The Drug-Controller General of India (DCGI) has given the go-ahead for the first clinical trials designed to test stem cell products. Stempeutics Research of Bangalore launched a combined phase 1 and phase 2 trial on April 22 to evaluate whether its stem cell products can benefit people who have experienced myocardial infarction and individuals with critical limb ischemia (CLI)—a condition that often requires amputation.

“These are the only two stem cell trials officially approved to date,” says Polani B. Seshagiri, a member of a government panel that made the recommendation to DCGI. There have been many claims in the past from Indian labs offering stem-cell therapy to treat a wide range of diseases, prompting criticisms that local regulators were failing to monitor the procedures (Nature 434, 259, 2005). But Seshagiri, who heads the Stem Cell and Transgenic Research Lab at the Indian Institute of Science in Bangalore, says, “none of these can be called a clinical trial.” The Stempeutics trials are the first randomized, double-blind, multicentric, placebo-controlled studies. Each trial will recruit 100 patients in batches.

“Our goal is to bring out affordable stem cell–based products as drugs in chemists' shops,” says Stempeutics' president Balu N. Manohar. The company extracts mesenchymal stem (MS) cells from the bone marrow of healthy donors and expands them in culture before infusing them back in. These MS cells are well tolerated by the recipient because they lack immunogenic major histocompatibility complex class II molecules on their surface, says Ramesh Bhonde, technical director for Stempeutics. The company has scaled up the production process to obtain 300 to 400 million MS cells of good manufacturing practice quality from a single donor. Preclinical animal toxicity studies, says Bhonde, confirm that ex vivo cultured adult MS cells are safe and can be used both in autologous and allogenic settings. Seshagiri does not expect any major obstacles in this form of therapy as long as the cells are clinical grade.