted 05 Jan
In vitro-derived medium spi…
By newseditor
Posted 04 Jan
Molecular and spatial signa…
By newseditor
Posted 30 Dec
Publications
HIF-1a accumulation in resp…
By newseditor
Posted 29 Jan
Electrical signals in the E…
By newseditor
Posted 29 Jan
Parathyroid hormone recepto…
By newseditor
Posted 29 Jan
Plasma biomarker profiles i…
By newseditor
Posted 29 Jan
Chemotherapy delivery time…
By newseditor
Posted 29 Jan
Presentations
Hydrogels in Drug Delivery
By newseditor
Posted 12 Apr
Lipids
By newseditor
Posted 31 Dec
Cell biology of carbohydrat…
By newseditor
Posted 29 Nov
RNA interference (RNAi)
By newseditor
Posted 23 Oct
RNA structure and functions
By newseditor
Posted 19 Oct
Posters
ASCO-2020-HEALTH SERVICES R…
By newseditor
Posted 23 Mar
ASCO-2020-HEAD AND NECK CANCER
By newseditor
Posted 23 Mar
ASCO-2020-GENITOURINARY CAN…
By newseditor
Posted 23 Mar
ASCO-2020-GENITOURINARY CAN…
By newseditor
Posted 10 Mar
ASCO-2020-GYNECOLOGIC CANCER
By newseditor
Posted 10 Mar