stem cell therapy and clinical

application Clinical Trial.Gov

Jeanne Adiwinata Pawitan

Department of Histology FKUI
2015

Jeanne Adiwinata Pawitan

Autism
 3. Autism
Safety and Efficacy of Stem Cell Therapy in
Patients With Autism
Responsible Party: Shenzhen Beike BioTechnology
Co., Ltd.
ClinicalTrials.gov Identifier: NCT01343511 History
of Changes
Other Study ID Numbers: BKCRAUTISM1.0(2009)
Study First Received: April 26, 2011
Last Updated: October 13, 2011
ClinicalTrials.gov processed this record on February
12, 2015
 Autism: 3-12 th
DSM IV CARS 30
Group 1: rehab th + hu UCB-MNC
Group 2: rehab th + hu UCB-MNC + hu
UC-MSC
6 months
Jl sel cara pemberian (-)
Publikasi: autism
 Publikasi: Yong -Tao Lv et al
4x pemberian - interval 5-7 hari
Allo-UCB-MNC 0.2-1% CD34- 2x 106 /kg
BB
Allo-UC-MSC - 1x 106 /kg BB
Normal saline
20 mL IV
2 mL intra thecal
UCB-MNC 1x IV 3x IT
UCB-MNC + UC-MSC 2x IV +IT 2x IT
 Autism- Yong -Tao Lv et al
Metode:
37 14 single 9 double -14 contr
Hasil:
no severe SE
CARS, ABC score, CGI:
double > single treatment > control
Alzheimer
 4. Alzheimer disease
Medipost co Ltd Seoul-Korea
50-75th dementia DSM IV criteria,
Alzheimer NINCDSADRDA criteria, KMMSE
score 10 24, PIBPET imaging (SUV > 1.5
cerebellum vs frontal lobe)
Feb-Dec 2011 - Safety-efficacy
Single group - open label study
Safety study maximum tolerated dose (MTD)
Efficacy study
 Alzheimer disease
Neurostem -AD (hu-UCB-MSC)
DOSE A: 250,000 cells /5 uL/ entry site - 3
million cells per brain
DOSE B: 500,000 cells/ 5 uL/ entry site - 6
million cells per brain
Side effect sesudah 12 minggu
Pemeriksaan: baseline 12 minggu:
ADAScog, SIADL, KMMSE, CGANPI,
serum transthyretin, amyloid beta and tau in
cerebrospinal fluid, PIBPET, FDGPET
Neurostem AD
Parkinsons disease
 1. Parkinsons disease
Inclusion
PD-motor complication Jaslok-India
min 5 th Auto BM-MSC
levoDOPA responsive- Dosis: ???
On-off - UPDRS
Cara: stereotaktik
(unified Parkinsons
disease rating scale) striatum
min 33% Fase 1: 18 bulan
35-70 th -Dll UPDRS + time test
Exclusion Mulai Juli 2009
Intra cranial surgery R/ 5 pasien
sp. 2th yl, dll
suspended
Cerebral palsy
Cerebral palsy
 Cerebral palsy-phase-1
18 pasien anak- G-CSF BM-TNC (total
nucleated cell) baseline - 6 bulan
Intrathecal: medianTNC: 13.12 108 (4.83-53.87) -
mengandung CD34+: 10.02 106 (1.02-29.9)
volume 7 mL (4-10.5)
IV: median TNC: 6.01 108 (1.36-17.85)
mengandung CD34+: 3.39 106
Side effect: headache, vomiting, fever and stiff neck
3 pasien dianggap aman
4.7 2.63 month increase in developmental age
according - Battelle Developmental Inventory
 4. Cerebral palsy
Intrathecal Stem Cells for Cerebral Palsy Phase II
(ISCII) randomized single blind (assessor)
Hospital Universitario Dr. Jose E. Gonzalez
Mulai Sept 2013 Juni 2016 - 60 subyek - (7 - 9th)
G-CSF 10 g/KgBB-3 hari berturut-turut auto
BM? buffy coat (10 mL) intrathecal
Acute side effect: malaise, headache, fever, and
nausea or vomit, etc
Outcome measures: progress in GMFCS (gross
motor function classification system) baseline- 1-
3-6 bulan
DM
Osiris therapeutics: BM-MSCIV
Prochymal (remestemcel-L)
DM
DM: beta cell viacyte
COPD
COPD
 COPD
Sao Paolo Brazil
COPD pulmonary emphysema -40-72 th
G-CSF 5 g/kg SC 3 days
BMP 151-202 mL MNC 108/mL 50
mL strainer 100m
IV- medial brachial vein 30 mL 20 menit
7 hr 1-2-3-6-12 bulan
Adverse effect (-)
Improvement
Liver cirrhosis
Liver cirrhosis
Liver cirrhosis
 20. Improvement of Liver Function in Liver Cirrhosis
Patients After Autologous Mesenchymal Stem Cell
Injection: a Phase I-II Clinical Trial
Shahid Beheshti Medical University-Tehran-Iran
Feb 2006 juni 2009 30 pasien
Randomized - single group - single blind
Safety efficacy study
D/ liver failure cirrhosis usia > 18 th, MELD score 10,
color Doppler: patent portal vein, serum fetoprot N,
liver USG no hepatocellular ca
Auto-MSC progenitor hepatocyte injection portal
vein USG guide - dosis???
Outcome: Liver function test, MELD score, mortality after 6
months
Kidney disease
1. Polykistic- chronic renal failure
Inclusion
Royan Institute - Iran
CRF
Autosom dominant PKD IV-auto BM-MSC
Genetic testing dose: 2.000.000/kgBB
Sonografi
Fase-1: 1-3-6-12-18 bl
GFR 25-60/men/1.73m2
mass formation
18-60th
GFR (6 bl)
Exclusion
recruiting 6 orang
Hamil-laktasi
Penyebab: DM, Ca,
Mulai April 2014
autoimun
DO
 Renal failure

Kidney transplant + HSC end stage

renal disease not yet recruiting
MSC kidney transplant chronic
allograft nephropaty unknown
SC + alemtuzumab + transplant
cegah rejection selesai belum
dilaporkan
 5. Chronic kidney disease
Inclusion Royan Institute - Iran
CKD symptom IV-Auto--BM-MSC
Serum-urine analysis Dosis: 2.000.000/kgBB
GFR 25-60/men/1.73m2
phase-1: 1-3-6-12-18bl
40-60 th
mass formation,
Exclusion creatinin (1 bl)
Hamil-laktasi GFR (6 bl)
penyebab: DM, Ca,
recruiting - 10 org
autoimun
DO Mulai april 2014
 SC macular degeneration
ESC retinal pigment epithelium
iPSC retinal pigment epithelium
iPSC Macular degeneration
Rheumatoid arthritis
 References
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