Blog Science on iPSCs
The Mighty Versatility of the iPS Cell
Nearly all the various parts of your body have adult stem cells that can be summonsed to repair or replace that specific tissue. But they are often not particularly good at it, especially as we get older. They could benefit from supplemental or replacement stem cells; unfortunately, the only adult stem cells that we have perfected how to supplement are blood-forming stem cells. Those hemopoietic stem cells are routinely used as part of cancer therapy and increasingly being tried for other blood and immune disorders.
Thanks to a discovery that won the 2012 Nobel Prize, now we do have a source of stem cells that could repair or replace any damaged or defective tissue in your body. Kyoto University researcher Shinya Yamanaka found a way to use four genes that are normally only active during embryo development and make any adult tissue revert to cells similar to embryonic stem cells. Those cells can become any part of our bodies. The new cells, dubbed induced Pluripotent Stem Cells (iPSCs) are now being tested in human clinical trials and in pre-clinical research for many diseases and injuries.
The best cells to get the job done
Researchers have shown that one of the cell types GoodCell stores, a type of white blood cell called an endothelial progenitor cell, can be converted to iPSCs highly efficiently. While researchers have steadily improved the methods of making iPSCs, it can still be hard to get a large quantity of converted cells, which makes using the best cells key. Another benefit of the progenitor cells derives from their spending most of their time deep inside your body where they are less prone to sun-induced mutations found in skin samples often used to make iPSCs.
Your own iPSCs also have the advantage of being an immunologic match so your body is unlikely to reject them. In Japan where much of the early clinical work has been done some of the more recent trials have used banks of iPSCs from donors. The Japanese population is generally much more genetically homogeneous than the US, so they believe they can create banks with enough genetic options for immunologic matches for most patients. But even with those matched cells, they are giving patients potentially dangerous immune suppressive drugs, which would not be needed with you own cells.
Having the youngest possible cells to work with seems important as well. Research has shown cells from elderly patients do not reprogram as efficiently as younger cells and have more mutations that can result in abnormalities in the reprogramed stem cells (link). These are some of the reasons GoodCell’s Scientific Advisory Board Members advise banking cells as early as possible. Your healthiest cells are the ones you have right now.
Clinical trials are underway
Age-related Macular Degeneration (AMD) robs the sight of nearly 11 million Americans over the age of 50. Early last year the National Institutes of Health began a clinical trial using patients’ own cells. After turning them into iPSCs they then use genetic signals to mature those cells into the type of cell that dies early in this form of blindness, which they inject into the back of the eye of the patients. Results have not been reported, but earlier trials started in Japan have indicated the procedure may restore some vision.
Several teams have begun or are about to begin clinical trials in Parkinson’s disease. These teams turn the iPSCs from patients into the dopamine-producing cells that are destroyed in the disease. A patient at Kyoto University became the first treated with iPSC-derived cells in October 2018 and that trial is ongoing. The San Diego company Aspen Neuroscience has begun final pre-clinical tests for a US trial. They also intend to launch a second trial for patients with a specific genetically-linked form of the disease in which they use gene editing technology to correct the gene prior to making the desired cells.
A similar approach marrying iPSC technology and gene editing is being used by a team at UCLA. They recently developed an approach showing promise in treating Duchenne muscular dystrophy, a disease caused by a mutation in the gene encoding a protein key to muscle strength, dystrophin. They used CRISPR-Cas9 to repair the gene in human iPSCs, turned them into skeletal muscle cells, and injected them into the muscle of dystrophin-deficient mice. They could see restored dystrophin in the muscle.
Help for those painful knees
While many clinics offer unproven therapies using a type of stem cell called an MSC to treat osteoarthritis in the knee, those treatments have not been shown to have a lasting effect. The short-term benefit often reported appears to be from an anti-inflammatory impact not creating the new cartilage patients need. Now a team at Kyoto University plans to make sheets of cartilage from iPSC type stem cells, which has been shown to produce the type of hard cartilage you want in your knees. And a collaboration among scientists at Duke, University of Washington and Stanford has developed a highly efficient way to use iPSCs to make sheets of the desired cartilage.
Here are a few of the other many therapies under development:
- The first patient needing cornea repair has been treated and seen vision improved;
- Patients at Osaka University have been treated with sheets of heart muscle after heart attack;
- Two industry leaders Blue Rock and NcardiaStemCell have teamed up for large scale production of heart muscle tissue;
- University of California at Davis researchers have turned iPSCs into the cells needed to produce the clotting factor missing in hemophilia, in mice;
- Swedish researchers have reprogrammed cells to become nerve that engrafted in the brains of mice and improved the damage from strokes;
- A team at University of Sydney has used iPS cells to create neurons that treat the intractable chronic pain of nerve damage in mice.
A rapidly moving field
The FDA has estimated it will receive more than 200 applications to begin new cell and gene therapy clinical trials per year beginning this year. These will add to the more than 670 cell and gene-modified cell therapies currently underway. The agency expects to approve 10-20 cell and gene products a year by 2025.
Led by some of the top scientists in regenerative medicine, GoodCell was founded on the belief that medical science will continue its rapid progress, and we have positioned our personal biobanking solution to help you benefit from that progress.
Learn more about how the science behind GoodCell puts you in control of your health journey