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

Adopt Me

I'm Fluffy

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Breed

Color

Age

Sex

Hair

mixed

Gray

Adult

Female

Medium

Adopt Me

I'm Ginger

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Breed

Color

Age

Sex

Hair

mixed

Orange

Adult

Male

Medium

Adopt Me