Northwestern researchers discover new protein to prevent & reverse damage in UMNs
The problem with having a rare disease is that almost no money goes towards research. With PLS it’s even more complicated by the fact that ALS – also a rare disease – tends to be the MND most likely to get research money. Of course, that is with good reason as ALS is a horrific disease that I would like to see cured as soon as possible. Unfortunately, even though it would be handy to have people with other UMN/LMN diseases grouped into ALS studies, rarely does that happen. I get it, but it is a bit demoralizing.
A group of researchers at Northwestern have done some research on UMNs leading to some interesting discoveries of late. One, they released a paper, Better understanding the neurobiology of primary lateral sclerosis which has a lot of great info about metabolism and mitochondria etc. The thing that really struck me though is that they posit that PLS is just ALS with some genetic or environmental thing preventing it from developing. I don’t know why that is really my important takeaway but it’s a question I get asked all the time.
Two, they released a second paper which has received a lot of hype (for good reason). Here is the Northwestern presser ALS neuron damage reversed with new compound. NU-9, a protein, seems to be well tolerated in mice models and will move forward with animal toxicology studies with human trials being about 1 – 2 years out. Still, Dr. Silverman – the man who engineered the compound – has started a drug company and hopefully there will be some movement soon.
The news is a welcome relief for many sufferers of MNDs and we are all super excited to see how quickly NU-9 can to human trials. As I joked on facebook, I will fly to Chicago every darn week if it means I can be included in them. Still, one of the researchers Dr. Hande Ozdinler cautions that the word “reverses” is a bit excessive. In their study the diseased neurons were still alive when they saw an improvement in them. So in that case the statement is correct. However, if the neurons are dead there is no evidence that this compound will bring them back to life. A better statement may be that it reverses the damage in diseased neurons, not dead ones. The mice studies also took place in a short period of time so there is no telling (yet!) how the compound works on subjects who have had an MND for a longer period of time. Also, probably people read the headlines and see “reverses” and they think that the compound “reverses” the disease itself. Unfortunately, there is no evidence that it cures the diseases themselves. It may very well be that this protein will be a drug you have to be on the rest of your life much like diabetics are on insulin.
Still, this is exciting news from the research community and I am looking forward to the further studies on NU-9. I am also curious if the “Right to Try” legislation will push the studies on this compound through a quicker process.
Here are two videos, the first one has Dr. Ozdinler discussing the research & the second is more of a snapshot social media promo video: