Scientists at the Florida campus of The Scripps Research Institute have identified a potential new treatment of osteoporosis. This new therapeutic approach could promote the development of new bone-forming cells in patients suffering from bone loss. These are great news for people suffering from osteoporosis, as this disease causes a lot of injuries as bones become much weaker and can break with minor stress or even spontaneously.
There are no highly effective treatments for osteoporosis. This new research focused on a protein called PPARy, known as the master regulator of fat. Scientists were researching its impact on the fate of stem cells derived from bone marrow (“mesenchymal stem cells”). As other stem cells, these cells can grow into some other types of cells. For example, fat, connective tissues, bone and cartilage. Therefore, they have a great therapeutic potential for number of different diseases and conditions, including osteoporosis.
As is often the case, mouse models were very useful in this research. Scientists noticed that a partial loss of PPARy protein led to increased bone formation in a genetically modified mouse. The researchers wanted to see if they can achieve similar effect with a drug candidate, so they used a wide variety of structural biology approaches in combination in order to create a new drug compound that would repress the biological activity of the PPARy protein. Scientists wanted to see if reducing activity of the protein would promote the formation of new bone cells.
And they got promising results from their research. Researchers found that the new compound, which they called SR2595, could affect human mesenchymal stem cells in such a way that there would be a statistically significant increase in osteoblast formation. It is a cell type known to form bone.
Patrick Griffin, one of the scientists that conducted this research, said that “these findings demonstrate for the first time a new therapeutic application for drugs targeting PPARy, which has been the focus of efforts to develop insulin sensitizers to treat type 2 diabetes”. Now, since scientists have already demonstrated that SR2595 is suitable for testing in mice, they are planning next steps. They will continue to research the drug with other animal models, taking into account such factors as bone loss, aging, obesity and diabetes.
As much as identifying a potential new therapeutic for bone loss is extremely important achievement, this new study may have even broader implications. This protein that was researched in the study is closely related to several other proteins that also have a role in the disease. This means that scientists could apply new knowledge to develop new compounds to a variety of therapeutic applications.
This research also helped to better the understanding about the osteoporosis – now scientists know more about how natural molecules in human body regulate metabolic and bone homeostasis, and how unwanted changes can underlie the pathogenesis of a disease.
Even though they are still just preliminary, the results of the study provide hope that science someday will be able to create effective therapeutics to treat diseases of bone loss. Osteoporosis is more common in older people, but can also affect younger people as well. Which is why treating it is an important goal, since it induces a lot of difficulties in moving and even pain.