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.