Hold your horses - HSCT is still an experimental treatment
ECTRIMS Online Library. Hauser S. 09/11/19; 279383; 31
Stephen Hauser
Stephen Hauser

Abstract: 31

Type: Hot Topic

Abstract Category: Hot Topic 4: HSCT and stem cell treatment in MS

S. Hauser

UCSF Weill Institute for Neurosciences, Department of Neurology, University of California, San Francisco, CA, United States

Marked improvements in the long-term course of MS are associated with the availability of increasingly effective therapies against the focal inflammatory (relapsing) component of the disease. This represents one of the great achievements of modern medicine. However, despite recent incremental successes, development of effective therapy against the degenerative (progressive) component of MS remains a largely unmet challenge. There is great interest in determining the value of highly aggressive therapy delivered as early as possible in the disease course, analogous to a cancer therapy model in which induction of a sustained or permanent remission is the goal.
Advantages of human autologous stem cell transplantation (HSCT) for MS are several: high effectiveness against relapsing disease; a single administration only can yield a prolonged benefit lasting years, an appealing prospect for patients and payers; and mechanistic studies indicating that some degree of 'reprogramming' of the immune system has taken place. Potential disadvantages must also be considered, including: serious safety concerns, especially with more intensive conditioning regimens; lack of reliable long-term follow-up; little evidence supporting effectiveness in advanced MS cases; and uncertain data that autoreactive T or B cells are permanently deleted with HSCT.
We are entering an era where relapses are well-controlled in the vast majority of patients. Looking ahead, the relative value of treatment options will likely be based primarily on their effect against progression, which occurs throughout the disease course and in all forms of MS. Demonstrating long-term protection against progression, monitored clinically but supported by MRI measures and useful biomarkers will be key. It is also reasonable to expect that 'cure' would require elimination of all evidence of immune hyper-responsiveness in the CNS, including heightened immunoglobulin production and oligoclonal bands in the CSF of treated patients, an outcome that has not yet been convincingly demonstrated with any MS therapeutic agent.
Disclosure: Dr. Hauser serves on the Scientific Advisory Board (SAB) for Symbiotix, Annexon, Bionure, Alector, and Molecular Stethoscope, and Board of Trustees (BOT) for Neurona Therapeutics. Dr. Hauser also has received travel reimbursement and writing assistance from F. Hoffman-La Roche Ltd. for CD20-related meetings and presentations.

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