Researchers at the University of Hong Kong’s LKS Faculty of Medicine (HKUMed) have developed an oral form of arsenic trioxide (Oral-ATO; ARSENOL) to treat acute promyelocytic leukemia (APL), a once-deadly type of blood cancer.
This breakthrough is not only a first for Hong Kong’s medical research, but also a major advancement in leukemia treatment globally. Oral-ATO has already received patents in the United States, Europe, and Japan, and has shown excellent results in clinical trials.
A Life-Saving Alternative to Traditional Treatments
APL was once a highly fatal disease, with up to 30% of patients dying early from complications. Traditional treatments often included bone marrow transplants, which are expensive, highly toxic, and not widely available. Now, after more than 20 years of research, HKUMed has successfully introduced Oral-ATO as a safer and more effective treatment.
A 15-year study on 400 patients with relapsed APL showed that Oral-ATO led to a 100% remission rate and an 80% five-year survival rate, all without the need for bone marrow transplants. When used for patients in early remission, 90% remained leukemia-free for five years, and the overall survival rate reached 97%.
In newly diagnosed APL patients, the results were even better—100% survival and disease-free rates after five years. This means Oral-ATO has the potential to completely change the way APL is treated.
Oral Treatment Instead of Chemotherapy
One of the most promising aspects of Oral-ATO is its ability to replace or reduce chemotherapy. Researchers have developed a fully oral treatment plan that includes:
- Oral-ATO
- All-trans retinoic acid (ATRA)
- Ascorbic acid (Vitamin C, AAA regimen)
This “AAA” therapy can be taken at home instead of requiring hospital-based chemotherapy. It is safe for both children and adults with APL and is effective for all risk levels of the disease.
International Recognition and Expanding Access
The success of Oral-ATO has led to recognition by major health regulators:
- The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Oral-ATO orphan drug designation (ODD), a key step toward its approval in these regions.
- The Guangdong Provincial Medical Products Administration (GDMPA) has already approved Oral-ATO for clinical use in China’s Greater Bay Area via the HKU-Shenzhen Hospital.
- Singapore, Malaysia, and Taiwan have joined the APL Asian Consortium, a research network led by HKUMed that is studying Oral-ATO across the region.
In the United Kingdom, a nationwide Phase 3 trial is set to begin, funded by Blood Cancer UK. The study will test the use of Oral-ATO as a frontline treatment for APL.
In North America and Europe, HKUMed researchers are working with industry partners to begin clinical trials this year.
A Curable Disease?
For decades, APL was considered a highly fatal leukemia. Now, thanks to Oral-ATO, APL is becoming a curable disease. This groundbreaking treatment is not only more effective and safer, but also cheaper and more accessible than traditional therapies.
Dr. Harinder Gill, the lead researcher, believes that Oral-ATO will change lives worldwide. “Our goal is to ensure that all APL patients have access to this life-saving treatment. Making APL a curable disease would be our greatest achievement.”
With continued research and international cooperation, Oral-ATO could become the new global standard for treating APL, offering patients everywhere a better chance at life.
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