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So You Heard About Rintega? Here’s What’s Next for Brain Tumor Drug Development

Last week, news broke that a promising clinical trial of a new type of treatment for a subset of glioblastoma (GBM) patients – an immunotherapeutic vaccine made by Celldex Therapeutics called RINTEGA (rindopepimut) – was discontinued because patients receiving the investigational therapy were not doing better than patients in the control arm.

As we said in our statement regarding this news, these results were a bit shocking and certainly disappointing. There had been high-hopes in the field for RINTEGA and it is frustrating for the whole community that a new treatment for newly diagnosed GBM patients did not come to pass.

However, there is still a lot to be encouraged by in brain tumor drug discovery and development.

At last count, 58 new potential medicines were in clinical development for brain tumor patients (PhRMA 2015 Report). This is up from 47 just the year prior (PhRMA 2014 Report)*.

Most of these treatments are either targeted therapies, in the field of “precision medicine,” or immunotherapies in one of the various categories of immuno-oncology, which remains very promising despite last week’s news.

And, while clinical trials remain a high-stake endeavor for sponsors and participants, and the historical rate of success in brain tumor clinical trials has been troubling, there is more hope and momentum for getting new and better treatment to patients now than ever before.

Importantly, many of these potential new treatments being developed and tested will benefit from improvements in brain tumor clinical trials that are beginning to be implemented.

NBTS is leading the way on many of these initiatives, as it has recognized the need to improve the brain tumor research and clinical trial process. Significant developments include:

  1. A new imaging protocol for brain tumor clinical trials was announced last year that will help clinical trial sponsors and regulators identify with more confidence which experimental treatments are working and those that aren’t. This may allow promising new therapies to reach approvals faster via an Accelerated Approval pathway, while making sure sponsors can move on from ineffective therapies sooner. NBTS spearheaded this effort together with Jumpstarting Brain Tumor Drug Development Coalition partners, as part of our Clinical Trial Endpoints Initiative after realizing there was a great opportunity to improve the way potential new brain tumor drugs were being evaluated in clinical trials.
  2. As part of the same umbrella initiative (Clinical Trial Endpoints), NBTS has also been a catalyst in helping the field come together to make trials more patient-centric and let the patient voice be heard in drug development. The hope is future clinical trials will help better meet the needs of patients.

Clinical trials for potential new brain tumor treatments in the future will become an even greater venue to access the most cutting-edge treatments. Moving forward, participating in clinical research will be more important than ever in ensuring the best new therapies get approved and make it to the market.

Leaders of the Defeat GBM Research Collaborative meet at the 2015 National Brain Tumor Society Scientific Summit

Now, to take a step back, it’s also important to note that future potential therapies entering clinical trials for brain tumors will also have benefited from new models of collaborative research happening much earlier in the R&D process – during the basic/discovery, translational, and preclinical phases of research – thus increasing their odds for success in clinical trials before they even enter them.

For example, NBTS’ Defeat GBM Research Collaboration allows some of the world’s foremost experts in glioblastoma (GBM) research – with complementary skill sets – to work together in a structure that cultivates unprecedented collaboration for research teams involved to regularly interact; share data, information, and ideas; work on multiple, interrelated projects simultaneously; and accelerate the time it takes to produce new and better treatments for GBM patients.

Defeat GBM’s Drug Development Core (team) illustrates how a novel approach to brain cancer research can positively impact future clinical trials. The Drug Development team, led by researchers at MD Anderson Cancer Center, has established a drug-screening infrastructure that is unrivaled in the brain cancer field:

  • Utilizing one of the most comprehensive sets of pre-clinical testing models the team has screened thousands of compounds (alone and in combinations) obtained from different biopharmaceutical companies.
  • Over 75,000 single drug and drug combinations have been screened across a range of 12 different GBM cell line lab models. This screening effort has prioritized the most active compounds to advance to further study in mice models. One compound in particular was found to have especially promising activity in both cell and animal models, and has been identified as a potential clinical candidate for a phase 1 clinical trial in GBM.
  • Additionally, this effort is allowing researchers to identify the type of molecular profiles that may predict whether a patient’s tumor is going to be sensitive or resistant to a targeted therapy – which could benefit any number of future clinical trials.

This type of broad-based effort is setting up a new mode for how investigators interact with each other to get, not just get trials done – I mean there are clinical trial operations all over the place – but to get real science done that then feeds into trials that are being designed as the science is being done by the people who are doing the science and doing the trial.

Attribution: Dr. Webster Cavenee of Ludwig Cancer Research, Defeat GBM researcher and advisor

Finally, a renewed interest in advancing cancer research in our nation’s capital should generate even more hope for what lies ahead for new brain tumor treatments. In the past two and a half years, we’ve seen President Obama announce major federal Precision Medicine Initiative; the bipartisan passage of the 21st Century Cures Act in the U.S. House of Representatives (with companion legislation currently being considered in the U.S. Senate); the White House and Vice President Joseph Biden announce a new Cancer Moonshot Taskforce; and Congress ending 2015 by passing a budget deal for FY16 that delivered the biggest increase in funding for the National Institutes of Health (NIH), the largest funder of brain tumor research in the U.S., in 12 years, along with increases for the National Cancer Institute, and the FDA.

There is great reason to be hopeful for continued momentum and progress for the brain tumor community in the months and years ahead. But there is still much work to be done. And so despite last week’s setback, we continue to fight on with renewed vigor and resolve.

*NOTE: these figures are just for medicines in development by biopharmaceutical companies. There are additional investigational agents/clinical trials underway sponsored by non-biopharmaceutical research groups.

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