Next Generation Drug Delays Onset of ALS Symptoms

A second generation drug may prove effective in treating an inherited form of amyotrophic lateral sclerosis (ALS), according to a study by the National Institute of Neurological Disorders and Stroke, a part of the National Institutes of Health.
 
The findings of the study, published in the Journal of Clinical Investigation, found that newer versions of a gene therapy, called antisense oligonucleotide, delayed the signs of ALS in rodents by silencing the gene, superoxide dismutase 1 (SOD1). 
 
Prior research found that older versions of an antisense oligonucleotide were able to turn off a form of ALS caused by mutations in SOD1, according to the authors of the current study. The treatment was found to attach to versions of the gene encoded in messenger RNA (mRNA), mark the genes for disposal, and stop the production of the SOD1 protein.
 
The researchers used a newer version of an antisense oligonucleotide previously tested in a phase 1 trial to treat rodents genetically modified to carry normal or disease-mutant versions of human SOD1. They found that the infusion of newer versions of the drug was more efficient in decreasing normal, human SOD1 mRNA levels in rats and mice.
 
The drug was also found to extend survival in rats that were genetically modified to carry a disease-causing mutation of SOD1 compared with an older version of an antisense oligonucleotide, according to the study.
 
The new formulation of the antisense oligonucleotide was also found to delay the age that mice carrying the mutant SOD1 gene began to have difficulty balancing on a rotating rod. The researchers also found that the drug apparently prevents muscle weakness and the loss of nerve and muscle connection.
 
Based on these findings and the belief that the next generation drug could help treat muscle activation problems in patients with ALS, the antisense oligonucleotide will be evaluated in a phase 1 clinical trial in patients with the disease.


Stay up to date on the latest news in specialty pharmacy by getting Specialty Pharmacy Times in your mailbox or inbox for free!

Click here to sign up for free for the bi-monthly Specialty Pharmacy Times print journal delivered to your address.

Click here to sign up for our email newsletters delivered every Monday, Wednesday, and Friday, in addition to breaking news alerts.

Click here to follow us on Facebook. 

Click here to follow us on Twitter. 

Click here to join our LinkedIn group. 


Related Articles

The use of digital technology and wireless communication systems has become more common in health care and has enabled patients to communicate important information about their vital signs, specific symptoms, and adherence to health care providers in multiple locations. Many patients with multiple sclerosis (MS) have expressed interest in the use of technology for their own self-management, including use of apps that help provide social support and self-management of their disease. Pharmacists need to understand the role of technology for patients with MS, including the use of smart auto-injectors as well as how innovative technology can be applied to improve health care decisions, improve adherence, and enhance engagement to improve patient outcomes. This program focuses on digital and mobile technology systems that are being utilized in patients with MS and explains how data may be applied for specific patient case- and population-based decisions.
Compared with interferon beta-1a, ozanimod reduced less cortical grey matter volume in patients with relapsing forms of multiple sclerosis.
An investigational therapy for amyotrophic lateral sclerosis has shown promise in slowing the progression of the disease in patients with a genetic form of the disease.
Company Profile >
Industry Guide >
Market News >
Peer Exchange >
Conferences >
Subscribe >
Specialty Times Resources
About Us
Advertise
Careers
Contact Us
Terms & Conditions
Privacy
MJH Associates >
Pharmacy Times
OTCGuide
American Journal of Managed Care
Cure
MD Magazine
ONCLive
Targeted Oncology
Physicians' Education Resource
Pharmacy & Healthcare Communications, LLC
2 Clarke Drive
Suite 100
Cranbury, NJ 08512
P: 609-716-7777
F: 609-716-4747

Copyright Specialty Pharmacy Times 2006-2018
Pharmacy & Healthcare Communications, LLC. All Rights Reserved.
 

$vacMongoViewPlus$