Translational Research in Stem Cell Treatment of Neuromuscular Diseases

Table 4

Table summarizing the clinical trials on the treatment of MS with stem cells.

Phase	Stem cell	Followup	Results	Reference
I/II	HSCs	15 months	The number of lesions decreased on MRI. Clinically, patients improved slightly or remained stable	[112]
—	HSCs	1–1.5 years	A fall in intensity of motor and coordination disorders. No recovery of cranial nerve function was observed	[113]
I/II	HSCs	16 months	Positive early results	[114]
II	HSCs	12 months	EDSS improved in some patients. Only two patients had relapses Disappearance of enhanced T1 lesions on MRI	[115]
II	HSCs	24 months	Important clinical issues in the use of HDIT and stem cell transplantation for MS were identified	[116]
II	HSCs	36 months	All patients showed clinical stabilization or improvement. Quality of life improved	[117]
—	HSCs	2 years	Long-term suppression of inflammatory activity in MS patients who received HSCs is associated with profound qualitative immunological changes	[118]
—	HSCs	3 years	HSCs can reduce BDNF levels to values associated with lower activity	[119]
—	HSCs	41.7 months	Improvement or stabilization of neurological conditions in 63% of patients. HSCs was shown as a promising procedure to slow down progression in a subset of patients affected by severe, progressive MS	[28]
—	BMCs	19 months	Some degree of improvement in the sensory, pyramidal, and cerebellar functions noted	[120]
I/II	HSCs	37 months	All patients were free from progression (no deterioration in EDSS), and 16 were free of relapses. Significant improvements were noted in neurological disability	[27]
I	BMCs	12 months	EDSS improvement in 5/7, stabilization in 1/7, and worsening in 1/7 patients. Hints of clinical but not radiological efficacy and evidence of safety with no serious adverse events	[26]
—	HSCs	48.92 months	Confirmed relapse-free survival rate was 62.9% and progression-free survival rate was 83.3%	[121]
I/II	BMCs	≤25 months	Transplantation of MSCs in patients with MS and ALS is a clinically feasible and relatively safe procedure and induces immediate immunomodulatory effects	[81]
—	BMCs	—	Expression of FoxP3 at 6 months after intrathecal injection of MSC was significantly higher than the levels prior to treatment. Such significant enhanced expression of FoxP3 associated with clinical stability	[122]
—	BMCs	12 months	Improvement of EDSS by 0.5–1 points in 6/8, stabilization in 1/8, and progression in 1/8 patients	[123]
IIa	BMCs	10 months	There were no safety issues of stem cell application. The evidence of structural, functional, and physiological improvement after treatment in some visual endpoints is suggestive of neuroprotection	[124]