Here’s a look at a couple of stories that caught our eye this week:
Jasper Therapeutics has had a busy couple of weeks. Recently they announced data from their Phase 1 clinical trial treating people with Myelodysplastic syndromes (MDS). This is a group of disorders in which immature blood-forming cells in the bone marrow become abnormal and leads to low numbers of normal blood cells, especially red blood cells. We blogged about that here.
The data showed that six patients were given JSP191 – in combination with low-dose radiation five of the six had no detectable levels of disease and the sixth patient had reduced levels.
This was a big deal for us because CIRM funded the early stage research and even a clinical trial that led to the development of JSP191.
Now Jasper has announced it is partnering with the National Institute of Allergy and Infectious Disease in a Phase 1/2 clinical trial using JSP191, as part of a treatment for chronic granulomatous disease (CGD). Congratulations to Jasper. And congratulations to us for helping them get there.
Oh, and just to toot our horn a little bit more – it is Friday after all – we have funded other approaches to CGD including one that resulted in curing Brenden Whittaker.
OK, enough about us.
To say that this last year has been a stressful one would be something of an understatement. But it’s not just people who get stressed. Stem cells do too. And, like people, when stem cells get stressed they don’t always behave in the way you would like them to. When some people get stressed they find a cocktail can help take the edge of it. Apparently that works for stem cells as well!
Now we are not talking about slipping a Manhattan or Mai Tai into a petri dish filled with stem cells. We are talking about a very different kind of cocktail.
Researchers at the National Institutes of Health have developed what they describe as a “four-part small molecule cocktail” that can help protect a specific kind of stem cell from stress. The cell is an induced pluripotent stem cell (iPSC), which has the ability to turn into any other kind of cell in the body. iPSC’s have great potential for treating a variety of different diseases and conditions, but they’re also sensitive and without the right conditions and environment they can get stressed and that in turn can damage their DNA and lead to them dying.
In a news release Dr. Ilyas Singeç, the lead researcher, says this NIH “cocktail” could help prevent that: “The small-molecule cocktail is safeguarding cells and making stem cell use more predictable and efficient. In preventing cellular stress and DNA damage that typically occur, we’re avoiding cell death and improving the quality of surviving cells. The cocktail will become a broadly used staple of the stem cell field and boost stem cell applications in both research and the clinic.”
The team hope this could enhance the potential therapeutic uses of iPSCs in finding treatments for diseases such as diabetes, Parkinson’s and spinal cord injury.
The study is published in the journal Nature Methods.
All cells are easily exposed to variety of risk factors such as high temperature, hypoxia, heavy metal, chemical agents and antineoplastic drugs. Therefore, DNA is permanently subject to damage and many DNA repair pathways are involved in recognition and removal of a diverse array of DNA lesions. Hence, DNA repair mechanisms are key to maintain genome stability. The stress of cells are mostly related with development of Heat Shock Proteins (HSPs)in their cellular level, HSPs have cytoprotective function and antiapoptotic role. They are present in all living cells to participate in protein homeostasis, preventing protein denaturation and unfolding under normal and stressful conditions. HSPs have direct role of inducible DNA repair based on observation of MNNG and MNU trigger the decrease of mutation rate after HS. HSP blocked high frequency of mutation rate in Saccharomyces Cerevisiae induced by high temperature, suggests enzymes involved in repair of premutational damage may be more resistant to denaturation. In addition, cells exposure to UV and g-ray induced Hsp70, which may bind to p53, suggesting regulating activity on cell cycle, DNA repair processes and apoptosis. This is in contrast to most of the cancer cells which commonly overexpressed HSPs supporting the role of HSPs to promote cancer cell proliferation and apoptosis inhibition.
The culturing of stem cells in large groups or single cell cloning in cell culture plate consistently induced stress conditions are unavoidable. In vitro, stem cells are growing into different sizes of organoids. The lack of blood circulation in organoids cause the inner part of cells undergoing stress condition by depletion of oxygen supply and tend to die. This phenomenon of hypoxia is a key obstacle factor to hinder the potential therapeutic uses of stem cells in regenerative medicine. The development of small-molecule cocktail may further improve health conditions of stem cells but cells cannot be survive without oxygen.