clinical trial Archives - NFCR

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Understanding Adaptive Clinical Trials

Testing new treatments is key to successfully treating cancer. Unfortunately, the current model for conducting clinical trials — evaluating the effectiveness of therapies on human subjects — is inefficient, expensive and suffers from high failure rates. To overcome these challenges, the practice of adaptive trials is emerging and has become one of the most promising advancements in cancer research today.

Challenges with traditional clinical trials

Currently, it takes approximately $2.5 billion dollars, 10+ years and thousands of patient volunteers to bring one drug to market.[i] Despite this high cost, over half of phase III oncology trials have negative results or fail to complete. In addition, traditional trials cannot be modified once they begin. Patients get only one opportunity and receive only one treatment.

Adaptive trials literally adapt as evidence accumulates

Adaptive trials do not require years of follow-up to determine whether a new experimental treatment is beneficial. They test multiple treatments and combinations of treatments on patients in parallel and are designed to be continuously updated as researchers use what they learn from each patient to make real-time adjustments as the trial proceeds.

They are flexible by design so adjustments can be made in terms of dosage, subject population or sample size as needed.

In an adaptive trial, if one treatment regimen seems to be more successful, researchers can increase the number of participants receiving that treatment. This is especially important for patients with aggressive cancers as life expectancies are too short for traditional trial timetables. With adaptive trials, ineffective treatments can be shut down early, and new treatments can be initiated quickly enabling patients to receive the most promising treatments faster.

In a latest article published on Science Magazine: “Congress and FDA nominee heap love on ‘adaptive trials”, GBM AGILE is featured as one of the ongoing clinical trials using this new adaptive trial design.

GBM AGILE revolutionizes brain cancer trials

Glioblastoma multiforme (also known as GBM) is the deadliest brain cancer and is widely regarded as incurable and universally fatal, killing 95% of patients within five years of diagnosis. To combat this deadly disease, NFCR is part of a robust, international coalition working on innovative ways to defeat GBM utilizing adaptive trials known as GBM AGILE.

Led by the best and brightest cancer researchers, GBM AGILE is a revolutionary global collaboration to test and develop new brain cancer treatments. Its personalized approach will allow us to accelerate the discovery of targeted treatments for individual patients. This global coalition has attracted over 150 participants from more than 40 leading cancer institutions across three continents. This revolutionary approach accelerates research for curing the aggressive form of brain cancer GBM and will serve as a new clinical research model for combating other cancers as well.

In addition to GBM AGILE, there are other adaptive trials underway for certain cancers and tumor types, with more coming soon.  Other trials include:

  • I-SPY TRIAL is an adaptive clinical trial for women with newly diagnosed, locally advanced breast cancer.
  • Lung-MAP is a large adaptive clinical trial testing several new treatments for patients who have advanced stage squamous cell lung cancer.
  • Precision Promise is the first large-scale precision medicine adaptive trial for patients with pancreatic cancer.

While adaptive trial features are not yet commonplace, Congress and the FDA are focused on accelerating their development to benefit patients.

[i] https://www.scientificamerican.com/article/cost-to-develop-new-pharmaceutical-drug-now-exceeds-2-5b/

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The First Successful Clinical Trial

Clinical Trial and translational medicine. Our CEO and son of the Founder of NFCR likes to talk history. Franklin C. Salisbury, Jr. notes the coincidence, perhaps, that the Salisbury name has long been associated with unheralded basic research that leads to major breakthroughs. Clinical trial and translational medicine –  bring work from lab to patient bedside and is not really a new trend.

HMS Salisbury site of first clinical trialThe HMS Salisbury was a 50 gun British Warship, built at East Cowes, Isle of Wight and launched  on January 29, 1746.  James Lind was the Royal Navy surgeon who studied treatments for scurvy “on board the Salisbury at sea” in 1747.1747 year of the first ever clinical trial

We all remember the story that lemons and oranges cured scurvy and was discovered aboard a navy vessel  but who besides our Franklin would think to connect HMS Salisbury with clinical trials done today. And yet, the comparison is an important one.

The Salisbury  was said to have some 30-40 members of the crew afflicted by scurvy.  “Yet the roll call shows at most one or two as sick during this entire voyage on which six men “departed this life”. This suggests a culture of official denial of sickness at sea, one of many possible reasons, perhaps, why Lind’s work was neglected says Graham Sutton in his essay James Lind aboard Salisbury (Sutton G (2004) James Lind aboard Salisbury)

Official Denial is an interesting phrase. One perhaps that has a place today in our world of blockbuster breakthroughs.There is an interesting bridge between Salisbury, Scurvy, Citrus, Vitamin C and the cancer research supported by NFCR. That connection is Albert Szent-Györgyi.

He was the co-founder of NFCR along with Franklin Salisbury’s dad. Szent-Györgyi was credited with discovering Vitamin C and received the Nobel Prize in 1937. He went on to state his belief that” Cancer is a disease that can be cured,” and the rest is history. For more than 43 years NFCR has been supporting basic lab research into the causes, prevention and treatment of cancer.  The Albert Szent-Györgyi Prize for Progress in Cancer Research is named in honor of Doctor Albert Szent-Györgyi and is a symbol of NFCR’s enduring commitment to uphold Dr. Szent-Györgyi’s vision of curing cancer through innovation and collaboration.

NFCR funded scientists can attest to the need for long term support of basic research. Today’s breakthroughs are often the result of decades of meticulous work built on the work of previous generations. Clinical trial make a difference.   The two men on board the Salisbury that were assigned to the citrus fruit treatment recovered. So too is our hope that those who participate in today’s clinical trials are the lucky ones.

 

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