Dr. Joshua Hare, MD, co-founder and Chief Science Officer of biotech company Longeveron, and founding director of the Interdisciplinary Stem Cell Institute at the University of Miami Miller School of Medicine, discusses two published positive clinical studies of stem cell treatment for aging frailty and how Longeveron is progressing with development of stem cell therapies for aging-related conditions to improve quality of life. www.longeveron.com
Dr. Joshua Hare co-founded Longeveron in 2014 utilizing intellectual property and technology exclusively licensed from the University of Miami, where he is also the founding Director of the university’s Interdisciplinary Stem Cell Institute (ISCI). At the University of Miami Miller School of Medicine, he is chief of the Division of Cardiology, and Louis Lemberg professor of medicine and professor of molecular and cellular pharmacology. Dr. Hare graduated from the University of Pennsylvania and has a medical degree from Johns Hopkins University. He did his residency at Hopkins and fellowships at Hopkins, Harvard University and The Brigham and Women’s Hospital in Boston. Dr. Hare was Professor of Medicine and Biomedical Engineering and director of cardiac transplantation at Johns Hopkins, prior to joining the faculty at University of Miami.
Neal Howard: Welcome to the program. I’m your host, Neal Howard here on Health Professional Radio. Glad that you could join us once again. Our guest is Dr. Joshua Hare Co-founder and Chief Sciences Officer of a company called Longeveron and he’s here today to talk about a couple of clinical studies of stem-cell treatments for aging frailty and how his company is aiming to improve quality of life for those of us who are aging. Welcome to the program, Dr. Hare. How are you?
Dr. Joshua Hare: I’m fine. Thank you for having me.
N: Now, other than being the Co-founder of Longeveron, give our listeners a bit of background about yourself.
J: Yes. I’m a practicing physician. I’m a cardiologist. I direct the stem-cell research program at the University of Miami and I’ve been studying the use of adult stem cells as a therapy for a variety of diseases for about 20 years now.
N: So, you’re very familiar with stem cells, some of the controversies, some of the recent advancements. Today, we’re talking about stem cell research and developments as they relate to aging. I understand that there’s a difference between successful aging and accelerated aging. I had never heard of either those terms. What is successful aging?
J: Yes. You have asked a very important question. Most of us think that aging is inevitable. Aging is inevitable but the rate at which we age is different amongst people. So if you take an example of people who are 80 years old, some of those people are in the nursing home or in a wheelchair requiring support and others remain very active. The difference between those two types of individuals in that age range is frailty. So some of us, as we age develop frailty at a greater rate and we think that’s because of stem cell depletion and inflammation. So we all have different levels of inflammation and stem cells and those of us with more inflammation and fewer stem cells are going to age faster and develop some symptoms that we call frailty.
N: Okay. So just to be clear, frailty – is that an all-inclusive term or is it a specific set of circumstances having to do with age?
J: Well, you can have frailty associated with other diseases. But in the case of aging frailty, it is a specific syndrome that geriatricians define and can categorize and are attempting to treat.
N: There are two studies. Do both of these studies that we’re going to talk about address frailty and successful aging or are the studies totally unrelated but still related to the quality of life as that quality of life relates to aging?
J: Well, our goal, they are related because we think if we treat people with frailty we’re going to convert them on to a trajectory of more successful aging. So if you think of frailty as a greater age-related decline in physical capacity, what we wanted to do in the studies was treat people with stem cells again because I recall I mentioned that aging frailty is associated with stem cell depletion. So our idea was to give the stem cells back and see if people are going to be more physically active and functional as a result of the treatment.
N: Where are these stem cells coming from?
J: They’re coming from a donor. So we’ve developed a technology where we can take themselves from a young healthy donor and amplify them in large quantity so that we can use them to treat many different patients. And what we found is that the cells are what we called immuno privileged or immunoevasive. So we can take them from one person and give them to another without matching. The cells came from a young healthy donor source.
N: Those are the stem cells that must be harvested or are those the stem cells that lend themselves best to this study? I thought that there were ways that a person’s own stem cells could be stimulated for new growth. Is that a myth or miss…?
J: No. There are different ways in which we can get stem cells from a person and give them back to them. However, that’s very cumbersome and also for an older person remember the frailty result is due to depletion of stem cells. So if we’re taking a person’s own stem cells and giving them back to them that’s A – cumbersome and B – may not work as well as, that’s why we give them a young healthy source. That’s the whole idea behind having what we call it off-the-shelf stem cell whereby we’ve prepared it in advance, we’ve quality controlled it. We know that it’s effective and therefore that is the idea behind our project.
N: Let’s talk about the first study and some of the results from that study.
J: Yes. So the first study was basically the phase 1 study which is designed to just show safety and we also did a dosing escalation. We gave three cohorts of patients, three different doses and our primary goal is to make sure it was safe but we were also measuring the people’s functional capacity, how far they could walk, how well their pulmonary function was, their lung function, how well their cognitive function was and their levels of inflammation. And we were very pleasantly surprised to find that first of all, it was very safe. Nobody had an untoward bad outcome and when we measured how well people were doing, lo and behold they were doing much better. Their physical capacity was improved on all of the measurements we listed. So we were very pleased with the results of that first study in which everybody got the stem cells at different doses.
N: Even cognitive skills were improved.
J: Yes. We did something called a Mini-Mental score which is a widely used score that neurologists use and it was improved by a statistically significant amount in this particular study.
N: Were there any patients who were dealing with Alzheimer’s either early onset or later stages or middle stages of Alzheimer’s that were improved on that level?
J: We specifically for this study did not include people who had cognitive impairments or Alzheimer’s disease. But because of the result, because the results were statistically significant we did and have gone on at Longeveron to now study specifically a cohort of people with cognitive impairment and Alzheimer’s disease. Because of the finding, we thought and did turn to a specific study of Alzheimer’s disease which is now ongoing and put at the present time.
N: It’s my understanding that there is currently no FDA-approved treatment for aging frailty even though it’s a serious and life-threatening geriatric syndrome as you pointed out.
J: That is correct.
N: Where does your company fall? I mean you have some successful studies here. What do you see on the horizon for approval of some of your developments?
J: We’ve applied to the FDA. We were working with the FDA to try to get a registration study done. Meaning a study that is successful that could lead to approval. So we firmly believe that this treatment should be and an FDA-approved product. Meaning that it fulfills all of the regulatory requirements of the FDA. We want to make sure that it is safe and effective and that we do all of the studies that the FDA requires. But we do believe that working with the FDA and particularly under some of the new provisions that they have enacted for regenerative therapies a very big development was the passage of the 21st Century Cures Act by Congress which provides specific guidelines for how stem cell therapies should be approved in the United States and we’re working with the FDA on those particular issues so that we could get this approved.
N: Well, with somewhere around 96 million Americans or so being the age of 65 or older by 2060 is some very exciting news on the horizon.
J: That is correct.
N: Where can we learn more about your company online?
J: We do have a website www.longeveron.com and that should be easy to find and we have a lot of publications in the medical literature that’s all listed on our website. So whether you’re a physician or a patient you can find out a lot of information about us on our website.
N: Thank you so much for talking with me today, Dr. Hare. It’s been a pleasure.
J: It’s a pleasure to be with you. Thank you very much.
N: You’ve been listening to Health Professional Radio. I’m your host, Neal Howard. Transcripts and audio of this program are available at healthprofessionalradio.com.au and also at hpr.fm. You can subscribe to this podcast on iTunes, listen in and download a SoundCloud. And be sure to visit our affiliates page at healthprofessionalradio.com.au and also at hpr.fm.