How it works?
It is an attempt to use the natural immune system response of RNA interference (RNAi) to block the replication of HCV in the liver by regulating certain genes. Here is a video explaining RNAi for the laymen...
This is my best explanation of how this would work with hepatitis C...
When the HCV virus invades a liver cell it begins to make copies itself (that is the main function of all viruses) as quickly as it only lives for hours. For the virus to make copies of itself it uses RNA to copy and distribute itself. When the virus is in this part of its viral life-cycle its RNA is double-stranded and long. What this treatment will attempt to do is have an enzyme that is called a 'Dicer' chop up (gene-splicing) any double-stranded RNA it finds into small bits that a group of proteins then can then unwind, leaving only one strand which the proteins can then carry away. The proteins than will look for any other RNA that has the same code as the one it is carrying and then bind to it. The binding process then leads to partial or full blockage of the virus RNA protein production. This stops the virus from replicating and spreading to more liver cells. Without replication the virus will quickly die off preventing the liver damage that occurs each time the virus replicates. Undetectable viral load and no further liver damage.
This would be an additional approach to the current DAA approach of stopping viral replication by interfering with the nonstructural region codes for the polypeptides NS2, NS3, NS4A, NS4B, NS5A, and NS5B. For example Sovaldi is a NS5B polymerase inhibitor.
First received: July 8, 2013 Last updated: January 20, 2014 recruiting has started
Official Title: A Phase I,II Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of Single Doses of TT-034 for Subjects With Chronic Hepatitis C (CHC) Infection
Subjects must a history of chronic HCV infection defined as documented HCV genotype 1 infection for at least 6 months.
among other requirements
Baseline HCV RNA level of > 100,000 IU/mL and:No evidence of cirrhosis at Screening At least 3 months since prior therapy for HCV A willingness to enroll in a 5 year follow-up safety study
Many exclusions to limit HCV patients to those in the best overall heath.
Duke Clinical Research Institute Durham, NC Recruiting
UCSD Antiviral Research Center an Diego Ca Not yet recruiting
contact info at the above link.
Phase 1 can be risky but someone has to go first. Phase 2a risk is a little lower. If you are considering please become well informed about the risks, If offered the trial after consulting with your hepatitis doctor make your own decision.
So it will be some time before we will see results. If successful then more phase 2b and/or 3 studies with HCV patients with less ideal and more advanced Hep C and other conditions. Then other genotypes etc. It could be a couple of years or longer before possible FDA approval if it works.
Thank you for your explanation.
My guess is that anyone with moderate or severe fibrous, cirrhosis or elevated chance of progression from F0/F1 shouldn't consider waiting for a couple of years to see if this tx gets approved. They should try to get tx with these new now approved and/or recommended off label drugs depending on their doctor's advice.
If TT-034 or similar one shot(or pills) tx is successful with few serious side effects it may be the way to go in a couple of years. Even if it was priced at $10,000 a shot or ? tx it's much lower than Sovaldi Opinion Gilead is trying to get as much as it can for Sovaldi now because of future competition that will dramatically lower the price in the next year or two. Even now I bet their average price is much lower than $1,000 pp if you include discounts to major health organizations, copay assistance and free drug no ins low income. .
How it works:
This is a first-time use of a method of therapy designed to transfer anti-HCV genetic sequences into the hepatocytes of subjects infected with HCV. The anti-HCV sequences will be comprised of three different short hairpin RNAs (shRNA) that have the ability to directly cleave the RNA genome of HCV by a process known as RNA interference. The transfer of the anti-HCV sequences will be accomplished using a "vector" that was made from an adenovirus-associated virus (AAV) by removing the viral genes and replacing them with a non-replicating genetic sequence that produces three different shRNA that target three different regions within the HCV genes.
Works differently from any other tx's.
However, the shra "medicine" continues to be produced in the cytoplasm of liver cells by the cells own "machinery". It is one shot with long lasting viral fighting effects.
You are all so smart.... I don't have a clue what anyone of you have said.
Simply it's using Immunotherapy to kill the virus. Basically it's creating an environment in which your own immune system destroys the enemy (virus)
They are using it also in the treatment of cancer with promising results. No doubt within the next 5 years many diseases will be treated without drugs that kill both healthy cells and toxic cells such as chemotherapy. Immunotherapy boosts our own immune system to attack damaged cells only.
It's extremely exciting days ahead for the medical community, and those of us that have chronic diseases.
We will soon see the face of medicine change in a big way!