Evidence of Therapy
Photo Credit: Wikimedia
A forum on stem cell therapy in Duchenne would have little meaning if there was no evidence stem cells play a role in the disease. Here we present a representative table of clinical administrations of stem cells to Duchenne patients and the results they report. We also commence a table summarizing comments and critiques from experts on those studies. Our conclusion from results reported in over 1000 DMD patients: (1) certain stem cell administrations mitigate Duchenne progression, (2) stem cell therapies probably result in some life extension and (3) propositions concerning DMD causation have changed dramatically .
Stem Cell Therapy Providers
India, Neurogen Brain and Spinal Institute
Address: Station Rd, Sector 40, Seawoods, Navi Mumbai, Maharashtra 400706, India
Phone: +91 99202 00400
Clinical Therapy: Autogolous Mononuclear Bone Marrow Transplant: CD 34 cells.
Ukraine, EmCell
Address: 37 A Syretska street, Kyiv 04073, Ukraine
Phone: +38 044 223 28 95
Clinical Therapy: Allogenic Fetal Stem Cells (donor derived)
United States, FDA Compassionate Care Exceptions.
Address and phone: Contact Individual Patients/physicians
Clinical Therapy: Allogenic Stem Cells (donor derived)
STUDY
Patients
Age
Cells
Cell number
Route
Evaluation factors
Efficacy
Adverse events
2007, Italy
5
10 (avg)
Allogenic muscle-derived CD133+ cells
20,000
One infusion. Intramuscular.
Clinical, safety, biopsy. Readouts at 2, 4 and 6 months.
Very limited/none
No
2013, India
125
11.5 (avg)
Autologous Bone Marrow MNCs (note 1)
BW * 106
One infusion. Intrathecal L4-L5 and IM.
Clinical, CPK, MRI, EMG. Readout at 12month. (note 2)
88.8% improvement (note 3)
No
2014, India
1
9
Autologous Bone Marrow MNCs (note 4)
33*106
Four infusions. L4-L5 and IM.
Clinical, CPK, MRI, EMG. Readouts over 3 years.
Improvement over 3 years (note 5)
No
2014, Ukraine
27 study
15 ctrl
13 (avg)
Allogenic, cryopreserved fetal liver or brain stem cells.
>100*106
One infusion. IV and sub-cutaneous.
Brooke, ECG, respiratory capacity, ALT, AST, CPK, LDH. Readouts at 6 and 12 months
Improvements in both control and study group (note 6)
No
2015, Italy
5
8 to 12
Mesangioblasts from brother.
8 to 35 *108
4-6 infusions. Intra-arterial in upper and lower limbs.
Clinical, muscle biopsy.
None
Yes (note 7)
2018, Turkey
9
7 to 14
Allogenic Wharton jelly cultured MSCs.
2*106 cells/kg/dose
8 infusions. 4 intra-muscular and 4 intra-arterial.
Clinical, MRI, EMG, muscle biopsy (note 8).
Yes, initially (note 9).
No
2020, Poland
3
11, 12, 22
Cultured BM-MSCs and cultured myogenic stem cells from muscle biopsy. Donors: father, father, sister.
700 to 820 *106
One infusion. Intramuscular.
Clinical, histology, ELISA, EMG (note 10)
Some motor improvement, some dystrophin and new muscle fibers detected (note 11)
No
2007, Italy
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Patients
Age
Cells
Cell Number
Route
mixed
Gray
Adult
Female
Medium
I'm Fluffy
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Breed
Color
Age
Sex
Hair
mixed
Gray
Adult
Female
Medium
I'm Ginger
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Breed
Color
Age
Sex
Hair
mixed
Orange
Adult
Male
Medium