Gene Therapy

Live bacteria Virus used for treatment of diseases

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Do you know that there are some diseases which can only be treated by injecting live bacteria and virus at the point of disease.

Bladder Cancer treatment with live atenuated microorganisms Bacillus Calmette-Guerin (BCG).  Gene Therapy assited by live virus vector, Novel Drug Delivery System.

One of such disease is bladder cancer which is treated by injecting live bacterial inoculum in to bladder. Bacillus Calmette-Guerin (BCG) is a atenuated (reduced virulence) strain of Mycobacterium bovis.

After injection of live bacterial solution ( Solution containing bacteria) with the help of catheter, it is left for hours to show it’s effect. The effect is very painful, bladder gets inflammation from bacterial invaders, swelling and even bleeding also is found.
Patients get fever, chilling and weeknes.

The benificial out come of the challaging the bladder tissue with live bacterial solution of BCG, is to enhance the local epithelial cells to produce immunity against cancerous cells present in bladder. It happens, after the live bacterial solution injection the bladder tissue start reactions to produce immunity against the bacteria. And also against cancerous cells.

After the treatment it is found that bladder cancer cells wanish in some cases, and do not propagate to other body tissues. Spreading to muscular and other tissues is the peculiar prognosis of cancer. Cancer cells propagate in other organs and damage them.

BCG is used as vaccine against Tuberculosis, every new born is given BCG vaccine to protect against Tuberculosis. Since it is live bacteria it swells and form infection at the site of injection. But the BCG strain used in the vaccine is attenuated or weekend so that it doesn’t casue any harmful effects.

Bladder Cancer Treatment With BCG and Gene Therapy
Bladder Cancer Treatment With BCG and Gene Therapy

Bladder Cancer Treatment with Virus Vector assited Gene Therapy.

Cancer of Bladder has got new treatment options in United States of America, US FDA have approved a new drug again a live virus vector carries a gene when injected in to bladder, the gene intercalate in to bladder epithelial cells and fixes Interferons producing gene inside the epithelium. And now the epithelium tissue starts producing Interferons which destroy cancer cells.

Detail update is available here Gene therapy for Bladder Cancer .

This Gene Therapy is approved for patients who do not respond to BCG treatment and the cancer cells have not moved to other organs and muscle tissue (non metastatic). The virus vector do not multiply in side the host cells and thus is not harmful.

New Drug A Live Virus Carrying Gene, A Gene Therapy approved by USFDA for treatment of Bladder Cancer that is non metastatic and don’t respond to other treatments like BCG

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Novel drug Adstiladrin that delivers genes through a live virus vector, approved by USFDA for treatment of non-muscle invasive  Bacillus Calmette-Guérin (BCG)-unresponsive bladder cancer.

This month the US FDA has approved a novel drug, a gene therapy that makes use of a live virus (vector) which carries the gene and delivers it to the point where it is required to provide treatment. This virus carrying the gene is rendered harmless, virus  does not multiply in human cells, it is used to carry gene that is required to be delivered at the point in the human body,  that is bladder.

This drug Adstiladrin is recommended to patients who are not responding to treatment with Bacillus Calmette-Guérin inoculation and their cancer is no metastatic non muscle invasive type (NMIBC).

The drug consists of human gene that code for interferon, and starts biosynthesis of interferon is delivered through this virus vector gene delivery system Nadofaragene firadenovec (also known as rAd-IFNa/Syn3)

Patients affected with Bladder Cancer that is no metastatic and it’s not responding to Bacillus Calmette-Guérin which is one of best treatment of Bladder Cancer, in such a situation the drug Adstiladrin is found to provide relief; it is manufactured by Ferring Pharmaceuticals A/S.

The approved gene therapy drug is a live virus carrying a gene that codes for human interferons which fight with cancerous cells and help to prevent spread of cancerous cells to other body tissue and muscles.

Other treatment for bladder cancer involves injecting Bacillus Calmette-Guérin live bacterias, which stimulate the local tissue to trigger its own defense mechanism against cancerous cells thereby eliminating the cancerous cells. In some patients this also does not work therefore such patients now can be provided with the gene therapy drug Adstiladrin which when injected into bladder will deliver genes that produce interferons into local epithelial cells and induce production of interferons that will eliminate cancerous cells.

New drug for Bladder Cancer Treatment
Bladder irrigation with Drug

 

USFDA approves first Gene Therapy Drug for Hemophilia B

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USFDA approved First Gene Therapy Drug for Hemophilia B

What is Hemophilia B : Patients affected with Hemophilia B suffer uncontrolled bleeding from any part of body from inside or from any damaged or wounded body part, some times without any bruise or any cut bleeding occurs from internal organs or oral cavity.

Symptoms of Hemophilia B Patients affected with Hemophilia B suffer bleeding in joints, and soft tissue, bleeding in stomach and frequent bleeding from nose, bleeding through gums, person get bleeding even with slight scratch on skin or any body part. Surgery is one point where there is risk of bleeding. Bloody urine blood in urine are the symptoms of Hemophilia B

Treatment of Hemophilia B, what is treatment available for Hemophilia B

Hemophilia B Patients are given injections or intravenous dosage of factor ix, which is only treatment available till yesterday before US FDA gave approval for a novel gene therapy drug. The factor ix intravenous dosage required is frequent just like patients of diabetes needs insulin, in same manner patients affected with Hemophilia B are required to give intravenous factor IX dosage to maintain the body level of factor IX so that they don’t suffer from bleeding.

Hemophilia
Hemophilia B

What is responsible for Hemophilia B Hemophilia Bis a disease condition occurring due to faulty gene which do not produce factor IX which is responsible for blood clotting process of formation of semisolid blood clot, in absence of factor ix, uncontrolled bleeding from damaged tissue occurs, since the clot formation do not occur in Hemophilia B patients, sometimes the bleeding is spontaneous, from inner organs, intestine or oral cavity, etc., it can occur at any site.

Hemophilia B is X chromosome linked disease, which is inherited mostly predominantly to males, some female too suffers from Hemophilia B, patients do not have a gene that produce factor IX.

First of its kind New drug gene therapy approved by US FDA to treat Hemophilia B adult patients.

It’s a first of its kind of drug approved by US FDA, it is a Gene Therapy, name of the Gene therapy product is Hemgenix it is approved by US FDA on 22-11-2022 for treatment of adult Hemophilia B patients.

Hemgenix is given in a single dose IV infusion, this gene therapy product consists of a virus vector carrying a gene for factor IX , which is not virulent to human, but when given in intravenous dosage it is able to deliver the gene to targeted delivery site in patient’s liver where gene is expressed for production of Factor IX, blood level of Factor IX is increased and patients risk of bleeding is eliminated.

Statement of US FDA Commissioner
Statement of US FDA Commissioner

Clinical evaluation of safety and efficacy of Hemgenix is done by its innovator company  CSL (ASX: CSL) https://www.csl.com/

Center for Disease Control US have published example of real people living with Hemophilia.

https://www.cdc.gov/ncbddd/hemophilia/stories.ht

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