I was put in contact with someone with MS who was about to spend ~£60,000 by going abroad for HSCT. She lives in one of the home counties, i.e. a county that abuts on the outer perimeter of London, and hence could be referred into one of the London MS Centres for an opinion. In summary, she is on natalizumab and had a relapse. She was told by her consultant that as her MRI had no enhancing lesions she was not eligible to be referred for HSCT. This is incorrect the London MS-AHSCT Collaborative Group’s current eligibility criteria for AHSCT (autologous haematopoietic stem cell therapy) state either the presence of Gd-enhancing lesions or an increase in T2-lesion load.
Please note that the London MS-AHSCT stress that they currently view this as an exceptional therapy for some people with MS, rather than a standard treatment; and that neither NICE nor NHS England have ‘green lighted’ this therapy for routine use in any form of MS. However, MS is green-lighted as one of the autoimmune diseases that could be treated by haematologists under their own NICE guidance. However, there is a current NICE health technology appraisal proposing this at present.
If you are interested in this treatment it is important that you consult with your Neurology Team in the first instance.
Patient Eligibility Criteria Adopted by the London MS-AHSCT Collaborative Group
The eligibility criteria are overall aimed at selecting patients who have failed approved treatments of high efficacy or have none available to them and have recently presented evidence of inflammatory CNS disease activity; and who could undergo AHSCT with an acceptable estimated level of risk of adverse events. Justification for each of criteria is supported by evidence from AHSCT trials and observational studies.
I. Diagnosis of MS made by a neurologist
II. Able to walk, needing at most bilateral assistance to walk 20m without resting
III. In relapsing MS (RMS), failed one licensed disease modifying drug of high efficacy (currently including alemtuzumab and natalizumab) because of demonstrated lack of efficacy
IV. New MRI activity within last 12 months
2. Disease duration ≤15 years from diagnosis of MS
3. Diagnosis of MS according to McDonald’s criteria
4. For PPMS, CSF OCB+
5. For RMS, failed at least one licensed disease modifying drug of high efficacy (‘Category 2’ as defined by Scolding N, Barnes D, Cader S, et al. Pract Neurol 2015;15:273–279; currently including alemtuzumab and natalizumab) because of demonstrated lack of efficacy (as evident from relapse, MRI activity as defined below at Point 7, or EDSS increase) after being on DMT for at least 6 months
6. EDSS score 0-6.5
7. Inflammatory active MS as defined by ≥1 Gd+ (>3mm) lesion (off steroids for one month) or ≥2 new T2 lesions in MRI within last 12 months
8. Approved by the MDT
a. Eligible for an ethically approved clinical trial where AHSCT is offered as one of the treatment arms
b. Unable to adequately understand risk and benefits of AHSCT and give written informed consent
c. Prior treatment with total lymphoid irradiation and autologous or allogeneic hematopoietic stem cell transplantation London MS-AHSCT Collaborative Group – Patient Eligibility Criteria Final V.3. – 8/12/2015
d. Contraindication to MRI including but not limited to metal implants or fragments, history of claustrophobia or the inability of the subject to lie still on their back
e. Poorly controlled depression or recent suicidal attempt
f. Presence of any active or chronic infection
g. Unable to walk 20mt with or without support, or wheelchair dependent
h. Any significant organ dysfunction or co-morbidity that the Investigators consider would put the subject at unacceptable risk