It is often said that the best science appears when one is looking for something. . . else, entirely. This is one of those times.
Novartis had been developing a small molecule it called Branaplam, which was a survival of motor neuron-2 (SMN2) RNA splicing modulator -- it hoped, for the treatment of spinal muscular atrophy. Researchers at Novartis discovered that Branaplam lowered mutant Huntington (mHTT) protein levels in both mouse models and in blood samples from the spinal muscular atrophy trials, over several years.
In a bit of pure luck, since the trial on SMA was failing, the team started to pursue Huntington's. Here's the bit, from PharmaLive, tonight:
. . .In a paper, published March 3 in Nature, Sivasankaran et al. reported the results of these studies, where they examined fibroblasts (skin cells) derived from HD patients. “We could see a clear lowering of both the Huntington mRNA as well as the mutant Huntington protein in response to the molecule,” he said.
Branaplam, “modulates splicing, which is the process by which a single messenger RNA can give rise to multiple protein isoforms by piecing together separate pieces of RNA called exons,” he explained. “In the case of the Huntington gene, it modulates splicing so as to include an exon that marks the RNA to be degraded. This, in turn, leads to the lowering of the Huntington gene as well as protein expression. . . .”
Onward. . . for yet another sublime ride around a helio-orbit, tomorrow. . . smile. Be excellent to one another. . . .
नमस्ते
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