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Cell therapy clinical trials 2009 – part II

Fresh digest of published clinical trials and cases, which captured my attention in last 2 months. The feature of today’s review – absence of trials done in USA.


C-peptide levels and insulin independence following autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus [1] (Brazil)

After a mean follow-up of 29.8 months following autologous nonmyeloablative HSCT in patients with newly diagnosed type 1 DM, C-peptide levels increased significantly and the majority of patients achieved insulin independence with good glycemic control.

leukemia – donor cells control

Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study [2] (Europe – International)

In a phase I—II, multicentre, non-randomised trial of haploidentical stem-cell transplantation, we infused donor lymphocytes expressing herpes-simplex thymidine kinase suicide gene (TK-cells) after transplantation
28 patients received TK-cells starting 28 days after transplantation; 22 patients obtained immune reconstitution at median 75 days (range 34—127) from transplantation and 23 days (13—42) from infusion. Ten patients developed acute GVHD (grade I—IV) and one developed chronic GVHD, which were controlled by induction of the suicide gene.

read more [3]

leukemia – MSC infusion

Infusion of mesenchymal stromal cells can aid hematopoietic recovery following allogeneic hematopoietic stem cell myeloablative transplant: a pilot study [4] (Belgium)

In this pilot study, we evaluated the safety and efficiency of donor expanded MSC infusion after allogeneic hematopoietic stem cell transplantation (HSCT) in six patients with poor hematopoietic recovery.
Two patients displayed rapid hematopoietic recovery (days 12 and 21), and four patients showed no response. The two patients who showed hematopoietic recovery were in first complete remission compared to the other heavily pretreated patients. There were no toxic side effects linked to MSC infusion.
Our data suggest that patients with a less damaged stroma could benefit from this approach.

gene therapy

VEGF gene therapy fails to improve perfusion of ischemic myocardium in patients with advanced coronary disease: results of the NORTHERN trial [5] (Canada)

A total of 93 patients with refractory Canadian Cardiovascular Society (CCS) class 3 or 4 anginal symptoms were randomized to receive 2,000 microg of VEGF plasmid DNA or placebo (buffered saline) delivered via the endocardial route using an electroanatomical NOGA guidance catheter.
Despite the intramyocardial administration of a high “dose” of plasmid DNA using a percutaneous guidance catheter system, there was no benefit of VEGF gene therapy at 3 or 6 months for any of the end points studied.

read one more gene therapy FAIL [6]

neurology – spinal cord injury

Stem cells in the treatment of chronic spinal cord injury: evaluation of somatosensitive evoked potentials in 39 patients [7] (Brazil)

Thirty-nine consecutive patients with diagnosed complete cervical and thoracic SCI for at least 2 years and with no cortical response in the SSEP study of the lower limbs were included in the trial. The trial patients underwent peripheral blood stem cell mobilization and collection.
Twenty-six (66.7%) patients showed recovery of somatosensory evoked response to peripheral stimuli after 2.5 years of follow-up.

cancer – immunotherapy

Combination therapy of in vitro-expanded natural killer T cells and α-galactosylceramide-pulsed antigen-presenting cells in patients with recurrent head and neck carcinoma [8] (Japan)

The aim of this clinical trial was to investigate the feasibility of intra-arterial infusion of in vitro-expanded Vα24 natural killer T (NKT) cells combined with submucosal injection of α-galactosylceramide (KRN7000; αGalCer)-pulsed antigen-presenting cells (APC). A phase I clinical study was carried out in patients with head and neck squamous cell carcinoma (HNSCC). Patients with locally recurrent HNSCC refractory to standard therapy were eligible.
Regarding the clinical responses, three cases exhibited a partial but significant response, four were classified as stable disease, and one patient continued to develop progressive disease. The use of the intra-arterial infusion of activated Vα24 NKT cells and the submucosal injection of αGalCer-pulsed APC has been shown to induce significant antitumor immunity and had beneficial clinical effects in the management of advanced HNSCC. The use of such therapeutic modalities may be helpful in the management of tumors and therefore needs to be explored in further detail.

tissue engineering

Effectiveness of haemodialysis access with an autologous tissue-engineered vascular graft: a multicentre cohort study [9] (International)

Application of a tissue-engineered vascular graft for small-diameter vascular reconstruction has been a long awaited and much anticipated advance for vascular surgery. We report results after a minimum of 6 months of follow-up for the first ten patients implanted with a completely biological and autologous tissue-engineered vascular graft.
Overall, primary patency was maintained in seven (78%) of the remaining nine patients 1 month after implantation and five (60%) of the remaining eight patients 6 months after implantation.

cases reports:
Allogeneic mesenchymal stem cells transplantation in treatment of multiple sclerosis [10]
Non-expanded adipose stromal vascular fraction cell therapy for multiple sclerosis [11]
Histological findings on fetal striatal grafts in a Huntington’s disease patient early after transplantation [12]


also read:
Cell Therapy Clinical Trials in 2008 – part I [13]
Cell Therapy Clinical Trials in 2008 – part II [14]
Cell therapy clinical trials in 2009 – part I [15]
more clinical trials [16]

picture credit: “Display Direct” via Flickr [17]

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