If you, a loved one or friend has faced the possibility of needing a stem cell transplant as part of therapy for blood cancer, you know it is a tough decision. The 50,000 transplants that occur worldwide each year most often save lives, but they come with a cost. The use of extreme chemotherapy or radiation to make room in the bone marrow for the new stem cells can cause infertility, new cancers later in life and extreme suppression of your immune system, leaving you vulnerable to life-threatening infections.
Current regimens for conditioning your bone marrow to accept the new cells are more like a shotgun than a targeted arrow. Several teams around the country are working to put exquisitely targeted arrows in the quiver of transplant teams. In fact, the leading science journal Nature recently published a reader-friendly review of three teams’ efforts presented at the year’s most significant blood science conference, the American Society for Hematology (ASH).
If research confirms the early results presented in December, the use of blood stem cell transplants could be greatly expanded to cure other forms of cancer, autoimmune conditions – lupus, rheumatoid arthritis, multiple sclerosis – and other non-life-threatening diseases.
Crafting the targeted arrow
The FDA anticipates receiving more than 200 applications for investigational new drugs (INDs) in cell and gene therapy per year starting in 2020, building upon its total of more than 800 active cell-based or directly administered gene therapy INDs currently on file. By 2025, the agency predicts it will be approving 10-20 cell and gene therapy products a year based on an assessment of the current pipeline and the clinical success rates of these products.
Three companies in particular are using antibodies – key elements in the immune system for fighting disease – that specifically target cells that need to be removed to make room for replacement stem cells.
Jasper Therapeutics has successfully treated four infants who needed the stem cell transplants to treat a rare immune disorder, and has tested the antibody in more than 80 healthy volunteers and patients. They plan to expand their clinical trials to patients with leukemia and the blood disorders known as myelodysplastic syndromes. The research originated from labs at Stanford University.
Another Stanford team’s work flowed into the company Forty Seven, which is using a combination of two antibodies, one similar to Jasper’s and one that blocks the action of immune cells that try to stop the first antibody. Their team says the two acting together should more easily clear the needed space in the bone marrow.
Finally, Magenta Therapeutics is collaborating with the National Institutes of Health to use a slightly different antibody combined with a toxin that it releases only after coming in contact with cells that need to be removed from the marrow. Studies in mice and primates indicate early success.
All three companies expect to move rapidly into clinical trials to bring new, safer transplants to many different groups of patients.
Storing your healthy blood stem cells
These scientific breakthroughs – and so many more – will be made possible with access to healthy, quality stem cells, which are the foundation to any cell or gene-based therapy. Interestingly, stem cells become less effective at doing their job as people age and are more likely to have mutations from environmental factors or just normal cell replication. For some cancers and other conditions, stem cells can actually harbor the disease once it manifests. In these situations donor stem cells must be used, and transplant teams generally seek out the youngest donors they can find. Donor cells, however, carry added risk – the condition known as Graft Versus Host Disease – which occurs when they create immune cells that attack your own tissue.
Storing your youngest, healthy cells can be a solution to both these barriers—aged cells and risks of donor cells— to increase the success rates of stem cell transplants.
One of the companies above, Magenta, along with at least three others, are working on methods to expand the number of stem cells in a small sample, similar to the number of cells GoodCell can currently store for its members, to create enough cells for transplant. Magenta presented data from a clinical trial at last year’s ASH meeting showing that the small number of cells indeed produced enough for a successful transplant. Additional trials are currently underway to bring these procedures to market.
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.
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