Stem cell stories that caught our eye: a tool toward safer bone marrow transplants, Parkinson’s and a Spinal cord clinical trial.

A stem cell treatment for TB? A report this week from Sweden suggests yes.

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. 

Artificial bone marrow. Stem cell transplants using cells originating in bone marrow have proven to be dramatic lifesavers in several forms of cancer. Problem is too few people needing donor cells can find a match or even a partial match and for those with only a partial match, the procedure is still often deadly because of immune complications. This has led countless teams to search for ways to mass produce matched bone-marrow stem cells, generally with no or minimal success. Part of this results from not knowing how stem cells interact with other cells in their natural niche: the bone marrow itself. Now a German team has created an artificial scaffold that mimics the structure of the marrow and seeded it with relevant support cells and shown they could get stem cells from cord blood to feel at home there and behave normally in the lab. This becomes a great research tool to let teams figure out how to grow the needed stem cells. It was written about on ScienceDaily.

Genetic cause of Parkinson’s. CIRM funded researchers at the Gladstone Institutes have used two new technologies, a one-of-a-kind robotic microscope and the ability to reprogram the skin cells of a patient into stem cells, and scrambled several assumptions in Parkinson’s disease. Using the microscope to peer into the inner workings of nerves grown from the Parkinson’s-derived stem cells they found that some commonly talked about players in the cause of the disease were not behaving as expected. The patients had the most common genetic mutation linked to the disease, LRRK2. Looking at that protein, as well as the suspect fellow culprits alpha-synuclein and inclusion bodies, the researchers found an interplay that bucked conventional wisdom. The biggest culprit was LRRK2 but if you eliminate its accomplice, the alpha-synuclein, it can’t murder the nerve. Gladstone released a press release about the study. You can read about other work CIRM funds in the area at our Stem Cells for Parkinson’s Fact Sheet page.

Stem Cells hint of hope in TB. The thought that stem cells could help treat multi-drug resistant tuberculosis is, quite frankly, counterintuitive for me. But Stockholm’s Karolinska Hospital is reporting preliminary—make that very preliminary—results suggesting stem cells might be able to significantly improve a patient’s ability to clear the TB bacteria with antibiotic therapy. They used the patients’ own mesenchymal stem cells, the second type of stem cell found in bone marrow. Those cells have a proven ability to tamp down inflammation and modulate our immune response. One hypothesis in this work suggests that the lungs of these patients are so inflamed it is hard for the antibiotics to do their job. Reducing this inflammation may help. Sixteen of 30 treated with the combo cleared the infection within 18 months. But only five of 30 in the antibiotic-only group did. Reuters ran one version of the story, and medpageTODAY ran a more detailed account reviewing some prior efforts to combine antibiotics and immune therapy for drug resistant TB.

First North American patient in spinal cord trial. It is always fun to report a company supported by CIRM is advancing its clinical trial work with patients. StemCells Inc. today announced that it had enrolled and treated its first North American patient in a spinal cord injury trial that had previously only been treating patients in Switzerland. A Canadian, treated at the University of Calgary, became the ninth person enrolled, and the company press release on the patient was picked up on Yahoo Finance. We wrote about the preliminary results from the first three patients in this spinal cord injury trial. CIRM funded some of the basic science underpinning this trial and our board had voted to help fund this North American trial, but the company decided to proceed with its own funding sources for the trial.

A personal tale of stem cell progress. For stem cell junkies, LifeScientist published a detailed interview with one of the stars of the field Martin Pera. He led the CIRM-funded stem cell center at the University of Southern California for the first half dozen years of its existence and now leads a similar effort in Australia—a return engagement for him. The U.S. born scientist had been working in Oxford when he was recruited to Australia in 1996 by the man who would later become our President, Alan Trounson. They set out to isolate human embryonic stem cells (hESCs), which they had done in 1998, but had not put all the finishing touches on the work prior to the now-landmark publication reporting isolation of hESCs by Wisconsin’s James Thomson. The piece is a nice personal take on how the field has progressed.

Video update on a trial for ALS/Lou Gehrig’s disease. Reuters has posted a video update of the Neuralstem clinical trial for patients with ALS that has been underway for the past couple years at Emory University in Atlanta. You can read about work CIRM funds on the disease at our ALS Fact Sheet.

Documents surface on questionable Italian clinical trial. We have written in the past about a clinical trial in Italy, with on-again-off-again support from the Italian government, that has rallied much of the nation’s scientific community against the intervention. Now, the journal Nature has been given documents that seem to verify many of the concerns raised over the past year. The journal shared its report with Scientific American’s web site.

Don Gibbons

A stem cell researcher’s dilemma, to tell or not to tell

Jackie Ward is a stem cell researcher at U.C. San Diego. She recently came across something that caused her to pause and think about her responsibilities as a scientist. It’s an issue that most scientists have to face at some point or other in their careers. This is her story

Among the multiple cat pictures that I receive weekly from my parents, my mother also recently forwarded me a link from our local small town newspaper. This article was about a fundraiser being held to financially support stem cell treatment for a young local resident who suffered from severe brain trauma. This is just one of several articles I’ve seen of this nature from my hometown in the past few years.

I am from a conservative Southern state and my hometown is far from any large research area. Growing up in my town of 15,000 people, I knew not a single scientist, much less anybody that studied stem cells. I wondered if this family had contacted anybody who was an expert in the stem cell field to discuss this treatment. Was it my responsibility to contact them?

I did a bit more research (that’s my nature as a PhD student after all) on the clinic this patient planned on attending. This clinic claims to isolate stem cells derived from body fat – also called adipose-derived – to treat many kinds of diseases. Though they do not directly claim to cure any diseases, there are links on the website that imply treatment of up to 30 different conditions, ranging from HIV to Parkinson’s disease to arthritis.

It is not stated anywhere how the stem cells are isolated, why they would be effective, or that long term analyses of the patients indicate any improvement in their condition. In short, they lack all the measures that would typically indicate that this procedure is a safe, effective, scientifically-based treatment.

I wouldn’t consider myself an expert on regenerative medicine, but I do have a scientist’s understanding of the field. As a PhD student, my work focuses mainly on using stem cells to model neurological diseases. However, thanks to many classes and general interest, I have learned a lot about regenerative medicine and what the current state of stem cell therapy is. The International Society for Stem Cell Research has a nice primer about this on its website; and the CBS-TV show “60 Minutes” also did a great segment on this. In summary, the only current proven use of stem cell replacement as therapy involves the use blood stem cells to treat disorders of the blood and immune system. This is not to say that stem cells won’t one day be used to treat a much larger range of diseases, it is simply that a new field requires more time to come to full fruition.

I asked myself “is it my responsibility as a stem cell scientist to disseminate these concepts to those in my community?” Normally I can answer this question quite easily. I believe I have an obligation to reach out to the community to translate complex scientific concepts and to offer my perspective on my field of expertise.

I had never before considered that doing so could be unwelcome or cause hardship but I wondered if by explaining my knowledge of regenerative medicine to this family, would I potentially be dashing their hopes of treatment for their son.

I wrestled with this question for some time and in the end I decided to write to the family representative of this patient. To me the benefits of doing so outweighed the detriments. It is still my responsibility as a scientist to communicate with the public in good situations or bad.

I explained my knowledge of the stem cell field and expressed concern at the risks—medical, financial, and emotional—associated with this kind of treatment. The brief reply from the family underlined their strong desire of instilling hope for the patient despite the risk that there would be no real medical benefit. There’s no argument that this is a valiant cause. Hope is a powerful force, and can have profound effects. However, I believe that hope can be instilled by other methods, and not expensive, scientifically unsound treatments.

My attempt at helping this family didn’t affect their decision or how they proceeded in any way. As a scientist, I believe the money raised for this cause could be better spent on effective treatments like intensive physical therapy or support groups or paying already incurred hospital bills. Yet, even though it did not have an impact on this family, that doesn’t mean I wouldn’t do it again. I feel that all scientists have an obligation to give back to society in whatever way they can. People may not always listen to us, but at least they will have had a chance to base their ultimate decision on sound science rather than just pure hope.

Jackie Ward

Jackie took part in our Elevator Pitch Challenge last year and described the kind of work she is doing and why it’s important.