This is a 4th issue of Cells Weekly. Please follow us for the most significant news in stem cell research and cell therapy!
1. Hematopoietic stem cell transplantation could increase survival in systemic sclerosis
Preliminary data from Phase III of ASTIS clinical trial were reported this week at the EULAR 2012 Annual Congress:
As of May 1, 2012, significantly more deaths have occurred in the conventional treatment group. Half of the deaths in the HSCT group occurred early and were deemed treatment-related according to an independent data monitoring committee. In the conventional treatment group in contrast, none of the deaths were deemed to be treatment-related; but more deaths occurred later and most were related to progressive disease.
2. Discussion on ESC-derived retinal cells transplantation in ACT trial
As you know, Advanced Cell Technology (ACT) is running 3 clinical trials, assessing embryonic stem cell-derived retinal cells in eye diseases. This week, the Guardian posted an interesting piece about stem cell trials and highlighted the 3rd patient in the ACT trial (and first UK patient) – Marcus Hilton. Guardian’s post uncovered some potential outcome of stem cell treatment:
He was diagnosed when he was about 10, after various pairs of glasses did no good at all. Now 34, he recalls: “I could never see the blackboard. I was blagging my way through school – native wit.”.
Very interesting discussion took place in Lancet – a medical journal, where ACT has published preliminary results of stem cell treatment of first two patients.
Based on the ACT trial design, the first group makes a gloomy predictions:
According to this model, transplanted hESC-derived retinal pigment epithelial cells would be unlikely to survive, exposed as they are to the same pathological mechanism that affects the host cells.
Since none of these mechanisms is corrected by the proposed treatment, any initial benefit could be followed by a progressive degeneration of transplanted retinal pigment epithelial cells.
The second group addresses the issue of clinical tests to measure outcome:
… Although these methods are standard clinical practice, they are unreliable and difficult to scale.
… Thus, the success of this surgery depends on the subjective observations of the patient with Stargardt’s disease. Although interesting, the finding needs objective confirmation.
And the authors replied:
We wish to clarify some points in response to Jinhai Huang and colleagues. At no point in our paper do we claim evidence of a therapeutic response to the transplantation of hESC-derived retinal pigment epithelium into our two patients. The report focuses on preliminary safety findings. Functional improvements seen in our patients are qualified as very difficult to measure and as perhaps being due to other factors, including the placebo effect.
One may wonder, why the a letter to editor and discussion in the journal has happened almost 6 months after publication? One may think, that scientist need time to think to write 2 paragraphs. Think hard and long time. As long as 5 months. Wouldn’t it be awesome if it could happen right the next day after publication online?
3. Bone marrow cells in hemorrhagic stroke – results of clinical study in China
You can read results of this study here. 100 patients were included, 60 underwent the procedure (40 served as control):
Autologous bone marrow mononuclear cell implantation reduced neurological impairment and improved activities of daily living in a selected group of ICH patients.
4. Adipose stem cell clinical trials analysis
Leeza Rodriguez has joined our cell therapy clinical trials network. She analyzed and posted adipose stem cell clinical trials, registered in ClinicalTrials.gov.
Each week I plan to publish data or charts from my spreadsheet. The charts below represent a few data sets from the spreadsheet. I have read and collected info about each clinical trial to include sponsor, trial status, country, donor source, disease indication, cell expansion, stem cell dosages, etc. so this could get very interesting!
Don’t miss her updates! Highly recommended!
5. The quality of hematopoietic cells after long-term cryogenic storage
The biggest study assessing the quality of bone marrow and mobilized blood products after 11-19 years or storage in liquid nitrogen, has been published. Interestingly, the authors demonstrated profound decline in viability, but not in colony-forming ability. The study brought up the question about differences in quality of hematopoietic products, derived from cord blood, bone marrow and mobilized blood after cryo-storage. Also, what the impact of these findings on clinical use and outcome? Of course, the study should be yet reproduced, but it was the first significant attempt to assess quality of hematopoietic cell products, derived from bone marrow and mobilized blood.
6. The field of stem cell-based R&D products gets even hotter
With 2 more new products coming on the market. The first product is actually a service provided by CDI – MyCellTM.
The second product is human iPS-derived neurons modified to carry Alzheimer’s disease related gene by ReproCELL.
Both products will be introduced to professionals next week on upcoming ISSCR annual meeting.
7. A press conference on the perspectives for treatment of HIV by CCR5-mutated cord blood
Lawrence Petz (a transplant physician) and Timothy Brown (the first patient who was treated from HIV) gave a public press conference on the future perspectives of HIV treatment by CCR5-mutated cord blood. The “Berlin patient” case has inspired some professionals to start to screen cord blood inventories for potential library of CCR5-mutated (HIV-resistant) samples.
“At the present time, I feel there’s no other way to cure a reasonable number of patients other than using cord blood,” Petz said.
Two weeks ago, a patient in the Netherlands was the first to undergo this potentially revolutionary treatment. As was the case with Brown, the transplant was primarily done to address another disease, but doctors specifically selected a unit of cord blood that contained the HIV-resistant gene in hopes of curing that as well. Another similar surgery is scheduled for a patient in Madrid within the month.
Petz explained that as of now, the treatment isn’t meant for all HIV patients. The inventory of cord blood units that carry the HIV-resistant gene – 100 out of 17,000 tested so far – needs to be built up over time.
Petz said he believes HIV patients with other hemolytic disorders, such as Brown and the Netherlands patient, and AIDS patients who do not respond well to current drugs on the market, should be considered for the transplants.
8. Adipose-derived stem/ progenitor cells facilitate cancer development
New study, published in online version of PNAS, made some buzz in the mass media. Nothing actually new here (read here, here, here, here and here), but the current widespread clinical use of “adipose tissue stem cells” captures attention to any new studies like that:
Stem cells often used in reconstructive surgery following mastectomies and other cancer-removal treatments may pose a danger…
9. Welcome a new stem cell blogger – Jennifer Wong
This week I was enjoying reading posts on Life-Dialogues blog. Significant part of the blog dedicated to stem cell research. I’d recommend you to read some of Jennifer’s posts:
Elusive Role of “Stemness” in Cancer
iPSC Reprogramming: Genomic Hardware Friendly but marred with Epigenetic Software Bugs
10. The best of the week in mass media
Our Misunderstanding of Stem Cells post by Rui Dai for HuffPost Science. Sobering debunking of the over-hyped news:
I love stem-cell science. That is what I plan to work on for my future career. However, our current understanding of stem cells is leading the public down the wrong path. The future cure for heart disease will not be found in 10 to 15 years by transplanting heart cells developed from skin cells.
It is “a beautiful but unworkable idea”, says Smith. “The cost of generating these kinds of therapies for an individual is completely unrealistic. At the present time we have no model of how that could be done. It is so expensive.”
“So the real use of IPS cells is to make them from diseased patients and use them in the lab to develop drugs,” says Smith
That’s all for today guys. It was very long, but I hope you will enjoy reading.
If you read it all to the end – a bonus track: