Monday December 16, 2019

Gene Therapy may Help Treat Chronic Kidney Disease

Chronic kidney disease is a slowly progressive disease, so that is an advantage

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DNA
Heavy drinking can change your DNA: Study

Scientists have found that administering gene therapy may reverse damage in kidney cells, suggesting a potential treatment for chronic kidney disease characterised by gradual loss of its functions.

The research showed that whether adeno-associated virus (AAV) — a relative of the virus that causes the common cold — could deliver genetic material to damaged cells in the kidneys.

They explained that diabetes, hypertension and other conditions cause chronic kidney disease, which occurs when damaged kidneys cannot effectively filter waste and excess fluids from the body.

“Chronic kidney disease is an enormous and growing problem. Unfortunately, over the years, we have not developed more effective drugs for the condition, and this reality is leading us to explore gene therapy,” said Benjamin D. Humphreys from the Division of Nephrology at Washington University in the US.

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Gene (Representational image). IANS

In the study, published in Journal of the American Society of Nephrology, the team evaluated six AAV viruses, both natural and synthetic, in mice and in stem-cell-derived human kidney organoids.

A synthetic virus, Anc80, created by the researchers proved successful in reaching two types of cells that contribute to chronic kidney disease by secreting proteins that gum up the organ and cause irreversible damage.

The results showed that the genetic material carried by Anc80 was transferred successfully to the targeted kidney cells and the same virus was also used in gene therapy strategies to treat mice with kidney scarring.

Also Read: Parkinson’s Identified Gene to Combat Alzheimer’s

“The interesting thing about the adeno-associated viruses is that they persist in the body for many months, potentially giving a therapeutic gene a chance to do its work,” Humphreys explained.

“Chronic kidney disease is a slowly progressive disease, so that is an advantage. After many more years of research, we could envision that patients would need injections maybe twice a year as opposed to every week, like with chemo,” Humphreys added. (IANS)

Next Story

Medicare Uses Breakthrough Gene Therapy to Cover Some Blood Cancers

Medicare covers more than 60 million seniors and people with disabilities

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blood cancer
FILE - In this Feb. 14, 2019, file photo, Centers for Medicare & Medicaid Services (CMS) Administrator Seema Verma speaks during a news conference in Washington. VOA

Expanding access to a promising but costly treatment, Medicare said Wednesday that it would cover for some blood cancers a breakthrough gene therapy that revs up a patient’s own immune cells to destroy malignancies.

Officials said Medicare would cover CAR-T cell therapies for certain types of lymphoma and leukemia, uses that are approved by the Food and Drug Administration. The cost can run to hundreds of thousands of dollars per patient, not counting hospitalization and other expenses. Medicare Administrator Seema Verma said the decision would provide consistent and predictable access nationwide, opening up treatment options for some patients “who had nowhere else to turn.”

Turbocharge, reprogram cells 

CAR-T uses gene therapy techniques to turbocharge the patient’s own immune system cells, reprogramming them to harbor a “receptor” that zeroes in on cancer, and then to grow hundreds of millions of copies. The revved-up immune cells are returned to the patient’s bloodstream and can continue to fight cancer for months or years.

blood cancers
Medicare covers more than 60 million seniors and people with disabilities. Pixabay

Although side effects can be severe, studies have shown the treatment to be highly effective against certain types of cancers. Researchers are working to add more types to that list. Medicare has been weighing the decision for months. The program often sets the tone for private insurance as well.

ALSO READ: After Effects of Articles 370: Pakistan Suspends Trade with India, Expels Indian High Commissioner

Special program

In its announcement, Medicare said it would cover CAR-T when the treatment is provided in institutions that are enrolled with the FDA in a special program to promote safety. It will also cover the treatment for other uses, if they are recommended by agency-approved medical research literature.

CAR-T uses a different strategy than other gene-therapy techniques. Instead of trying to fix disease-causing genes, it focuses on the patient’s immune system, specifically the T cells that battle foreign substances in the body. The problem with cancer is that malignant cells can often evade detection by the patient’s T cells. CAR-T helps the body’s own T cells do a better job of spotting tumors. Medicare covers more than 60 million seniors and people with disabilities. (VOA)