Stem cell treatment cost 2.0: legit therapy

stem cells costWe hear so much about exciting potential stem cell therapies. Some of these are rigorously evaluated ones in the FDA clinical trial pipeline and others are available right now mainly through predatory stem cell clinics. Earlier this year I posted about the cost of the offerings of dubious stem cell clinics.

In this post, I address the cost of a future, legitimate, FDA-approved stem cell therapy. How high will that be?

This is a critical question because if many patients cannot afford a stem cell therapy then the impact of that therapy is reduced. Cost is inversely related to access. On the other hand, stem cell biotechs must make some profit or they will go out of business. Investors, who are often enthusiastic boosters of the stem cell field, will lose large sums of money and confidence in the field too in that scenario if stem cell treatments are priced “too low”. What is the “right” price?

There is likely to be increasing pressure on biologics drug prices as well from the federal government. Witness Hillary Clinton’s recent tweet on this topic below that sent people into a tizzy.

At the state level, such as here in California, the question of stem cell treatment cost is also becoming more pressing including for our state stem cell agency, CIRM. As CIRM-funded clinical trials advance, which is a wonderful thing, at the same time we get closer to where someone will have to decide on stem cell price tags.

We can look at what other cellular drugs have cost as guidance for the price tag range for stem cell treatments. For instance, Prochymal (its old name under Osiris) that is now rebranded as TEMCELL from Mesoblast/JCR likely will cost about $200K for a full treatment for GVHD (HT to Alexey). Provenge, the cellular prostate cancer drug from the controversial biotech Dendreon had (has?) a price tag of $93K. The most expensive drug in history, the gene therapy med Glybera will cost around $1.5 million per patient.

Realistically, a typical legit stem cell therapy could easily be $100K per patient. A personalized cellular medicine such as an autologous stem cell-based therapy could easily run into the hundreds of thousands per patient. Some therapies could go as high as $500,000 (see this helpful piece by David Jensen) or even into the millions.

Irv Arons

Irv Arons (@iarons) has come up with a great table of cost estimates focused in the area of vision therapy (free registration required). Thanks to Irv for permission to use it here (above).

How will patients afford such expensive therapies?

Will such therapies be covered by governmental agencies or insurance companies? They should.

We should also be keeping in mind the current costs of treating today’s patients with major and sometimes chronic diseases. These costs run into the hundreds of billions or above a trillion dollars each year in the US alone. That’s important context and rightly indicates that the costs of stem cell therapies to society may be appropriate even if at an individual level they seem high.

How does this compare to stem cell treatments at predatory clinics?

Such “treatments” range from $5,000-$20,000 each and most patients with whom I have talked either received or were pitched at least two such treatments, amplifying the total cost. The cost to the clinic of the treatment itself can be as low as $500-$1,000. Some clinics claim to have treated thousands of patients suggesting they are making millions in profits.

Why are stem cell clinic offerings typically relatively cheaper than legit treatments? Frankly, it is because they don’t follow the rules or do the necessary studies to prove safety and efficacy. Ten thousand dollars is still a lot to pay for something that doesn’t work and could even be harmful.

Even so some consumers may perceive dubious stem cell treatments as the way to go because of the lower cost, particularly if the legitimate stem cell field fails to do a good job at educational outreach and the FDA continues to effectively do nothing about the stem cell clinic problem.

The bottom line remains a question. Where’s the stem cell price sweet spot where we can help the most patients, but also generate a needed profit for the biotechs?

We need to find an answer to this question soon.

What is the top stem cell biotech of 2014? Take our poll

What is the most exciting, important, promising, all-around awesome stem cell biotech of 2014? Vote and tell us why in the comments.

Book Excerpt: Top 4 Stem Cell For-Profit Good Citizen Biotechs

The below is an excerpt from my book, Stem Cells: An Insider’s Guide.

I hope you enjoy this excerpt and the book, which I think is a really good deal at under $27 paperback or under $17 for E-book version.

Having our innovation and ethics too

In the stem cell field you can have it both ways. As a company, you can be ethical and help patients. There are many examples of stem cell-related corporations doing “the right thing” when it comes to developing therapies based on growing stem cells in the lab. Below I discuss a few of these companies (Disclosure: I have no financial interests in these companies, which are listed in alphabetical order).

Advanced Cell Technology

As one example, let me mention a company called “Advanced Cell Technology” or ACT. I would argue that, especially in the last half dozen or so years, the company has been a model citizen in the for-profit stem cell field.ACTC

ACT’s most advanced product in the pipeline toward the clinic is an embryonic stem cell-based therapy for macular degeneration, the leading cause of blindness. ACT grows the embryonic stem cells in culture and differentiates them through a complex process into special cells called retinal pigmented epithelial cells (RPEs). Macular degeneration robs people of their sight because their endogenous RPEs die off. Therefore, the principle behind ACT’s therapy is to replace the endogenous RPEs with exogenous ones. So far, ACT has reported no major negative outcomes from its trials of transplants of embryonic stem cell produced-RPE. I am discussing ACT here because it follows FDA rules and works in an ethical manner to protect patients. It publishes its data and engages patients.


Athersys is an adult stem cell company developing allogeneic products to treat a number of important human diseases. The company has five clinical trials listed in the government database. AthersysThe target diseases include stroke, heart attack, blood cancers, obesity, and ulcerative colitis. Their top-line product, MultiStem, is described by the company in one of their clinical trial write-ups as follows:

“MultiStem(r) is a new biological product, manufactured from human stem cells obtained from adult bone marrow or other nonembryonic tissue sources. Factors expressed by MultiStem cells are believed to reduce inflammation and regulate immune system function, protect damaged or injured cells and tissue, promote formation of new blood vessels, and augment tissue repair and healing.”

Athersys has a good reputation in the stem cell field for transparency including regularly publishing their data and following FDA regulations.


Mesoblast is another good citizen in the stem cell field and has an unusually large number of stem cell products in the pipeline. Their work is based on a type of cell that they call the “Mesenchymal Progenitor Cell” or MPC. Interestingly, while typically progenitor cells have less potency than stem cells, as we discussed earlier in this book, Mesoblast’s MPCs are not your ordinary progenitor cells. 


I recently heard a talk by Dr. Paul Simmons of Mesoblast who reported that MPCs are in fact more potent than MSCs, which is an interesting paradox of nomenclature. Mesoblast is conducting FDA-approved clinical trials for a host of human diseases. I found 9 clinical trials listed for Mesoblast including for conditions as variable as spinal disc injury and heart attacks.


NeuralStem is a fourth good citizen in the stem cell for-profit world. They are a model citizen for the field when it comes to transparency says Alexey, who I trust on this a great deal, publishing data and even publicly releasing their patient consent form, a rarity in the stem cell field. NeuralStem currently has four clinical trials listed in the database: two on depression, one on spinal cord injury, and one on Amyotrophic Lateral Sclerosis (ALS; Lou Gehrig’s Disease).NeuralStem

The key, positive roles of investors in good-citizen companies

It is important to also highlight the crucial role of investors in making safe, effective, ethical, and compliant stem cell treatments a reality. For-profit stem cell companies including the good citizens of the corporate stem cell world need large amounts of cash to make stem cell-based medicine a reality.

The money comes from investors, who are hoping that some of the exciting stem cell biotech companies become profitable. I know from talking with many of the investors that they are choosing to invest in the stem cell companies not just because they believe that they will be profitable, but also because the stem cell products of those companies will potentially help people suffering from diseases and injuries. I believe that the investors in publicly traded stem cell companies fulfill a key role in accelerating stem cell cures. They tend to be a highly educated, engaged group of people as evidenced by their posts on a website for stem cell investors where I sometimes blog as well.

Investors in privately owned companies can also have positive roles, but I am concerned that in that context the lack of transparency may lead to a more complex, potentially ethically problematic influence.

The main overall challenge in a for-profit setting is to create a business regulatory environment in the stem cell field that enables good actors to succeed.

Stem Cell & Cell Med Industry Briefs

Mesoblast_LtdWhere do things stand with some players in the for-profit world of stem cell and cellular medicine companies? Here are a few brief news items with quick analysis. Mesoblast is dominating the news of late.

Mesoblast reported good news from its Phase II safety trial for its mesenchymal progenitor cell (MPC) product in patients with Type II Diabetes. There were no treatment-related adverse outcomes and as a bonus there were hints of efficacy.

Osiris Going in New Direction. Cellular medicine company Osiris has been in the news quite a lot lately. The latest is that CEO & President Randall Mills is stepping down after about 10 years as the company’s leader. Personal reasons were cited, but the move could reflect a change in direction for the company. About a month ago we all learned that Osiris had received an untitled letter from the FDA and a few weeks before that it sold off its stem cell portfolio to Mesoblast.

Celgene Partners with OncoMed on the cancer stem cell front. In a case of one plus one is probably more than two, Celgene shelled out big bucks to partner with cancer stem cell biotech OncoMed to team up against cancer stem cells. OncoMed has been on a roller coaster since its IPO, but has an interesting, promising product in Phase II trials in the Notch pathway inhibitor Demcizumab.

Japanese Regenerative Medicine Regulatory Change Creates Buzz. The Japanese government has implemented regulatory changes that could speed the development of regenerative medicine therapies. The changes interest Cell Med biotechs including Mesoblast:

Mesoblast chief executive and founder Professor Silviu Itescu told Inside Business the bills enable Japan’s government to approve new products conditionally, providing their safety is confirmed in clinical trials, even if their efficacy has not yet been verified.

There are concerns, however, that a lack of data on efficacy may expose more patients to new products that have little chance to help them.

Disclosure: I don’t own stock in and am not affiliated with any of the companies mentioned above.

My response to ‘Our Bodies, Our Cells’ Harvard Law blog post on stem cell deregulation

Over at the Harvard Law “Bill of Health” blog, Mary Ann Chirba and Alice A. Nobel posted a piece a few days ago that has generated a lot of discussion about FDA regulation of stem cell interventions.

Their piece really has two parts.

In the first part they go through the US v. Regenerative Sciences Inc. (RSI) case providing helpful and insightful perspectives on the history of the case and their prediction as to the likely outcome on appeal. They feel that the FDA will prevail.

In the second half of the piece, Chirba and Nobel build an argument for weakening of FDA regulation of autologous adult stem cell therapies. While I agree with them on the need for changes at the FDA (in my upcoming book I go out on a limb and call for 5 specific reforms at the FDA related to stem cells), I believe they do not make a strong case and that the specific proposed changes that they have put forth would do more harm than good.

Here is my comment on their post:

The historical and legal perspectives in the first half of the article are spot on and helpful.

However, parts of the later portion where you argue for weakening of the regulation of propagated adult stem cell therapies greatly concern me and you have left out some very important elements that are key to understanding the ongoing debate over the appropriate level of regulatory oversight for stem cell therapies. For example, you pretty much gloss over the safety concerns related to these stem cell interventions.

It seems to me that changes are definitely needed at the FDA in some respects related to stem cells such as expanded compassionate use of stem cells for patients with fatal diseases and a push for more openness. So we agree on a need for change at the FDA on stem cells. But the weakening of regulatory standards for propagated adult stem cell interventions would greatly increase patient risk. Further, as the numerous outstanding adult stem cell biotechs such as Athersys and Mesoblast have shown, in the stem cell field a company can have its innovation and be compliant too.

Your statement, “Conditioning the extent of regulation on the degree of manipulation may make sense on paper but is vague and confusing in practice, especially in the dynamic field of cellular therapies” is very puzzling.

In fact, the degree of manipulation is operationally (not just on paper) extremely important from a patient safety perspective and it makes perfect common sense that stem cells manipulated in different ways and to different degrees should be subject to different regulations. I do not see what’s vague or confusing about that at all.

On the other hand, your argument that smaller companies producing stem cell drugs should not be subject to the same regulations as larger companies is a dangerous one. Since when is the law variable depending on the size of the entity that should be following that law? Just as small and large drug manufacturers of pill (chemical) drugs have to follow the same rules to provide data on safety and efficacy, smaller companies selling stem cell drug interventions should have to follow the same rules and laws as big companies. To do otherwise would put patients at great risk. Don’t underestimate the number of patients collectively that stem cell clinics are treating and putting at risk these days: the numbers are in the many thousands and growing.

Paul Knoepfler

UC Davis