CIRM-Funded Clinical Trials Targeting Cancers

Welcome to the Month of CIRM!

As we mentioned in last Thursday’s blog, during the month of October we’ll be looking back at what CIRM has done since the agency was created by the people of California back in 2004. To start things off, we’ll be focusing on CIRM-funded clinical trials this week. Supporting clinical trials through our funding and partnership is a critical cornerstone to achieving our mission: to accelerate stem cell treatments to patients with unmet medical needs.

Over the next four days, we will post infographics that summarize CIRM-funded trials focused on therapies for cancer, neurologic disorders, heart and metabolic disease, and blood disorders. Today, we review the nine CIRM-funded clinical trial projects that target cancer. The therapeutic strategies are as varied as the types of cancers the researchers are trying to eradicate. But the common element is developing cutting edge methods to outsmart the cancer cell’s ability to evade standard treatment.

For more details about all CIRM-funded clinical trials, visit our clinical trials page and read our clinical trials brochure which provides brief overviews of each trial.

Confusing cancer to kill it

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Thomas Kipps, MD, PhD: Photo courtesy UC San Diego

Confusion is not a state of mind that we usually seek out. Being bewildered is bad enough when it happens naturally, so why would anyone actively pursue it? But now some researchers are doing just that, using confusion to not just block a deadly blood cancer, but to kill it.

Today the CIRM Board approved an investment of $18.29 million to Dr. Thomas Kipps and his team at UC San Diego to use a one-two combination approach that we hope will kill Chronic Lymphocytic Leukemia (CLL).

This approach combines two therapies, cirmtuzumab (a monoclonal antibody developed with CIRM funding, hence the name) and Ibrutinib, a drug that has already been approved by the US Food and Drug Administration (FDA) for patients with CLL.

As Dr. Maria Millan, our interim President and CEO, said in a news release, the need for a new treatment is great.

“Every year around 20,000 Americans are diagnosed with CLL. For those who have run out of treatment options, the only alternative is a bone marrow transplant. Since CLL afflicts individuals in their 70’s who often have additional medical problems, bone marrow transplantation carries a higher risk of life threatening complications. The combination approach of  cirmtuzumab and Ibrutinib seeks to offer a less invasive and more effective alternative for these patients.”

Ibrutinib blocks signaling pathways that leukemia cells need to survive. Disrupting these pathways confuses the leukemia cell, leading to its death. But even with this approach there are cancer stem cells that are able to evade Ibrutinib. These lie dormant during the therapy but come to life later, creating more leukemia cells and causing the cancer to spread and the patient to relapse. That’s where cirmtuzumab comes in. It works by blocking a protein on the surface of the cancer stem cells that the cancer needs to spread.

It’s hoped this one-two punch combination will kill all the cancer cells, increasing the number of patients who go into complete remission and improve their long-term cancer control.

In an interview with OncLive, a website focused on cancer professionals, Tom Kipps said Ibrutinib has another advantage for patients:

“The patients are responding well to treatment. It doesn’t seem like you have to worry about stopping therapy, because you’re not accumulating a lot of toxicity as you would with chemotherapy. If you administered chemotherapy on and on for months and months and years and years, chances are the patient wouldn’t tolerate that very well.”

The CIRM Board also approved $5 million for Angiocrine Bioscience Inc. to carry out a Phase 1 clinical trial testing a new way of using cord blood to help people battling deadly blood disorders.

The standard approach for this kind of problem is a bone marrow transplant from a matched donor, usually a family member. But many patients don’t have a potential donor and so they often have to rely on a cord blood transplant as an alternative, to help rebuild and repair their blood and immune systems. However, too often a single cord blood donation does not have enough cells to treat an adult patient.

Angiocrine has developed a product that could help get around that problem. AB-110 is made up of cord blood-derived hematopoietic stem cells (these give rise to all the other types of blood cell) and genetically engineered endothelial cells – the kind of cell that lines the insides of blood vessels.

This combination enables the researchers to take cord blood cells and greatly expand them in number. Expanding the number of cells could also expand the number of patients who could get these potentially life-saving cord blood transplants.

These two new projects now bring the number of clinical trials funded by CIRM to 35. You can read about the other 33 here.

 

 

 

New stem cell approach targeting deadly blood cancers

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Every four minutes someone in the US is diagnosed with a blood cancer. It might be lymphoma or leukemia, myeloma or myelodysplastic syndromes (MDS). While we have made great strides in treating some of these over the years, we still have a long way to go. Need proof? Well, every nine minutes someone in the US dies from a blood cancer.

Because of that need, the CIRM Board last week approved $3.5 million to help fund the search for a more effective, more efficient way to treat people suffering from blood cancer.

The Board funded a program by Angiocrine Biosciences, a San Diego-based company that is developing a new method for transplanting cord blood into patients.

Now cord blood transplants have been around for decades and they can be very effective. But they can also cause serious, even life-threatening complications. And they have limitations. For example some cord blood units are small and don’t have as many stem cells as the doctors would like. As a result, patients may need to spend longer in the hospital recovering from the procedure, putting them at increased risk of viral infections or pneumonia. Alternatively, doctors could use more than one cord blood unit for each transplant and while that seems to be an effective alternative, some studies suggest it can also carry an increased risk for serious complications such as Graft-versus-host disease (GVHD) where the newly transplanted cells attack the patient’s body.

To get around these issues, Angiocrine is developing a product called AB-110. This takes stem cells from cord blood, uses a specialized manufacturing facility to expand their numbers and then mixes them with genetically modified endothelial cells, the kind of cell that forms the lining of blood vessels.

It’s hoped that AB-110 will reduce the complications and increase the chances the transplanted cells will successfully engraft, meaning they start growing and creating new, healthy, blood cells.

In a news release CIRM’s President and CEO, C. Randal Mills, PhD, says this program fits in perfectly with our mission of accelerating stem cell treatments to patients with unmet medical needs:

“This project aims to do precisely that, speeding up the body’s ability to create new white blood cells and platelets – both essential qualities when treating deadly diseases like leukemia and lymphoma. Under CIRM 2.0, we are trying to create a pipeline of products that move out of the lab and into clinical trials in people, and we’re hopeful this program will demonstrate it’s potential and get approval from the Food and Drug Administration (FDA) to begin a clinical trial.”

Everyone at Angiocrine and CIRM will work as hard as we can to move this research toward a clinical trial as fast as we can. But in the meantime there are tens of thousands of critically ill people in desperate need of a life-saving transplant.

One way of helping those in need is for new parents to donate their child’s umbilical cord blood to the state’s umbilical cord blood collection program. This is a safe procedure that doesn’t harm the baby but could save someone’s life.

The cord blood program is housed at the UC Davis Institute for Regenerative Cures – a facility CIRM helped build and where we fund many great projects. This program is particularly important because it collects and stores cord blood units that reflect the state’s diverse communities, and that are available to all those in need of a transplant.

The bank also is a rich source of cord blood units for research, particularly for stem cell research, which will hopefully lead to even more effective therapies in the future.