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CosmeticSurg Blog

Dr. Rodriguez discusses Plastic Surgery, Medicine, and Stem Cell Research

2012 NYC Stem Cell Summit: It’s Darkest Before the Dawn

The 7th annual New York Stem Cell Summit was held on February 21, 2012. The conference was sponsored by Robin Young, former Wall Street Biotech analyst, and featured presentations by numerous public and private companies involved in the stem cell industry. After attending last year’s Stem Cell Summit, we reported on the industry awareness that adipose tissue has more stem cells than bone marrow .

This year Robin Young presented his 2020 U.S. sales forecast for stem cell therapies. Likewise, he took everyone through a look back on what happened in 2011. A total of 27 companies each gave top level overviews of their products, clinical trial statuses, and business models. The majority of the companies are developing allogeneic stem cell therapies, which are based on cells from a donor and scaled up in the manufacturing process.

2011: The Darkness Before the Dawn

It is no secret to biotech investors, that stem cell stocks suffered dramatic declines in 2011. To be more specific, they were taken out to the woodshed and beat to smithereens. Robin Young summarized that the market capitalization of all the public stem cell sectors declined by 25% in 2011 compared to the previous year. In 2011, total market cap of the entire publicly traded stem cell sector was only $ 3.4 Billion. Every single publicly traded stem cell stock declined in value, except for one company, Mesoblast (ASX: MBX).

Robin Young attributed the steep sector decline to ‘FDA regulatory pushback‘ – which is code speak to mean that the FDA reversed some of the previous favorable decisions from the past. In one example, an allograph product from Allosource was already approved, but now the FDA was reclassifying the product from tissue to drug, causing major upheaval in the industry.

However, the good news is that 2011 is now over! The thinking is that the stem cell space is emerging from the ‘valley of death’, or the’ trough of disillusionment’, which is commonly seen in new industries years after their initial hype period. Several cell therapy manufacturing companies are reporting increased commercial activity and clinical trial activity is ramping up.

contract activity in the cell therapy space is heating up

Lonza, a huge Swiss cell therapy company, reported that contract activity is brisk and that they are expanding cGMP manufacturing capacity based on existing and extrapolated demand coming out of clinical trials. In fact, many companies presenting at the conference appeared to be using Lonza as their manufacturer.

According to conference host Robin Young, Mesoblast is now considered the largest and most prolific stem cell company in the industry as measured by clinical trial activity. Mesoblast is actively involved in phase I, II and III clinical trials. Mesoblast is so positive about the future marketplace for commercial stem cell therapy that it has contracted with Lonza to make it’s own separate manufacturing facility. And Gamida, an Israeli company, detailed how, by manufacturing their approved StemEx® product with Lonza, they reduced the cost of goods sold (COGS). StemEx® is the first approved allogeneic stem cell product to get FDA approval for the marketplace.

Based on the number of times ‘Lonza’ was mentioned during all the presentations, it appears that Lonza will be ready to scale up manufacturing and assist the vast majority of cell therapy companies to get their products out to the marketplace. If this were the railroad industry, Lonza would be the supplier of the train tracks.

MSC’s as a Cure : Curing Disease vs. Managing Disease

Arnold Caplan, PhD, a prolific publisher of stem cell research and an Osiris founder, presented the keynote address to the group. Caplan is largely responsible for coining the term MSC, or Mesenchymal Stem Cells, in the late 1980’s. MSC’s are one of the prominent types of stem cell cells responsible for healing and curing disease.

Caplan reiterated that stem cells usher in a new era of Regenerative Medicine.

Regenerative medicine is a new paradigm because stem cells will cure diseases, and not just manage them.

Up to now, diseases have only been managed by drugs mass produced by the pharmaceutical industry. Regenerative medicine is a new paradigm because stem cells will cure diseases, and not just manage them. As an example, Caplan reminded the group that diabetes has been managed with insulin for 60 years, but that insulin has not been able to cure diabetes. In another example, nitroglycerin has been used since the 1880’s to treat heart disease, but it has not been able to cure heart disease. According to Dr. Caplan, stem cells will do the curing!

MSC’s: Mesenchymal Stem Cells Medicinal Signaling Cells

Dr. Caplan called for a name change for the stem cell acronym ‘MSC’, which he created back in the late 80’s. He said that since he created the term, he should have the right to change the wording based on what we have learned since then. The current MSC term is widely known to mean “Mesenchymal Stem Cells”. However, Dr. Caplan feels that a better description of MSC is Medicinal Signaling Cells. This term much better reflects the cells ability to become your own personal medicine cabinet to heal and cure disease.

How do MSC’s work their magic?

Much of the original work in the stem cell world over the past decades focused on what stem cells can differentiate into, or turn into. Dr. Caplan believes that while differentiation does occur, we have largely been missing the boat on the magical part of what stem cells do. The magic of stem cells occurs when they leave their niche and secrete a medicine chest of cytokines and other molecules which are capable of healing the body.

According to Dr. Arnold Caplan, all MSC’s sit on blood vessels as pericytes. Every blood vessel has pericytes. MSC’s are pericytes. Whenever a blood vessel is inflamed or broken, the pericyte/MSC will leave the blood vessel and travel, (or ‘home’) to the site of injury. As MSC’s leave the blood vessel, they release tons of cytokines and molecules. These cytokines and molecules become your own personal drug store eligible to treat and cure over 40 diseases. Even more exciting is Caplan’s belief that the medicine chest is self renewing—it is capable of restocking itself! After an MSC has performed its duty to repair or cure, the MSC returns to its native niche on the blood vessel, capable of performing its magical healing all over again.

Animal studies point the way to success

There are basically three phases of clinical trials: Phase I, Phase II, and Phase III. Prior to embarking on any Phase I trial, clinical investigators must show results using animals. We are happy to report that the animals are doing great! Vet Stem has treated over 5,000 animals for tendinitis with MSC’s derived from adipose fat. The cure rate is an amazing 89%.

Mindblower for MSC applications: Use as an Antibiotic

Mindblower: Stem Cells as AntibioticsDr. Caplan said the mindblower of all applications is MSC’s use as an antibiotic. When MSC’s upregulate, they secrete a molecule called hcap18-LL37, which acts like an antibiotic. Antibiotics treat infections. There is currently a clinical trial on the condition known as ‘sepsis’ which is a life threatening form of infection in the blood stream. Caplan stated that early trial results using MSC’s are extremely encouraging. Speaking to the point of MSC’s inherent capability as an antibiotic, Caplan pointed out that if you were to go back and look at the databases for allographs, which are simply a matrix seeded with adult stem cells, you would see that these have incredibly low, nearly zero, infection rates. According to Dr. Caplan, this is due to the antibiotic properties of MSC’s.

FDA regulatory guidelines

Dr. Arnold Caplan called on the FDA to create a new category in the regulations for stem cell therapy with MSCs. He suggested that stem cells are neither tissues, nor drugs (in the pharma sense), but their own unique category. Additionally the idea of performing phase I, II, and III drugs trials for each indication is not realistic because stem cells are multi functional multi purpose, multi reactive agents, as opposed to single agent drugs that have been the standard in the pharma industry. He called for a distinct new classification in the FDA regulations to help mobilize the stem cell sector. He emphasized that we can not shape the new era of stem cell therapy to the existing FDA regulatory code. This sounded like an awesome idea to me, but inquiring minds want to know who has the authority to make that change? How do we put that in motion?

Market Projections and Crystal Ball Gazing

The total U.S. stem cell market is projected to be $6 Billion by year 2020. However, this assumes less than a 3% penetration rate for nearly all indications and is based on 44 million procedures. By all accounts, this 3% projection is an extremely conservative estimate of penetration. Top level category breakdowns for the US market in 2020 include orthopedic, cardiovascular, dental, diabetes, nerve repair, and others. An interesting application from the ‘others’ category included a presentation by Celtigen whereby stem cells repair the hair shaft and restore hair growth.

2012: Looking forward
After a year of contracted growth in 2011, according to Robin Young, this is the inflection point in the growth curve.

In 15 years, stem cell therapies will eliminate 80% of all spine surgery.

In 2012 the stem cell sector will embark on its prolific and steady rise, resulting in higher market caps and stock price for stem cell companies. The sector will not look back.

Top Treatment Category for Year 2020: Neurological diseases
Robin Young predicts that in 2020, cell therapy treatments for neurological diseases will be the biggest sector of the stem cell economy. The sector will command the highest prices per treatment. He estimates that pricing for neuro treatments will be $35K-$40K.

Orthopedic Applications: Stem Cells to trounce Spine Surgery
How do you spell disruptive? According to one orthopedic surgeon involved in a clinical trial for lower back pain, cell therapy will one day own the orthopedic market. Dr.Kenneth Pettrine of the Spine Institute has teamed with SpineSmith and they are using Mesoblast’s mesenchymal precursor cells (MPC’s). Dr. Pettrine predicts that in 15 years, stem cell therapies will eliminate 80% of all spine surgery.

Stem Cell Clinical Trials

Number of stem cell trials: According to Dr. Caplan, there are currently 182 active clinical trials using MSC’s for over 40 different applications.

Conditions being treated: heart attack, ischemia, stroke, asthma, Crohn’s disease, acute renal failure, tendon and meniscus repair, diabetes, arthritis, Lupus, ALS, MS, sepsis.

Tissue sources used to isolate stem cells: adipose fat, bone marrow, cord blood.

Stem Cell Delivery Methods: In the marketplace, stem cells will be delivered by two methods. Many companies will be distributing products in both categories.

  1. Direct Delivery to the injury site by injection or placement of a matrix. This method of cell delivery is used in orthopedics and cardiac.
  2. IV infusion delivery. Disease indications using this delivery method include asthma, osteoporosis, inflammatory joint disease, multiple sclerosis, and acute radiation syndrome.

Dosing and timing of injections matter: Mesoblast reports that there is a clear dose response curve in cardiac applications. In their phase II trial they reported that a single dosage of 150 Million MPC cells in their Revascor project significantly reduced hospitalization and death rates for congestive heart failure patients, compared to lower dosages of 25 Million, or 75 Million cells.

one week after injections with MSC’s patients are euphoric

In orthopedic applications, Dr. Pettine reported using dosages of 6 Million and 12 Million  stem cells for disc repair. He reported that one week after injection with MSC’s, patients are euphoric.

Jason Kolbert, a former Wall Street Biotech analyst and now VP of Neostem, stated that dosing must be given with enough time for the cells to migrate to the site of injury or disease. Neostem has identified that period as day 4 or 5 for one of its cardiac applications.

Are stem cell therapies safe?

On the topic of safety, Allosource, a maker of allographs, which is a matrix seeded with stem cells, reported that there have been NO reported adverse events with over 4,000 procedures.

The Future of Healthcare

In closing remarks to the conference attendees, Robin Young noted that he believes stem cell therapy will ultimately be less expensive for the healthcare system. He gave an example whereby a $40K orthopedic procedure will be replaced with a single session $5K cell therapy injection.

Best soundbite heard

If Don Draper of Mad Men were hired to run a PR campaign for public awareness of stem cell therapies, he would likely have zoomed in on this sound bite spoken during the course of the conference. It’s quite simple, yet accurate:

“Stem cells are a way to naturally heal your body.”

Nuff said. Onward with the revolution!

Posted in Fat Stem Cells

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  • wrote
    April 17, 2012 at 4:02 pm

    Point well taken, Dr. Rodriquez; as you say the process is more complicated than just circumventing regulatory impediments, it’s about expertise and the implementation of protocols, process and the rigorous application of the metrics of scientific method to the results. Without these controls, the floodgates are loosened and harm may result while opportunites to do good are potentially forefeited.
    It is riveting, however, to see how the political dichotomy is playing out. While I hesitate to ascribe ulterior motives to the paradox of progressives’ new found reactionary resistance to autologous adipose stem cell therapy, it doesn’t seem to much of stretch to say it smacks of the financial influence of K Street and big Pharma. Cui bono. Thank you!

  • wrote
    April 17, 2012 at 2:54 pm

    The answer is not very simple, as there are safeguards that need to be implemented. Otherwise there will be a gold rush by unscrupulous operators who may not be as knowledgeable as needed.
    We are becoming involved in the political process so clinical protocol guidelines, facility accreditation processes and treatment outcome registries get implemented. This will ensure patient access to the latest advances, but at the same time ensure safety and monitoring of results.
    What is interesting is that the roles of the proponents of stem cell therapy have reversed. The Christian evangelicals who were very opposed to embryonic stem cell research are now very supportive of adult stem cell clinical trials. The progressives who thought science should go forward even if it meant sacrificing embryos are now vehemently opposed to adult stem cell therapy as an “unproven” therapy.

  • wrote
    April 17, 2012 at 12:06 pm

    Was bowled over a couple days ago while checking stock indicators in the stem cell sector when I was led to article about adult stem cell therapy and Texas govenor Rick Perry. The gist of it was he may endeavor to sidestep FDA strictures and “liberalize” the state’s medical regulatory climate in relation to the practice adult stem cell therapy.

    Apparently his effusive public policy pronouncements that he hopes to make Texas the future center for advanced adult stem cell procedures was engendered by his personal success with treatment for his chronic back problem. The irony, based on my personal political predlications, was startling to say the least. Is this the thin edge of the proverbial wedge?

  • wrote
    March 28, 2012 at 12:58 pm

    Excellent state of the union address. Thanks.

  • wrote
    March 16, 2012 at 1:38 pm

    Interesting questions. I am a member of IFATS, have presented at IFATS, and was at that meeting.
    First of all Kacey Marra is highly respected in the field. She runs the lab for Dr. Peter Rubin, a leader in the field of adipose tissue science. He is the chairman of Plastic Surgery at Univ of Pittsburgh.
    As far as your specific questions, the over all issue is: Does the delivery vehicle affect graft take?
    The answer is yes, it does. There are many vehicles being used, but then other issues arise. Does it work in the body as well as in the lab? And if it does (and I give Marra’s work full credibility), then there are regulatory issues with the FDA. So even if this or that product works, they are years away from seeing the light of day.
    A secondary issue is, do you want the fat graft to “take”, or do you want the stem cells in it to perform another function, for example, healing radiation tissue (my research interest). In this case, my aim would be not necessarily to improve graft take, but to make cells behave a certain way.
    Finally, there are injectables in the market now, such as Hyaluronic Acid (Restylane, Juvederm), that help fat take by providing a 3-D tissue matrix that helps the cells adhere to it.
    All this is really fun, and I applaud you for having found the IFATS site. If you are a Physician, or in the industry, join IFATS, as it is where a lot of the cutting edge work in fat grafting and stem cells is being presented.

  • wrote
    March 12, 2012 at 1:02 pm

    Hi, Dr. Rodriguez, it’s rather off-topic, but speaking of summits, I was wondering if you were aware of this: http://www.ifats.org/wp-content/uploads/2011/11/IFATS-Program-book-Web.pdf

    Some really interesting stuff in there, most of it relevant to what you do. One that I found particularly interesting, on page 86, is a paper that was presented by Kacey Marra titled “Encapsulated Adipogenic Factors Effect In Adipose Graft Retention.” Basically, they mixed suctioned fat with microspheres containing insulin or dexamethasone before reinjecting it, so that the microspheres would then slow-release the insulin/dexamethasone into the fat graft. Apparently they found that this significantly increased the fat graft “take” in all cases. Unfortunately, I don’t know how much it improved take, because they don’t say in the abstract, and I can’t find the paper they presented online anywhere.

    I also remember not that long ago that some researchers found that NPY injections directly into fat had an extremely pronounced effect, both in increasing graft survival and in causing new fat to grow by itself, and NPY antagonists had the opposite effect (ie, causing spot reduction where they were injected by triggering the fat to devascularize and shrink itself). So I would guess that microspheres containing NPY would work even more effectively than insulin or dexamethasone.

    Anyhow, just curious, what’s your take on when this sort of thing makes its way into actual plastic surgery practice?