Empowered Patient Podcast

Joy Duemke, Director of Marketing North America at Terumo, BCT, and Barry Liden, President and Managing Advisor of Patient Voice Advisors, highlight the importance of medical device companies engaging with patient associations to incorporate patient feedback into the design and development of their products. Patients can provide unique insights to engineers and researchers that may lead to better outcomes and patient experiences. The discussion focuses on the collaboration related to improving blood therapy delivery for sickle cell patients.

Barry explains, "So if medical device companies want to ensure that their technologies are going to get to the people that need them and that those folks want to be able to take potentially some risks of having the device implanted or used on them, they need to listen to the patient and understand what kind of outcomes are important to them and what they're willing to tolerate."

Joy elaborates, "With medical devices, it's very personal to the patient. It's not a bag that's hung or a pill that's taken, but it's typically something that might be, as Barry mentioned, implanted in their body or interacting very closely with their body. So the processes are very technical. There's often a high amount of training that has to happen with those operators, and it's often even a specialty department that might utilize those devices. So for me, at least on the medical device side, I think it's even more important that we understand what that patient impact is going to be with the innovations that we're launching into the market and making sure that they have a positive experience and that they can access those therapies."

"So what's interesting about blood therapy for sickle cell disease is it takes a lot of players to come to the table to successfully enable care. As we get excited about some of the transformative therapies on the horizon for sickle cell, we still need to manage those patients well with blood therapy like automated red cell exchange. And to get that, you need to bring together the patient, the prescriber, a service provider, and blood donor to bring that blood to the table because it's very, very specialized and matched to the donors that donate those cells."

#TerumoBCT #PatientVoiceAdvisors #MedicalDevices #MedTech #SickleCell

terumomedical.com

patientvoiceadvisors.com

Listen to the podcast here

Joy Duemke, Director of Marketing North America at Terumo, BCT, and Barry Liden, President and Managing Advisor of Patient Voice Advisors, highlight the importance of medical device companies engaging with patient associations to incorporate patient feedback into the design and development of their products. Patients can provide unique insights to engineers and researchers that may lead to better outcomes and patient experiences. The discussion focuses on the collaboration related to improving blood therapy delivery for sickle cell patients.

Barry explains, "So if medical device companies want to ensure that their technologies are going to get to the people that need them and that those folks want to be able to take potentially some risks of having the device implanted or used on them, they need to listen to the patient and understand what kind of outcomes are important to them and what they're willing to tolerate."

Joy elaborates, "With medical devices, it's very personal to the patient. It's not a bag that's hung or a pill that's taken, but it's typically something that might be, as Barry mentioned, implanted in their body or interacting very closely with their body. So the processes are very technical. There's often a high amount of training that has to happen with those operators, and it's often even a specialty department that might utilize those devices. So for me, at least on the medical device side, I think it's even more important that we understand what that patient impact is going to be with the innovations that we're launching into the market and making sure that they have a positive experience and that they can access those therapies."

"So what's interesting about blood therapy for sickle cell disease is it takes a lot of players to come to the table to successfully enable care. As we get excited about some of the transformative therapies on the horizon for sickle cell, we still need to manage those patients well with blood therapy like automated red cell exchange. And to get that, you need to bring together the patient, the prescriber, a service provider, and blood donor to bring that blood to the table because it's very, very specialized and matched to the donors that donate those cells."

#TerumoBCT #PatientVoiceAdvisors #MedicalDevices #MedTech #SickleCell

terumomedical.com

patientvoiceadvisors.com

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Dr. Simrit Parmar, Founder of Cellenkos, is developing T regulatory cell therapies from cord blood to treat aplastic anemia, myelofibrosis, and acute lymphoblastic leukemia. Their three-prong strategy focuses on resolving inflammation and alleviating the burden of transfusions. Treg cells from cord blood are naturally tolerant and do not risk rejection, meaning they can be administered to patients without the need for matching. The cells can be consistently manufactured in a scalable way and distributed globally.

Simrit explains, "Tregs, the T regulatory cells, are actually regulators of our immune system. So if you think about it every day as a human being, we face many challenges. We face many insults and injuries to our body, both externally and internally, but our body has a way to maintain that balance. The T regulatory cells are the cells that are the mastermind of making sure that any response by our body, for example, to get rid of an antigen or to get rid of an irritant does not overstay the welcome because our body utilizes the mechanism of inflammation to get rid of these injuries or these insults."

"So umbilical cord blood is, or the umbilical cord is, the connection between the baby, the fetus, and the mother. And if you think about it, the baby is 50% mismatched to the mother, and nature allows it. Nature allows a permissive environment where the baby can grow and eventually give birth, and the whole species is advanced. What happens is that nature has allowed the tolerance, and one of the mechanisms by which this tolerance, the maternal-fetal tolerance, is induced by the very T regulatory cells that populate the conduit between the mother and the baby, is the cord blood. So what we did is harness the power that nature has bestowed upon these cells to just do one job and one job only, which is to resolve inflammation. This is the fundamental reason we went after cord blood as a starting material because as a physician and drug developer, we wanted to make sure that the product, the cells we're giving into the patient, are doing its job."

#Biotechnology #DrugDevelopment #LifeSciences #AplasticAnemia #Myelofibrosis #GVHD #ALS #Tregs #CellTherapies #TargetedTherapies #AutoimmuneDiseases

cellenkosinc.com

Listen to the podcast here

Dr. Simrit Parmar, Founder of Cellenkos, is developing T regulatory cell therapies from cord blood to treat aplastic anemia, myelofibrosis, and acute lymphoblastic leukemia. Their three-prong strategy focuses on resolving inflammation and alleviating the burden of transfusions. Treg cells from cord blood are naturally tolerant and do not risk rejection, meaning they can be administered to patients without the need for matching. The cells can be consistently manufactured in a scalable way and distributed globally.

Simrit explains, "Tregs, the T regulatory cells, are actually regulators of our immune system. So if you think about it every day as a human being, we face many challenges. We face many insults and injuries to our body, both externally and internally, but our body has a way to maintain that balance. The T regulatory cells are the cells that are the mastermind of making sure that any response by our body, for example, to get rid of an antigen or to get rid of an irritant does not overstay the welcome because our body utilizes the mechanism of inflammation to get rid of these injuries or these insults."

"So umbilical cord blood is, or the umbilical cord is, the connection between the baby, the fetus, and the mother. And if you think about it, the baby is 50% mismatched to the mother, and nature allows it. Nature allows a permissive environment where the baby can grow and eventually give birth, and the whole species is advanced. What happens is that nature has allowed the tolerance, and one of the mechanisms by which this tolerance, the maternal-fetal tolerance, is induced by the very T regulatory cells that populate the conduit between the mother and the baby, is the cord blood. So what we did is harness the power that nature has bestowed upon these cells to just do one job and one job only, which is to resolve inflammation. This is the fundamental reason we went after cord blood as a starting material because as a physician and drug developer, we wanted to make sure that the product, the cells we're giving into the patient, are doing its job."

#Biotechnology #DrugDevelopment #LifeSciences #AplasticAnemia #Myelofibrosis #GVHD #ALS #Tregs #CellTherapies

#TargetedTherapies #AutoimmuneDiseases

cellenkosinc.com

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Jay Hartenbach, the President and COO of Diakonos Oncology, is developing a dendritic cell therapy to treat late-stage and aggressive cancers like glioblastoma and pancreatic cancer. This approach uses the patient's cells and tumor tissue to generate a personalized treatment. The resulting vaccination tricks the immune system to attack cancer cells by making them look like a virus which the immune system knows how to eliminate.

Jay explains, "Our vaccine works by using something called a dendritic cell from the patient. And so the dendritic cell is what we like to think of as the master of the immune system and somewhat the general that directs immune responses against threats, whether a bacterial threat or a viral, fungal threat, whatever it may be. The dendritic cell sits on top of the immune hierarchy. So, we take a patient's dendritic cell, and we also take a sample of that patient's tumor. We replicate effectively a viral infection with those tumor-derived antigens, the tumor-derived specific genetic material."

"By mimicking how a virus would infect those dendritic cells with the specific tumor antigens, we're able to trick the body into thinking that the cancer cells are virally infected cells. And from there, the body's good at eliminating those cells because we've all fought viruses before, and we're all standing and walking around and alive today. We just take over or hijack into that immune response except instead of killing infected cells, the body is now targeting those cancer cells."

#Diakonos #Vaccines #Cancer #Oncology #Glioblastoma #PancreaticCancer #Tumors #DendriticCells

diakonosoncology.com

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Jay Hartenbach, the President and COO of Diakonos Oncology, is developing a dendritic cell therapy to treat late-stage and aggressive cancers like glioblastoma and pancreatic cancer. This approach uses the patient's cells and tumor tissue to generate a personalized treatment. The resulting vaccination tricks the immune system to attack cancer cells by making them look like a virus which the immune system knows how to eliminate.

Jay explains, "Our vaccine works by using something called a dendritic cell from the patient. And so the dendritic cell is what we like to think of as the master of the immune system and somewhat the general that directs immune responses against threats, whether a bacterial threat or a viral, fungal threat, whatever it may be. The dendritic cell sits on top of the immune hierarchy. So, we take a patient's dendritic cell, and we also take a sample of that patient's tumor. We replicate effectively a viral infection with those tumor-derived antigens, the tumor-derived specific genetic material."

"By mimicking how a virus would infect those dendritic cells with the specific tumor antigens, we're able to trick the body into thinking that the cancer cells are virally infected cells. And from there, the body's good at eliminating those cells because we've all fought viruses before, and we're all standing and walking around and alive today. We just take over or hijack into that immune response except instead of killing infected cells, the body is now targeting those cancer cells."

#Diakonos #Vaccines #Cancer #Oncology #Glioblastoma #PancreaticCancer #Tumors #DendriticCells

diakonosoncology.com

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Dr. Pol Boudes, CMO of Rectify Pharmaceutical, highlights the importance of membrane proteins, specifically the role transporters play in how cells interact with their environment. Rectify is working on developing positive functional modulators (PFM) to address diseases related to dysfunctional transporters the first being primary sclerosing cholangitis, a rare liver disease with no current treatment options. The PFM can potentially restore the normal function of transporters involved in bile composition and secretion, which are key mechanisms in PSC.

Pol explains, "So you have different types of membrane proteins, but what we're working with are called transporters, so they're proteins that are based on the membrane but channel components out and in the cells. So basically, it's a way for the cell to interact with its environment, and you have multiple types of transporter proteins. We're specifically working or started to work with one group of family of proteins that are called the ABC transporters. They're a very important function, and they can either be disrupted because there is a mutation in the protein, so it's a genetic disease, or they are potentially disrupted because there is just a functional deficit of this transporter. The protein is normal, but it's not functioning properly. So it's a functional deficit of what we call the wildtype protein."

"So with the PFMs, what we're doing is to use small molecules that you can administer orally, and they bind to the transporter, and by doing so, they can modify the three-dimensional structure of the transporter and consequently the way this protein behaves within the cellular environment. So we correct the function of the transporter by changing the size, if you want, of the shape of the transporter. So we started to focus on this type of proteins and you have many potential diseases due to transporter deficit."

"We're focusing on one disease, liver disease. That's our lead PFM for a disease called primary sclerosing cholangitis, which is a disease of the liver. This very severe liver disease is also a disease that has no treatment available, which is a little bit frustrating because the progression of this disease leads to liver cirrhosis. And the only thing you can do at this stage is liver transplantation. As you might know, liver transplantation is problematic because it's a very complex process, it's also very expensive, and unfortunately, there is a shortage of transplants. So we're trying to address this problem."

#RectifyPharma #PrimarySclerosingCholangitis #PSC #RareDisease #LiverDiseases #BileProduction #Transporters

rectifypharma.com

Listen to the podcast here

Dr. Pol Boudes, CMO of Rectify Pharmaceutical, highlights the importance of membrane proteins, specifically the role transporters play in how cells interact with their environment. Rectify is working on developing positive functional modulators (PFM) to address diseases related to dysfunctional transporters the first being primary sclerosing cholangitis, a rare liver disease with no current treatment options. The PFM can potentially restore the normal function of transporters involved in bile composition and secretion, which are key mechanisms in PSC.

Pol explains, "So you have different types of membrane proteins, but what we're working with are called transporters, so they're proteins that are based on the membrane but channel components out and in the cells. So basically, it's a way for the cell to interact with its environment, and you have multiple types of transporter proteins. We're specifically working or started to work with one group of family of proteins that are called the ABC transporters. They're a very important function, and they can either be disrupted because there is a mutation in the protein, so it's a genetic disease, or they are potentially disrupted because there is just a functional deficit of this transporter. The protein is normal, but it's not functioning properly. So it's a functional deficit of what we call the wildtype protein."

"So with the PFMs, what we're doing is to use small molecules that you can administer orally, and they bind to the transporter, and by doing so, they can modify the three-dimensional structure of the transporter and consequently the way this protein behaves within the cellular environment. So we correct the function of the transporter by changing the size, if you want, of the shape of the transporter. So we started to focus on this type of proteins and you have many potential diseases due to transporter deficit."

"We're focusing on one disease, liver disease. That's our lead PFM for a disease called primary sclerosing cholangitis, which is a disease of the liver. This very severe liver disease is also a disease that has no treatment available, which is a little bit frustrating because the progression of this disease leads to liver cirrhosis. And the only thing you can do at this stage is liver transplantation. As you might know, liver transplantation is problematic because it's a very complex process, it's also very expensive, and unfortunately, there is a shortage of transplants. So we're trying to address this problem."

#RectifyPharma #PrimarySclerosingCholangitis #PSC #RareDisease #LiverDiseases #BileProduction #Transporters

rectifypharma.com

Download the transcript here

Neetu Rajpal, the CEO, and Co-Founder of Lilac Software, brings data and analytics tools to healthcare payers with a specific focus on addressing health disparities and improving benefits for Medicare Advantage members. The Medicare Advantage Star Rating program is a key incentive for health plans to be more innovative about data analysis to improve quality measures and receive financial incentives. Timely, reliable information in an accessible format and better data analytics improve decision-making, patient outcomes, and engagement with providers and patients.

Neetu explains, "Health plans tend to have lots of data. They have lots of valuable tools, but those tools have data that is locked inside those tools themselves. So if you're, for example, part of an actuarial team, you may actually be getting PDFs, you may be getting spreadsheets, you may be getting access directly to a CSV file. You may get all these things on a one-off basis, and you have to make sense of all this data. The burden of cleaning and making use of this data falls on you."

"This is exactly where lots of energy is lost, lots of labor costs are lost, and lots of efficiency is lost. So with Lilac, we're trying to make sure that all of this stuff of banal value is just available behind the scenes. This is exactly what tech is supposed to be doing for you. Help you operate at the top of your license. If you're an actuary, do actuarial things and let tech make sure that the data you need to do your work is available to you when you need it, and you can just rely on and trust it."

"Yes, unfortunately, there is still a lot of paper in the process. Some of the paper is regulatorily required. So if you're a payer, you're required to send any of your members a directory of providers that are available. If they ask for it in paper, you are required to send them ID cards that can be paper. We all know about the fax machines and all of those things on paper."

#LilacSoftware #DataAnalytics #HealthAI #AI #MedicareAdvantage #MedicareStarRating #HealthPlan #HealthcarePayers #HealthcareInsurance

lilacsoftware.com

Listen to the podcast here

Neetu Rajpal, the CEO, and Co-Founder of Lilac Software, brings data and analytics tools to healthcare payers with a specific focus on addressing health disparities and improving benefits for Medicare Advantage members. The Medicare Advantage Star Rating program is a key incentive for health plans to be more innovative about data analysis to improve quality measures and receive financial incentives. Timely, reliable information in an accessible format and better data analytics improve decision-making, patient outcomes, and engagement with providers and patients.

Neetu explains, "Health plans tend to have lots of data. They have lots of valuable tools, but those tools have data that is locked inside those tools themselves. So if you're, for example, part of an actuarial team, you may actually be getting PDFs, you may be getting spreadsheets, you may be getting access directly to a CSV file. You may get all these things on a one-off basis, and you have to make sense of all this data. The burden of cleaning and making use of this data falls on you."

"This is exactly where lots of energy is lost, lots of labor costs are lost, and lots of efficiency is lost. So with Lilac, we're trying to make sure that all of this stuff of banal value is just available behind the scenes. This is exactly what tech is supposed to be doing for you. Help you operate at the top of your license. If you're an actuary, do actuarial things and let tech make sure that the data you need to do your work is available to you when you need it, and you can just rely on and trust it."

"Yes, unfortunately, there is still a lot of paper in the process. Some of the paper is regulatorily required. So if you're a payer, you're required to send any of your members a directory of providers that are available. If they ask for it in paper, you are required to send them ID cards that can be paper. We all know about the fax machines and all of those things on paper."

#LilacSoftware #DataAnalytics #HealthAI #AI #MedicareAdvantage #MedicareStarRating #HealthPlan #HealthcarePayers #HealthcareInsurance

lilacsoftware.com

Download the transcript here