Sickle Cell Treatment Shows 100% Response

A phase I/II clinical study has shown that an anticancer compound generated a 100% response rate in sickle cell patients. The data were presented at the annual meeting of the American Society of Hematology in San Francisco (CA, USA).

The 36-week study enrolled six sickle cell patients, five of whom had shown no response to the current standard treatment (Hydroxyurea, HU) after one year and one patient who was not treated with HU. Each of the six patients treated with the new compound, decitabine, experienced elevated levels of fetal hemoglobin, which prevents sickle-shaped cells from congregating, allowing them to move freely throughout the body.

Following systemic administration of six cycles (36 weeks) of decitabine, average fetal hemoglobin levels increased more than 22%. There were no reports of any sickle cell episodes during the course of treatment. Decitabine is the product of SuperGen (San Ramon, CA, USA).

"The study concludes that decitabine is a highly effective alternate drug for the treatment of sickle cell anemia, especially for nonresponders,” said Joseph DeSimone, Ph.D., University of Illinois (Chicago, USA), who presented the data.



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