From discovery to medicine
Part three: What we’re doing to move lab discoveries into the clinic

In the past few weeks, VAI Voice has discussed what it takes to translate a promising laboratory discovery into a life-changing, approved treatment. In our final post in this three-part series, we’re going to take a look at the many ways that the Institute is involved in the clinical trial process. Be sure to check out part one and part two!

Van Andel Research Institute (VARI) takes its mission to improve the health and enhance the lives of current and future generations to heart.

That’s why we’re deeply committed to supporting clinical trials, which are critical steps in transforming innovative lab discoveries into approved treatments that better patients’ lives. Although the Institute isn’t a medical center, it serves as a connector that brings together the best of the best to streamline the therapeutic development process and help move urgently needed treatments into clinical use. We do this by designing and facilitating clinical trials in collaboration with organizations across the U.S. and abroad.

Currently, the Institute supports more than a dozen clinical trials in cancer and Parkinson’s disease through two of our largest collaborations. Here’s how we’re working to make a difference:

Cancer

The VARI–SU2C Epigenetics Dream Team has seven ongoing clinical trials at medical centers across the U.S. and in Copenhagen, Denmark. Two of these trials — non-small cell lung cancer and bladder cancer — are supported by two of 10 inaugural SU2C Catalyst^® grants, totaling nearly $5.5 million. The trials are evaluating new combination treatments for:

Acute myeloid leukemia (AML), an aggressive blood cancer that is notoriously difficult to treat and has poor long-term survival.

Bladder cancer, a tough-to-treat cancer that is the sixth most common type of cancer diagnosed in the U.S.
*Supported by an SU2C Catalyst^® grant

Liver, pancreatic, bile duct and gallbladder cancers, a group of cancers with few available treatment options.

Metastatic colorectal cancer, the second leading cause of cancer death in men and women combined in the U.S.

MDS and AML, which also are the subject of a small pilot study that is investigating whether a simple addition to the standard care regimen may improve the current therapy’s ability to impede cancer cell growth and destroy cancer cells. This combination is also being explored in patients with clonal cytopenia of undetermined significance (CCUS) — thought to be a potential precursor to MDS in some patients.

Myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML), two blood cancers that are incurable with current drugs and that may progress to AML, a much more aggressive cancer.

Non-small cell lung cancer, the most common type of lung cancer, which accounts for more than 80 percent of cases. Lung cancers are a major public health problem and claim more lives annual than any other type of cancer.
*Supported by an SU2C Catalyst^® grant

Parkinson’s disease

The Linked Clinical Trials initiative, spearheaded by The Cure Parkinson’s Trust and supported by the Institute, aims to shift the paradigm on Parkinson’s treatment from managing symptoms to slowing or stopping the disease’s progress. By investigating medications that are already approved to treat other conditions and that impact the same biological processes at play in Parkinson’s, scientists hope to cut the time it takes for new, more effective medications to be approved, getting them to the people who need them faster.

Medications being investigated by LCT include:

Ambroxol, a medication originally developed to treat respiratory ailments, which has shown promise in correcting an underlying molecular problem in Parkinson’s.

Deferiprone, a medication that removes excess iron from the blood and that is being investigated for its potential to reduce high iron levels in the area of the brain most affected by Parkinson’s.

EPI-589, an experimental drug originally designed to treat rare mitochondrial diseases in children.

A growing body of evidence suggests similar dysfunctions in mitochondria, the power plants of cells, may also contribute to Parkinson’s.

Exenatide, a Type 2 diabetes medication that has shown outstanding promise in lab experiments and clinical trials as a therapy that may slow the progression of Parkinson’s. Following positive results from a phase II trial reported in 2017, plans for a larger, phase III trial are in the works.

Liraglutide, another Type 2 diabetes medication currently being investigated as part of a clinical trial at Cedars-Sinai Medical Center in Los Angeles, Calif.

Nilotinib, a medication originally developed to treat the blood cancer leukemia. This multi-center trial is supported by the Michael J. Fox Foundation for Parkinson’s Research, The Cure Parkinson’s Trust and VARI.

Simvastatin, a cholesterol-lowering medication that is part of the PD-STAT trial, which is underway at 21 medical centers across the United Kingdom.

Closing thoughts

Clinical trials are immensely complex, and require an extraordinary level of teamwork between scientists, physicians, patients, pharmaceutical companies, research organizations and the FDA to ensure safe and effective therapies make it into the doctor’s office. By collaborating with colleagues and organizations around the world, we are working hard to efficiently translate lab discoveries into new therapies that improve human health.

Learn more about our efforts at vai.org/clinical-trials.

Did you know? Some of our scientists also are physicians who treat patients at local hospitals, using their research to inform treatment and taking what they learn in the clinic back into the lab (read more about our physician-scientists here.)