Researchers at Oregon State University announced today that they have essentially stopped the progression of amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, for nearly two years in one type of mouse model used to study the disease - allowing the mice to approach their normal lifespan.
The findings, scientists indicate, are some of the most compelling ever produced in the search for a therapy for ALS, a debilitating and fatal disease, and were just published in Neurobiology of Disease.
"We are shocked at how well this treatment can stop the progression of ALS," said Joseph Beckman, lead author on this study, a distinguished professor of biochemistry and biophysics in the College of Science at Oregon State University, and principal investigator and holder of the Burgess and Elizabeth Jamieson Chair in OSU's Linus Pauling Institute.
In decades of work, no treatment has been discovered for ALS that can do anything but prolong human survival less than a month. The mouse model used in this study is one that scientists believe may more closely resemble the human reaction to this treatment, which consists of a compound called copper-ATSM.
Copper delivery to the CNS by CuATSM effectively treats motor neuron disease in SODG93A mice co-expressing the copper-chaperone-for-SOD (DOI: 10.1016/j.nbd.2016.01.020)
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First step toward CRISPR cure of Lou Gehrig's disease
University of California, Berkeley scientists have for the first time used CRISPR-Cas9 gene editing to disable a defective gene that causes amyotrophic lateral sclerosis, or Lou Gehrig's disease, in mice, extending their lifespan by 25 percent.
[...] The mice were genetically engineered to express a mutated human gene that in humans causes about 20 percent of all inherited forms of the disease and about 2 percent of all cases of ALS worldwide. Though the genetic cause is not known for all cases of ALS, all are accompanied by the premature death of motor neurons in the brain stem and spinal cord. The neurons allow the brain to control muscles, so loss of this connection means loss of muscle control.
[...] The UC Berkeley research team used a virus that Schaffer's team engineered to seek out only motor neurons in the spinal cord and deliver a gene encoding the Cas9 protein into the nucleus. There, the gene was translated into the Cas9 protein, a molecular scissors that cut and disabled the mutant gene responsible for ALS.
In this case, Cas9 was programmed to knock out the mutated gene SOD1 (superoxide dismutase 1). The onset or start of the disease was delayed by almost five weeks, and mice treated by the gene therapy lived about a month longer than the typical four-month lifespan of mice with ALS. Healthy mice can live a couple of years.
Lou Gehrig's disease = amyotrophic lateral sclerosis (ALS).
In vivo genome editing improves motor function and extends survival in a mouse model of ALS (open, DOI: 10.1126/sciadv.aar3952) (DX)
Previously: New Therapy Halts Progression of Lou Gehrig's Disease in Mice
(Score: 0) by Anonymous Coward on Saturday January 30 2016, @03:44PM
The next shoe to drop will be a bumper crop of fly-by-night drug clinics catering to professional athletes with CTE. There will be guys willing to pay to be guinea pigs.
(Score: 2, Insightful) by mechanicjay on Saturday January 30 2016, @04:45PM
About 20 years too late for my Grandpa, but I truly hope this leads to a real treatment. Horrifying disease.
My VMS box beat up your Windows box.
(Score: 2) by bzipitidoo on Saturday January 30 2016, @05:30PM
I know how you feel. My mother has Alzheimer's. She could still drive and shop for groceries through 2009. We took the car keys away in March 2010. Too many suspicious paint marks on the bumper that she had no idea how they got there. Wasn't able to sign her name by the end of '12, and in Oct '13, she forgot her potty training. She can still talk a little, though fragmentary and incoherent, read very slowly, and walk and use a spoon. Still very able physically, which, sadly, works against us, as she is quite capable and prone to wandering away and getting lost, or digging around in cupboards, the fridge, and the trash, spilling detergent and food on the floor, dropping and breaking glass, and dragging a blanket through the mess then tracking it all over the house. Have to keep an eye on her every waking moment to stop that sort of thing. And it goes on and on, there's no break, no vacation from caring for her, and every sign it could continue for another 3 or 5 years or longer. We have caretakers in the home part of the day. Could use more, but that is costly.
There doesn't seem to be any hope for real progress on Alzheimer's in time to help my mother. Merely preventing or arresting it is now no longer enough to help her, it would have to be a cure so powerful that it can reverse it even in cases that have progressed to her level of severity.
(Score: 2, Interesting) by mechanicjay on Saturday January 30 2016, @06:20PM
Yeah, my Grandpa was diagnosed with ALS in the late 80's. A vibrant presence, highly intelligent, captain of industry (CEO of a company you've definitely fucking heard of). He finally passed around 2003 or so. I say finally, because we all watched his body die bit by bit over the course of 15 years, trapping his mind inside an increasingly useless shell. At the end, he was confined to bed, controlling a Stephen Hawking type computer voice, by blinking his eyelid, because it's the last piece of his body he was able to actually move, even that was a very slow, painfully deliberate movement.
In contrast, I watched my other grandfather loose his mind to some kind of dimensia, eventually becoming almost a vegetable with a strong healthy (for a 90 yr old) body.
I'm honestly not sure which was worse to watch.
My VMS box beat up your Windows box.
(Score: -1, Troll) by Anonymous Coward on Saturday January 30 2016, @05:20PM
Has anyone collected a bunch of these breathless press releases from 10-20 years ago and done a "where are they now"? That would be interesting.
(Score: 2, Troll) by wonkey_monkey on Saturday January 30 2016, @05:21PM
...of finding a mouse named Lou Gehrig?
systemd is Roko's Basilisk
(Score: 2) by Gravis on Saturday January 30 2016, @05:31PM
I'm all for treating disease but I fear that simply enabling people to live longer could encourage the proliferation of it by via offspring. Since it's a genetic condition, the only way to really beat ALS is to prevent it from being passed onto offspring. It's encoded in your DNA, therefore it's a feature, not a bug.
(Score: 0) by Anonymous Coward on Saturday January 30 2016, @07:17PM
Quick find something negative to say about good news!fuck off
(Score: 0) by Anonymous Coward on Saturday January 30 2016, @07:40PM
so your honest opinion is that if someone has a genetic disease, they and their offspring automatically cannot make any useful contribution to society?
humanity lives in groups because there is an evolutionary advantage to caring for the sick and weak.
or maybe you're trying to say that rather than saving the genetically diseased, we should first take care of the starving, of which there are many more?
since these researcher get funding, my guess is that society has already decided it cares more about these diseases than about the starving.
I don't know if it makes sense for us to try and label this as "good" or "bad", but you can try to do it anyway; I doubt it will make a difference to anyone.
(Score: 0) by Anonymous Coward on Sunday January 31 2016, @07:17PM
You can test for this disease. So a possible solution is to not have any children with the disease.
There are already more than seven billion people on this overpopulated planet ( http://www.overshootday.org/ [overshootday.org] ). Do contribute usefully to society while you're alive but if your genes suck or you're not going to be a good parent, please don't have children.
Are there statistics showing that those with the ALS genes are tend to be much better than average in some things? No? Then what's the big deal if you don't have kids?
If yes, well they might cure those stats soon if they aren't careful... Plus I'm sure there's more than one way to get better at stuff than having the ALS tendency.
(Score: 3, Insightful) by HiThere on Saturday January 30 2016, @08:34PM
Thus the advantage of CRISPR (etc.) on human gene-line cells. Of course, first they need to understand exactly what they are changing.
The thing about ALS is it *doesn't* diminish reproductive success. It usually doesn't take effect until the main reproductive years are past. The only way to eliminate is that isn't horrendously inhumane is gene-line surgery. The same is true of Altzheimer's. And probably several other less well known diseases. And if you're doing gene-line surgery anyway, why not get rid of hemophilia, beta-thalassanemia, etc.
Javascript is what you use to allow unknown third parties to run software you have no idea about on your computer.
(Score: 2) by Gravis on Saturday January 30 2016, @10:38PM
The only way to eliminate is that isn't horrendously inhumane is gene-line surgery.
you should be more careful when using the words like "only" because it's quite common for problems to have alternative solutions. simply put, genocide and restrictions on human reproduction could swiftly put many genetic disorders to an end.
(Score: 2) by HiThere on Sunday January 31 2016, @07:13PM
So you don't consider genocide horrendous?
Javascript is what you use to allow unknown third parties to run software you have no idea about on your computer.
(Score: 2) by Gravis on Sunday January 31 2016, @11:12PM
of course i do but when talking in absolutes, you must consider all options.
(Score: 2) by takyon on Saturday January 30 2016, @09:07PM
Human evolution no longer involves natural selection. Now even the weakest of individuals can survive and procreate, instead of being killed or lost in childhood.
There will only be 1-3 generations to go before gene editing is in widespread practice. There's not enough time for your fears to matter.
[SIG] 10/28/2017: Soylent Upgrade v14 [soylentnews.org]
(Score: 2) by Gravis on Saturday January 30 2016, @10:44PM
Human evolution no longer involves natural selection.
if that were true then anyone would be willing to mate with another person. the criteria for natural selection in humans has changed drastically.
There will only be 1-3 generations to go before gene editing is in widespread practice.
i hope you are right.
There's not enough time for your fears to matter.
assuming that nothing interrupts or impedes the progression of our sciences.
(Score: 1) by tftp on Sunday January 31 2016, @07:28AM
if that were true then anyone would be willing to mate with another person
You mean, it is not so? I'd think that discovery of the pill facilitated exactly that behavior. GP is right; natural selection and humans had parted their ways at least a century ago. You can even say that social selection has far greater impact on the end result.
(Score: 2) by Gravis on Sunday January 31 2016, @12:23PM
if that were true then anyone would be willing to mate with another person
You mean, it is not so? I'd think that discovery of the pill facilitated exactly that behavior.
i should allow me to clarify because by "another person" i meant "any other person".