Empowered Patient Podcast

Chris Hoyt, CEO of KeifeRx,  talks about the oral medication being developed that uses tyrosine kinase inhibitors (TKIs) and autophagy to remove unwanted proteins.  Research done by Georgetown University on leukemia, led by Dr. Charbel Moussa, found that lower doses of drugs like nilotinib and bosutinib could cross the blood-brain barrier and remove toxic proteins on an intracellular basis.  This research has been used to develop treatments for neurodegenerative conditions with promising results in reducing cognitive decline. Chris explains, "Essentially, the way TKIs work in the body, and particularly in the case of the neurodegenerative conditions that we're using them to treat at KeifeRx, is they trigger a mechanism called autophagy, which essentially is the cell's garbage disposal mechanism. What we're doing with TKIs is using that mechanism to remove toxic proteins. TKIs have mostly been used historically in cancer, and particularly in leukemia. The classic use would be in cancer to try to remove as muc

Simon Allen is the CEO and Director of Anebulo Pharmaceuticals, which focuses on finding an effective treatment for acute cannabinoid intoxication or ACI, particularly in an emergency department setting. ACI is a condition where an individual has consumed or smoked too much cannabis or products that contain THC or synthetic cannabis and is experiencing symptoms of psychosis, anxiety, elevated heart rate, or is unresponsive. Their drug ANEB-001 is designed to address the cause of the symptoms without the need for traditional methods like benzodiazepines and beta blockers, which have their risks.  Simon explains, "What we are seeing is individuals taking these high-potency edibles, for example, and also smoking and vaping. These are other forms that can create ACI outcomes. And essentially, they're out for several hours, if not overnight, in the hospital. This can get as serious as the escalation to a psychiatric ward if the individual is so psychotic, agitated, and anxious that the emergency department can't

Nancy Lurker, CEO and Board Director of EyePoint Pharmaceuticals, aims to prevent the progression of blindness by developing technology that addresses retinal eye diseases. These back of the eye diseases are age-related and an increasing threat to populations worldwide. Current treatments require regular injections in the eye. The micro inserts developed by EyePoint will allow the drug to work for six months, allowing patients more flexibility to maintain their vision.  Nancy explains, "We know that if you look at the current eye treatments on the market today, wet AMD, retinal vein occlusion, diabetic macular edema, and diabetic retinopathy, all of those are running close to $30 to $40 billion being spent to help to treat these terrible eye diseases which may lead to blindness." "So, what causes these diseases? There's a genetic component at times, but also age plays a factor. And then, of course, diabetic macular edema and diabetic retinopathy can be caused for patients who have diabetes. So. what happens

Dr. Gavin Samuels, Chief Business Officer at CinRx, a holding company currently with six portfolio companies, each focused on developing one drug. Providing these companies with the right resources at the right time makes for more efficient use of resources and can speed up drug development.  Their strength is understanding challenges and opportunities approximately a year before an IND is filed to help shepherd the drug through the IND-enabling study and through clinical development.  Gavin elaborates, "The disease areas that we're involved in, cardiovascular disease, metabolic disease, inflammatory diseases, these are problems that present a real challenge to patients and a real challenge to society and a real challenge to humanity. We want to try and address those problems by bringing innovative drugs and developing them as quickly and efficiently as possible and bringing to the patient." "The criteria we use is that it has to fit a specific unmet medical need. We have to have a large group of patients th

Jennifer Quinn, Head of global pricing and health economics and outcomes research at Debiopharm, emphasizes the importance of reimbursement, not just regulatory approval, for a drug to be available to patients.  With patient-centered outcomes research, drug developers can use data for risk-benefit assessment relevant to regulators that reflect how patients consider the risks and whether they will undergo a procedure. Jennifer explains, "My team really tries to make sure that, when we're thinking about a clinical development program, we make sure we're capturing those value markers throughout from a very early stage. That is so that when someone out-licenses it and becomes our partner, they have the data they need not just to get through regulatory approval but to have the drug reimbursed and made available to patients." "It's so important that patients have not just the agency in their care, but we understand how treatments are impacting patients -- impacting their lives, impacting how they feel, impacting t

Ohad Arazi, CEO of Clarius, a medical imaging startup with a mission to bring accurate, easy-to-use, AI-assisted, and affordable hand-held ultrasound tools to a broad range of medical professionals. Traditional ultrasound is costly, stationary, and requires specialized training. The portable Clarius device communicates with an iPad or an Android or iOS phone. The ultrasound produces highly nuanced grayscale real-time images that, with the help of artificial intelligence, can be used safely in a wider point-of-care environment to make informed decisions. Ohad explains, "We're all about bringing together high-performance ultrasound imaging, cloud data, and artificial intelligence into this powerful ecosystem that improves patient care enabled by medical imaging in every setting. The key differentiation is that handheld ultrasound is not about replacing legacy systems in places where they're already well entrenched, like radiology or cardiology. It is rather about bringing medical imaging to new care settings an

Alina Czekai, Vice President of Strategic Partnerships at Cohere Health, points out the need to transform the often painful prior authorization process. The traditional approach takes a piecemeal view of requests and provides a yes or no response. The Cohere view looks at the process as a multidimensional challenge that requires understanding the entirety of care and leveraging technology and AI to help guide behavior toward what is most clinically appropriate. Alina explains, "You can think of it as an authorization for a credit card. Ensuring that what is going to be done or what medication or devices are going to be ordered is actually going to be paid for. The process and payments in the healthcare system today have become so complex. It's complex not only for the health plan that's administering payments for care but also for physicians and their staff. Most importantly, it's a confusing and stressful process for patients." "We believe that prior authorization is a process that can be automated, that sh

Jared Crapo, Head of Product and Technology at Kalderos, focuses on drug discounts paid by drug manufacturers to pharmacy benefit managers, commercial or government payers, and healthcare providers. Congress created the 340B Drug Discount Program and the Medicaid Drug Rebate Program to support healthcare for underserved patients. While these programs have dramatically grown, so too have the complexities and confusion about discounts and disagreements about payments. Jared explains, "The 340B program was created by Congress, and it's a subsidy from drug manufacturers to underserved healthcare providers. The Medicaid Drug Rebate Program is a similar program created by Congress that is a source of financial support for state Medicaid agencies, also provided by drug manufacturers. So drug manufacturers give a discount to state Medicaid agencies for drugs prescribed to Medicaid patients." "One way to think about it is there's a lot of information asymmetry, which means some stakeholders have access to a lot more

Jessica Ackerman, the Vice President for Strategic Partnerships and Impact at Responsum Health, is focused on the insights that patients can provide drug companies and other people newly diagnosed or suffering from a chronic condition. Responsum is a digital-based educational community providing accurate scientific information, peer support, access to clinical trials, and a path by which drug companies can get real-world data.  Jessica explains, "Every disease focus that we have started has come from a partnership, some vested interest, from either a commercial sponsor or a pharmaceutical company, to build this robust community. Their interest is to gain the patient's voice and get insights, market research data, and real-world data from patients and family members to understand their gaps in care. So we have created a number of communities, like kidney disease CKD. We have glaucoma. We have pulmonary fibrosis, which is considered a rare disease, so we are in the rare disease and orphan disease space as well

 Dr. Phil Wagner, Founder and CEO of Sparta Science, is developing a movement health intelligence solution to better understand the overall health of a patient and their risk of injury.  They are interweaving movement ability and balance ability to analyze meaningful biomarkers to ensure top performance and safe mobility. From his experience as a sports trainer, Phil warns about making assumptions about performance at any age. Phil explains, "We do a balance assessment to assess that movement health, and it takes about 20 seconds on each side, and that will actually generate about a million data points on our device. This feeds into software that uses machine learning to compare you against yourself but also around others in a similar cohort, whether that's similar ages or sex at birth. So, being able to compare yourself against your own previous baselines and assessments, but also compare yourself for norms against others." "We're working with the military on a TBI biomarker, traumatic brain injury because

Dr. Clark Chang is a cornea and keratoconus, KC, specialist at Wills Eye Hospital in Philadelphia and the Director of Global Medical Affairs at Glaukos. Keratoconus is a disease of the eye where tissue thins and gradually bulges outward into a cone-like shape.  While changes in the cornea are small, vision becomes very blurry, and the underlying cause may be overlooked. Glaukos has introduced the iLink procedure, a minimally invasive outpatient procedure to slow or halt the progression of this disease and help patients preserve their vision. Clark explains, "It is associated with age, but it's in the different group of people than most of your audience is thinking. We're very familiar with conditions like age-related macular degeneration. Everybody knows that because it's very impactful to one's vision, and the fear of loss of vision has frequently been rated as one of the top one or two biggest fears in a person's life. Most diseases are age-related in that they can occur with time. This disease more common

Dr. Antonin (Tony) de Fougerolles, CEO of Evox Therapeutics, highlights that exosomes, small nanoparticles, are produced by every cell in the body.  They contain RNA and proteins that the cell decides to put into the exosome, and they're secreted out into the environment and act as a way that cells communicate with each other. Evox is excited about using exosome therapeutics to load genetic medicines such as RNA interference, small RNA drugs, gene therapy, or genome editing into exosomes and effectively delivering them inside cells. This approach provides a new way to treat rare diseases with an underlying genetic cause. Tony elaborates, "Exosomes were first discovered or noticed in the scientific literature in the '80s and the early '90s, and at first were thought really just to be vesicles that cells excreted full of what people at the time thought was garbage, things the cell didn't need. In more recent times, in the last 10, 15 years, it's become clear that these exosomes can in some cases serve a functio

Dr. Eugene Chan, Chairman and Co-Founder of Abpro, talks about the next generation of antibody therapies that can target more than one target to activate the immune system in the fight against cancer. Abpro is also working on a prophylactic antibody therapy to help prevent immunocompromised patients from getting COVID. Eugene explains, "Abpro has developed the antibodies, in a lot of these cases, to be able to target more than one target. This basically activates the immune system more so that whatever you're targeting is cleared more rapidly from the body. This applies to the cases of cancer that we are focused on, and we've basically developed these antibodies called bispecific antibodies - bi for targeting two different sites. These antibodies essentially have more and greater activity than conventional antibodies." "We developed a program and antibodies that bind to HER2+, which is one of the molecular markers on breast and gastric cancers. This particular therapy brings the immune system, the body's own

Dr. Edith Perez, Chief Medical Officer at Bolt Biotherapeutics, describes the innovative approach Bolt is taking to bring together the precision of antibodies against the tumor with the power of the innate and adaptive immune system by activating the immune system at the tumor site.  This approach can minimize side effects from systematic activation of the immune system and reinforce long-term anti-tumor efficacy. Edith explains, "The myeloid cells are a wide range of cells in the body, and initially, the myeloid cells can start attacking the tumors right away. But as important, or even more importantly, is that they secrete the cytokines and chemokines to teach the T-cells to essentially develop an immunological memory to be able to fight these foreign bodies for a long time."  "First of all, there are the abnormalities of the tumor themselves, and we target those with monoclonal antibodies. Then we actually link that with what we call a non-cleavable and non-cell permeable linker. Again, the idea is to bri

Dr. Romain Micol, CEO, and Dr. Tom VanCott, Chief Scientific Officer, at Combined Therapeutics, are excited about the opportunities to design mRNA vaccines that target specific tissue cells to restrict protein expression and have stronger potency in the fight against infectious disease and cancer. They have designed their vaccine platform to keep the vaccine antigen expression at the injection site, protecting the liver, kidney, and heart.  Romain explains, "So mRNA vaccines are highly effective both for infectious diseases and, more recently, to treat cancer. It's an ideal technology that is relatively easy to make and to manufacture. It could be very rapid and can get expression in vivo where protein would be folded properly. We can currently use the mRNA vaccine to deliver multiple materials to prevent infectious diseases and also cure cancer." "The main challenge we see at Combined Therapeutics, both for vaccines for infectious disease and for cancer treatment, is in their biodistribution and potency. Ou

Nick Hedges is the CEO of Raintree Systems, a provider of software-as-a-relationship to help therapy and rehab companies enhance their patient engagement, provide access to an electronic medical record system, and improve revenue cycle management. Using predictive models and a digital approach to billing and reimbursement, Raintree is reducing the friction for patients, providers, and payers. Nick explains, "A lot of people describe software these days as delivered primarily through the internet as software-as-a-service. However, service doesn't really tell you the full story about how we interact with our clients. We see our software as being a partnership. We work very closely with our clients to make sure that it delivers exactly what they need, and we build our product in unison with our clients. That's why we call it software-as-a-relationship." "Healthcare, in general, is quite far behind in terms of the digital patient experience or the digital consumer experience. One of the things that Raintree seek

Whitney Stewart, the Director of Clinical Project Management at Curebase,  and Megan Coder, the Chief Policy Officer of the Digital Therapeutics Alliance, have worked together on a white paper Setting the Stage for a Fit-For-Purpose DTx Evidentiary Standard. The goal is to outline foundational principles that, in some cases, are unique to the digital therapeutics category. Their focus is on baseline expectations related to procedures and the type, quality, and timing of clinical trials necessary to evaluate and widely implement DTx therapies.   Megan explains, "Given some of their development pathways and processes, given how they're regulated as a medical device and not a drug, we've really started talking with Curebase and some of our other members. We are looking at, and we recognize that, these digital therapeutic products have great value to patient care. They can be used alongside or sometimes even in place of some of these traditional therapies. But what became evident to us pretty quickly is that the

Karen Smith is the Founder of Catie's Cause, a nonprofit charity dedicated to building awareness about the safe use of over-the-counter pain medications.  After the death of her daughter Catie from liver failure due to overuse of Tylenol, Karen founded this organization to shine a light on the unintended and intentional side effects of easy access to these medicines. Karen explains, "The research shows that there are four main groups of individuals who are impacted by acetaminophen poisoning. Young adults definitely are a large group, oftentimes because they're on their own and they don't realize the dangers of medication. Some of them do begin to abuse it and they take it way too regularly. Sadly, about 50% of the deaths are from suicide, so obviously intended, but 50% of them are unintended. So young adults are a big group."  "Another group is young children because parents tend to freak out when their little ones get a headache, and they overmedicate. The third group impacted are the elderly because they

Dr. Jing Wang is the Dean and Professor at Florida State University College of Nursing and is focused on bringing a high-tech, high-touch approach to patient care in the education of nurses. The idea is to leverage technology and digital health tools to support nurses so that they can spend more quality time with patients. The newest generation of students are digital natives, and one goal is to help them use that knowledge to smooth the transition for the less technology savvy. Jing explains, "I think it's all about how technology is introduced into the learning in our current nursing education and health profession education. That is critically important. That's what we are doing here at Florida State University College of Nursing. We would like to do it in the right way, to introduce technology as a tool to enhance care and facilitate care versus this is a fancy technology, we must use it. I think that's core, and that's the attitude that we are hoping our graduating students will have." "We have launched