Wednesday January 29, 2020

Medicare to Cover for Some Blood Cancers Breakthrough Gene Therapy

Officials said Medicare would cover CAR-T cell therapies for certain types of lymphoma and leukemia

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Medicare, Blood Cancers, Gene Therapy
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 

Medicare, Blood Cancers, Gene Therapy
Expanding access to a promising but costly treatment, Medicare said Wednesday that it would cover for some blood cancers a breakthrough gene therapy. Pixabay

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.

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.

Special program

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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)

Next Story

Scientists Develop “Special Molecular Switch” for Gene Therapy

In a study, the researchers demonstrated the power of their new switching technique by incorporating it into a gene therapy that produces the hormone erythropoietin

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Gene
However, gene therapies have been viewed as inherently risky because once they are delivered to a patient's cells, they cannot be switched off or modulated. Pixabay

Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing.

The feat, published in the journal Nature Biotechnology, offers gene therapy designers what may be the first viable technique for adjusting the activity levels of their therapeutic genes.

The lack of such a basic safety feature has helped limit the development of gene therapy, which otherwise holds promise for addressing genetically based conditions.

“I think that our approach offers the only practical way at present to regulate the dose of a gene therapy in an animal or a human,” said lead researcher Michael Farzan from the Scripps Research Institute in Jupiter in Florida, US.

Gene therapies work by inserting copies of a therapeutic gene into the cells of a patient, if, for example, the patient was born without functional copies of the needed gene.

The strategy has long been seen as having enormous potential to cure diseases caused by defective genes. It could also enable the steady, long-term delivery to patients of therapeutic molecules that are impractical to deliver in pills or injections because they don’t survive for long in the body.

Gene
Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing. Pixabay

However, gene therapies have been viewed as inherently risky because once they are delivered to a patient’s cells, they cannot be switched off or modulated. As a result, only a handful of gene therapies have been approved to date.

In this study, the researchers demonstrated the power of their new switching technique by incorporating it into a gene therapy that produces the hormone erythropoietin, used as a treatment for anaemia.

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They showed that they could suppress expression of its gene to very low levels with a special embedded molecule, and could then increase the gene’s expression, over a wide dynamic range, using injected control molecules called morpholinos that the US Food and Drug Administration has found to be safe for other applications. (IANS)