Endofound Medical Conference 2017
"Breast, Ovary and Endometriosis"
October 28, 2017 - Lotte New York Palace Hotel
“Cells Are the New Cure: The Cutting-Edge Medical Breakthroughs That Are Transforming Our Health”
Max Gomez, PhD.
Award-winning broadcast journalist, CBS News Medical Correspondent
Hi guys. You awake still? How's your blood sugar? Getting a little low? All right. Since this is right before lunch I will try to avoid showing you some massive bloody tumors. No endometriomas or anything of that sort, but I do have some videos that I think are going to be fun to show and they'll kind of illustrate I think some of the things that we're talking about.
I want to be talking about cells today and cells that don't necessarily directly impact endometriosis, ovarian cancer and breast cancer and so forth. I'm going to take a little bit different kind of a point of view here, more of a 60,000 foot view if you will of cells, and how cells I believe are going to be the next drugs and the next cures and as you may have heard, that's the title of our new book that's outside. And so before I start I always want to make sure that I acknowledge my friend and colleague Dr. Robin Smith. She's a co-author on the book and without whom the book and the scientific conferences that we helped organize at the Vatican actually of all places, that gave rise to the book, that would not have been possible.
So let me start out with a couple of questions for you. How many of you here want to live to be 100? Boy that's a lot less than I usually get. Then never mind, 110? I know why because you don't want to live to be 100 the way we normally think of 100, right? Which is, oh, there goes my talk. Wearing diapers in a wheelchair, not even being able to remember your own name or your kids' names. Hang on. Now I know why they use Power Point. That's not what I'm talking about. It's going to be a lot better. Anti-aging and aging is going to be a lot better than that. How about a question that perhaps hits a little closer to home? How many people here have or have had cancer? Don't tell me because that's a HIPPA violation probably somewhere along the line. I don't want to know. But you know someone who's lost someone to cancer, you know someone who's had cancer. Of course you do, we all have. We all know that.
How about a little less depressing thought? Do your joints ache and creak? Yeah, I know mine do. My knees sound like a bowl of Rice Krispies when I get up in the morning and I walk across the hall. One more question. Know anyone with diabetes? A leading killer and a major cause of heart disease, stroke, blindness, kidney failure, amputations? Well I believe that all of these health problems that we're talking about, old age, cancer, diabetes, arthritis and many others are about to be totally transformed converted into in some cases chronic diseases, in other cases completely cured. We're actually on the brink I really believe of a revolution in medicine or in that often used but rarely accurate phrase a paradigm shift in medicine. I'll explain. But first one last question, how many of you take a prescription drug? That's okay, almost all of us do, right?
According to the Mayo Clinic, seven out of 10 Americans take at least one prescription drug and in 2013, listen to this, in 2013, this number stunned me, people between the ages of 50 and 64 had an average of more than 19 prescriptions per person, 19 prescriptions for drugs per person. So I think that's about to change. I think that in the not too distant future, when you go to the doctor, he or she may not give you a prescription for pills or capsules, he or she is going to give you a prescription for a living drug, living human cells. Let me explain what I mean by that.
I've been a medical correspondent on TV since I left my post-doctoral fellowship at Rockefeller University 37 years ago, and trust me, it hurts to say that. I can honestly say every researcher I speak to, Sloan Kettering, NYU, Yale, Mount Sinai, all over the place they all agree with what I've been saying, which is more has happened in medical research in the last five years than in the previous 30. That's how fast things are moving. That's how fast the pace of medical advancements and medical research and it's accelerating. In fact, it's almost impossible for the average physician, let alone the general public to keep up. Or in the case of the public, even be aware of how close we are to the proverbial, as Malcolm Gladwell would put it, the tipping point. That's one reason why we wrote the book, that and I really want people to know how close we are to what Vice President Biden said to us, at the last Vatican Conference. He said, "We are at an inflection point in mankind's development of effective treatments and even cures for some of the most devastating diseases and conditions afflicting the planet."
Now obviously I can't cover everything that that covers, but let's hit a couple of areas I think you'll be interested in. I want to start out with something that was in the news recently, what just may be a cure for blood cancers like leukemias, lymphoma and multiple myeloma. It's a treatment called CAR T cells. That's shorthand for Chimeric Antigen Receptor cells, T cells. Now the name is not that important but here's what it did for some children, lovely about 11 minutes remaining for the enhanced dictation. I don't need enhanced dictation. All right, let's see. I'm gonna show you this one. Here's what it did for a young woman and some others who were dying of leukemia ... Or not. I'm not getting audio out of here, right? It's always a tech problem.
[Video 00:06:16] All battling leukemia-
There we go. Let me go back here and start from the beginning then.
[Begin video 00:06:20] Emily Whitehead looks like your typical 12 year old girl now, but five years ago, she was dying of cancer. Seven year old Kishawn Malhorn and 16 year old Emma Collins were facing the same fate, all battling leukemia and having failed traditional treatments.
But then came a second chance, a game-changing experimental therapy that utilized their own bodies' immune systems to kill the cancer.
The volume is all the way up.
And now thanks to a unanimous vote by an FDA expert panel yesterday, these kids won't be the only ones to benefit.
Ten years ago I don't think that anybody thought that you could productively use the immune system to fight cancer.
Dr. Stephen Grupp conducted the clinical trial for this revolutionary treatment call CAR T Therapy.
Now in addition to chemotherapy, radiation and surgery, this is the fourth thing, immunotherapy.
In the lab, the patient's own immune T cells are genetically modified by a virus to recognize a patient's cancer. Then those custom made white blood cells are given back to the patient. A single dose has brought longterm remissions in more than 82% of the most seriously ill patients treated.
When they find something that they recognize like the B cell leukemia, they kill that off.
Better yet, some of the CAR T cells remember what the leukemia looks like to fight any recurrence.
Can certainly lead to at least five years of survival and we hope can lead to cures.
Cures for thousands of children around the world, like Emily.
Now I'm five years cancer free.
This CAR T therapy can have serious side effects but remember the children who got it were literally on death's doorstep. It's been effective mainly against leukemia and lymphoma but researchers are working to use it against so-called solid tumors like breast, colon and lung. Dr. Max Gomez, TV 1055. [End video 00:08:16]
1055 is our sister station. I actually work for Channel Two, CBS here. There are several things I want you to remember from this video or at least the parts that you saw there. One is as I'm sure many of you know, physicians in general and oncologists in particular are very hesitant to use the C word. The C word is not cancer. The C word is cure. Dr. Hunger, who you saw there at the end, is one of the leading pediatric oncologists in the country. He used the C word for us.
Two things I want you to remember about Emily Whitehead was she was on death's doorstep, literally, when doctors threw the Hail Mary. She was the first child to get this CAR-T therapy for her leukemia. Doctors said that they thought she had somewhere around several pounds of leukemia cells circulating in her body when they did that, when they did the CAR-T therapy. And the second thing I want you to remember is that just a couple of months ago, back in August, Emily had her five year check-up. She is five years cancer free after being literally on death's doorstep for her leukemia. She had failed already, by the way, two full rounds of chemotherapy, so it wasn't like they went to this right out of the box.
And then just last week, the FDA approved another CAR-T therapy. This one for a type of non-Hodgkin's Lymphoma in adults. Again, another important advancement in the treatment of very difficult to treat cancers. These CAR-T therapies are huge against what we call liquid tumors or blood tumors. It's going to be a little tougher, as I said, against solid tumors, because solid tumors tend to ... They're kind of like a medieval city, and they form walls around themselves, and they protect themselves from the immune system, but we're working on that, including a whole new approach to CAR-T cells. It's called armored CAR-T cells of all things.
So, let's talk about another area where cells have the potential to make a huge difference against disease ... Let me take a step back. You know, the most common way we treat disease these days is of course with a medication. Infections, cancer, mood disorders, erectile disfunction, overactive bladder. I don't know if there's an app for that, but I guarantee you there is a drug for that. But drugs are fairly crude instruments, generally. Drugs generally address one biochemical pathway, but cells are really different. You and I right now, there's an estimate that every second, every cell in our body has about a billion biochemical reactions going on. Every one of our cells, every second, right? Each cell is constantly adjusting to its environment to maintain homeostasis.
So, why is that important? Because cells are constantly adapting, they also, if you'll forgive me to an anthropomorphism here, they kind of "know" what to do in a given circumstance. If you put them int he right neighborhood, and they look around and say, hmm, I kind of see what's going on here. I think I know what to do. I'll give you an example of that, a very dramatic example. It's about not this summer, but the summer before. A neurosurgeon from Stanford published a study where he took patient who had had chronic stroke. They were chronic stroke patients, and most of you know that the conventional wisdom is, whatever function you recover after the first six months or a year after a stroke, that's about all you're going to get. We know now that there is some plasticity left over, and you can get more, but that's the conventional wisdom.
So, these were all chronic stroke patients. There's a woman who was in a wheel chair, another one who had a paralyzed arm, could barely speak. He did a craniotomy, and using stereotactic surgery, injected generic stem cells, these were mesenchymal stem cells from a couple of donors, into the area of the stroke. And these people, all these chronic stroke patients, recovered function, and in some cases even hard to understand neurologically how they recovered the function. The woman in the wheel chair was up and walking. I saw this video. Woman with the paralyzed arm, could barely speak, she's up and about, she was in her 30s, she's got married, she's had a baby. It is absolutely remarkable. They put stem cells into the area of the stroke. It's not clear, and they probably didn't form any new brain cells, but they acted as promoters of healing in the area there, and promoted new connections between the cells that were left over. This is neuroplasticity, I guess, at its best. Again, you put them in the right place, they sort of know what to do.
So, think about the implications, now, of cells being able to adapt and impact the biological neighborhood they're being put into. This really sets a stage for cells being the new drugs. They're going to, I believe, eliminate autoimmune diseases, beginning with type 1 diabetes. Now let's see if we have any better luck here with this. Alright, now I don't have my ... here we go. Why don't I have ... My desktop, my QuickTime player, let's try this. Let's see if we can make it happen this way. And the answer is no. Okay, how do I drag that ... oh, that's me. How do I bring that video down to where we can see it? See, I'm in the wrong union. I'm not allowed to touch any of the machines, the cameras, anything else.
Where's the video?
That's it. That should be it right there, I believe.
[Begin video 00:14:17] Type one diabetes has become a global health crisis. Solve the diabetes puzzle. From technological approaches like the artificial pancreas to so called smart insulin, that is automatically activated or suppressed in response to blood sugar levels. And several groups are developing beta cell implants that utilize a variety of immunoprotective strategies, including stem cells and non encapsulation to protect those replacement beta cells.
But for our purposes today, we'll focus on several cell based approaches aimed at restoring immune tolerance of beta cells. Spencer Frank was 23, backpacking in Southeast Asia, when he became very fatigues. His blood sugar had gone to 300. When he returned to grad school, he volunteered for a phase one diabetes clinical trial at UCSF, the University of California at San Francisco, under the direction of diabetologist, Dr. Steve Gidomen. While allowing the apparent stabilization of Spencer's diabetes, even in a phase one safety trial, is based on work in the UCSF lab of Dr. Jeff Bluestone. He and colleagues have learned how to identify and isolate from a diabetic patient a subpopulation of T cells in the immune system called T-Regs, short for T-Regulatory, that are defective in type one diabetics. When these T-Regs are greatly expanded in number in the lab and given back to the same diabetic patient, something remarkable happens.
We put back in a billion or two billion of these cells. We put in a lot of them, but also we know, after this two week period in which we grow them, that they work better. We've fixed some of the defects that we identified in these patients to begin with, and the cells that come out the other end are better than the cells that we took out of the person's arm, so we're not just putting back more of the cells. We're putting back better cells.
And it looks like this resets the delicate balance in the immune system to prevent autoimmunity. A phase two trial is getting underway to validate the concept.
A different approach to rebalancing the immune system appears to have helped young Ivan Trinovsky, a brutal type one diabetic. When conventional therapies didn't help Ivan, his mom took him to China for an innovative and unconventional immune system reset. That approach is now being elaborated in this lab, at Hackensack University Medical Center, where the innovator, Dr. Yong Zhao has come to prepare his technique for a US clinical trial. It uses umbilical cord stem cells to reset T-reg cells into tolerance.
Dr. Zhao accomplished the T-reg reeducation by adhering umbilical cord stem cells onto the bottom of a series of stacked Petri dishes. After extracting the patient's T cells, he percolates them through the dishes, where they interact with the stem cells. What makes this process even more noteworthy is what happens when these reeducated T cells are returned to the patient.
Once those cells get back in the body, they go find all the other activated cells, and they tell those cells, wait a minute guys, you're off base. You don't want to attack that. That's part of us, and it shuts the whole thing down.
But whatever method is used to shut down the autoimmune attack, the destroyed beta cells still need to be replaced. There is some evidence for beta cell regeneration, but most type one diabetics will likely need a fresh supply of insulin producing cells.
And that's where those other stem cells will come in. We've been learning how to take stem cells and differentiate them into insulin producing beta cells. But, think about what we just talked about here. This is a relatively straightforward approach to rebalancing the immune system. In Jeff Bluestone's approach, part of the trick is of course identifying just the T-regulatory cells, but he just takes them in the lab and expands them and creates billions of them, where he started with a few, and in the process of doing this expansion of the T cells, they revert back to their default genetic program, if you will, and they say, okay, I kind of know now what I'm supposed to do. I'm supposed to keep these T-effector cells, the ones that kill bacteria and viruses and in this case are attacking the beta cells, hold them in check and bring them back to where they're supposed to be doing, so now you've rebalanced autoimmunity.
Likewise, with Dr. Yong Zhao's approach there, the stem cells, they just touch and interact with these T cells. You're not putting any stem cells back in, but the stem cells again are kind of slapping the T cells around a little bit and saying, wake up guys. This is not what you're supposed to be doing and gets them to revert back to their default program, so they kind of know what they're supposed to be doing.
So think about this. And the reason, by the way, that they do this study in type one diabetes is because it's really easy to tell if you're having any impact on autoimmunity. You can take a look at blood sugar, you can take a look at how much insulin people need, and so it's fairly easy to tell whether you're having any impact on the autoimmunity here. But, if it works in type one diabetes, and you can rebalance the immune system and shut down autoimmunity, what's next? Rheumatoid arthritis, Crohn's, lupus, psoriasis maybe, MS. They might all be treatable with this autoimmune, cellular rebalancing or reeducation, so I think we've just started to scratch the surface here of how medicine is going to change in the 21st century.
I believe that cells will become not exclusively, but will become the new drugs or better yet, I think cells are going to be the new cure. Cells can be taken from your own body and re engineered, or in some cases, they'll be off the shelf cells, being manufactured and stored. That's happening right now. A number of companies are doing that right now. Generic cells, if you will, that are not going to be rejected. They can be used as drugs.
Cells know what to do. We just have to find the right ones, put them in the right environment at the right time, and then sit back and watch what nature, God, whatever you believe in, whatever you want to call it, do its thing. What it does best, and that's heal what ails us. It's an amazing revolution that I think is going to happen in our lifetimes. Maybe not mine, it'll happen in yours. You look a lot younger than I am. This is going to happen. I just hope that I live long enough to live longer and see it all happen. Thank you.
Should I take any questions, or are we going to lunch? What do you want to do? I'll take a question or two, if ...
Any questions? stem cell issue is such a ... was a hot issue for a while, we thought it was going away, now you're giving us some interesting and exciting news. ... stem cells are responsible, because they are the active soldiers of wound healing. Recognize the unwanted elements as far as individual cells are concerned, and then they initially want to kill them. However, once they realize they are one of theirs, they let them go and those cells that they let go kill the host in a way. It's passive host versus graft reaction, actually, and stem cells could be fooled. Endometriosis is a disease where stem cells are fooled. That's the say I see it. I hope everyone has an opinion on this, but ...
And by the way, just to ... because there's a lot of confusion, especially amongst lay people about stem cells. We're not talking about embryonic stem cells. Cells that come from embryos. And that in order to use them, you end up having to destroy an embryo, and that's they the Catholic church and a lot of other people are against using embryonic stem cells. We're talking about adult stem cells, here. When I went to medical school, there was really no such thing as an adult stem cell. We were taught you were born with all the brain and heart cells you're ever going to have, and then of course, you spend most of your college years trying to kill them off with tequila. And some of us were better at it than others. That is not what we're talking about.
It turns out, we have a lot of stem cells left over in our bodies after we're born, from birth on. We're talking about stem cells. We have stem cells in our brain, in our heart. Fat turns out to have lots and lots of stem cells, fortunately. In muscle, lining blood vessels. We've got stem cells everywhere. The trick here is getting enough of them, activating them, having them in the right place, so that they kind of know what to do and go out and repair the damage, so adult stem cells are where all the action is, and that's really I think one of the reasons why the Vatican was okay with these stem cell conferences that we've held. We've done three of them, now, and we're planning the fourth one for next spring. We got lucky. There's adult stem cells. You don't have to mess with embryonic stem cells.
There's a question from an anonymous individual. Anyone in New York who's using stem cells for neuropathy? Or neurogenic ... regeneration.
I can't say that I know off the top of my head. I should know that. Neuropathy is a little more complicated than putting some cells in and getting something and fixing something, but I don't know, I'm afraid.
I'm sure there are more questions. Any questions? Dr. Oktay.
This guy looks like he's got a tough question for me.
Dr. Gomez, this is Dr. Oktay. We met many years ago, but maybe you don't remember. My question is actually, I was intrigued by the stroke cases. I was wondering if those were adult stem cells injected into lesions, is that right?
Are those published cases? What was the source of that?
Yes. I don't remember ... it might have been the journal of neurosurgery, but they were definitely published. This was not just case studies.
It's fascinating. The references.
These were published in a high and peer reviewed journal. And again, the reason these adult stem cells are in most cases preferable to the embryonic stem cells, I want to make sure people get this. First of all, adult stem cells were thought to be only able to kind of the type of tissue that they came from. We've now learned how to de-differentiate them and re-differentiate these adult stem cells, so you can kind of make any stem cell tissue, any tissue that you want out of these adult stem cells.
We can also do now what Tenacka and Thompson independently figure out how to do, is take adult skin cells, use a virus to insert, the original technique used, 4 genes, and it turns an adult cell, not even an adult stem cell, an adult cell reverts back into an embryonic like state. They're called IPS cells. So, you can actually make, essentially, embryonic cells from adult cells by doing some genetic manipulation, so basically, we can do almost everything you can do with embryonic cells with adult cells or some version of an adult cell.
And, by the way, embryonic cells, one of the reasons that people aren't very clear on, embryonic cells have a dark side that not everybody knows about, and that's that embryonic cells sometimes form tumors. And not everybody realizes that, but if you think about it, an embryonic stem cell, and embryo starts as a fertilized egg becomes two cells, four cells, eight, sixteen and so forth. Multiplies like crazy until the time that a baby is born, you've got billions and billions and billions of cells, right? So, in an embryo, in a fetus, there are mechanical and biochemical signals back and forth that tell some cells to keep dividing, other cells to slow down, you can stop, alright we're ready. You can be bone, you can be muscle, you can be brain, you can be gut. That sort of thing.
If you take an embryonic cell out of its normal environment and now put it into a strange environment, an embryonic cell doesn't always know what to do, and it looks around and it says, I don't know what the hell's going on here, and it reverts back to its default genetic program, which is to multiply and to multiply and to multiply. That's cancer. So, it doesn't always happen, and we don't really know how often that happens, but that is the potential dark side, and that's why all the action these days is in adult stem cell research.
How difficult it is to isolate stem cells? Adult particular. Adult and placental stem cells same thing? I think placental is a cell that really sits in location and already ready to differentiate, right? And adult in both bone marrow and everything wandering around.
From bone marrow, it's easy. You can get stem cells out of bone marrow pretty easily. In fact, most bone marrow transplants are actually stem cell transplants. You can take placental cells, umbilical cord blood cells, most of those are stem cells, and that's actually become a very rich source for stem cells. Placental cells turn out to be really good sources of generic stem cells. They're being used for anti-aging strategies. Taking placental cells and it turns out, if you take these placental cells and infuse them or mix them up with old stem cells, you rejuvenate the entire stem cell population. Fat, there's lot of stem cells in there, and so extracting it ... it depends on which type of stem cell you're looking for, but it's not all that hard. There's probably gallons of liposuction fat being taken out even as we speak over here on Park Avenue, where you can throw a rock and you can't miss a plastic surgeon's office. There's lots and lots of stem cells that are probably being thrown away every single day.
Fascinating. Dr. Seckin, do you have any questions? Dr. Bulun, any comment, questions?