HSCT (Stem Cell) Treatment for Multiple Sclerosis (MS) in Ireland

Stem cell therapy has been under investigation since the 1990's but was initially deemed risky. In the early 2000's, the mortality rate during treatment was as high as 1 in 25. Over time, advancements in treatment protocols and patient selection criteria have significantly reduced these risks. Today, mortality rates are less than 1% in approved centres.

The treatment was previously considered a last resort, often offered to patients with advanced disease. Research has shown that HSCT is more effective when administered earlier, particularly in individuals with relapsing-remitting MS (RRMS) who have active inflammation. This shift in understanding has made it a viable option for a broader group of patients, provided the timing and criteria for treatment are optimal.

HSCT involves the collection and reinfusion of a patient’s own haematopoietic stem cells. The treatment comprises of several stages:

1. Conditioning Phase: High-dose chemotherapy is administered to suppress the immune system. This step eliminates the malfunctioning immune cells responsible for attacking the nervous system.
2. Stem Cell Collection: Stem cells are harvested from the patient’s blood or bone marrow. These cells are then processed and stored.
3. Reinfusion: The harvested stem cells are reinfused into the patient. These cells migrate to the bone marrow and begin producing new, healthy immune cells.
4. Recovery: Patients undergo a critical recovery period, during which their immune system rebuilds. This stage requires close monitoring to manage potential complications. Early complications can occur within the first year of treatment and longer term problems, such as other autoimmune diseases, can emerge at a later date.

This process aims to halt disease activity by resetting the immune system. However, the recovery phase can be physically demanding, often requiring weeks of isolation and ongoing medical support. Patients are closely monitored for infections, and supportive treatments, such as antibiotics and antivirals, are administered to reduce risks.

Studies have demonstrated promising results:

• Patients with relapsing-remitting MS tend to benefit the most, with up to 83% achieving "no evidence of disease activity" (NEDA) after two years, and over 60% at five years.
• In progressive MS, outcomes are less favourable, though some benefit has been observed in cases with active inflammation.

Research indicates that younger patients with active inflammation and fewer mobility issues respond better to HSCT. For patients with very aggressive MS, where multiple relapses occur within a short period, HSCT can sometimes be considered as a first-line treatment, although this is rare.

It is important to be aware HSCT is an effective treatment for inflammation in MS but it is not a cure, further relapse or progression can occur at a later date, and it does not result in remyelination or repair to previously damaged nervous tissue.

Ongoing trials such as the BEAT-MS and STAR-MS studies aim to compare HSCT with modern disease-modifying therapies (DMTs). These trials will provide critical insights into the long-term effectiveness of HSCT compared to existing treatments. Earlier research highlighted that HSCT offers better outcomes than many standard treatments, particularly in patients with relapsing MS who have failed other high-efficacy therapies.

For people living with MS in Ireland, the pathway to HSCT involves multiple steps:

1. Consultation: Person living with MS discusses HSCT with their neurologist if their current treatment is ineffective. This step often follows evidence of new relapses, disease progression, or new lesions on MRI scans.
2. Referral: Cases are reviewed by a multidisciplinary team (MDT) in Ireland to assess eligibility based on NHS criteria. The MDT includes neurologists, haematologists, and MS nurse specialists.
3. Approval: Suitable candidates are referred to a further MDT and if approved for treatment are referred to a specialised centre for further evaluation. Both the MDT and specialised centres are located in London
4. Treatment Abroad: Once approved funding is sought via the E112 Treatment Abroad Scheme, candidates undergo the procedure and are monitored closely during recovery. Post-treatment follow-up typically occurs in Ireland.

Criteria for HSCT include:

• Aged between 18 and 55 (up to 65 in some cases)
• MS duration under 15 years (preferably under 10)
• Evidence of active inflammation
• Prior treatment with high-efficacy DMTs

Candidates must also have adequate family or social support during recovery, as the treatment can lead to significant physical and emotional challenges.

HSCT is an aggressive treatment with potential complications, including:

• Short-term: Hair loss, infections, nausea, and fatigue are common. Patients may also experience a temporary worsening of MS symptoms during the recovery phase.
• Long-term: Thyroid issues, shingles, and rare cases of infertility or increased cancer risk have been reported. While these risks are low, they highlight the need for careful patient selection and monitoring.

Fertility preservation is an important consideration, as the treatment may impact reproductive health. Options such as egg, embryo, or sperm freezing should be discussed prior to treatment. In Ireland, fertility preservation options for non-cancer patients remain limited, adding to the complexity of planning for HSCT. Even if funding for HSCT itself is approved, fertility preservation may have to be self-funded.

While HSCT has shown significant potential, patient experiences vary. Short-term recovery can be challenging, with many reporting fatigue, cognitive issues, and emotional struggles. However, long-term quality of life often improves, particularly for those who achieve disease stabilisation or remission.

Neuropsychological studies have shown that patients frequently report an improvement in overall quality of life after treatment, despite initial difficulties. Key factors influencing recovery include age, the severity of MS before treatment, and the presence of supportive care during recovery.

Those considering HSCT are encouraged to discuss it openly with their neurologist if this is something they are interested in learning more about. 

Things to discuss with your neurologist include:

• Your understanding of HSCT and its potential benefits
• Specific reasons you believe you may be suitable candidate
• Any new relapses, lesions, or progression despite current treatment

Neurologists may recommend further assessments or refer the case to Ireland’s MDT for review. It may be the case that other interventions would need to be explored before HSCT could be considered. 

Even if HSCT is not immediately suitable, it may remain an option if circumstances change.

While the criteria for HSCT generally require less than 15 years since diagnosis, flexibility exists in certain cases. Patients with clear inflammatory activity and relapses, even beyond 15 years, may still be considered. However, the absence of new lesions or inflammatory activity significantly reduces eligibility, particularly for progressive forms of MS.

Some people explore commercial HSCT options abroad. It is essential to ensure the chosen facility adheres to recognised standards, such as JACIE accreditation. You should:

• Understand the specific protocols used at the centre
• Confirm the safety data and outcomes of the facility
• Coordinate follow-up care with their local healthcare providers

While Irish centres are generally supportive of people living with MS seeking HSCT abroad, it is crucial to have a clear understanding of the treatment and any follow-up requirements before proceeding. Please speak to your neurologist before making any plans to access stem cell therapy commercially. It is very important that a local haematologist also agrees to continue your care and monitoring when you return after a transplant to prevent or identify potentially serious complications.

There is limited data on the long-term efficacy of HSCT beyond five years. Some patients experience sustained remission, while others may see new disease activity after several years. Research indicates variability in outcomes, highlighting the importance of individualised care and ongoing monitoring.

Efforts are underway to establish a HSCT service within Ireland, reducing the need for candidates to travel abroad. A comprehensive Irish service would focus on:

• Adequate staffing and infrastructure
• Individualised patient care
• Monitoring both clinical outcomes and quality of life measures

Establishing a local service would not only improve accessibility to HSCT treatment in Ireland but also reduce the financial and logistical burdens on people living with MS and their families.

BEAT-MS Trial 

STAR-MS Trial 

A qualitative study on the experiences of autologous haematopoietic stem cell transplant for Multiple Sclerosis

Neuropsychological outcomes following HSCT in MS: A systematic review