We buy insurance because we all want to make sure that if something awful happens, we are prepared. Many years ago I was asked to be on the scientific advisory board of a company that was collecting stem cells from patients for future use. The venture never really worked out, primarily because this was like buying insurance when it was unlikely to pay out. Let me explain.
Why do I take the time to do these blogs? The big reason is that I get inundated with e-mails, patient in clinic questions, and online queries about each and every technology on which I blog. Rather than going through a detailed 20-minute lecture each time, it’s easier to review something once and place my analysis online. That way, I can just send someone a link.
The company I worked with used a mobilized peripheral blood apheresis protocol. This meant that they gave the patient a drug to knock stem cells loose from the bone marrow and into the bloodstream. They then used a machine process called apheresis to collect a cell fraction from the blood that would hopefully contain those cells and then deliver the rest of the blood product back to the patient. At one point we were asked if we could grow stem cells from their apheresis product. Regrettably, no mesenchymal stem cells grew, so we concluded that whatever stem cells they were collecting were “barely there.”
The company also had a bigger issue. They had a hard time selling patients on these collections because the cells they stored couldn’t legally be used in the U.S. to treat anything. So this was like buying an expensive insurance policy that would never pay off.
We see many physicians making the claim that they are performing “stem cell therapy” merely by collecting a blood sample. Regrettably, this is mostly fiction as blood is very poor in any active stem cell content. In fact, what the patient is really getting is a hyperexpensive platelet-rich plasma therapy.
The process behind LifeVault sounds intriguing. You send in a blood sample, and according to their website, they separate the blood sample into “DNA,” “Stem Cells,” and “Blood Plasma.” The site then claims (as an incentive to save your blood sample) that stem cells are being used currently in research studies to treat ALS, multiple sclerosis, Alzheimer’s disease, leukemia, Parkinson’s disease, and cardiovascular disease.
There’s just one little problem here, and it’s called reality. Most of the research being done in these diseases use mesenchymal stem cells, which aren’t generally found in blood. So what stem cell type is LifeVault isolating? Try as I might, there is no info available on the site to answer that question. On their LinkedIn feed, they recently listed an Alzheimer’s study that involved adipose-tissue stem cells that were isolated and culture expanded. Given that none of that has anything to do at all with the blood being stored by LifeValut, it’s unclear why this study was posted. Another study listed on their feed used a unique protein that turns on myelin repair in the body, again having nothing to do with what LifeVault is doing.
I did get this response from the LiftVault CEO, Trevor Perry: “We are isolating specifics cells for induced pluripotent stem cell creation and hematopoietic stem cell progenitors.” That looks the same as this news story.
Induced pluripotent stem cells (iPSC) are based on the idea that we can take adult stem cells and coax (or hammer) them, in the lab, back to their primitive stem cell state. The technology is new this past 5–10 years, and there have been significant concerns that these cells may not be all they’re cracked up to be. The biggest issue is that we have almost no clinical data that these “lab manufactured” stem cells will help any disease.
Forever Labs takes a different approach, in that at least they’re using a stem cell source that has a significant number of stem cells and has early clinical data suggesting efficacy. They take a bone marrow sample, and their advertising is focused on storing your stem cells when you’re young. Their focus in on anti-aging. Whether this would work or not is anybody’s guess, but at least there is some research that shows that giving younger stem cells to an older person could possibly work to improve health.
One concern with this company came in a call I got from a colleague. Like all stem cell-storage plays I have ever seen, they always branch out. This colleague asked me if I thought that this was a good service to offer his patients. His intent was saving some of their bone marrow for a future treatment. For example, he would perform one bone marrow aspiration and then use some for the first treatment and save some for a future therapy. What I told him was that while at first blush this may sound like a smart idea, given that bone marrow stem cells are dose sensitive, for most clinical applications involving large joints and the spine, there wouldn’t be enough cells left over to store. In fact, the only “stem cell storage” concept that makes sense is if you’re growing the stem cells to larger numbers, but this isn’t legal in the U.S. This is only offered in our licensed Grand Cayman facility.
Is either company offering something that is likely to be useful to someone who is young now but as he or she ages acquires an awful and incurable disease? To get to that answer, I created a decision tree that asks the tough questions.
First, the biggest issue where all of these companies fail is that FDA approval for stem cells used to treat disease is VERY SPECIFIC. Based on the existing regulatory structure, there will NEVER be a blanket approval of a cell type (like mesenchymal stem cells or iPS cells) for therapy. That approval will tie a very specific lab process used to collect, isolate, and culture those cells to treat a specific disease. Hence, if you don’t use that lab process, there is no approval. This means that it’s VERY UNLIKELY that these stored cells will ever receive any FDA approval for use in any disease. To do that, these two companies would have to submit a several-hundred-million dollar IND to treat every specific disease. That’s VERY UNLIKELY to happen.
Below is the decision tree for LifeVault’s “GoodCell” product. As you can see, there are MANY issues, not the least of which are that while the company CEO replied that they will be creating iPS cells from this collection, and that may be possible, we have no early clinical data that iPS cells would be able to cure any of the diseases listed. It’s also a concern whether iPS cells could be created after a sample of blood is shipped across the country, isolated, stored for 20 years, and then thawed. While it’s likely you could make that work, it’s not a sure thing (which I why I have “Maybe” at that step). However, even bigger issues happen on the regulatory side. Meaning, if iPS cells could be approved for some future clinical use of a specific disease, that approval will be tied to a very specific collection and lab process and company performing that process. If that company is not LifeVault, these cells may not be able to be legally used for that purpose.
Forever Labs has an easier decision tree, but again gets hung up on the regulatory side. So while there are MSCs in bone marrow and there is good early-stage research showing these may help treat multiple disease types, the same issues happen on the regulatory side. Any MSC approval will be tied to a specific company and specific process of isolating and growing cells.
While this could be theoretically possible, this has not happened in any FDA cellular product approval to date. The process of isolating and storing the cells also goes through the FDA approval process and is as integral as creating or growing cells. Hence, it’s VERY UNLIKELY that any company that eventually gets an FDA approval to use stem cells to treat disease would be able to use stored cells from either company.
The upshot? Nobody likes to buy an expensive insurance plan that may never pay off. While it’s possible that either company’s product may work to someday cure disease, that seems very, very unlikely sitting here in the early part of the 21st century. Hence, please think before you leap.
Note, I did get a detailed response from one of LifeVault’s founders, Harvard professor David Scadden, that I thought in all fairness I felt I should publish:
“Thank you for your note, Chris.
About the Author
Christopher J. Centeno, M.D. is an international expert and specialist in regenerative medicine and the clinical use of mesenchymal stem cells in orthopedics. He is board certified in physical medicine as well as rehabilitation and in pain management through The American Board of Physical Medicine and Rehabilitation.…