Reflecting on Regenerative Medicine As A Brand

Stem cell-based Regenerative Medicine has huge power for good in the clinic and at the same time great potential for economic development. Those two things go hand in hand.

As I argued in my stem cell book, biotech commercialization of stem cells is not a bad thing, but rather if it is done responsibly it is a good, necessary part of advancing therapies to help patients.

From an economic perspective  stem cell-based Regenerative Medicine is a new, exciting brand.

It’s a brand, but a unique one because it is shared by so many diverse parties and influenced by tens of thousands of people and companies around the world. “Regenerative Medicine” is not owned by any one person or company.

Regenerative Medicine brand

A good analogy would be to “Soft Drinks” rather than to “Coke” or “Pepsi”. Soft Drinks are what we might call an “umbrella brand”. A higher-level brand. It’s important to point out that if “soft drinks” as an umbrella brand get a bad name as being linked to diseases and being harmful to health, for example, it is bad for all soft drink companies…or to extend the analogy all car companies or all airline companies, etc. .

The same kind of thing is true for Regenerative Medicine as an umbrella brand. If some folks give it a bad name, it hurts everyone with an interest in the brand including current and future patients.

There is a shared collective interest in promoting this brand and enhancing the public’s knowledge and positive, evidence-based perceptions of Regenerative Medicine. Of course there are specific organizations with the words “Regenerative” and “Medicine” in their name such as the great journal Regenerative Medicine (disclosure, I’m on the editorial board), CIRM, the Alliance for Regenerative Medicine (ARM) and many others so perhaps the stakes are highest for them on this brand, but it still has a powerful, more global impact. Without commercialization of stem cells we collectively cannot get safe, effective, evidence-based therapies to patients.

Our brand “Regenerative Medicine” faces some serious challenges even as it advances. These challenges include threats to the perception of the brand as scientific and trustworthy. These come from scandals such as STAP and others that do not reflect the ethos of our field but disproportionately and negatively influence the Regenerative Medicine brand.

Publicity is good for Regenerative Medicine, but hype isn’t so we need to draw a line. As much as we all want to spread the word about stem cells and how exciting they are, there is such a thing as going too fast, cutting corners, or hype.

Dubious stem cell clinics are another threat to the Regenerative Medicine brand. I don’t think the legit stem cell/regenerative medicine field is doing enough to counter these clinics as they spread like mushrooms all over the world including in the US.

As much as there are threats and challenges to Regenerative Medicine, it is also an incredibly, exciting and even revolutionary time for this brand and its associated fields of science. I usually don’t think of Regenerative Medicine as a brand, but I believe that now and then viewing it from that perspective is a helpful, healthy exercise.

Disclosure–I own no stock or positions in stem cell or regen med biotechs.

Some cool recent stem cell papers: recommended reading

What have been some recent stem cell and/or regenerative medicine papers that are worth some extra thought and reading?

There have been some cool ones.

There was of course the very important Mitalipov group paper comparing NT hESCs to IVF hESCs and hIPSCs. See my review here.

There was a great paper on the role of HDACs in mESCs.

A paper reporting making endothelial and hematopoietic cells from pluripotent stem cells with a combination of GATA2 and other individual transcription factors.

In vivo reprogramming in pigs reportedly leads to transient production of pacemaker cells in hearts.

Planarian worms have the remarkable ability to regrow entire worms when sliced into tiny pieces and they do so via pluripotent stem cells called neoblasts. A new paper demonstrates that not all neoblasts are created equal and different clones exhibit specific fate preferences.

What recent papers would you recommend on stem cells or regenerative medicine?

 

StemCells, Inc. Faces Lawsuit With Serious Allegations

stemcellsinc-logoStemCells, Inc. is one of those stem cell-related biotech companies that we in the field hope will become a big success and help many patients in the future via stem cell technology.

There are a lot of obstacles facing such companies. Some are privately held, while others such as StemCells, Inc. are publicly traded (stock symbol STEM). Unfortunately, StemCells, Inc. has had to face two rather serious problems in the last few weeks.

First, former CIRM President Alan Trounson joined the leadership of StemCells, Inc. only days after leaving CIRM despite the fact that CIRM had granted the company almost $20 million during Trounson’s tenure as President. This move has faced criticism (e.g. see here) because of the appearance of a potentially serious conflict of interest. It appears CIRM was not aware of Trounson’s joining the StemCells, Inc. board until the news came into the public domain.

Second, today Courthouse News Service announced that a former StemCells, Inc. manager, Rob Williams, has filed suit against the company. David Jensen has also reported on this breaking case here and here.

Williams alleges potentially serious problems with the company’s procedures. He also claims wrongful termination and retaliation. The latter allegations center on the claim that once he made upper management aware of his concerns about the stem cell lines and manufacturing practices that he was terminated shortly thereafter. The actuall full lawsuit file can be read here (PDF; hat tip to Jensen).

From the suit, quoted on Jensen’s blog, come very serious and as yet unsubstantiated accusations:

“’Shortly after beginning his employment, plaintiff noted poor sterile technique, failure to adhere to current Good Manufacturing Practices in the company’s manufacturing process, and substantial deficiencies in the company’s Manual Aseptic Processing of HuCNS-SC (Human Central Nervous System Stem Cells) cell lines – failure and deficiencies that put patients at risk of infection or death during ongoing clinical trials,’ Williams says.

Update: StemCells, Inc. via Jensen’s blog, has issued a statement denying the charges:

“The Company has reviewed the complaint filed by Mr. Williams, a former employee whose employment was terminated for performance deficiencies, and finds no merit to the allegations.”

 

Of course, as with any ongoing litigation, those of us who are not parties to the case do not know the facts and StemCells, Inc. will certainly have its side to this case. Therefore, I’d encourage people not to rush to judgment. However, this is a concerning development for the company and the stem cell field.

Top 5 possible natural stem cell boosts

Stem Cell BoostHow might we all boost our own stem cells?

If possible isn’t that a better, simpler, & safer idea than getting a transplant of stem cells?

Think of it as preventative medicine via stem cells.

It’s not a sure thing by any means. And, yeah, it is not as simple as asking for a boost in your Jamba Juice (see at right).

However, below are five possible simple ways that research suggests theoretically might be helpful to give your existing so-called endogenous stem cells a boost. You can also read more on this in my new stem cell book.

Important: consult with your doctor before considering any of these ideas. This blog post is not meant as medical advice.

1. Exercise

The human body seems designed to increase stem cell numbers when we are more active. This makes great sense if you consider that the more active that we are the more new cells that we’ll need as we are likely to lose more of our older cells by exercising. Here are two papers backing up the idea of exercise boosting our stem cell numbers.

2. Caloric restriction and fasting

An article recently came out saying that fasting boosts stem cell numbers. The team from USC found that even just a few days of fasting increased the number of stem cells in our blood system. This potential connection between fasting and stem cell numbers also makes sense as our body probably needs to be more efficient at times of low food availability and boost resistance to infection.

3. Tai chi

Researchers in China have reported that people who practice the martial art Tai Chi saw a several-fold boost in their stem cell populations (by which they meant a specific type of cell called a “Progenitor CD34+Cells” cell. This seems a little too good to be true in terms of magnitude, but could fit in with the exercise boost discussed above.

4. Sleep

Although I wrote earlier about how activity could boost stem cell levels, it also kind of makes sense that stem cells may do their thing while we are asleep. Stem cell science also supports this idea as well.

5. Protect yourself and your stem cells from radiation

When you read this you probably are thinking of dental or chest x-rays or CT scans, but I think a far more important source of radiation for most of us is UV light from the sun.

It is smart to protect your skin stem cells. Become an educated user of sunscreens and about sun exposure. Their use is complex and in fact may be harmful if misused as most of us do. In any case, the best protection from skin cancer is shade or if you have to be out in the sun, clothing. Do not let sunscreen increase your sun exposure dramatically or its use will backfire.

Finally, something that is not recommended. Stem cell supplements are not of any use based on today’s clearest evidence. They are at best a huge waste of money and at worst a risk to your health.

Duke Launches Large, Debated Stem Cell Trial for Autism

Can stem cells be used to treat autism?

At this point the jury remains out on that question, but a growing number of kids are nonetheless getting such “treatments” at for-profit dubious clinics. Academic clinical researchers are interested in this area as well.

Still, the potential use of stem cells to treat autism is a highly controversial area even in a clinical trial setting.Dawson Kurtzberg

Today it was announced in a piece on the website of the Simons Foundation Autism Research Initiative that a dramatically larger study involving hundreds of autistic children and adults is now being run by a team at Duke led by Drs. Joanne Kurtzberg, M.D. and Geraldine Dawson, Ph.D. (pictured above in image from Duke PR).

A very surprising and intriguing aspect of the article on the Kurtzberg trial from the Simons Foundation is that it alternately quoted Kurtzberg and Arnold Kriegstein, Director of the Broad Center of Regenerative Medicine and Stem Cell Research in what feels like a debate format. Kriegstein is highly skeptical of the Duke trial, which he is quoted as calling “premature” and “more like a ‘Hail Mary pass’ than a rational therapy.”

The article quotes him as to why he is skeptical:

“These are not cells that can treat a laundry list of diseases,” he says. Because the stem cells are similar to those that normally give rise to blood cells, he says, it is unlikely that they can repair or replace neurons in the brain. Also, because autism results from errors during development, it is unlikely that the stem cells can reverse those effects.

It is unusual and valuable to quote researchers in this way with different opinions. I find it surprising how blunt Kriegstein is in his concerns about this trial. The trial was also announced on the Duke Medicine website here. According to the Duke PR, Dawson was “who was the founding director of the University of Washington Autism Center and then chief science officer at Autism Speaks before joining the Duke faculty in August 2013.” Dawson and Kurtzberg are two top-notch biomedical scientists.

The $40 million stem cell autism trial is funded in part by a $15 million gift from the Marcus Foundation in Atlanta. The official name of the trial is “Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)”.

The primary outcome measure is safety, while secondary measures are potential efficacy as measured by cognitive and behavioral tests.

The trial will also use umbilical cord stem cells to treat other conditions such as stroke, but the team aims to treat 390 autistic people in that arm.

In fact, the trial apparently already started enrolling patients last month including 20 children ages 2-5 years old treated in an autologous fashion.

For more background on the use of umbilical cord stem cells for autism and other disorders see my two-part interview with Kurtzberg here and here.

Two of the difficult aspects of the idea of using stem cells to treat autism are (1) that autism spectrum disorder is a diverse umbrella group of disorders and (2) that it remains unknown what the various causes might be. Another challenge is that stem cells infused IV rarely if ever make it into the brain. At least in part for these reasons, I’m skeptical that stem cells used in the manners proposed can help autism.

autism stem cells

In 2012 a small trial right here in Sacramento by Sutter was launched using umbilical cord blood stem cells to treat autism. You can see the SacBee headline announcing it that I read at the breakfast table.

The Duke trial official clinicaltrials.gov listing can be read here for more details.

For example, the dose used with be 10-50 million nucleated cells/Kg of subject weight.

I wish we knew more about the pathogenesis of autism, which is a fancy word for saying what causes it, before we proceeded with these kinds of trials. On the other hand, I can see where such a trial could greatly advance knowledge and there is an urgent and seemingly growing need. Overall, I have very mixed feelings.

What are your thoughts on this trial? On the idea of using stem cells to treat autism? Perhaps we can have pro and con readers have a fruitful debate right here in the comments as well.