In recent news, researchers have found methylene blue to be a promising drug candidate for the treatment of progeria. This is one of the oldest of modern synthetic medical compounds, as it is getting on for 140 years since it was first created by chemists. Nearly a decade ago it was shown to delay cellular senescence and enhance mitochondrial activity in cell cultures. The results for progeria to date look very good in much the same sort of cell studies, but the important qualifying next stage of animal studies has yet to happen.
Progeria is often called an accelerated aging condition. It is not accelerated aging, however, but a form of genetic damage to lamin-A, a protein central to the correct operation of cells. This global dysfunction results in degenerative conditions that have many similarities to those of the later stages of aging, and which are fatal over the course of ten to twenty years. Few patients live past their teens. It is thought that the same damage as runs rampant in progeria is present to a much lesser degree in normal aging, though it is an open question as to whether there is enough of it to have any meaningful impact in comparison to the contribution of other forms of cell and tissue damage. Still, this small shared commonality is why it is worth paying attention to progress towards therapies for progeria, as you'll sometimes see results like these:
Research suggests that a common, inexpensive and safe chemical called methylene blue could be used to treat progeria - and possibly the symptoms of normal aging as well. A new study shows for the first time that small doses of methylene blue can almost completely repair defects in cells afflicted with progeria, and can also repair age-related damage to healthy cells. "We tried very hard to examine the effect of methylene blue on all known progeria symptoms within the cell. It seems that methylene blue rescues every affected structure within the cell. When we looked at the treated cells, it was hard to tell that they were progeria cells at all. It's like magic."
Progeria results from a defect in a single gene. This gene produces a protein called lamin A, which sits just inside the cell's nucleus, under the nuclear membrane. Healthy cells snip off a small piece of each new lamin A molecule - a small edit that is necessary for lamin A to work properly. Cells with progeria, however, skip this important editing step. The defective lamin A interferes with the nuclear membrane, causing the nucleus to form bulges and deformations that make normal functioning impossible. Cells with progeria also have misshapen and defective mitochondria, which are the small organelles that produce energy for the cell. Although previous studies suggested damage to mitochondria in progeria cells, the current study is the first to document the nature and extent of this damage in detail. A majority of the mitochondria in progeria cells become swollen and fragmented, making it impossible for the defective mitochondria to function.
The team found that methylene blue reverses the damages to both the nucleus and mitochondria in progeria cells remarkably well. The precise mechanism is still unclear, but treating the cells with the chemical effectively improved every defect, causing progeria cells to be almost indistinguishable from normal cells. The researchers also tested methylene blue in healthy cells allowed to age normally. The normal aging process degrades mitochondria over time, causing these older mitochondria to resemble the mitochondria seen in progeria cells. Once again, methylene blue repaired these damages. "So far, we have done all of our work in stem cell lines. It is critical to see whether the effect extends to whole animals. We also want to see if methylene blue can repair specific effects of progeria in various cell types, such as bone, skin, cardiovascular cells and others. Further down the line, other groups might begin human clinical trials. It's very exciting."