Rare Disease Gets Big Boost from California’s Stem Cell Agency

UC Irvine’s Dr. Leslie Thompson and patient advocate Frances Saldana after the CIRM Board vote to approve funding for Huntington’s disease

If you were looking for a poster child for an unmet medical need Huntington’s disease (HD) would be high on the list. It’s a devastating disease that attacks the brain, steadily destroying the ability to control body movement and speech. It impairs thinking and often leads to dementia. It’s always fatal and there are no treatments that can stop or reverse the course of the disease. Today the Board of the California Institute for Regenerative Medicine (CIRM) voted to support a project that shows promise in changing that.

The Board voted to approve $6 million to enable Dr. Leslie Thompson and her team at the University of California, Irvine to do the late stage testing needed to apply to the US Food and Drug Administration for permission to start a clinical trial in people. The therapy involves transplanting stem cells that have been turned into neural stem cells which secrete a molecule called brain-derived neurotrophic factor (BDNF), which has been shown to promote the growth and improve the function of brain cells. The goal is to slow down the progression of this debilitating disease.

“Huntington’s disease affects around 30,000 people in the US and children born to parents with HD have a 50/50 chance of getting the disease themselves,” says Dr. Maria T. Millan, the President and CEO of CIRM. “We have supported Dr. Thompson’s work for a number of years, reflecting our commitment to helping the best science advance, and are hopeful today’s vote will take it a crucial step closer to a clinical trial.”

Another project supported by CIRM at an earlier stage of research was also given funding for a clinical trial.

The Board approved almost $12 million to support a clinical trial to help people undergoing a kidney transplant. Right now, there are around 100,000 people in the US waiting to get a kidney transplant. Even those fortunate enough to get one face a lifetime on immunosuppressive drugs to stop the body rejecting the new organ, drugs that increase the risk for infection, heart disease and diabetes.  

Dr. Everett Meyer, and his team at Stanford University, will use a combination of healthy donor stem cells and the patient’s own regulatory T cells (Tregs), to train the patient’s immune system to accept the transplanted kidney and eliminate the need for immunosuppressive drugs.

The initial group targeted in this clinical trial are people with what are called HLA-mismatched kidneys. This is where the donor and recipient do not share the same human leukocyte antigens (HLAs), proteins located on the surface of immune cells and other cells in the body. Around 50 percent of patients with HLA-mismatched transplants experience rejection of the organ.

In his application Dr. Meyer said they have a simple goal: “The goal is “one kidney for life” off drugs with safety for all patients. The overall health status of patients off immunosuppressive drugs will improve due to reduction in side effects associated with these drugs, and due to reduced graft loss afforded by tolerance induction that will prevent chronic rejection.”

71 for Proposition 71

Proposition 71 is the state ballot initiative that created California’s Stem Cell Agency. This month, the Agency reached another milestone when the 71st clinical trial was initiated in the CIRM Alpha Stem Cell Clinics (ASCC) Network. The ASCC Network deploys specialized teams of doctors, nurses and laboratory technicians to conduct stem cell clinical trials at leading California Medical Centers.

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These teams work with academic and industry partners to support patient-centered for over 40 distinct diseases including:

  • Amyotrophic Lateral Sclerosis (ALS)
  • Brain Injury & Stroke
  • Cancer at Multiple Sites
  • Diabetes Type 1
  • Eye Disease / Blindness Heart Failure
  • HIV / AIDS
  • Kidney Failure
  • Severe Combined Immunodeficiency (SCID)
  • Sickle Cell Anemia
  • Spinal Cord Injury

These clinical trials have treated over 400 patients and counting. The Alpha Stem Cell Clinics are part of CIRM’s Strategic Infrastructure. The Strategic Infrastructure program which was developed to support the growth of stem cell / regenerative medicine in California. A comprehensive update of CIRM’s Infrastructure Program was provided to our Board, the ICOC.

CIRM’s infrastructure catalyzes stem cell / regenerative medicine by providing resources to all qualified researchers and organizations requiring specialized expertise. For example, the Alpha Clinics Network is supporting clinical trials from around the world.

Many of these trials are sponsored by commercial companies that have no CIRM funding. To date, the ASCC Network has over $27 million in contracts with outside sponsors. These contracts serve to leverage CIRMs investment and provide the Network’s medical centers with a diverse portfolio of clinical trials to address patients’’ unmet medical needs.

Alpha Clinics – Key Performance Metrics

  • 70+ Clinical Trials
  • 400+ Patients Treated
  • 40+ Disease Indications
  • Over $27 million in contracts with commercial sponsors

The CIRM Alpha Stem Cell Clinics and broader Infrastructure Programs are supporting stem cell research and regenerative medicine at every level, from laboratory research to product manufacturing to delivery to patients. This infrastructure has emerged to make California the world leader in regenerative medicine. It all started because California’s residents supported a ballot measure and today we have 71 clinical trials for 71.

 

 

How CIRM support helped a promising approach to type 1 diabetes get vital financial backing

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The “Valley of Death” sounds like a scary place from “Lord of the Rings” or “Game of Thrones” that our heroes have to navigate to reach safety. The reality is not that different. It’s the space that young companies have to navigate from having a good idea to getting financial backing, so they can move their projects towards the clinic. At the other side of the Valley are deep-pocket investors, waiting to see what makes it through before deciding if they want to support them.

It’s a Catch 22 situation. Without financing companies can’t make it through the Valley; but they need to get through before the folks with money will considering investing. As a result many companies languish or even fail to make it through the Valley of Death. Without that financial support promising therapies are lost before they even get a chance to show their potential.

CIRM was created, in part, to help those great ideas get through the Valley. That’s why it is so gratifying to hear the news today from ViaCyte – that is developing a promising approach to treating type 1 diabetes – that they have secured $80 million in additional financing.

The money comes from Bain Capital Life Sciences, TPG and RA Capital Management and several other investors. It’s important because it is a kind of vote of confidence in ViaCyte, suggesting these deep-pocket investors believe the company’s approach has real potential.

In a news release Adam Koppel, a Managing Director at Bain, said:

“ViaCyte is the clear leader in beta cell replacement, and we are excited about the lasting impact that it’s stem cell-derived therapies can potentially have on improving treatment and quality of life for people living with insulin-requiring diabetes. We look forward to partnering with ViaCyte’s management team to accelerate the development of ViaCyte’s transformative cell therapies to help patients.”

CIRM has been a big supporter of ViaCyte for several years, investing more than $70 million to help them develop a cell therapy that can be implanted under the skin that is capable of delivering insulin to people with type 1 diabetes when needed. The fact that these investors are now stepping up to help it progress suggests we are not alone in thinking this project has tremendous promise.

But ViaCyte is far from the only company that has benefitted from CIRM’s early and consistent support. This year alone CIRM-funded companies have raised more than $1.0 billion in funding from outside investors; a clear sign of validation not just for the companies and their therapies, but also for CIRM and its judgement.

This includes:

  • Humacyte raising $225 million for its program to help people battling kidney failure
  • Forty Seven Inc. raising $113 million from an Initial Public Offering for its programs targeting different forms of cancer
  • Nohla Therapeutics raising $56 million for its program treating acute myeloid leukemia

We have shown there is a path through the Valley of Death. We are hoping to lead many more companies through that in the coming years, so they can bring their therapies to people who really need them, the patients.

 

 

 

Stories that caught our eye: Is a Texas law opening up access to stem cell treatments working? Another CIRM-funded company gets good news from the FDA.

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Texas Capitol. (Shutterstock)

In 2017 Texas passed a sweeping new law, HB 810, which allowed medical clinics to provide “investigational stem cell treatments to patients with certain severe chronic diseases or terminal illnesses.” Those in favor of the law argued that patients battling life-threatening or life-changing diseases should have the right to try stem cell therapies that were involved in a clinical trial.

Now a new study, published in the journal Stem Cells and Development, looks at the impact of the law. The report says that despite some recent amendments t there are still some concerns about the law including:

  • It allows treatment only if the patient has a “severe, chronic” illness but doesn’t define what that means
  • It doesn’t have clearly defined procedures on tracking and reporting procedures so it’s hard to know how many patients might be treated and what the outcomes are
  • There is no Food and Drug Administration (FDA) oversight of the patients being treated
  • Because the treatments are unproven there are fears this will “open up the state to unsavory and predatory practices by individuals preying on vulnerable patients”

The researchers conclude:

“While HB 810 opens up access to patients, it also increases significant risks for their safety and financial cost for something that might have no positive impact on their disease. Truly understanding the impact of stem cell based interventions (SCBI) requires scientific rigor, and accurate outcome data reporting must be pursued to ensure the safety and efficacy behind such procedures. This information must be readily available so that patients can make informed decisions before electing to pursue such treatments. The creation of the SCBI registry could allow for some level of scientific rigor, provide a centralized data source, and offer the potential for better informed patient choices, and might be the best option for the state to help protect patients.”

Another CIRM-funded company gets RMAT designation

Poseida

When Congress approved the 21st Century Cures Act a few years ago one of the new programs it created was the Regenerative Medicine Advanced Therapy (RMAT) designation. This was given to therapies that are designed to treat a serious or life-threatening condition, where early clinical stage trials show the approach is safe and appears to be effective.

Getting an RMAT designation is a big deal. It means the company or researchers are able to apply for an expedited review by the FDA and could get approval for wider use.

This week Poseida Therapeutics was granted RMAT designation by the Food and drug Administration (FDA) for P-BCMA-101, its CAR-T therapy for relapsed/refractory multiple myeloma. This is currently in a Phase 1 clinical trial that CIRM is funding

In this trial Poseida’s technology takes an immunotherapy approach that uses the patient’s own engineered immune system T cells to seek and destroy cancerous myeloma cells.

In a news release Eric Ostertag, Poseida’s CEO, welcomed the news:

“Initial Phase 1 data presented at the CAR-TCR Summit earlier this year included encouraging response rates and safety data, including meaningful responses in a heavily pretreated population. We expect to have an additional data update by the end of the year and look forward to working closely with the FDA to expedite development of P-BCMA-101.”

This means that five CIRM-funded companies have now been granted RMAT designations:

CIRM-funded medical research and development company does $150M deal to improve care for dialysis patients

Fresenius & Humacyte

Nearly half a million Americans with kidney disease are on dialysis, so it’s not surprising the CIRM Board had no hesitation, back in July 2016, in funding a program to make it easier and safer to get that life-saving therapy.

That’s why it’s gratifying to now hear that Humacyte, the company behind this new dialysis device, has just signed a $150 million deal with Fresenius Medical Care, to make their product more widely available.

The CIRM Board gave Humacyte $10 million for a Phase 3 clinical trial to test a bioengineered vein needed by people undergoing hemodialysis, the most common form of dialysis.

Humacyte HAV

The vein – called a human acellular vessel or HAV – is implanted in the arm and used to carry the patient’s blood to and from an artificial kidney that removes waste from the blood. Current synthetic versions of this device have many problems, including clotting, infections and rejection. In tests, Humacyte’s HAV has fewer complications. In addition, over time the patient’s own stem cells start to populate the bioengineered vein, in effect making it part of the patient’s own body.

Fresenius Medical Care is investing $150 million in Humacyte, with a plan to use the device in its dialysis clinics worldwide. As an indication of how highly they value the device, the deal grants Fresenius a 19% ownership stake in the company.

In an interview with FierceBiotech, Jeff Lawson, Humacyte’s Chief Medical Officer, said if all goes well the company plans to file for Food and Drug Administration (FDA) approval in 2019 and hopes it will be widely available in 2020.

In addition to being used for kidney disease the device is also being tested for peripheral artery disease, vascular trauma and other cardiovascular indications. Lawson says testing the device first in kidney disease will provide a solid proving ground for it.

“It’s a very safe place to develop new vascular technologies under clinical study. From a regulatory safety standpoint, this is the first area we could enter safely and work with the FDA to get approval for a complete new technology.”

This is another example of what we call CIRM’s “value proposition”; the fact that we don’t just provide funding, we also provide support on many other levels and that has a whole range of benefits. When our Grants Working Group – the independent panel of experts who review our scientific applications – and the CIRM Board approves a project it’s like giving it the CIRM Good Housekeeping Seal of Approval. That doesn’t just help that particular project, it can help attract further investment in the company behind it, enabling it to expand operations and create jobs and ultimately, we hope, help advance the field as a whole.

Those benefits are substantial. To date we have been able to use our funding to leverage around $2 billion in additional dollars in terms of outside companies investing in companies like Humacyte, or researchers using data from research we funded to get additional funding from agencies like the National Institutes of Health.

So, when a company like Humacyte is the object of such a lucrative agreement it’s not just a compliment to the quality of the work they do, it’s also a reflection of our ability to pick great projects.

Timing is a critical factor in kidney development

Through countless studies, it’s clear that genes and environmental factors are important for determining cellular identity. Now, a research team at the University of Southern California  (USC) have found that timing is another critical factor in determining cell fate during organ development.

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Developing human nephron, the filtering unit of the kidney. Image by Nils O. Lindstrom and Tracy Tran/McMahon Lab USC Stem Cell

In findings published in Development Cell, Dr. Andy McMahon’s group shows that development of the nephron, the filtering structure of the kidney, is acutely dependent on when cells arrive in that developmental region. Cells that arrive in the developing nephron early become part of the tubule, which is responsible for reabsorption of water and salt, whereas cells that arrive late become part of the glomerulus, the structure that is responsible for filtering blood.

The scientists verified that timing influenced cell identity with a combination of microscopy, which allowed them to follow particular cell types as they developed, and single-cell RNA sequencing, which allowed them to determine how gene expression changes in a population of cells.

In a press release, Dr. McMahon details the importance of these findings:

“By studying normal human nephron development, we’re gaining important information about how to replicate this intricate process in the laboratory. The hope is that laboratory-grown nephrons can be used to further study the process of development, screen potential therapies to treat disease, and eventually provide the building blocks to assemble functional kidneys for transplantation into patients.”

Understanding kidney development is crucial because approximately 30 million people suffer from chronic kidney disease and it is the ninth leading cause of death in the United States alone. Insights into the basics of kidney biology can provide important advances to develop novel therapeutics for this devastating condition.

Stem cell study holds out promise for kidney disease

Kidney failure

Image via youtube.com

Kidney failure is the Rodney Dangerfield of diseases, it really doesn’t get the respect it deserves. An estimated 660,000 Americans suffer from kidney failure and around 47,000 people die from it every year. That’s more than die from breast or prostate cancer. But now a new study has identified a promising stem cell candidate that could help in finding a way to help repair damaged kidneys.

Kidneys are the body’s waste disposal system, filtering our blood and cleaning out all the waste products. Our kidneys have a limited ability to help repair themselves but if someone suffers from chronic kidney disease then their kidneys are slowly overwhelmed and that leads to end stage renal disease. At that point the patient’s options are limited to dialysis or an organ transplant.

Survivors hold out hope

Italian researchers had identified some cells in the kidneys that showed a regenerative ability. These cells, which were characterized by the expression of a molecule called CD133, were able to survive injury and create different types of kidney cells.

Researchers at the University of Torino in Italy decided to take these findings further and explore precisely how CD133 worked and if they could take advantage of that and use it to help repair damaged kidneys.

In their findings, published in the journal Stem Cells Translational Medicine, the researchers began by working with a chemotherapy drug called cisplatin, which is used against a broad range of cancers but is also known to cause damage to kidneys in around one third of all patients. The team found that CD133 was an important factor in helping those damaged kidneys recover. They also found that CD133 prevents aging of kidney progenitor cells, the kind of cell needed to help create new cells to repair the kidneys in future.

Hope for further research

The finding opens up a number of possible lines of research, including exploring whether infusions of CD133 could help patients whose kidneys are no longer able to produce enough of the molecule to help repair damage.

In an interview in DD News, Dr. Anthony Atala, Director of the Wake Forest Institute for Regenerative Medicine – praised the research:

“This is an interesting and novel finding. Because the work identifies mechanisms potentially involved in the repair of tissue after injury, it suggests the possibility of new therapies for tissue repair and regeneration.”

CIRM is funding several projects targeting kidney disease including four clinical trials for kidney failure. These are all late-stage kidney failure problems so if the CD133 research lives up to its promise it might be able to help people at an earlier stage of disease.

It’s World Kidney Day: Highlighting CIRM’s Investments in Treating Kidney Failure

WKD-Logo-HiToday is World Kidney Day. Hundreds of events across the globe are taking place “to raise awareness of the importance of our kidneys to our overall health and to reduce the frequency and impact of kidney disease and its associated health problems worldwide.” (Side note: in recognition that today is also International Women’s Day, World Kidney Day’s theme this year is “Kidney’s & Women: Include, Value, Empower.)

To honor this day, we’re highlighting how CIRM is playing its part in that mission. The infographic below provides big picture summaries of the four CIRM-funded clinical trials that are currently testing stem cell-based therapies for kidney failure, a condition that affects well over 600,000 Americans.

When a person’s kidneys fail, their body can no longer filter out waste products and extra fluid from the blood which leads to life-threatening complications. About 30% of those affected in the U.S. have organ transplants. Due to the limited availability of donor organs, the other 70% need dialysis, a blood filtration therapy, that requires several trips a week to a special clinic.

Both treatment options have serious limitations. Organ recipients have to take drugs that prevent organ rejections for the rest of their lives. Over time, these drugs are toxic and can increase a patient’s risk of infection, heart disease, cancer and diabetes. In the case of dialysis treatment, the current procedure uses a plastic tube called a shunt to connect to a patient’s vein. These shunts are far from ideal and can lead to infection, blood clots and can be rejected by the patient’s immune system. These complications probably play a role in the average life expectancy of 5-10 years for dialysis patients.

Four CIRM-funded clinical trials aim to circumvent these drawbacks. Humacyte has received over $24 million from the Agency to support two clinical trials that are testing an alternative to the plastic shunt used in dialysis treatment. The company has developed a bioengineered vessel that is implanted in the patient’s arm and over time is populated with the patient’s own stem cells which develop into a natural blood vessel. The trials will determine if the bioengineered vessel is superior to the shunt in remaining open for longer periods of time and with lower incidence of interventions due to blood clots and infections.

The other two CIRM-funded trials, one headed by Stanford University and the other by Medeor Therapeutics, aims to eliminate the need for long-life, anti-rejection medicine after kidney transplant. Both trials use a similar strategy: blood stem cells and immune cells from the organ donor are infused into the patient receiving the organ. If all goes as planned, those donor cells will engraft into and mix with the recipient’s immune system, making organ rejection less likely and ending the need for immune-system suppressing drugs.

For more details visit our Clinical Trial Dashboard.

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Stem Cell Roundup: Lab-grown meat, stem cell vaccines for cancer and a free kidney atlas for all

Here are the stem cell stories that caught our eye this week.

Cool Stem Cell Photo: Kidneys in the spotlight

At an early stage, a nephron forming in the human kidney generates an S-shaped structure. Green cells will generate the kidneys’ filtering device, and blue and red cells are responsible for distinct nephron activities. (Image/Stacy Moroz and Tracy Tran, Andrew McMahon Lab, USC Stem Cell)

I had to take a second look at this picture when I first saw it. I honestly thought it was someone’s scientific interpretation of Vincent van Gogh’s Starry Night. What this picture actually represents is a nephron. Your kidney has over a million nephrons packed inside it. These tiny structures filter our blood and remove waste products by producing urine.

Scientists at USC Stem Cell are studying kidney development in animals and humans in hopes of gaining new insights that could lead to improved stem cell-based technologies that more accurately model human kidneys (by coincidence, we blogged about another human kidney study on Tuesday). Yesterday, these scientists published a series of articles in the Journal of American Society of Nephrology that outlines a new, open-source kidney atlas they created. The atlas contains a catalog of high resolution images of different structures representing the developing human kidney.

CIRM-funded researcher Andrew McMahon summed it up nicely in a USC news release:

“Our research bridges a critical gap between animal models and human applications. The data we collected and analyzed creates a knowledge-base that will accelerate stem cell-based technologies to produce mini-kidneys that accurately represent human kidneys for biomedical screening and replacement therapies.”

And here’s a cool video of a developing kidney kindly provided by the authors of this study.

Video Caption: Kidney development begins with a population of “progenitor cells” (green), which are similar to stem cells. Some progenitor cells (red) stream out and aggregate into a ball, the renal vesicle (gold). As each renal vesicle grows, it radically morphs into a series of shapes — can you spot the two S-shaped bodies (green-orange-pink structures)? – and finally forms a nephron. Each human kidney contains one million mature nephrons, which form an expansive tubular network (white) that filters the blood, ensuring a constant environment for all of our body’s functions. (Video courtesy of Nils Lindstorm, Andy McMahon, Seth Ruffins and the Microscopy Core Facility at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at the Keck School of Medicine of USC)


Lab-grown hamburgers coming to a McDonald’s near you…

“Lab-grown meat is coming, whether you like it or not” sure makes a splashy headline! This week, Wired magazine featured two Bay Area startup companies, Just For All and Finless Foods, dedicated to making meat-in-a-dish in hopes of one day reducing our dependence on livestock. The methods behind their products aren’t exactly known. Just For All is engineering “clean meat” from cells. On the menu currently are cultured chorizo, nuggets, and foie gras. I bet you already guessed what Finless Foods specialty is. The company is isolating stem-like muscle progenitor cells from fish meat in hopes of identifying a cell that will robustly create the cell types found in fish meat.

Just’s tacos made with lab-grown chorizo. (Wired)

I find the Wired article particularly interesting because of the questions and issues Wired author Matt Simon raises. Are clean meat companies really more environmentally sustainable than raising livestock? Currently, there isn’t enough data to prove this is the case, he argues. And what about the feasibility of convincing populations that depend on raising livestock for a living to go “clean”? And what about flavor and texture? Will people be willing to eat a hamburger that doesn’t taste and ooze in just the right way?

As clean meat technologies continue to advance and become more affordable, I’ll be interested to see what impact they will have on our eating habits in the future.


Induced pluripotent stem cells could be the next cancer vaccine

Our last story is about a new Cell Stem Cell study that suggests induced pluripotent stem cells (iPSCs) could be developed into a vaccine against cancer. CIRM-funded scientist Joseph Wu and his team at Stanford University School of Medicine found that injecting iPSCs into mice that were transplanted with breast cancer cells reduced the formation of tumors.

The team dug deeper and discovered that iPSCs shared similarities with cancer cells with respect to the panel of genes they express and the types of proteins they carry on their cell surface. This wasn’t surprising to them as both cells represent an immature development stage. Because of these similarities, injecting iPSCs primed the mouse’s immune system to recognize and reject similar cells like cancer cells.

The team will next test their approach on human cancer cells in the lab. Joseph Wu commented on the potential future of iPSC-based vaccines for cancer in a Stanford news release:

“Although much research remains to be done, the concept itself is pretty simple. We would take your blood, make iPS cells and then inject the cells to prevent future cancers. I’m very excited about the future possibilities.”

 

In a stem cell first, functioning human kidney structures grown in living animals

One of the ultimate quests in the stem cell field – growing organs to repair diseased or damaged ones – took a significant step forward this week. In a first, researchers at the University of Manchester, in the U.K., showed that human embryonic stem cell-derived kidney tissue forms into functional kidney structures, capable of filtering blood and producing urine, when implanted under the skin of mice.

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Cross-section of human stem cell-derived kidney tissue grown in mouse. When injected in blood, dextran (green) was taken up by the kidney structure, proving it’s functional. (Credit University of Manchester/ Stem Cell Reports)

When a person has end-stage kidney disease, their body can no longer filter out waste products and extra fluid from the blood which leads to serious health complications, even death. Blood filtration therapy, called dialysis, can substitute for a kidney but the average life expectancy is only about 10 years for patients receiving dialysis. Kidney transplants are another answer for treating kidney disease, but organ availability is in limited supply. About 2.2 million people die worldwide from a lack of access to these treatment options. So other therapeutic approaches to help end-stage kidney disease sufferers are sorely needed.

The current study, published in Stem Cell Reports, used human embryonic stem cells to grow kidney tissue in the lab. While the lab-grown tissues showed hallmarks of kidney structures, they were unable to fully develop into mature kidney structures in a culture dish. So the scientists tried implanting the human kidney tissue under the skin of mice and left it there for 12 weeks. The team showed that kidney structures, called glomeruli, which play a key role in filtering the blood, formed over that time and had become vascularized, or connected with the animal’s blood supply. The team further showed those structures were functional by injecting a fluorescently tagged substance called dextran. Tracing the fate of the dextran in the blood showed that it had been filtered and taken up by tubular structures in the kidney tissue which indicates urine production had begun.

Professor Sue Kimber, one of the leaders of the study, summed up the significance and current limitations of these results in a press release:

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Sue Kimber

“We have proved beyond any doubt these structures function as kidney cells by filtering blood and producing urine – though we can’t yet say what percentage of function exists. What is particularly exciting is that the structures are made of human cells which developed an excellent capillary blood supply, becoming linked to the vasculature of the mouse.

Though this structure was formed from several hundred glomeruli, and humans have about a million in their kidneys – this is clearly a major advance. It constitutes a proof of principle- but much work is yet to be done.”

To be sure, curing a person suffering from end-stage kidney disease with a stem cell-grown kidney is some ways off. But, on the nearer horizon, this advance will provide a means to study the human kidney in a living animal, a powerful tool for uncovering insights into kidney disease and new therapeutic approaches.