Stem Cell Transplant

What are Stem Cells?

Stem cells are cells which are able to differentiate into other types of cells and are capable of ‘self-renewal’ or multiplying to produce greater numbers. Stem cell treatments are any therapies which target or use stem cells – usually to replace or repair damage.

What is Stem-Cell Treatment?

Stem cell therapy is any treatment that uses or targets stem cells. This is usually to help replace or repair damaged cells or tissues, but can also be used to prevent damage from happening in the first place.

Autologous Haematopoietic Stem Cell Transplant (AHSCT)

The aim of this treatment is to “reset” the immune system, so that it will stop attacking the person’s own central nervous system. AHSCT is a high-risk procedure, it has traditionally been used for the treatment of life-threatening cancers. Trials for MS have been taking place for over 10 years. Much of the scepticism surrounding HSCT is due to the early transplants raising a number of concerns about the safety and efficacy with several long term of life threatening complications. However more recent trials are defining the criteria for whom success is most likely to be achieved and greater emphasis on safety and efficacy.

Process of AHSCT

AHSCT is an aggressive and high-risk treatment which involves a number of steps, including:

• Collecting or ‘harvesting’ the AHSCs from the bone marrow or blood of the patient
• Purifying and concentrating the AHSCs in the laboratory
• Freezing or ‘cryopreserving’ the cells in the laboratory until they are required
• Administering high dose chemotherapy to deplete the patient’s immune system by destroying the remaining cells within the bone marrow (conditioning)
• Returning/reintroduction of the thawed AHSCs to the patient by infusion. Within the peripheral blood they should develop into red and white blood cells and re-establish their immune system.

Recommendations

Stem cells have generated a lot of excitement because the potential therapeutic benefits. There is real hope that stem cell transplants may assist in remyelination and may help protect the nervous system from immune attacks. HSCT is not currently available in New Zealand for the treatment of MS. MSNZ recommends that:

• Evidence supports benefits for those with early and active relapsing MS who are not responding to currently available treatments.
• Evidence does not seem to support positive use outside of these criteria.
• Currently funded DMTs should be used in the first instance for relapsing MS.
• Stem cell treatments should only be done in established centres that strictly adhere to the International Society for Stem Cell research guidelines and should be done in the context of a clinical trial where benefits can be measured and patients closely monitored for adverse outcomes.

Further Information

MS Australia

‘Stem Cell Treatments – a Quick Guide for Medical Practitioners’ – Australian National Health and Medical Research Council (NHMRC)

‘Stem Cell Treatments – Frequently Asked Questions’ – Australian National Health and Medical Research Council (NHMRC)