CRISPR Makes Another Advance in ALS
Could the CRISPR-cas9 gene-editing system be the key to a cure for amyotrophic lateral sclerosis (ALS)? A new paper written by a group of Stanford graduate students and just published in the journal Nature Genetics suggests a very hopeful maybe. Here’s the background.
Back in 2011, a paper was published delineating the cause of ALS, at least in a group of families with a proven autosomal dominant genetic feature. The cause in these cases is a repeating series of six nucleotides in a gene called C9orf72. It’s sort of like a broken record in the gene, or a hiccup. It’s called a hexanucleotide repeat, a section of DNA code that cycles over and over between 30 and up to thousands of times in affected patients. Scientists believe that when these repeats are read and translated into proteins, they cause the proteins to stack up and accumulate, leading to damage where the accumulation occurs. In the case of ALS, these deformed proteins accumulate in motor neurons.
ALS by the way is not the only disease caused (or at least that can be caused) by the accumulation of abnormal proteins in areas of the body where they should not be. Huntington’s Disease is another. Creutzfeldt Jakob Disease, the human form of mad cow, another. Amyloidosis, the accumulation of beta amyloid protein in the body a third. Alzheimer’s Disease has the same beta amyloid protein accumulating into deposits in the brain. Alzheimer’s also has an autosomal dominant genetic form that strikes in late middle age just like Huntington’s.
Some of these diseases are always genetic like Huntington’s, while others can be genetic like Alzheimer’s and ALS, while still others like CJD are caused by ingested prions, or deformed proteins that cause other healthy proteins to become deformed and start to stack as well.
Back to Stanford, this group of young scientists just succeeded in isolating the repeating nucleotides that cause ALS and cutting them away using CRISPR-cas9. Tests were done on human cell cultures to see if cutting out the repeats was able to protect motor neurons, and it was found that using a specific editing bracket, they indeed did protect them in human cell cultures.
Now, cell cultures are a long way from a cure, and the cure for ALS has not been discovered by any means yet. This is just a first step, but an encouraging one that goes to the root of the disease and not just symptomatic treatment.
What are the implications for investors? As I covered recently, CRISPR stocks have been on a tear, and new developments and improvements with the gene editing technology have been coming out pretty quickly. CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT) and Intellia Therapeutics (NASDAQ:NTLA) all exploded onto the scene in 2016 and all have market caps over the $1 billion mark. All three of them went public almost immediately after the discovery of the technique, spending almost no time in private equity. The founders of these companies were all directly involved in the initial discoveries and wanted to strike while the iron was hot.
It is unclear if any of the CRISPR companies currently public can stake a claim through patents on any treatment developed for ALS using the CRISPR-cas9 method, and as of now none of the three are directly involved in this new Stanford research on ALS. The point here is not necessarily that any of the three have a cure for ALS in their back pocket, but rather that paradigm-shifting developments in this space are unlikely to slow down.
Any human trials using CRISPR in any one of these indications, University or industry sponsored, are likely to generate enormous attention that will bleed into these stocks regardless of whether any of them are directly involved. It is likely though that at some point, one or more of them will be involved by the time any of this new research gets past initial clinical trials. Universities do not have the money to sponsor large scale clinical trials.
The larger point here is that this might be the early stages of a paradigm shift in the drug discovery process. How so? The discovery of monoclonal antibodies (mabs) for example gave the biotech industry an enormous boost and directed start-up after start-up to dig for gold in mabs. AbbVie’s (NYSE:ABBV) Humira, for example, a monoclonal antibody, is far and away the best-selling drug ever, and how many new drugs ending in the suffix mab do we hear of in the drug development world these days?
Just as mabs were a paradigm shift in medicine that led to hundreds of billions of dollars in sales, the next paradigm shift in drug development could come from CRISPR. As more research comes to light, it looks increasingly likely that this could be the case. As biotech investors realize this, these stocks will garner more and more attention from investors in the sector.
Disclosure: No positions.
Published at Mon, 12 Mar 2018 14:39:39 +0000