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.

A Noble pursuit; finding the best science to help the most people

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Mark Noble. Photo by Todd Dubnicoff

Mark Noble, Ph.D., is a pioneer in stem cell research and the Director of the University of Rochester Stem Cell and Regenerative Medicine Institute in New York. He is also a member of CIRM’s Grants Working Group (GWG), the panel of independent scientific experts we use to review research applications for funding and decide which are the most promising.

Mark has been a part of the GWG since 2011. When asked how he came to join the GWG he joked: “I saw an ad on Craigslist and thought it sounded fun.”  But he is not joking when he says it is a labor of love.

“My view is that CIRM is one of the greatest experiments in how to develop a new branch of science and medicine. If you look at ventures, like the establishment of the National Institutes of Health, what you see is that when there is a concentrated effort to achieve an enormous goal, amazing things can happen. And if your goal is to create a new field of medicine you have to take a truly expansive view.”

Mark has been on many other review panels but says they don’t compare to CIRM’s.

“These are the most exciting review panels in which I take part. I don’t know of any comparable panels that bring together experts working across such a wide range of disciplines and diseases.   It’s particularly interesting to be involved in reviews at this stage because we get to look at the fruits of CIRM’s long investment, and at projects that are now in, or well on the way towards, clinical trials.

It’s a wonderful scientific education because you come to these meetings and someone is submitting an application on diabetes and someone else has submitted an application on repairing the damage to the heart or spinal cord injury or they have a device that will allow you to transplant cells better. There are people in the room that are able to talk knowledgeably about each of these areas and understand how the proposed project might work in terms of actual financial development, and how it might work in the corporate sphere and how it fits in to unmet medical needs.  I don’t know of any comparable review panels like this that have such a broad remit and bring together such a breadth of expertise. Every review panel you come to you are getting a scientific education on all these different areas, which is great.”

Another aspect of CIRM’s work that Mark admires is its ability to look past the financial aspects of research, to focus on the bigger goal:

“I like that CIRM recognizes the larger problem, that a therapy that is curative but costs a million dollars a patient is not going to be implemented worldwide. Well, CIRM is not here to make money. CIRM is here to find cures for unmet medical needs, which means that if someone comes in with a great application on a drug that is going to cure some awful disease and it’s not going to be worth a fortune, that is not the main concern. The main concern is that you might be able to cure this disease and yeah, we’ll put up money to help you so that you might be able to get into clinical trials, to get enough information to find out if it works. And to have the vision to go all the way from, ‘ok, you guys, we want you to enter this field, we want you to be interested in therapeutic development, we are going to help you structure the clinical trials, we are going to provide all the Alpha Stem Cell Clinics that can talk to each other to make the clinical trials happen.

The goal of CIRM is to change medicine and these are the approaches that have worked really well in doing this. The CIRM view clearly is:

‘There are 100 horses in this race and every single one that crosses the finish line is a success story.’ That’s what is necessary, because there are so many diseases and injuries for which new approaches are needed.”

Mark says working with CIRM has helped him spread the word back home in New York state:

“I have been very involved in working with the New York state legislature over the years to promote funding for stem cell biology and spinal cord injury research so having the CIRM experience has really helped me to understand what it is that another place can try and accomplish. A lot of the ideas that have been worked out at CIRM have been extremely helpful for statewide scientific enterprises in New York, where we have had people involved in different areas of the state effort talk to people at CIRM to find out what best practice is.”

Mark says he feels as if he has a front row seat to history.

“Seeing the stem cell field grow to its present stage and enhancing the opportunity to address multiple unmet medical needs, is a thrilling adventure. Working with CIRM to help create a better future is a privilege.”

 

A year in review – CIRM’s 2017 Annual Report focuses on a year of accelerating stem cell treatments to patients

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At CIRM we have our focus very clearly on the future, on accelerating stem cell therapies to patients with unmet medical needs. But every once in a while, it’s a good idea to look back at what you have already done. Knowing where you came from can help you get to where you are heading.

So, it’s with a sense of accomplishment that we are unveiling our 2017 Annual Report. It’s a look back at another banner year for the stem cell agency, the research we funded, the partnerships we created and, most importantly, the lives we touched.

It features profiles of several people who received stem cell therapies in CIRM-funded clinical trials and the impact those therapies are having on them. But it also looks at some of the other individuals who are such a vital part of the work we do: patient advocates, researchers and a member of our Grants Working Group which reviews applications for funding. Each one, in their own way, contributes to advancing the field.

The report also highlights some of the less obvious ways that our funding is benefitting California. For example, the additional $1.9 billion dollars our funding has helped generate through co-funding and partnerships, or the number of projects we are funding that have been awarded Regenerative Medicine Advanced Therapy Designation from the Food and Drug Administration (FDA), making them eligible for accelerated review if their results continue to be promising.

It’s a look back at a successful year.

But we are not resting on our laurels. We are already hard at work, determined to make 2018 even better.