Teams of researchers from Summa Health System, IC-MedTech and other institutions have completed the first ever FDA-approved human clinical trial of Apatone®. The drug Apatone makes use of a new strategy wherein it selectively lowers the level of compounds in the tumor cells that otherwise enables energy production and protection against chemotherapy. Thus the drug employs a non-toxic approach weakens and kills cancers in a novel way.
Apatone was discovered by Dr. Henryk Taper from the Catholic University of Leuven in Brussels, Belgium and was developed by Dr. James Jamison and Dr. Jack Summers, both of Summa Health System, and Dr. Jacques Gilloteaux, now with the American University of the Caribbean in St. Maarten. Their groundbreaking discovery found that moderate doses of Apatone eliminate many types of cancer cells, including prostate, bladder, renal and ovarian.
"This strategy targets cancer cells by their inflammatory response," explains Dr. Jamison. "It's a different approach than most other anti-tumor drugs, which target dividing cells or the development of blood vessels within the tumor. Since normal cells use sugars or fats for energy and cancer cells rely on glucose, the real key here is that Apatone resembles glucose. As Apatone preferentially accumulates in cancer cells, it also supplies quinone that weakens and can destroy the cancer cell from within.
Licensed in 2004 to IC-MedTech, Inc., a California-based biotechnology company, the first clinical trial began in 2005 to evaluate the drug in prostate cancer patients. The clinical studies, which were conducted at Summa Health System in Akron, Ohio and with Dr. Ananias Diokno at William Beaumont Hospital in Royal Oak, Mich., examined the safety and effectiveness in 17 end-stage prostate cancer patients for 12 weeks. These patients took Apatone orally each day. The trials were supported by the Beaumont Foundation, Summa Health System and IC-MedTech.
Throughout the trial, investigators monitored prostate-specific antigen (PSA) levels, PSA velocity and PSA doubling times. Although PSA is a protein normally produced by the prostate gland, individuals with prostate cancer have increased levels. PSA velocity is the change of PSA levels over time and PSA doubling time is the time it takes for a patient's PSA level to double.
"The results of the trial are very promising," said Dr. Jamison. "Sixteen of the 17 patients responded positively to the Apatone and 13 showed a decrease in PSA velocity and an increase in PSA doubling time. At the end of the treatment period, 15 patients opted to continue treatment."
Showing delays in the biochemical progression in end-stage prostate cancer patients, the trial successfully demonstrated the safety and efficacy of orally administered Apatone. Research is continuing and insights into how this drug works have lead to collaborations and discoveries in the field of liquid crystal compounds. Apatone is a Liquid Crystal PharmaceuticalTM and has resulted in research sponsored at Summa and with Dr. Chun-che Tsai at Kent State University. This work shows great promise for cancer and other diseases.
"Ultimately, Apatone is intended to be administered intravenously prior to chemotherapy so it can break down the substances in a tumor that protect it from the chemotherapy and allow a greater cell kill," continued Dr. Jamison. "Between cycles and following completion of chemotherapy, Apatone will be taken orally to help prevent or slow tumor regrowth."
Although the researchers are still working to receive FDA approval for chemotherapy in conjunction with Apatone, the FDA granted orphan drug status to IC-MedTech this year for the use of Apatone as treatment for metastatic, or locally advanced, stage III and IV bladder cancer. An orphan drug designation grants special status to a product to treat a rare disease or condition.
Additional clinical trials are planned for intravenous administration of Apatone in patients who have failed chemotherapy.