Communities Weekly Rewind: Hope and Innovation in Nephrology

By Sri Lekha Tummalapalli, MD, MBA, Nephrology Fellow, UCSF

Weekly Rewind 1024x512 (004)_FINAL_2.pngOn March 27, 2018, members of American Society of Nephrology (ASN) and the American Association of Kidney Patients (AAKP) joined together in Washington, D.C. for Kidney Health Advocacy Day.  Patients and physicians joined together to meet with Senate and House Congressional Offices in Capitol Hill to support innovation in kidney health.  Months of efforts by ASN staff culminated on April 26, 2018, when ASN signed a Memorandum of Understanding with the Department of Health and Human Services to establish the Kidney Innovation Accelerator, KidneyX.

We had a lively discussion on ASN Communities on the future of innovation in kidney disease.  Thank you to Drs. Mukta Baweja, Lauren Stern, Terrence O’Neil, Andrew Malone, Richard Glasscock, Alejandro Diez, and all others for contributing your insights. Read the full thread on ASN Communities, entitled “Urgency and Hope: Advocating for Essential Innovation on Capitol Hill.” This discussion covered the opportunities and methodology for innovation in the kidney space and how we can take advantage of it today.

Next Generation Renal Replacement Therapy (RRT) Technologies
Innovation to create an implantable self-contained device or a miniaturized wearable device were discussed as next generation RRT technologies. The Wearable Artificial Kidney at University of Washington and The Kidney Project at UCSF are two leading innovators in this area.  Dr. Alejandro Diez discussed that another ideal breakthrough would be antigen-free organs to allow for immunosuppression-free renal transplantation.

Dr. Andrew Malone discussed innovation in medical devices, drawing from learnings from the 1987 symposium entitled “New Medical Devices: Factors Influencing Invention, Development, and Use,” organized by National Academy of Engineering (NAE) and the Institute of Medicine (IOM).[1]  Insights from the symposium included:

  • Medical device innovation is usually incremental and based largely on engineering problem solving by individuals and small firms.
  • A close relationship between the manufacturer and the user is critical for innovation, and clinical contact between medical device companies and academic centers is strongly correlated with the degree of technological innovation.
  • Collaboration between small and large companies allows for innovation while providing access to resources, channels of distribution, experienced with regulatory issues, and greater market reach.


Novel Therapeutics
“We also do not truly have a strong therapeutic option of preventing progression of CKD other than controlling risk factors - an area which is truly starved for innovation.” - Dr. Mukta Baweja

Several novel therapeutic candidates were discussed, sparked by successes in HIV and HCV therapeutics.  Many had enthusiasm for CRISPR-Cas9 genome editing in the treatment of genetic kidney diseases. The technology is being studied in polycystic kidney disease and has the potential to make xenotransplantation possible.[2]

Single cell 'omics' to define single cell phenotypes in normal and diseased kidney was also discussed. There is potential to couple these new single cell insights with engineering and mechanics advances such as 3D printing and nanotechnologies.  Using mesenchymal stem cells for kidney regeneration is another promising innovation.

APOL1 was also discussed as a possible therapeutic target. Mechanisms of disease and novel therapeutic approaches from the Heymann et. al article “Therapeutics for APOL1 nephropathies: putting out the fire in the podocyte” were discussed.[3]

New Care Models to Decrease Incident ESRD
Multidisciplinary integrated care was proposed as one possible innovation to delay the progression of CKD. The paper "Person-Centered Integrated Care for Chronic Kidney Disease" by Valentijn et al. performed a systematic review and meta-analysis, which concluded that person-centered integrated care may have little effect on mortality or quality of life.[4]  We discussed the major limitation of the short follow-up time (mean of 12 months) of the studies included.

Dr. Terrence O’Neil pointed out, “In the US, putting a Nephrologist co-located with a Nurse Educator, a Pharmacist, a Social Worker, a Data Analyst and a Dietitian costs about $1.1 million USD annually (VHA FTE cost data, 2017)...Delaying only 15 patients from reaching ESRD at $80,000 USD per patient for one year more than pays for those personnel.” However, challenges including institutional inertia and misaligned incentives limit these new care models from taking hold.

Electronic Health Record (EHR) Solutions
Opportunities to generate CKD registries and risk stratify patients using the EHR were also discussed as much-needed health IT innovations.  Innovations in EHR interoperability would allow for seamless data sharing between outpatient nephrologists, transplant nephrologists, inpatient medical teams, and Medicare for reporting requirements.

We look forward to seeing the innovations fostered by KidneyX, the Kidney Innovation Accelerator. By mitigating regulatory and financial barriers, KidneyX will bridge the gap between research and commercialization to develop new treatments for patients with kidney disease.

 

Resources


[1] Ekelman, Karen B., ed. New medical devices: Invention, development, and use. National Academies, 1988.

[2] Miyagi, Ayano, Aiwu Lu, and Benjamin D. Humphreys. "Gene editing: powerful new tools for nephrology research and therapy." Journal of the American Society of Nephrology 27.10 (2016): 2940-2947.

[3] Heymann, Jurgen, et al. "Therapeutics for APOL1 nephropathies: putting out the fire in the podocyte." Nephrology Dialysis Transplantation 32.suppl_1 (2017): i65-i70.

[4] Valentijn, Pim P., et al. "Person-Centered Integrated Care for Chronic Kidney Disease A Systematic Review and Meta-Analysis of Randomized Controlled Trials." Clinical Journal of the American Society of Nephrology 13.3 (2018): 375-386.

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Sri Lekha Tummalapalli, MD, MBA, Nephrology Fellow, UCSF
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Weekly Rewind 1024x512 (004)_FINAL_2.pngOn March 27, 2018, members of American Society of Nephrology (ASN) and the American Association of Kidney Patients (AAKP) joined together in Washington, D.C. for Kidney Health Advocacy Day.  Patients and physicians joined together to meet with Senate and House Congressional Offices in Capitol Hill to support innovation in kidney health.  Months of efforts by ASN staff culminated on April 26, 2018, when ASN signed a Memorandum of Understanding with the Department of Health and Human Services to establish the Kidney Innovation Accelerator, KidneyX.

We had a lively discussion on ASN Communities on the future of innovation in kidney disease.  Thank you to Drs. Mukta Baweja, Lauren Stern, Terrence O’Neil, Andrew Malone, Richard Glasscock, Alejandro Diez, and all others for contributing your insights. Read the full thread on ASN Communities, entitled “Urgency and Hope: Advocating for Essential Innovation on Capitol Hill.” This discussion covered the opportunities and methodology for innovation in the kidney space and how we can take advantage of it today.

Next Generation Renal Replacement Therapy (RRT) Technologies
Innovation to create an implantable self-contained device or a miniaturized wearable device were discussed as next generation RRT technologies. The Wearable Artificial Kidney at University of Washington and The Kidney Project at UCSF are two leading innovators in this area.  Dr. Alejandro Diez discussed that another ideal breakthrough would be antigen-free organs to allow for immunosuppression-free renal transplantation.

Dr. Andrew Malone discussed innovation in medical devices, drawing from learnings from the 1987 symposium entitled “New Medical Devices: Factors Influencing Invention, Development, and Use,” organized by National Academy of Engineering (NAE) and the Institute of Medicine (IOM).[1]  Insights from the symposium included:

  • Medical device innovation is usually incremental and based largely on engineering problem solving by individuals and small firms.
  • A close relationship between the manufacturer and the user is critical for innovation, and clinical contact between medical device companies and academic centers is strongly correlated with the degree of technological innovation.
  • Collaboration between small and large companies allows for innovation while providing access to resources, channels of distribution, experienced with regulatory issues, and greater market reach.


Novel Therapeutics
“We also do not truly have a strong therapeutic option of preventing progression of CKD other than controlling risk factors - an area which is truly starved for innovation.” - Dr. Mukta Baweja

Several novel therapeutic candidates were discussed, sparked by successes in HIV and HCV therapeutics.  Many had enthusiasm for CRISPR-Cas9 genome editing in the treatment of genetic kidney diseases. The technology is being studied in polycystic kidney disease and has the potential to make xenotransplantation possible.[2]

Single cell 'omics' to define single cell phenotypes in normal and diseased kidney was also discussed. There is potential to couple these new single cell insights with engineering and mechanics advances such as 3D printing and nanotechnologies.  Using mesenchymal stem cells for kidney regeneration is another promising innovation.

APOL1 was also discussed as a possible therapeutic target. Mechanisms of disease and novel therapeutic approaches from the Heymann et. al article “Therapeutics for APOL1 nephropathies: putting out the fire in the podocyte” were discussed.[3]

New Care Models to Decrease Incident ESRD
Multidisciplinary integrated care was proposed as one possible innovation to delay the progression of CKD. The paper "Person-Centered Integrated Care for Chronic Kidney Disease" by Valentijn et al. performed a systematic review and meta-analysis, which concluded that person-centered integrated care may have little effect on mortality or quality of life.[4]  We discussed the major limitation of the short follow-up time (mean of 12 months) of the studies included.

Dr. Terrence O’Neil pointed out, “In the US, putting a Nephrologist co-located with a Nurse Educator, a Pharmacist, a Social Worker, a Data Analyst and a Dietitian costs about $1.1 million USD annually (VHA FTE cost data, 2017)...Delaying only 15 patients from reaching ESRD at $80,000 USD per patient for one year more than pays for those personnel.” However, challenges including institutional inertia and misaligned incentives limit these new care models from taking hold.

Electronic Health Record (EHR) Solutions
Opportunities to generate CKD registries and risk stratify patients using the EHR were also discussed as much-needed health IT innovations.  Innovations in EHR interoperability would allow for seamless data sharing between outpatient nephrologists, transplant nephrologists, inpatient medical teams, and Medicare for reporting requirements.

We look forward to seeing the innovations fostered by KidneyX, the Kidney Innovation Accelerator. By mitigating regulatory and financial barriers, KidneyX will bridge the gap between research and commercialization to develop new treatments for patients with kidney disease.

 

Resources


[1] Ekelman, Karen B., ed. New medical devices: Invention, development, and use. National Academies, 1988.

[2] Miyagi, Ayano, Aiwu Lu, and Benjamin D. Humphreys. "Gene editing: powerful new tools for nephrology research and therapy." Journal of the American Society of Nephrology 27.10 (2016): 2940-2947.

[3] Heymann, Jurgen, et al. "Therapeutics for APOL1 nephropathies: putting out the fire in the podocyte." Nephrology Dialysis Transplantation 32.suppl_1 (2017): i65-i70.

[4] Valentijn, Pim P., et al. "Person-Centered Integrated Care for Chronic Kidney Disease A Systematic Review and Meta-Analysis of Randomized Controlled Trials." Clinical Journal of the American Society of Nephrology 13.3 (2018): 375-386.

Date:
Friday, April 27, 2018