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EryDex is a slow-release system, he says, and research has found that it ''prevents the typical steroid side effects." These can include thinning of bones, high blood sugar, cataracts, and high blood pressure.
Research has shown the treatment can delay symptoms and the course of the disease. In one study, 18 patients given monthly infusions for 6 months had a better score on a commonly used scale to assess motor skills and many other aspects of daily living. The company is conducting its final study before seeking FDA approval. Results of that study are expected by the second half of 2019.
The treatment has been granted orphan drug status by the FDA. That means the drug is designed to treat rare diseases or is unlikely to ever be profitable to a drug manufacturer. Besides ataxia telangiectasia, the company is looking at other diseases, including cancer, their system could treat.
Rubius Therapeutics in Cambridge, MA, is also studying superblood treatment for rare diseases, cancers, and immune system disorders. It has raised more than 0 million in financing for research.
The less-toxic superblood approach also allows the drugs to target tumors more directly, experts say. The drugs also stay in the body longer, so superblood may be more effective in the long term.
Experts say the new technology builds on another treatment advance known as CAR T-cell therapy. CAR T-cell therapy draws on the power of the body's T cells, known as the workhorses of the immune system, due to their ability to find and kill diseased cells.
In the CAR T process, blood is drawn from a patient, T cells are separated and genetically engineered to boost their tumor-fighting ability, then returned to the patient through an IV.
Compared to CAR T, superblood has more advantages, experts say. The superblood treatment is easier to use, can be made more quickly, and is long lasting.