Japanese researchers have started the world’s first human trial for a drug designed to regenerate teeth, following successful tests in animals. The drug works by blocking a protein called USAG-1, which normally stops tooth development. By preventing this protein from doing its job, the drug helps stimulate the growth of new teeth from hidden tooth buds in the jaw. The clinical trial, which began in October 2024 at Kyoto University Hospital, is testing the drug on healthy adult males aged 30 to 64 who are missing at least one tooth. The main goal is to check if the drug is safe to use in humans. If the trials go well, the researchers hope to make the drug available to the public by 2030 as a possible alternative to dentures or implants.

The drug works by targeting USAG-1, a protein that blocks pathways needed for tooth growth. When the drug stops USAG-1 from working, it reactivates dormant tooth germs in the jaw, which allows new teeth to grow. This approach is based on the same process that naturally happens when teeth develop. In earlier studies with animals like mice, ferrets, and dogs, a single dose of the drug triggered new tooth growth, without causing significant side effects. The drug specifically blocks USAG-1, meaning it affects only the teeth and avoids harming other parts of the body.

The clinical trial in humans will initially involve 30 healthy male adults between 30 and 64 years old, all of whom are missing at least one tooth. The researchers will give the participants different doses of the drug to see if it’s safe and how the body reacts to it. After that, a second phase of the trial will test the drug on young children who are missing teeth from birth. If everything goes as planned and the drug proves safe and effective, it could offer a new solution for people who have lost teeth, replacing traditional treatments like dentures and implants. The hope is that this treatment could be widely available by 2030, changing how we think about dental care.