Capricor's Cell Therapy Trial Shows Promising Results for Muscular Dystrophy
With more than a century of extensive research, muscular dystrophy has remained a disease group that is characterized by a shortened lifespan, with most treatment approaches failing to even slow down their progression. They would mostly strike a person at a tender age, and most of them would not cross their 30's.
Most cases of muscular dystrophy would be diagnosed between the ages of 5-12, with the majority of them being males. Drug therapy along with physio would only serve to slow down the disease a bit, but there is no cure yet. In most cases, the ailment would start with the weakness of limbs, further progressing to affect the respiratory system and heart. Finally, many of them would die due to progressive cardiac failure.
All dystrophies are caused by inherited genes, which would lead to the disruption in the structure of the various muscles. Either science must find a way to suppress or alter these genes, or a way to regrow and strengthen the muscular tissues. Modern research has made lots of progress in both the directions.
A specific group of researchers is trying hard to find the safe way to edit the defective genes in the muscles, mostly by deploying technologies like CRISPR, while other researchers are trying to harness the power of stem cell therapy.
Capricor therapeutics a biotechnology company is carrying out the clinical trial to find out the safety and effectiveness of cell therapy in muscular dystrophy. For this purpose, they are using CAP-1002, a type of allogeneic cardiosphere-derived cells.
Cell therapy that may eventually change the way we treat muscular dystrophies
CAP-1002 is fundamentally a type of stem cell therapy. Stem cell therapy can trace its origins back to 1960 when the first bone marrow transplant was completed in those suffering from a plastic anemia after chemotherapy.
It has been well known that our blood cells continue to grow throughout our lives, due to the presence of specific seed cells in the bone marrow. However, by 1990 lots was learned about these cells that can differentiate and grow to form the various tissues and organs, giving birth to the field of stem cell therapy. These seed cells are present in almost every organ of our body, playing an essential role in the organ repair. But they are either not that active in other organs when compared to bone marrow or are in very small numbers. So, other body organs have limited the ability to keep growing or repair. Same is true for muscular tissues.
Until the end of the 20th century, it was not known that such seed cells are present in almost every organ. So, the stem cells were mostly derived from either the embryo, the umbilical cord of the newborn, or bone marrow. But, now the advancement of technology has not only allowed us to identify and extract such seed cells from other organs, but it has also enabled us to multiply them by artificial means so that we could have enough supplies and the ability to use them out of the shelf.