A Breakthrough in Osteoporosis Treatment

(Posted on Tuesday, July 23, 2024)

In a world where an aging population faces increasing health challenges, a silent epidemic is quietly eroding the quality of life for millions. Osteoporosis affects one in three women and one in five men over 50 worldwide, weakening bones and leaving them vulnerable to life-altering fractures. However, as the global burden of this condition continues to grow, a glimmer of hope emerges in osteoporosis treatment.

At this forefront is a class of molecules called oligodeoxynucleotides, with one promising candidate, iSN40, showing potential. This molecule targets osteoblasts, the cells responsible for building and strengthening our skeletal framework, potentially offering a new lease on life for those grappling with fragile bones.

Osteoporosis causes up to 37 million fragility fractures in individuals aged over 55, which equates to about 70 fractures per minute globally. The need for effective treatments has never been more urgent. This research promises to enhance the lives of millions and alleviate the enormous economic burden on healthcare systems worldwide.

Understanding Osteoporosis

Osteoporosis is a condition characterized by weakened bones, which makes them fragile and more susceptible to fractures. Osteoporosis develops when the body loses too much bone, produces too little bone, or both. This process occurs gradually over time, earning osteoporosis the moniker of a “silent disease.”

As we age, our bones lose some of their density and strength due to a disrupted balance between bone breakdown and rebuilding in osteoporosis. This is partially because after 35, bone breakdown outpaces bone rebuilding. Also, hormonal changes start to take place as we age, particularly in women after menopause, when estrogen levels drop. Other lifestyle factors can also play a role, such as lack of exercise, poor diet, smoking, excessive alcohol consumption, and certain medical conditions and medications.

Many osteoporosis treatments involve complex and expensive medications containing particular proteins or hormones that aid in bone building or slow bone loss. Now, we are getting closer to gene-based treatments known as osteogenetic oligodeoxynucleotides or osteoDN.

Introduction to Osteogenetic Oligodeoxynucleotides

Osteogenic oligodeoxynucleotides, or osteoDNs, are derived from genomic sequences and are short DNA fragments designed from genetic material found in bacteria. These molecules work by targeting and activating osteoblasts, which are the cells responsible for building new bone tissue. By stimulating these cells, osteoDNs could help restore the balance of bone formation and breakdown.

The research team screened a library of ODNs derived from the genome of a beneficial bacteria called Lacticaseibacillus rhamnosus GG. They identified an ODN named iSN40 that showed a remarkable ability to promote bone cell development. iSN40 demonstrated several positive effects on bone cells, such as stimulating the expression of genes crucial for bone development and enhancing calcium deposition in bone cells.

Caption: The most stable of the ODNs in water. (A) iSN40. (B) MT01.

Interestingly, iSN40’s bone-promoting effects do not rely on a specific DNA sequence called the CpG motif, which is known to trigger immune responses. This suggests that iSN40 could stimulate bone growth without causing unwanted immune reactions.

The discovery of iSN40 is fascinating because nucleic acid-based drugs like ODNs offer several advantages over traditional protein-based treatments. They are easier and less expensive to manufacture, have better stability during storage, and can be designed to target specific cells or processes in the body. This research opens up new possibilities for developing innovative treatments for osteoporosis.

The next steps involve testing iSN40’s effectiveness in animal models and, eventually, in clinical trials. If successful, this approach could lead to more accessible and affordable treatments for the millions of people affected by osteoporosis and other bone disorders.

Toward the Future

The discovery of iSN40 represents a milestone in osteoporosis research. This osteogenetic oligodeoxynucleotide offers a new and efficient way to stimulate bone formation, giving hope to the millions affected by the disease. While translating these findings into clinical practice presents challenges, the potential benefits are enormous. With the emergence of iSN40 and similar oligodeoxynucleotides, the future of osteoporosis treatment looks promising.

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This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged by disease, injured by trauma, or worn by time to normal function. I include a full spectrum of chemical, gene, and protein-based medicines, cell-based therapies, and biomechanical interventions that achieve that goal.

In this subseries, we focus specifically on gene therapies. We explore the current treatments and examine the advances poised to transform healthcare. Each article in this collection delves into a different aspect of gene therapy’s role within the larger narrative of Regenerative Medicine. 

To learn more about regenerative medicine, read more stories at www.williamhaseltine.com