What is the most exciting, important, promising, all-around awesome stem cell biotech of 2014? Vote and tell us why in the comments.
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).
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.
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. The 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).
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.
Where 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.
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.
Both talks were outstanding.
Robins went first. He started by saying how Viacyte had a productive pre-IND meeting with the FDA last year.
I believe a target for them is to have IND in Q1 of 2014 and hopefully move to the clinical trials soon after.
I gotta say I love Viacyte’s technology. Having a frozen product that then can be recovered in culture for 3 days before loading into the capsule seems like a big plus.
Simmons also gave an interesting talk on Mesoblast’s status. Quite impressive.
I find it astounding just how big their pipeline is. They have perhaps as many as 10 products. They also have an impressive number of trials already ongoing based on allogeneic off-the-shelf products based on mesenchymal progenitor cells (MPCs).
Simmons posed a hypothetical question that people sometimes ask the company related to trophic factors made by MPCs, “Why don’t you just use the trophic factors that are secreted as the therapy”. Simmons’ reply was both humorous and encouraging, “These cells are quite intelligent!”
MPCs secrete just the right combo of factors as instructed by a given environment.
Both talks were great and I think these companies have strong potential to help thousands of patients within a decade.
Disclosure: I am not an investor in either company nor currently any company in the stem cell field.