How is Research Translated From the Bench to the Bedside?

New techniques or treatments are identified through new insights into a disease, existing treatments that have unanticipated effects and new technologies that may be applicable for a specific disease.

Discovery stage work from our researchers:

Discovery to Drug Development: Expanding the Role of Academic Centers

As more researchers at academic centers become involved in drug development, institutions are responding with support and guidance.

Immune Profiling Leads to Implications for Immunotherapy for NF1-Associated Tumors

Profiling reveals histologic subtype distinctions and heterogeneity of neurofibromatosis type 1-associated tumors.

Researchers do experiments to understand how the treatment is done to determine exactly how the treatment or drug works. Something they look for are:

• How the treatment should be administered
• What the best dosage might be
• Any potential side effects there might be
• How it interacts with other drugs or treatments

Before drugs and treatments can be tested in patients, researchers study to make sure the drug won't have the potential to cause serious harm to patients. This is done in a very closely monitored environment that have strict regulations for personnel, facilities, equipment, and procedures. 

Preclinical stage research from our researchers:

Combining Immunotherapies Effective Against Mouse Model of Cancer

Recent study results suggest that combining virotherapy and PD-1 blockades may be more effective than either approach alone.

Two Investigational Antitumor Agents Work Better Together Against MPNST and Neuroblastoma

The synergistic effects of a kinase inhibitor and an oncolytic herpes virus show promise for difficult-to-treat neuroblastomas and malignant peripheral nerve sheath tumors. 

Several phases of clinical trials are done to answer specific research questions about the drug or treatment and make sure it is working as expected in a small group of patients. 

Clinical stage research from our researchers:

The First Study of Oncolytic HSV-1 in Children and Young Adults With Cancer Indicates Safety and Tolerability

The phase 1 trial shows safety and tolerance of HSV1716 in the pediatric population; evidence of viral replication in the blood and acute inflammation on PET/CT scans suggest a phase 2 trial for dosage and efficacy is warranted.

After the research team has gathered preclinical and clinical data that indicate a drug is safe and effective for its intended use, it is approved by the FDA for use in other patients.