Prochymal

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Prochymal is a stem cell therapy product developed by Osiris Therapeutics, a leading company in the field of regenerative medicine. It is the first stem cell drug to receive approval from any regulatory agency in the world. Prochymal is used for the treatment of acute graft-versus-host disease (GvHD) in children, a life-threatening complication that can occur after a bone marrow transplant.

History[edit | edit source]

Prochymal was developed by Osiris Therapeutics, a company founded in 1992 with the aim of harnessing the potential of stem cells for therapeutic purposes. The development of Prochymal began in the early 2000s, and it was granted Orphan Drug status by the Food and Drug Administration (FDA) in 2003. In 2012, Prochymal became the first stem cell drug to be approved by a regulatory agency when it was approved by Health Canada for the treatment of acute GvHD in children.

Mechanism of Action[edit | edit source]

Prochymal is composed of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors. These cells have the ability to differentiate into a variety of cell types, and they also have immunomodulatory and anti-inflammatory properties. In the case of GvHD, the MSCs in Prochymal are believed to work by suppressing the immune response that causes the disease, thereby reducing inflammation and tissue damage.

Clinical Use[edit | edit source]

Prochymal is used for the treatment of acute GvHD in children who do not respond to steroids. GvHD is a serious complication that can occur after a bone marrow transplant, when the donor's immune cells attack the recipient's body. Prochymal has been shown to improve survival rates in children with severe GvHD, and it is currently the only approved treatment for this condition.

Future Directions[edit | edit source]

While Prochymal has been approved for use in Canada, it is still considered an investigational drug in other countries, including the United States. However, clinical trials are ongoing, and it is hoped that Prochymal will be approved for use in other countries in the near future. In addition to GvHD, Prochymal is also being investigated for the treatment of other conditions, including heart disease, liver disease, and diabetes.


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Contributors: Prab R. Tumpati, MD