Andrew Rosen, Chief Executive Officer of the National Ataxia Foundation, has a dual mission of accelerating research for treatments and cures for Ataxia while supporting the patients affected by this rare disease. Ataxia describes both a group of hereditary genetic diseases and symptoms of lack of coordination seen in other conditions. While genetic tests exist for hereditary Ataxia, a significant challenge in drug development has been the lack of a reliable biomarker. To encourage drug development, NAF has funded translational research and the world's most extensive study for SCAs, Spinocerebellar Ataxias, providing new data on the progression of the disease and for the design of future trials.

Andrew explains, "NAF has been around for a long time. We were founded in the late 1950s by a neurologist in western Minnesota who had a disease called Ataxia in his family. And NAF's mission has really been twofold ever since that. Our formal statement is that we are trying to accelerate the development of treatments and a cure while working to improve the lives of those living with Ataxia. And so we really focus on research and support. We do a lot in the research world. We fund grants for researchers who are looking into the various types of Ataxia. You'd be hard pressed to find an Ataxia researcher in the world, I think I can say now at this point that hasn't received a grant from NAF at one time or another in their career. We also do a lot of translational-type research. We fund the largest natural history study in Ataxias."

"So, the term ataxia itself is even complex because it both describes a genetic disease, and I think of that as capital 'A' - Ataxia, but it's also a symptom of many other conditions. So small 'a', if you will. Ataxia just means a lack of coordination. It comes from a Greek word. And so what I mean by both hereditary and other things is if you, for instance, have too much to drink, you would show signs of Ataxia. You might stumble when you walk, and your speech might be slurred. Those are the two classic symptoms of hereditary Ataxia. Right. So that's really the Ataxias that we at NAF focus on. So hereditary, as in passed from parent to child, there are several hundred forms of hereditary Ataxia, and they continue to find more as more specific genetic mutations are discovered year after year now."

#NationalAtaxiaFoundation #Ataxia #PatientAdvocacy #RareDisease #SCA

ataxia.org

Download the transcript here

Anne Meneghetti, Executive Director of epocrates, is focused on medication management and providing tools for clinicians to better handle challenges from the increasing population of patients who take multiple drugs and are treating a wide variety of conditions. The epocrates app provides access to drug information, pill identification, checks for interactions, and calculates dosing, reducing medication errors and patient confusion. Polypharmacy patients are further at risk when they are taking over-the-counter drugs and supplements, requiring regular medication reconcilation to avoid serious drug interactions.

Anne explains, "So epocrates has been around since the late 1990s, and it's the number one mobile medical app in the country in terms of physician usership. And it's both a free model and a subscription model. The main reason why clinicians use epocrates is because of the drug information. So, quickly looking up a drug dose, for example, if a drug needs to be prescribed by weight, what is the calculation for that? And there's no way that clinicians can memorize 8,000 drugs. So having it in a quick, easy-to-use app is really crucial in modern times. And sometimes it's not that the clinician doesn't know the dose of the drug, it's just that validation. When you pick up an app and look and see, yep, that's what I thought it was. And that validation, that sense of confidence, is something really precious for a busy clinician. We also use it for drug interactions."

"We find that clinicians really value drug interactions because you can't memorize 8,000 drugs interacting with 8,000 other drugs. And your EHR might tell you if you're trying to prescribe two drugs at the same time, your EHR might tell you, oh, red light, green light, yellow light. But a really good drug reference, like epocrates can tell you, if you're going to prescribe these two drugs together, you need to cut the dose of this one in half and you need to monitor the potassium levels. We find that clinicians really value drug interactions because you can't memorize 8,000 drugs interacting with 8,000 other drugs. And your EHR might tell you if you're trying to prescribe two drugs at the same time, your EHR might tell you, oh, red light, green light, yellow light. But a really good drug reference, like epocrates can tell you, if you're going to prescribe these two drugs together, you need to cut the dose of this one in half and you need to monitor the potassium levels."

#epocrates #MedicationManagement #Polypharmacy #DrugInteractions #DigitalHealth

epocrates.com

Download the transcript

Dr. Adam Kilian, Director of the Rheumatology Fellowship Training Program at the St. Louis University School of Medicine, focuses on IgG4-related disease, a rare multi-organ disease that has only recently been recognized due to significant diagnostic challenges. The MITIGATE trial is a landmark study that demonstrated that the first FDA-approved treatment, UPLIZNA from Amgen, provides an effective, targeted, steroid-free therapy. The approval of this drug is driving awareness in the medical community about IgG4-RD and whether the disease should be considered after inconclusive results for other suspected conditions.

Adam explains, "IgG4-related disease is a rare disease that affects many organ systems. It's a chronic systemic, fibroinflammatory disease that can affect almost any organ system. And it will usually present with these tumor-like inflammatory masses that can cause scarring and lead to organ failure."

"Our understanding of it continues to evolve, and it's had a really interesting story over the last century because IgG4-related disease affects so many different organ systems. Over the past century, there were actually many different diagnoses that were recognized, which now all fall under the umbrella of IgG4-related disease. Different scientists and physicians around the world over the years recognized the different manifestations of the disease in the pancreas, in the lymph nodes, in the head and neck, in the kidneys, or in the blood vessels. And it's only been in the last 20 years or so that we have recognized that actually all of these different disease entities from the last century are actually all manifestations of this disease, IgG4, IgG4-related disease."

"The clinical trial is called the MITIGATE trial, and it's a very seminal clinical trial in rheumatology from the last several years. This was the first-ever phase three double-blind, placebo-controlled, randomized controlled trial in IgG4-related disease. This was a huge trial. It was global. It was conducted in 22 countries with multiple specialties coordinating this trial. It was 52 weeks long, and its purpose was to evaluate the effectiveness and the safety of UPLIZNA in patients who have IgG4-related disease."

#MITIGATETrial #IgG4RelatedDisease #IgG4RD #UPLIZNA

UPLIZNA

Download the transcript here

Dr. Thomas Wisniewski, Director of NYU Langone's Alzheimer's Disease Research Center, shares insights from a recent study that projects new dementia cases in the US will double by 2060. New diagnostic tools have made it easier to identify early signs of Alzheimer's, and disease-modifying therapies are being approved, which can be effective if patients are treated in the earliest stages of the disease. AI is seen as a tool to help identify at-risk patients and routinely screen patients to manage the growing need for access to dementia care.

Thomas explains, "This was a study that was led by the NY Optimal Institute, which is directed by Dr. Joe Resh, who's really a leader in this area and many public health issues. He did a very thorough analysis along with his team that the annual number of incident dementia cases in the United States is projected to increase from current estimates of 500,000 to around 1 million in 2060. So basically doubling in white adults. Furthermore, in African Americans, this incidence of dementia rate is expected to triple, with the largest absolute increases in dementia cases going to be in the oldest old population."

"There was perhaps a lack of awareness of the prevalence of dementia in past decades. But now the importance of making an accurate diagnosis and recognition of dementia is becoming much more prominent in the medical literature and in the medical profession. And that message, I think, is permeating to the lay public as well. So there hasn't been a change in the definition so much, but there is an increase in knowledge in the medical profession about the importance of making this diagnosis, and people living alone."

"It's really been a dramatic change. So, apart from being a cognitive neurologist, I'm also a board-certified neuropathologist, and it used to be that making the definitive diagnosis of Alzheimer's disease required a chunk of brain. I'm delighted I don't need those chunks of brain anymore to make a diagnosis of Alzheimer's disease. There are now very clear clinical criteria and biomarker definitions for making a very accurate diagnosis of Alzheimer's disease."

#NYULangoneAlzheimers #AlzheimersDisease #DiagnosingAlzheimers #AlzheimersResearch #MedAI

med.nyu/centers-programs/alzheimers-disease-research/

Download the transcript here

Pedro Sanchez de Lozada, CEO of Canid, highlights the significant financial risks that pediatricians face in managing their vaccine inventory. There are administrative burdens associated with ordering, patient demand, tracking, reporting, and filing for insurance reimbursement. The Canid platform was designed to be a vaccine-as-a-service model, removing risk and administrative work from pediatric practices, allowing small and independent practices to achieve economies of scale, and freeing up more time to spend with patients and their parents.

Pedro explains, "When managing a vaccine program, the expectation is that the pediatrician will buy and maintain the stock in their office until a patient comes in and gets vaccinated. So, the refrigeration, the temperature logging, the stocking up, and the cost of the vaccine always fall as liability to the pediatrician. And what happens there is that you have a fairly small margin on these vaccines. Now I'm sure we're going to get into this, but when people say pediatricians make money on vaccines, they're usually cherry-picking."

"But in reality, as a pediatrician, you actually have to stock all the vaccines. And not all of them have decent margins. Some of them have negative margins. And so as a portfolio, you end up making a pretty thin margin, and then that doesn't even include all the different challenges you might have along the way. So let's take an example: a vaccine that expires or is dropped, and unfortunately needs to be wasted."

"So the way that we like to think about it is we want to make it so that the pediatrician can just give vaccines. That's all they need to think about. And what that means is, let's just imagine for a second, a refrigerator that automatically restocks itself, and you just take out the vaccines, you give it to a child, and you go on your merry way. That's obviously a little bit of an idealistic way of viewing things, but it's the experience that we want to accomplish."

#Canid #Vaccinations #Pediatricians #Vaccines #PediatricVaccineManagement

canid.io

Download the transcript here

Alicia Silver, senior director at ADVI Health, highlights the evolving landscape of cell and gene therapy and the need to improve patient access and payment for these treatments. Availability of these therapies for solid tumors and genetic diseases like sickle cell disease is expanding due to the transition from inpatient to outpatient and community settings. The FDA's decision to remove REMS requirements for specific therapies has accelerated the growth of facilities to provide care, particularly for vulnerable populations.

Alicia explains, "We work with a number of different cell and gene therapy clients throughout the sector. So we work with manufacturers who have commercialized cell and gene therapy products. So they have products that are currently on the market, manufacturers who are going through the process of clinical trials right now, working with the FDA to get approved products. But we also work with trade organizations that are working behind the scenes at the sector level, trying to get different policies and access changes for patients."

"To date, there's probably close to a couple of dozen FDA-approved cell and gene therapies, and they treat everything from blood cancers, which were the first approvals in something called CAR T. We saw blood cancers as the first approvals, and then everything through to solid tumors in oncology. But also, we have newer gene therapies for conditions like sickle cell disease. And that's an area that's been incredibly underserved and definitely will benefit from a durable gene therapy that hopefully corrects some of the issues that patients with sickle cell disease have, like pain crises that end up in a hospital. So from that perspective, we see a really wide range of treatments available to patients today and many more on the horizon."

"I think the price tag is definitely somewhat of sticker shock for people who don't understand how cell and gene therapy products are valued. And so what we do a lot of times, educating on, is helping payers understand that it's not necessarily $2 million for a treatment that's going to be a recurrent payment, but something that's kind of an investment in the patient's and the plan's future."

#ADVIHealth #CellTherapy #GeneTherapy #AcesstoCellGeneTherapy #ClinicalTrials

advi.com

Download the transcript here

Dr. Stacy Lindborg, President and CEO of IMUNON, has developed a DNA-based immunotherapy candidate for the treatment of ovarian cancer. This therapy works by administering the drug, which utilizes IL-12, a powerful anti-cancer cytokine, directly into the cavity where the cancer resides, thereby affecting the tumor microenvironment. Trials are showing that the drug can produce significant life extension when used in combination with standard chemotherapy.

Stacy explains, "There are about 300,000 women who are newly diagnosed with advanced ovarian cancer every year, about 20,000 in the US, and the frontline standard of care hasn't seen a change in the treatment in about 25 years. So this is for newly diagnosed women. The very first treatment that they would have, which we call frontline treatment, is a platinum-based chemotherapy. So carboplatin and Paclitaxel are both administered through IV over an hour for carboplatin, about three hours for Paclitaxel. So most women go directly to chemotherapy and then surgery, and then chemotherapy. Some women will go straight to surgery and then have this chemotherapy afterwards."

"Our approach is a DNA-based immunotherapy candidate that we have in Phase 3. We refer to the lead candidate as IMNN-001, and this is a non-viral nanoparticle that is administered directly into the cavity of interest. So, what we call the micro-tumor environment is the peritoneal cavity, where the cancer actually resides, and it's delivered through a catheter. And it basically has a very powerful anti-cancer fighting cytokine, IL-12, that is encoded in this immunotherapy. And it causes the cells that are within each woman's body, both cancer and non-cancer alike, to start producing activities that will help fight these complex cancer cells that exist."

#IMUNON #OvarianCancer #DNABasedImmunotherapy #WomensHealth #CancerAwareness

imunon.com

Download the transcript here

Donovan Campbell, CEO of Medbridge, empowers clinicians through a platform that is focused on movement-based medicine and a holistic approach to patient care. MedBridge has developed AI tools to perform triage, act as a care coordinator, and serve as a patient-facing coach, offloading administrative tasks and providing better support to patients. These tools are designed to support clinicians by flagging potential issues and increasing patient engagement by delivering immediate digital access to care.

Donovan explains, "We serve primarily physical therapists. And those physical therapists can work either in what's called a private practice outpatient setting or in a hospital setting, whether that's inpatient rehab or outpatient rehab. We also service physical therapy and adjacent practitioners, such as athletic trainers, speech and language pathologists, or occupational therapists. With the release of our new hybrid care platform, we're moving into different care categories, which is really exciting. So we're increasingly working with advanced primary care companies that service the employer health market. That's exciting. And we're exploring other opportunities within women's health and within orthopedics."

"Movement-based medicine is relevant to a really holistic approach to patient care, and it spans multiple clinical specialties. So think about the elderly patient who's just had a right knee replacement. Movement-based medicine is critical to getting them back up and on their feet. For a woman postpartum, pelvic health and movement-based medicine are really important. And pelvic health, movement-based medicine that she can access at home, is really important in her recovery and meeting her where she's at in her phase of life. And then physical therapy obviously is all about movement-based medicine."

#Medbridge #DigitalHealth #HealthcareAI #FutureAI #MovementBasedMedicine

medbridge.com

Download the transcript here