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Stem cells can develop into many cell types and act as the body’s repair system. They replace or restore damaged tissues, offering new possibilities for treating diseases.
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The treatment landscape for Myelodysplastic Syndromes is stratified strictly by risk. The philosophy divides patients into two broad categories: those for whom the goal is symptom management and quality of life (typically lower-risk MDS), and those for whom the goal is altering the disease course and extending survival (higher-risk MDS). Regenerative medicine, in the form of stem cell transplantation, plays a central role in the curative strategy, representing the only established method to permanently eradicate the disease clone.
For many patients, particularly the elderly with low-risk disease, the backbone of treatment is supportive care. The goal is to manage the cytopenias.
For patients with higher-risk disease or those who fail ESAs, “epigenetic therapy” is the standard of care. Drugs like Azacitidine and Decitabine are hypomethylating agents.
Administration: These drugs are typically given via injection or IV for several days in a row, every 4 weeks. They are not curative but can control the disease for months or years and delay progression to leukemia.
For the specific MDS subtype with isolated del(5q), Lenalidomide is highly effective.
This is the pinnacle of MDS treatment and the primary regenerative intervention. It is the only potential cure.
Graft-vs-Leukemia Effect: The cure relies not just on the new stem cells making blood, but on the “Graft-vs-Tumor” effect. The donor’s immune cells (T-cells) recognize the remaining MDS cells as foreign and attack them. This immunological surveillance is crucial for preventing relapse.
Historically, transplants used “myeloablative conditioning” (MAC)—extremely high doses of chemo that wiped out the marrow completely. This was too toxic for older MDS patients.
The field is rapidly advancing.
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Currently, no. Drug therapies like Azacitidine or Lenalidomide can control the disease, improve blood counts, and extend survival, often for years. However, they do not permanently eradicate the abnormal stem cell clone. Eventually, the disease usually becomes resistant. Allogeneic transplant remains the only proven curative modality.
Standard chemotherapy works by killing all rapidly dividing cells, which is very toxic. Azacitidine is a “hypomethylating agent” or epigenetic therapy. It works by reprogramming the cancer cells—switching active genes back on—to encourage them to mature and function normally, rather than just killing them outright.
Transplant is a high-risk procedure with significant potential for complications, including graft-versus-host disease and severe infection. For older patients with multiple other health issues or those with very low-risk MDS, the risk of dying from the transplant procedure may be higher than the risk posed by the MDS itself.
Patience is required. Unlike intensive chemo, which clears cells quickly, drugs like Azacitidine often take 4 to 6 months (cycles) to show their maximum benefit. It is crucial not to stop the therapy prematurely if no immediate improvement is seen in the first couple of cycles.
Patients who receive frequent red blood cell transfusions accumulate excess iron, as the body has no natural way to excrete it. This iron can damage the liver and heart. Chelation therapy involves taking medications (pills or infusions) that bind to the excess iron and help the body excrete it through urine or stool.
Multiple sclerosis (MS) is a disease that harms the brain’s control over the body. It has four main types: Relapsing-remitting MS (RRMS), Primary-progressive MS (PPMS),
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