Interesting post from the No More Excuses facebook page:
Michelle Lorenz 8 hrs
HUGE NEWS about #NurOwn & gene UNC13A
Abstract published today on MDA website discusses MORE Biomarker data that will be presented by Dr. Merit Cudkowicz at the MDA Conference on March 16th. https://mdaconference.org/NODE/1672 Brainstorm analyzed the genetics of 124 people in their study. They found a connection with people who had the A/C or C/C variant of UNC13A. 53% of the NurOwn responders were found to have one or two copies of this SNP in UNC13A This conclusion met the statistical significance threshold (0.016). There may be other genetic connections, but as with most ALS trials, the sample size wasn't large enough to reach statistical significance for that analysis. Brainstorm's Abstract stated: "Our results suggest that NurOwn treatment may influence disease progression in ALS patients who possess this risk allele and provides a basis for further genetic characterization in clinical trials." This finding is very important to a potential BLA for Accelerated Approval of NurOwn. Why?
Just last week, two different researchers published studies about the importance of TDP43 mislocalization and how it impact splicing errors in UNC13A and other genes as well. In discussing these studies published in NATURE, the NIH said:
Genetic changes in UNC13A "Absolutely fundamental" to disease process "Critical factor" in ALS "POTENTIAL THERAPEUTIC TARGET" in ALS
IMPORTANT: UNC13A does not CAUSE ALS. It impacts the progression of the disease. It may not have been checked if you had a genome panel done for genes that cause ALS.
Importantly, TDP43 mislocalization may impact ALZ, CTE too Look forward to Dr. Cudkowicz' discussion of these findings in the NurOwn trial & what the proposed clinical mechanism could explain how NurOwn may be impacting TDP-43 & UNC13A.
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