Empowered Patient Podcast Karen Jagoda

Salome Juethner, Senior Medical Director, Head of Rare Genetics, and Interim Head of Rare GI at Takeda, discusses HAE, hereditary angioedema, a rare genetic disorder that causes painful and unpredictable swelling attacks that can be life-threatening. Salome emphasizes the need to educate physicians to consider HAE as a potential diagnosis in children as young as two years old and underrepresented racial and ethnic groups. To treat HAE, Takeda offers Takhzyro, a medication that can be used on-demand and as a preventative therapy, administered at home through a subcutaneous injection. 
Salome explains, "Hereditary angioedema, or HAE, is a rare genetic disorder with a prevalence of one in 50,000. It can cause very painful, unpredictable swelling attacks that can occur in the throat, abdomen, hands, feet, and face. And as you can imagine, a throat attack could be potentially life-threatening. There are different triggers for these attacks. Some can just be stress and that could be positive stress like a wedding or going to prom or an illness and others. It can be quite limiting when you have to live thinking if I do something, is that going to trigger an attack? The symptoms can change over time, and HAE may not necessarily look the same for each person. Typically, people with HAE are missing an important protein in their blood called a C1 inhibitor. Either they're missing it or have very low levels, and it just may not work the way it should."
"For people who have never heard of HAE, things get a lot more difficult because they rely on their physician, who may or may not be aware of this very rare disease and consider it part of the differential diagnosis. So it is not uncommon to hear patients talk about going for years before they ever got their diagnosis and that they were experiencing symptoms during that time, going to the emergency room, and maybe even being misdiagnosed with another condition until they finally saw someone who thought, oh, you know what? We should screen you for HAE."
"Screening is really just a blood test. So, you would screen for a C4 level, which would be low or normal. If you're thinking about whether this could be HAE, then you would go ahead and check a C1 inhibitor level and a functional C1 level, and then those would be low as well. It's really about educating people, though, to consider it as part of the differential diagnosis." 
#Takeda #HAE #HereditaryAngioedema #RareDisease
takeda.com
Listen to the podcast here

Salome Juethner, Senior Medical Director, Head of Rare Genetics, and Interim Head of Rare GI at Takeda, discusses HAE, hereditary angioedema, a rare genetic disorder that causes painful and unpredictable swelling attacks that can be life-threatening. Salome emphasizes the need to educate physicians to consider HAE as a potential diagnosis in children as young as two years old and underrepresented racial and ethnic groups. To treat HAE, Takeda offers Takhzyro, a medication that can be used on-demand and as a preventative therapy, administered at home through a subcutaneous injection. 
Salome explains, "Hereditary angioedema, or HAE, is a rare genetic disorder with a prevalence of one in 50,000. It can cause very painful, unpredictable swelling attacks that can occur in the throat, abdomen, hands, feet, and face. And as you can imagine, a throat attack could be potentially life-threatening. There are different triggers for these attacks. Some can just be stress and that could be positive stress like a wedding or going to prom or an illness and others. It can be quite limiting when you have to live thinking if I do something, is that going to trigger an attack? The symptoms can change over time, and HAE may not necessarily look the same for each person. Typically, people with HAE are missing an important protein in their blood called a C1 inhibitor. Either they're missing it or have very low levels, and it just may not work the way it should."
"For people who have never heard of HAE, things get a lot more difficult because they rely on their physician, who may or may not be aware of this very rare disease and consider it part of the differential diagnosis. So it is not uncommon to hear patients talk about going for years before they ever got their diagnosis and that they were experiencing symptoms during that time, going to the emergency room, and maybe even being misdiagnosed with another condition until they finally saw someone who thought, oh, you know what? We should screen you for HAE."
"Screening is really just a blood test. So, you would screen for a C4 level, which would be low or normal. If you're thinking about whether this could be HAE, then you would go ahead and check a C1 inhibitor level and a functional C1 level, and then those would be low as well. It's really about educating people, though, to consider it as part of the differential diagnosis." 
#Takeda #HAE #HereditaryAngioedema #RareDisease
takeda.com
Download the transcript here

Lorie Spence and Carolyn Pritchard, Co-Founders of Bridge Medical Communications, focus on developing tools and resources to support healthcare professionals and patients in collaborating and making informed treatment decisions. They emphasize the importance of patient-centered care, participatory medicine, and patient engagement to help address therapeutic challenges and barriers that impact patient outcomes. By applying healthcare communication strategies and providing practical and functional tools for use at the point of care, they are bridging the gap between providers and patients.
Lorie explains, "Some tools we developed through Bridge Medical Communications through CONNECT really support healthcare professionals at the point of care. With the dynamic shift in the marketplace, with precision medicine and the need to engage the multidisciplinary team, we've developed tools like flow sheets that can be integrated into the EMRs to help assist and prompt through the steps of care from assessment of all the way to therapeutic onboarding and management."  
Carolyn elaborates, "Connecting is ensuring that the patient's voice and that the patient is, as you mentioned, participating in their healthcare journey. So we're trying to support industry and stakeholders in developing these tools so they can collaborate with the patients. As we've mentioned, it might be a patient counseling tool, like a transition tool, these types of things provide continuity of care critical between the patient, the healthcare provider, and often, caregivers."
#BridgeMedComms #PatientsVoice #PatientCentric #HealthcareProfessionals #ParticipatoryMedicine #HealthcareCommunications
bridgemedcomms.ca
Listen to the podcast here

Lorie Spence and Carolyn Pritchard, Co-Founders of Bridge Medical Communications, focus on developing tools and resources to support healthcare professionals and patients in collaborating and making informed treatment decisions. They emphasize the importance of patient-centered care, participatory medicine, and patient engagement to help address therapeutic challenges and barriers that impact patient outcomes. By applying healthcare communication strategies and providing practical and functional tools for use at the point of care, they are bridging the gap between providers and patients.
Lorie explains, "Some tools we developed through Bridge Medical Communications through CONNECT really support healthcare professionals at the point of care. With the dynamic shift in the marketplace, with precision medicine and the need to engage the multidisciplinary team, we've developed tools like flow sheets that can be integrated into the EMRs to help assist and prompt through the steps of care from assessment of all the way to therapeutic onboarding and management."  
Carolyn elaborates, "Connecting is ensuring that the patient's voice and that the patient is, as you mentioned, participating in their healthcare journey. So we're trying to support industry and stakeholders in developing these tools so they can collaborate with the patients. As we've mentioned, it might be a patient counseling tool, like a transition tool, these types of things provide continuity of care critical between the patient, the healthcare provider, and often, caregivers."
#BridgeMedComms #PatientsVoice #PatientCentric #HealthcareProfessionals #ParticipatoryMedicine #HealthcareCommunications
bridgemedcomms.ca
Download the transcript here

John Celebi, the President and Chief Executive Officer of Sensei Biotherapeutics, emphasizes the need to develop better patient selection strategies and next-generation therapeutics for cancer patients. Through their Tumor Microenvironment-Activated Biologics platform (TMAb), Sensei develops conditionally active therapeutics designed to be active only within the low-pH tumor microenvironment.  Current pipeline candidates are designed to be active only within the low-pH tumor microenvironment and show potential to overcome the challenges of VISTA, a promising checkpoint target.
John explains, "As an industry, we have some tough choices. Other strategies, both within and outside of IO, have made much better progress in targeting which patients will respond and which patients won't respond to treatment. Those are decisions that we have to make as an industry to keep up."
"The second one I would point to is that we're now in a decade after the first immuno-oncology drug approval. And so, we have a whole generation of patients now that have been exposed to checkpoint therapy, and that are now many of whom resistant to checkpoint therapy. We call that acquired resistance. And so the game has really changed. It's an open question: whether the first PD-1 drugs would be approved today if all of those patients had been treated with some other checkpoint therapy prior to it raises the bar. This means new strategies are needed to treat patients with acquired resistance to immunotherapy."  
"One of the interesting things about VISTA is that it does play a key role in the tumor microenvironment. The tumor microenvironment is unique because it's an environment inside the body in which cells proliferate very rapidly. That leads to a whole host of subsequent changes that affect the outcome of therapy, the fact that when cells are dividing rapidly, the pH in the area tends to drop, and it becomes more acidic. And that's directly related to the function of VISTA. It's one of the things that makes VISTA unique because VISTA is only activated as an immune checkpoint under lower pH conditions."
#SenseiBio #ImmunOncology #PatientSelection #Immunotherapy #Cancer #Tumors #ImmuneCheckpointTargets #VISTA
SenseiBio.com
Listen to the podcast here

John Celebi, the President and Chief Executive Officer of Sensei Biotherapeutics, emphasizes the need to develop better patient selection strategies and next-generation therapeutics for cancer patients. Through their Tumor Microenvironment-Activated Biologics platform (TMAb), Sensei develops conditionally active therapeutics designed to be active only within the low-pH tumor microenvironment.  Current pipeline candidates are designed to be active only within the low-pH tumor microenvironment and show potential to overcome the challenges of VISTA, a promising checkpoint target.
John explains, "As an industry, we have some tough choices. Other strategies, both within and outside of IO, have made much better progress in targeting which patients will respond and which patients won't respond to treatment. Those are decisions that we have to make as an industry to keep up."
"The second one I would point to is that we're now in a decade after the first immuno-oncology drug approval. And so, we have a whole generation of patients now that have been exposed to checkpoint therapy, and that are now many of whom resistant to checkpoint therapy. We call that acquired resistance. And so the game has really changed. It's an open question: whether the first PD-1 drugs would be approved today if all of those patients had been treated with some other checkpoint therapy prior to it raises the bar. This means new strategies are needed to treat patients with acquired resistance to immunotherapy."  
"One of the interesting things about VISTA is that it does play a key role in the tumor microenvironment. The tumor microenvironment is unique because it's an environment inside the body in which cells proliferate very rapidly. That leads to a whole host of subsequent changes that affect the outcome of therapy, the fact that when cells are dividing rapidly, the pH in the area tends to drop, and it becomes more acidic. And that's directly related to the function of VISTA. It's one of the things that makes VISTA unique because VISTA is only activated as an immune checkpoint under lower pH conditions."
#SenseiBio #ImmunOncology #PatientSelection #Immunotherapy #Cancer #Tumors #ImmuneCheckpointTargets #VISTA
SenseiBio.com
Download the transcript here