Blood Cancer Therapy Successful in 75% of Trial Patients

New data from an ongoing Phase 1/2 clinical trial has revealed an experimental immunotherapy led to successful response rates in 73% of patients suffering from multiple myeloma, a deadly form of blood cancer. Based on this promising data, an application to the US Food and Drug Administration (FDA) has been filed to bring the drug to market.

 

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How to Destroy Cancer by Training Immune Cells

While it sounds like the stuff of science fiction, a cancer treatment in which a patient’s own cells are engineered to hunt down and wipe out their disease — and then linger in the body to stop the cancer returning — is helping to save patients’ lives. The results of the treatment, known as CAR T-cell therapy, have been astonishingPatients who had exhausted all other options and been told they had just months to live have gone into remission. Others have even been cured by the one-off dose.

In trials, all signs of cancer disappeared in more than 80 per cent of patients with acute lymphoblastic leukaemia — the most common cancer in children — after receiving CAR T-cellsSuccess stories include Emily Whitehead, now 16, who in 2012 became the first child in the world to take part in a CAR T-cell trial. Emily, who only had weeks to live when her leukaemia became resistant to conventional therapies, had the revolutionary treatment at the Children’s Hospital of Philadelphia in the U.S. when she was six years old. She is still cancer-free today.

First given in the NHS two years ago to children with a rare blood cancer, CAR T-cell therapy is now used to treat four forms of the disease — and more could follow. It is also being trialled in a number of other blood cancers, such as myeloma, non-Hodgkin lymphoma and chronic lymphocytic leukaemia, and could be available soon for these patients. Early research suggests it can also tackle solid tumours. A new study showed that a new generation of CAR T-cells with more advanced genetic engineering could help treat mesothelioma, ovarian cancer and the deadly brain cancer glioblastoma in mice, without side-effects, reported the journal Science Translational Medicine.

The current uses of CAR T-cell therapy are ‘just the tip of the iceberg’, says Dr Andrew Furness, a consultant medical oncologist at the Royal Marsden Hospital in London. ‘Doctors and scientists are working tirelessly to expand its reach to many more patients.’

CAR T-cell therapy (or chimeric antigen receptor T-cell therapy) is a form of immunotherapy, using the power of a patient’s immune system to fight the disease.

Source: https://www.dailymail.co.uk/

How Gene-edited White Blood Cells Are Helping Fight Cancer

For the first time in the United States, a gene editing tool has been used to treat advanced cancer in three patients and showed promising early results in a pilot phase 1 clinical trial. So far the treatment appears safe, and more results are expected soon. To develop a safer and more effective treatment for cancer patients, scientists from the University of Pennsylvania, the Parker Institute for Cancer Immunotherapy in San Francisco and Tmunity Therapeutics, a biotech company in Philadelphia, developed an advanced version of immunotherapy. In this treatment, a patient’s own immune cells are removed from the body, trained to recognize specific cancer cells and then finally injected back into the patient where they multiply and destroy them.

Unlike chemotherapy or radiation therapy, which directly kills cancer cells, immunotherapy activates the body’s own immune system to do the work. This team used a gene editing tool called CRISPR to alter immune cells, turning them into trained soldiers to locate and kill cancer cells. By using this technique, the team hoped to develop a more effective form of immunotherapy with minimal side effects.

Better CRISPR-based gene editors for the diagnosis and treatment of cancer and other disorders, . combining chemistry, biology and nanotechnology, are used to engineer, control and deliver gene editing tools more efficiently and precisely.

The first step in making these tumor-killing cells used in the cancer drug trial was to isolate the T-cells – a type of white blood cells that fights pathogens and cancer cells – from the blood of the cancer patients. Two patients with advanced multiple myeloma and one patient with myxoid/round cell liposarcomav were enrolled for this study.

To arm the T-cells and bolster their tumor-fighting skills without harming normal cells, scientists genetically engineered the T-cellsdisabling three genes and adding one gene – before returning them to the patients.

The first two of these deleted genes encode T-cell receptors, which are proteins found on the surface of the T-cells that can recognize and bind specific molecules, known as antigens, on cancer cells. When these engineered T-cells bind to these antigens, it allows them to attack and directly kill the cancer cells. But the problem is that a single T-cell can recognize multiple different antigens in the body, making them less focused on finding the cancer cells. By eliminating these two genes, the T-cells are less likely to attack the wrong target or the host, a phenomenon called autoimmunity, In addition, they disrupted a third gene, called programmed cell death protein 1, which slows down the immune response. Disabling the programmed cell death protein 1 gene improves the efficiency of T-cells.

The final step in the transformation of these cells was adding a gene which produces a new T-cell receptor that recognizes and grabs onto a specific marker on the cancer cells called NY-ESO-1. With three genes deleted and one added, the T-cells are now ready to fight cancer.

Source: https://theconversation.com/