Stem cells have the potential to treat a wide range of diseases. Here, discover why these cells are such a powerful tool for treating disease—and what hurdles experts face before new therapies reach patients.
How can stem cells treat disease?
When most people think about about stem cells treating disease they think of a stem cell transplant.
In a stem cell transplant, stem cells are first specialized into the necessary adult cell type. Then, those mature cells replace tissue that is damaged by disease or injury. This type of treatment could be used to:
- Replace neurons damaged by spinal cord injury, stroke, Alzheimer’s disease, Parkinson’s disease or other neurological problems;
- Produce insulin that could treat people with diabetes or cartilage to repair damage caused by arthritis; or
- Replace virtually any tissue or organ that is injured or diseased.
But stem cell-based therapies can do much more.
- Studying how stem cells develop into heart muscle cells could provide clues about how we could induce heart muscle to repair itself after a heart attack.
- The cells could be used to study disease, identify new drugs, or screen drugs for toxic side effects.
Any of these would have a significant impact on human health without transplanting a single cell.
What diseases could be treated by stem cell research?
In theory, there’s no limit to the types of diseases that could be treated with stem cell research. Given that researchers may be able to study all cell types they have the potential to make breakthroughs in any disease.
How can I learn more about CIRM-funded stem cell research in a particular disease?
CIRM has created disease pages for many of the major diseases being targeted by stem cell scientists. You can find those disease pages here.
You can also sort our complete list of CIRM awards to see what we’ve funded in different disease areas.
What cell therapies are available right now?
While there are a growing number of potential therapies being tested in clinical trials there are only a few stem cell therapies that have so far been approved by the FDA. Two therapies that CIRM provided early funding for have been approved. Those are:
- Fedratinib, approved by the FDA in August 2019 as a first line therapy for myelofibrosis (scarring of the bone marrow)
- Glasdegib, approved in November 2016 as a combination therapy with low dose are-C for patients 75 years of age and older with acute myelogenous leukemia
Right now the most commonly used stem cell-based therapy is bone marrow transplantation. Blood-forming stem cells in the bone marrow were the first stem cells to be identified and were the first to be used in the clinic. This life-saving technique has helped thousands people worldwide who had been suffering from blood cancers, such as leukemia.
In addition to their current use in cancer treatments, research suggests that bone marrow transplants will be useful in treating autoimmune diseases and in helping people tolerate transplanted organs.
Other therapies based on adult stem cells are currently in clinical trials. Until those trials are complete we won’t know which type of stem cell is most effective in treating different diseases.
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