The success rate of hematopoietic stem cell transplantation (HSCT) for multiple sclerosis (MS) varies depending on several factors, including the type of MS, the patient’s condition before treatment, and the specific HSCT protocol used. Generally, HSCT is considered a promising treatment for certain forms of MS, especially relapsing-remitting MS that has not responded well to conventional therapies.
HSCT involves collecting a patient’s own stem cells, then administering high-dose chemotherapy or immunosuppressive therapy to wipe out the faulty immune system that attacks the nervous system in MS. After this, the collected stem cells are reinfused to rebuild a new, healthier immune system. This process aims to “reset” the immune system to stop it from attacking the myelin sheath that protects nerve fibers.
Studies and clinical trials have shown encouraging results. For example, in patients with treatment-resistant relapsing MS, HSCT has demonstrated a significant reduction in disease activity and progression. Some trials report that about 70% to 74% of patients remain free from disease progression five years after undergoing HSCT. This means that a large majority of patients experience long-term stabilization or improvement in their symptoms following the procedure.
The success of HSCT is often measured by progression-free survival, which refers to the length of time patients live without their MS worsening. In addition to halting progression, many patients also experience improvements in neurological function, such as better mobility, reduced fatigue, and improved cognitive abilities. These benefits can dramatically enhance quality of life.
However, HSCT is not without risks. The treatment involves intense chemotherapy that can cause serious side effects, including infections, organ damage, and in rare cases, treatment-related mortality. Therefore, careful patient selection is crucial. Typically, HSCT is recommended for younger patients with active relapsing forms of MS who have not responded to other therapies and who do not have significant heart or other organ diseases.
The procedure’s success also depends on the center’s experience and the specific conditioning regimen used before stem cell infusion. Some protocols use more aggressive immunosuppression, which may increase the chance of success but also raise the risk of complications. Less intense regimens might be safer but potentially less effective.
Long-term follow-up is essential to monitor patients after HSCT. While many remain stable for years, some may experience disease reactivation or late complications. Ongoing research and clinical trials continue to refine the approach, aiming to maximize benefits while minimizing risks.
In summary, HSCT offers a high success rate for carefully selected patients with relapsing MS, with many achieving long-term remission and improved neurological function. The treatment represents a powerful option when other therapies fail, but it requires thorough evaluation and management due to its complexity and potential risks.
