Gene therapy.

[Cherry]

Hi, just read this, always looking for some good news.

Best wishes to all, Cherry.



Another breakthrough on the horizon -- one for ALS. People who get the devastating diagnosis are often told they only have a few years to live. Now, gene therapy could add years to their life.



"We hope to slow disease progression and, in our wildest dreams, we hope to dramatically to slow disease progression," added Don Cleveland, Ph.D., a neuroscientist at the University of California, San Diego.



Researchers have tested a molecular therapy in rats that turns off the bad gene in ALS. The drug is infused right into the spinal cord. It could be the first really effective treatment for the disease, and the first gene-silencing therapy used in people for a neurological disorder. The therapy could also be applied to diseases like Parkinson's and Alzheimer's.



"That will be quite different -- if we are successful -- quite different from therapies available," Dr. Cleveland said.



These two different solutions are helping researchers get one step closer to a cure. The diabetes and ALS trials in people will start around 2008. They've already proven effective in animals with virtually no side effects.



For more information:

org



ALS, or Lou Gehrig's Disease:
Neurosciences Department University of California San Diego ranks first NIH grant funding committed Excellence in Research, Teaching, Clinical Care

[John1]

Thanks for posting this Cherry. I followed your link and looked around the site but couldn't find anything on this. Can you be more specific as to where it is? I notice that trials were expected to start in 2008. I wonder if there is anything more up to date.

John

[halfin]

I wonder if they might be referring to "antisense" therapy. That creates a molecule which is sort of the "anti" or opposite version of a particular gene, and neutralizes it. They are going to do trials soon for familial PALS who have the SOD1 gene mutation. I think it was going to require a pump to direct the medicine directly into the spinal cord. Here is an article on antisense therapy for ALS:

Antisense Oligonucleotide Therapy for SOD1 Related Familial ALS :: WUSM Department of Neurology

[GlenBrittle]

I looked at the website and could not find Don Cleveland in the faculty. So , I searched his name and found he has his own lab at the UCSD.


In the News - Don Cleveland

[Cherry]

I found it here John. WSIL TV • Breakthrough for Diabetes, ALS

Regards, Cherry.

[John1]

Thanks Cherry.

-John