Mustang Bio

Stem cell therapy for deadly childhood immune disorder goes four for four

/ Kevin McCormack / Leave a comment

The gold standard for any new therapy in the U.S. is approval by the Food and Drug Administration (FDA). This approval clears the therapy for sale and often also means it will be covered by insurance. But along the way there are other designations that can mean a lot to a company developing a new approach to a deadly disease.

That’s what recently happened with Mustang Bio’s MB-107. The therapy was given Orphan Drug Designation for the treatment of X-linked Severe Combined Immunodeficiency (SCID) also known as “bubble baby disease”, a rare but deadly immune disorder affecting children. This is the same therapy that CIRM is funding in a clinical trial we’ve blogged about in the past.  

Getting Orphan Drug Designation can be a big deal. It is given to therapies intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. It comes with some sweet incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. And, if the product becomes the first in its class to get FDA approval for a particular disease, it is entitled to seven years of market exclusivity, which is independent from intellectual property protection.

This is not the first time Mustang Bio’s MB-107 has been acknowledged as a potential gamechanger. It’s also been given three other classifications both here in the US and in Europe.

  • Rare Pediatric Disease Designation: this also applies to treatments for diseases affecting fewer than 200,000 people in the US that have the potential to provide clinically meaningful benefits to patients. It provides the company with a “voucher” that they can use to apply for priority review for another therapy they are developing. The hope is that this will encourage companies to develop treatments for rare childhood diseases that might not otherwise be profitable.
  • Regenerative Medicine Advanced Therapy (RMAT) designation: this allows for faster, more streamlined approvals of regenerative medicine products
  • Advanced Therapy Medicinal Product classification: this is granted by the European Medicines Agency (EMA) to medicines that are based on genes, tissues or cells and can offer groundbreaking opportunities for the treatment of disease.

Of course, none of these designations are a guarantee that Mustang Bio’s MB-107 will ultimately get FDA approval, but they’re a pretty good indication that a lot of people have confidence they’ll get there.

California gets first royalty check from Stem Cell Agency investments

/ Kevin McCormack / Leave a comment

COH image

CIRM recently shared in a little piece of history. The first royalty check, based on CIRM’s investment in stem cell research, was sent to the California State Treasurer’s office from City of Hope. It’s the first of what we hope will be many such checks, helping repay, not just the investment the state made in the field, but also the trust the voters of California showed when they created CIRM.

The check, for $190,345.87, was for a grant we gave City of Hope back in 2012 to develop a therapy for glioblastoma, one of the deadliest forms of brain cancer. That has led to two clinical trials and a number of offshoot inventions that were subsequently licensed to a company called Mustang Bio.

Christine Brown, who is now the principal investigator on the project, is quoted in a front page article in the San Francisco Chronicle, on the significance of the check for California:

“This is an initial payment for the recognition of the potential of this therapy. If it’s ultimately approved by the FDA as a commercial product, this could be a continued revenue source.”

In the same article, John Zaia, Director of the City of Hope Alpha Stem Cell Clinic, says this also reflects the unique nature of CIRM:

“I think this illustrates that a state agency can actually fund research in the private community and get a return on its investment. It’s something that’s not done in general by other funding agencies such as the National Institutes of Health, and this is a proof of concept that it can work.”

Maria Millan, CIRM’s President & CEO, says the amount of the payment is not the most significant part of this milestone – after all CIRM has invested more than $2.5 billion in stem cell research since 2004. She says the fact that we are starting to see a return on the investment is important and reflects some of the many benefits CIRM brings to the state.

“It’s a part of the entire picture of the return to California. In terms of what it means to the health of Californians, and access to these transformative treatments, as well as the fact that we are growing an industry.”

 

Taking a new approach to fighting a deadly brain cancer

/ Kevin McCormack / Leave a comment

Christine Brown DSC_3794

Christine Brown, Ph.D., City of Hope researcher

CIRM’s 2017 Annual Report will be going live online very soon. In anticipation of that we are highlighting some of the key elements from the report here on the Stem Cellar.

One of the most exciting new approaches in targeting deadly cancers is chimeric antigen receptor (CAR) T-cell therapy, using the patient’s own immune system cells that have been re-engineered to help them fight back against the tumor.

Today we are profiling City of Hope’s Christine Brown, Ph.D., who is using CAR-T cells in a CIRM-funded Phase 1 clinical trial for an aggressive brain cancer called malignant glioma.

“Brain tumors are the hardest to treat solid tumors. This is a project that CIRM has supported from an early, pre-clinical stage. What was exciting was we finished our first milestone in record time and were able to translate that research out of the lab and into the clinic. That really allowed us to accelerate treatment to glioblastoma patients.

I think there are glimmers of hope that immune based therapies and CAR-T based therapies will revolutionize therapy for patients with brain tumors. We’ve seen evidence that these cells can travel to the central nervous system and eliminate tumors in the brain.

We now have evidence that this approach produces a powerful, therapeutic response in one group of patients. We are looking at why other patients don’t respond as well and the CIRM funding enables us to ask the questions that will, we hope, provide the answers.

Because our clinical trial is a being carried out at the CIRM-supported City of Hope Alpha Stem Cell Clinic this is a great example of how CIRM supports all the different ways of advancing therapy from early stage research through translation and into clinical trials in the CIRM Alpha Clinic network.

There are lots of ways the tumor tries to evade the immune system and we are looking at different approaches to combine this therapy with different approaches to see which combination will be best.

It’s a challenging problem and it’s not going to be solved with one approach. If it were easy we’d have solved it by now. That’s why I love science, it’s one big puzzle about how do we understand this and how do we make this work.

I don’t think we would be where we are at without CIRM’s support, it really gave the funding to bring this to the next level.”

Dr. Brown’s work is also creating interest among investors. She recently partnered with Mustang Bio in a $94.5 million agreement to help advance this therapy.