Triple Punch Gene Therapy For AIDS Patients Clears Its First Safety Test In Humans

Filed in archive Gene Therapy on June 17, 2010

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A triple punch gene therapy has cleared its first safety test in humans as it gives human stem cells three ways to defy HIV. Four AIDS patients were injected with these cells and they tolerated the treatment and for up to two years, the cells produced anti HIV weapons.

Researchers are optimistic that after further clinical trials, combination therapy can replace or complement anti-retroviral drugs for treatment of HIV patients.

The trial piggybacked on a standard treatment where AIDS patients were given transplants of their previously saved blood stem cells for prevention of development of blood cancer. Besides normal blood stem cells, patients were also injected with cells in which three types of RNA based gene therapies were carried by a lentivirus. In the present trial, researchers combined genetic resistance into stem cells for replacing an immune system susceptible to HIV with one which can resist attack of the virus.

The study has been published in Science Translational Medicine.