Urgent Need for New Drugs to Treat Sickle Cell Disease, Says ICMR Scientist

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New Delhi— There is a critical need to develop new and more effective drugs for treating sickle cell disease, as the current standard therapy, hydroxyurea, does not work for all patients, said Dr. Manisha Madkaikar, Director of the ICMR–Centre for Research Management and Control of Haemoglobinopathies (CRHCM) in Nagpur.

Speaking to IANS, Dr. Madkaikar emphasized that although hydroxyurea remains the only drug with proven long-term benefits for patients, it has significant limitations.

“We have known about sickle cell disease for many decades, and yet hydroxyurea is the only treatment that has stood the test of time,” she said. “It improves quality of life and reduces complications, but unfortunately, not all patients respond to it. Plus, it requires lifelong use, which underscores the urgent need for better therapies.”

Hydroxyurea has been included in the National Health Mission’s list of essential medicines, but the lack of alternatives has left a gap in treatment options for non-responding patients.

The comments come on the heels of a major government initiative aimed at addressing the treatment gap. Last week, the Ministry of Tribal Affairs announced a ₹10 crore reward for developing a new drug for sickle cell disease, in partnership with AIIMS Delhi. The funding will be awarded to the most promising proposal through a national competition, according to Union Minister of State for Tribal Affairs Durgadas Uikey.

The minister also announced the establishment of the Bhagwan Birsa Munda Prize, which will honor breakthroughs in the development of treatments for sickle cell disease.

Sickle cell disease is a hereditary blood disorder that affects hemoglobin in red blood cells, leading to severe health complications, and is especially prevalent in tribal populations across India. Approximately one in every 86 births in Scheduled Tribe (ST) communities is affected by the condition.

Dr. Madkaikar also noted that while treatments such as bone marrow transplants and gene therapy are showing promise globally, they are not yet available in India — adding urgency to the search for accessible and effective alternatives.

“We don’t currently have any other drug with the effectiveness of hydroxyurea,” she said. “There is a tremendous need for breakthrough medications that can truly transform the lives of people living with sickle cell disease.” (Source: IANS)