Stanford Medicine Researchers Receive $18 Million to Advance Kidney Transplant and Gene Editing Treatments

Recent developments in medical research have been nothing short of revolutionary. The ability to use stem cells for transplantation and gene-editing techniques for genetic disorders has opened up a whole new world of possibilities for treating diseases. Stanford Medicine is at the forefront of these advancements, as they have just received $18 million in funding from the California Institute for Regenerative Medicine (CIRM) for two projects aimed at advancing cutting-edge treatments for children.

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The First Project

The first project, led by Alice Bertaina, MD, PhD, is a clinical trial for a treatment that allows for kidney transplantation without the need for long-term immunosuppression. The treatment involves a child receiving a stem cell transplant followed by a kidney transplant from the same donor, usually a parent. The stem cells provide the recipient with the donor’s immune system, allowing the kidney to be accepted without the need for long-term immunosuppression.

This treatment relies on a method Bertaina pioneered to process donor stem cells before infusing them in the recipient, known as alpha-beta T cell depletion. The method greatly reduces the risk of complications such as graft-versus-host disease and enables stem cell transplants between donors and recipients who are matched on only half of their genetic markers, such as parents and children.

According to an early report by Bertaina’s team, which focused on three children with a rare condition called Schimke immuno-osseous dysplasia, providing stem cells and a matching kidney can free recipients from the need to take immune-suppressing medications, which have significant long-term risks. Schimke immune-osseous dysplasia is a genetic disease that causes bone marrow failure, meaning patients need a stem cell transplant, as well as kidney failure.

The CIRM funding will allow Bertaina and her team to expand the primary diseases treated by this approach to include cystinosis and systemic lupus erythematosus. Cystinosis is a genetic disease that interferes with metabolism of cystine, causing long-term kidney damage. Lupus is an autoimmune disease that causes kidney failure in some patients. The researchers will also study the technique in patients who have rejected a previous kidney transplant due to focal segmental glomerulosclerosis, a form of scarring in the kidneys that is a common cause of transplant failure.

If this trial is successful, the number of patients who could benefit from this innovative approach will grow tremendously. The CIRM funding will also support extensive studies of the immunological mechanisms that enable the new technique to work, possibly allowing it to improve transplantation of other organs such as the liver and intestine.

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The Second Project

The second project, led by Natalia Gomez-Ospina, MD, PhD, is a study of a gene-editing technique aimed at a rare, severe genetic disease known as mucopolysaccharidosis type 1, or Hurler syndrome. Children with the disorder lack an enzyme that allows their cells to break down large, complex sugar molecules known as mucopolysaccharides or glycosaminoglycans. These sugars build up inside their cells, causing organ damage. Patients with the disorder have a life expectancy of around 10 years.

The Stanford Medicine team will genetically edit patients’ own blood-forming stem cells to restore the missing enzyme. The goal of the CIRM-funded trial is to show that the team can manufacture the cells and complete the safety studies needed to gain Food and Drug Administration authorization for a clinical trial.

“The funding will pave the way for trials in people to realize a more effective therapy for this devastating disease,” Gomez-Ospina said.

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Reference

Stanford Medicine teams awarded $18 million to improve kidney transplant and gene-editing techniques

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About the Author

Rich Foreman brings over 30 years of technology leadership to his role of CEO and Co-Founder of KidneySoft.  As founding CTO, Rich led the team that developed the CordicoShield / CordicoFire Wellness App. Cordico was honored with the Sacramento Innovation Award in 2021. After achieving a 7 digit ARR, Cordico was acquired by Lexipol in 2020. Rich has a BS in Industrial Engineering from the University of Washington, an MPA from Troy State University and was an officer in the U.S. Navy. Rich co-authored his book, "Tap into the Mobile Economy." Rich's blog was listed in Top 20 Marketing Mobile Blogs of 2014. He has been featured on KCRA3, NEWS10, 1170 Tech AM PowerDrive, Business Radio Money 105.5, SiliconIndia, the Sacramento Business Journal, and the Sacramento Bee. Rich is also the Founding Director of the Sacramento Chapter of Startup Grind and served a term as Utility Commissioner for the City of Folsom. Rich is a regular contributor to TechWire.net and StartupSac. Rich was the Co-founder of Apptology which was named Small Business of the Year in 2014 by the Sacramento Asian Pacific Chamber. He was also the Founding Chief Technology Officer at Cordico. Cordico was acquired by Lexipol in 2020.  Rich also served 4 years as a Naval Officer in the Civil Engineer Corps.

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Patient Education Disclaimer

This material is for informational purposes only. It does not replace the advice or counsel of a doctor or health care professional. KidneyLuv makes every effort to provide information that is accurate and timely, but makes no guarantee in this regard. You should consult with, and rely only on the advice of, your physician or health care professional.

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