The Greatest Challenges in Cell Therapy Today

Joining us in this Cell Therapy Bioprocessing & Commercialization Podcast is Jeff Karp - Associate Professor of Medicine at Brigham and Women's Hospital, Harvard Medical School. Jeff talks about the challenges facing the cell therapy space, how to improve cell homing following transplantation, and control cells among other things.

Listen to the Podcast | Access the complete transcript

What are some of the greatest challenges in cell therapy today? 

I think that we’re getting to a point in time where we can obtain almost any cell type in unlimited quantities with a few exceptions. This is using reprogramming, programming, different differentiation, protocols that have recently been worked out. So, I really think we are getting to that point where we can obtain nearly any cell type that can then be delivered to patients for treatment. 

While I think we’re there, I think one of the greatest challenges that remains is that once we transplant cells, we lose control over the cells. So, when we’re working with cells in a Petri dish, for example, we can pattern the media, we can put cells on all different types of textured substrates, we can control the media and the environment exquisitely. But, when we transplant cells, they are entirely at the mercy of the biological. So, if they end up in different tissues in the body, they are going to behave completely differently. So, we lose control of those cells following transplantation.

So, I think one of the greatest challenges is how can we now take the cells that we worked so hard to derive in vitro and then transplant those cells into patients and exhibit control so that the cells get to the right location and can perform their function when they get there.

[The above was an excerpt from the podcast]

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What Some Are Calling the “Holy Grail” of Stem Cell Research

Australian researchers have made what some are calling one of the most significant discoveries in the history of stem cell research. 

Thanks to a recent study involving zebrafish, researchers have uncovered how hematopoietic stem cells, one of the most critical types of stem cells, are formed.  These cells, also known as HSCs, are essential in replenishing the body’s supply of blood and immune cells and are key in transplants for patients with blood cancers.  The cells are thought to have the potential to treat a range of conditions because of their ability to transform into muscle, bone and blood vessels. 

An understanding of how these cells develop and regenerate is considered by many to be the “holy grail” of stem cell research and has applications for the treatment of spinal cord injuries, diabetes and degenerative diseases. 

The research team, led by Professor Peter Currie, from the Australian Regenerative Medicine Institute at Victoria’s Monash University, was able to uncover a major part of the cells development—something that had previously been a mystery to scientists.  The team had originally been studying muscle mutations in zebrafish but upon closer examination, they came across something perhaps more significant.  Thanks to transparent larvae of the fish, they noticed that a “buddy cell” appeared to help in the formation of HSCs.  These helper cells, known as Endotome cells, act as a “comfy sofa for pre-HSCs to snuggle into, helping them progress and even become fully fledged stem cells,” explained Currie. 

Currie said the focus of research can now turn towards finding the signals present in Endotome cells responsible for HSC formation in the embryo. 

“Then we can use them in the lab to make different blood cells on demand for all sorts of blood-related disorders,” he said.

While some are in fact calling this discover the “holy grail” of stem cell research, there is still some work to be done.  Said Georgina Hollway of the Garvan Institute of Medical Research in Sydney, “It’s difficult to say exactly how close we are, but we have uncovered a vital step in the process.”

What else is new in the field of cell therapy?  Join us for the Cell Therapy Bioprocessing conference, September 15-16, Boston, MA.  Download the agenda to see what’s on tap.

SAVE 20%* off the standard rate as a reader of this blog. Register here and use code XB14188BLOG.

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Researchers Regenerate Cornea, Successfully Grow Tissue from Stem Cells

A team of Boston researchers has identified a way to leverage limbal stem cells in order to regenerate human corneal tissue—One of the first examples of developing tissue from an adult-derived human stem cell.  The research, a collaboration between Massachusetts Eye and Ear, Boston Children’s Hospital, the VA Boston Healthcare System and Brigham Women’s Hospital, provides hope for those with eye-damaging injuries or diseases. 

The loss of limbal stem cells, which help maintain and restore corneal tissue, is one of the leading causes of blindness. 

It’s long been known that limbal stem cells have powerful regenerative potential, but the issue has been locating these stem cells.  The breakthrough came when Markus Frank, M.D. and Natasha Frank, M.D., co-senior investigators on the study, discovered the existence of ABCB5 molecules in limbal stem cells as well as their role in the maintenance and survival of the cell.  Scientists were then able to leverage the ABCB5 molecule as a marker for these cells and more easily identify them.  

The Frank team then put this theory to the test on two groups of mice--A group with fully functioning limbal cells and those without.  Using the presence of the ABCB5 molecule, researchers were able to locate and then extract these cells from donor tissue and transplant them into the corneal tissue of one of the groups.  They found that this group of mice was able to re-generate fully functioning corneas.  

The control group was divided in two with half the mice being given limbal cells that were ABCB5 negative and half receiving no limbal cells.  Both of these groups failed to repair the damaged tissue.  This helped to confirm ABCB5 molecule status as being responsible for the regenerative properties of these stem cells.  

“ABCB5 allows limbal cells to survive, protecting them from apoptosis [programmed cell death],” said Markus Frank.  “The mouse model allowed us for the first time to understand the role of ABCB5 in normal development, and should be very important to the field in general,” added Natash Frank. 

What’s next for the team?  Said Frank in a Fox News interview, “For the first step, we’re really working towards an autologous graft in patients who are blind in one eye.  And then the second step, we’d really work towards using donor derived cells to transplant in a similar manner that may require immune suppression – but it may not.” 

What else is new in the field of cell therapy?  Join us for the Cell Therapy Bioprocessing conference, September 15-16, Boston, MA.  Download the agenda to see what’s on tap.

SAVE 20%* off the standard rate as a reader of this blog. Register here and use code XB14188BLOG.

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What is the main challenge with the informed consent process as it relates to genetic research process?

Next week, the TIDES Event will take place in Providence, Rhode Island.  This week, we're revisiting some of the speaker interviews we conducted to prepare for the event. Today we look at the interview with Bertha deLanda, CIP - IRB/SCRO Panel Manager, Research Compliance Office at Stanford University.  She will be presenting Informed Consent and Genetic Research as a part of the Nucleic Acids Technologies for Molecular Diagnostics Workshop.

Today, Bertha shares her thoughts on the question:

What is an IRB and what do they have to do with the consenting process?

Well, in IRB or Institutional Review Board, it is the independent ethics review board that’s responsible for reviewing, approving, continuing with or rejecting any research that involves either human subjects or their identifiable private health information. It is composed of at least five individuals, one who must be an unaffiliated or public member, one who must be a non-scientist and other members who are doctors, lawyers, teachers, businessmen, etc. Because my panel also oversees studies that involve stem cell research, my panel includes BioE Thesis and researchers with a strong stem cell background. Also, sometimes when necessary, a panel member who is experienced in assisted reproductive technology. 

My background is in the genetics research field. Many of the PCR drug technologies I was involved with were genetic research-based. Their purpose was to diagnose, treat, identify or even, in some cases, cure diseases so that they would inevitably go to human clinical trials. Therefore, before writing a protocol that involves work with human samples, the scientists and researchers should do their best to avoid the pitfalls and retrospective issues that can occur when consenting, potential participants or their samples.

Read the rest of Bertha's interview here.

If you would like to learn more about this topic, join Bertha next week, May 12-15, 2014, in Providence, Rhode Island for the TIDES event. As a reader of this blog, when you register to join us and mention code XB14180BLOG, you can save 20% off the standard rate. Have any questions? Reach out to Jennifer Pereira.

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