My plans for 2018 are to continue the medical practice 2 days a week (really 1.5 days) while I begin a new job as a partner
in a new Venture Fund. My role is to evaluate companies for possible investment, and from time to time help in their governance.
I have already started in this new opportunity, and I am thrilled at the potential; it has taken me around the country and put me in a position
to both learn and consult with many brilliant people in science…and in the business of science. Topics we cover range from stem cells to really cool
medical devices to therapeutics for serious diseases of all sorts. I hope that readers of this blog and web site will stick around to learn a bit about what I am learning.
Now that it is established that there are stem cells in the interstices of fat (the “pericytes”), easy methods for counting such cells are in the offing. This would be a precursor to introducing better science to the administration of injections derived from fat (lipoaspirates). There are now at least 3 such technologies on the market, which differ not so much in the harvesting of fat (easy) but in the preparation of the injectable material; do you wash it, squeeze it, emulsify it, blend it in a Vitamix? What is the best method? This issue needs to be resolved by comparing methods and counting the purported stem cells in the preparation, which, not surprisingly, may be vastly different in different people. As with PRP, until a better level of analysis is used, it will be impossible to opine on the effectiveness of these injections with anything other than anecdotal evidence.
One further point: the PRP results at present are so good that any improvement will require lots of patients in a trial to demonstrate superiority. this is a good problem to have!
The recent internatonal stem cell meeting in Boston seen from the perspective of the first meeting (17 years ago) in that city showed great progress in the understanding and the utility of stem cells, for cancer, regenerative medicine, and inflammatory disease; the common origins of such issues (including normal aging) are appearing more and more likely. In terms of treatment, we are at the cusp- but not yet the reality- of validated stem cell treatments. So far, animal models- not human studies- have the best data. But it is coming.
One issue I follow is the measurement of stem cells, be it from fat or bone marrow, at the point of service; what is the patient actually getting. There are now several inexpensive ways to validate the stem cell content of a preparation. Like we do with PRP, I beleive that this will be essential in measuring the dose of treatment, and it will be an important way to characterize what we are doing, as you would with any drug.
Dr.Marcus is teaching “Funding a New Venture” for the Johns Hopkins Entrepreneuership Program Summer 2017.
At the recent FDA meeting last month, scientists, physicians and lay people with severe chronic diseases all had a chance to tell their stories and offer opinions to the agency about the use of “stem cell therapy”. When a person with MS or Parkinson’s alleges or reports a benefit, it is often hard to separate the science from natural ups and downs of the disease. When a for profit company makes a claim, it is often hard to separate out the obvious conflict of interest from a real potential game-changer. Very few at the meeting were there to report negative results; although one doctor from Miami showed 3 cases of patients who had lost their vision from “stem cell” injections given for macular degeneration. ALso, there was one report, from abroad, of embryonic cells causing a spinal tumor.
The FDA is in a tough position; the dictum of physicians is primum non nocere; this above all do no harm. On the other hand, there is obviously potential for disease modification or cure, if we could just figure it out.
Some patients strongly feel that if the cells come from their own bodies, it is nobody’s business who does what to whom…this would be a libertarian approach. The problem is, if this turns out to be a total flim flam, or worse, doesn’t the FDA have a responsibility to intercede?
The most pressing observation is this: many of the clinics are harvesting not stem cells but patient’s money. They are good at the latter but not the former. Stem cell therapy should begin with the VALIDATION (by 3 biomarkers) that we are, in fact, working with stem cells and not just fat, or blood, or a dose so small (of cells) as to be irrelevant. Second, we are far more likely to get good data from a not-for-profit entity (like the NIH). Third, we should widely circulate information, as in this blog, that helps patients make good decisions (and saves them money). These principles would protect the public to a large degree and would not hinder advancement of this very important field.
On Sept 12 Dr. Marcus is to present at the FDA conference on Cells and Tissues that will help determine the new guidelines on the use of stem cells and growth factors.
This fall semester, Dr. Marcus will once again teach “Funding a New Venture” through the Advanced Acadmeic Programs Department of Johns Hopkins. This is an online course with worldwide participation.
So far the first 11 patients with “amniotic” injections are doing fine, no complications and 10/11 experiencing little if any symptoms; in other words, PRP plus the amniotic fluid appears to be as good as PRP alone. It is still too early to determine if the addition of the amniotic fluid is better than PRP alone. Since this is not a controlled, blinded study, we will have to use prior knowledge and expectations to make a judgement of this kind.
As to the fluid, it contains a variable number of cells that appear in the cell counter as either lymphocytes or granulocytes. As expected, quality control of cell number is problematic and is not being done at the tissue bank on each lot. We are in the process of analyzing the growth factors in the fluid, which of course may be equally or more important than the cells. Stay tuned.
Dr. Marcus to teach E & E at George Mason University this Spring Semester 2016.
On Fri Nov 13 Dr. Marcus spoke to the entrepreneurs at Johns Hopkins Medical School about concepts in funding new ventures. The Group is Called Hexcite!