Hope for new MS drug which could repair damaged cells.

Researchers from the private biotech firm ENDECE Neural have just announced the development of a new compound they believe may have the potential to repair damage caused by multiple sclerosis (MS).

MS is the most common neurological disorder affecting young adults in the western hemisphere. Although scientists are still unsure of what causes the disorder, it is known that symptoms stem from damage to the fatty covering surrounding nerve cells, known as the myelin sheath. It is believed that in the early stages of the disease the body’s own immune cells (cells usually primed to seek out and destroy foreign agents within the body, such as viruses and parasites) mistake myelin for a foreign body and launch an attack. Since myelin is essential for fast neural communication and cell protection, the symptoms of MS stem from a slowing of neural communication and ultimately nerve cell damage.

oligoThe myelin surrounding cells in the brain and spinal cord is provided by cells called oligodendrocytes. These cells reach out a number of branching arms which wrap around segments of surrounding neurons, forming the myelin sheath. The majority of drugs available for treatment of MS aim to reduce initial damage to this sheath. However, researchers from ENDECE are now investigating treatments which can increase the number of oligodentrocytes in the central nervous system, thus leading to remyelination of damaged cells. Dr. James G. Yarger, CEO and co-founder of ENDECE notes, “For decades, researchers have been seeking ways to induce remyelination in diseases such as MS that are characterized by demyelination,”. And now this dream may be becoming a reality.

ENDECE’s work revolves around their pipeline drug NDC-1308. Although the name isn’t likely to turn any heads, its properties just might. Following the observation that pregnant women typically do not experience the symptoms of MS during their third trimester, a number of researchers have been exploring a possible role for estrogen in the treatment of MS. ENDECE researchers created 40 separate estradiol analogues (substances similar in structure to estradiol but with a range of key modifications) and assessed their biological effects. From this work they found that one analogue (NDC-1308) had a particularly potent effect on oligodentrocyte precursor cells (OPCs – cells with the ability to become mature oligodentrocytes), causing them to differentiate into mature oligodendrocytes. In follow-up studies researchers found that treatment with NDC-1308 led to remyelination in a mouse model of MS, specifically showing a 20% increase in myelination in the hippocampus (a region of the brain known to experience demyelination in this model). NDC-1308 was also found to cause remyelination in the rat and to induce cultured OPC cells to differentiate into mature oligodendrocytes. Taken together, these findings suggest that NDC-1308 may prove effective in restoring the lost myelin sheath on damaged axons in patients with MS.

Dr. Yarger states, “We envision NDC-1308 being administered either alone or in combination with current therapeutics that target the immune response and/or inflammation associated with MS. By inducing remyelination, it may be possible to restore muscle control, mobility, and cognition in patients with MS. Therefore, a drug that induces remyelination, such as NDC-1308, can potentially double the size of the current market for MS therapeutics.”

NDC-1308 is still in late preclinical development, and has yet to go through rigorous safety screening and clinical trials. However, as a drug that potentially stimulates remyelination, it represents a whole new strategy for the pharmaceutical treatment of MS patients in the future.

Post by: Sarah Fox