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Putting Discoveries into Practice

The scope of Penn's hematology and medical oncology clinical research enterprise is very broad, spanning all phases of clinical research, including pre-clinical work and discovery, phase 1 and 2 studies and leadership of national phase 3 trials intended to change the standard of care.

Penn clinical investigators regularly publish high profile and important findings in diverse fields, ranging from the most fundamental cellular investigations, to leading edge translational and clinical research.

Notable Recent Advances

  • In a cancer treatment breakthrough 20 years in the making, Penn researchers announced in August 2011 they have shown sustained remissions of up to a year among a small group of advanced chronic lymphocytic leukemia (CLL) patients treated with genetically engineered versions of their own T cells. The protocol, which involves removing patients' cells and modifying them in Penn's vaccine production facility, then infusing the new cells back into the patient's body following chemotherapy, provides a tumor-attack roadmap for the treatment of other cancers including those of the lung and ovaries and myeloma and melanoma. 
  • Penn investigators observed unprecedented clinical activity of the selective BRAF inhibitor, vemurafenib (PLX4032), in patients with advanced melanoma. Penn investigators were also involved with the design and conduct of a national phase 3 randomized trial to support FDA registration for vemurafenib in advanced melanoma. 
  • Penn investigators in the Breast Program observed that poly (ADP-ribose) polymerase (PARP) inhibition is a viable therapeutic option in the treatment of women with BRCA-deficient breast cancers. 
  • Penn investigators in the Cancer Genetics Program presented data showing that women who are carriers of the BRCA1 and BRCA2 genetic mutations have reduced mortality if they undergo prophylactic oophorectomy. 
  • Collaborating teams in the Immunology and Phase 1 Programs showed that the combination of an agonist CD40 antibody with gemcitabine chemotherapy in a small cohort of patients with surgically incurable pancreatic cancer led to tumor regressions in some patients. 
  • Penn investigators led a group establishing consensus guidelines on stem cell collection for patients with myeloma. 
  • Penn investigators demonstrated that sorafenib is highly active in advanced differentiated thyroid cancer. Sorafenib is currently being studied in a national phase 3 study to support possible FDA registration for its use in this disease. 

Hematology/Oncology Clinical Research Today 

  • National leadership on studies sponsored by the Eastern Cooperative Oncology Group studying novel treatments for advanced kidney cancer. 
  • Clinical investigations in advanced melanoma are building on the highly encouraging results with selective BRAF inhibition, including strategies in the clinic to overcome resistance to these inhibitors.
  • A series of clinical trials is underway evaluating autophagy inhibition as a strategy to overcome resistance to chemotherapy in several refractory hematologic and solid malignancies. 
  • New signaling inhibitors are being studied alone and in combination in patients with advanced thyroid cancer, building on the results seen with sorafenib.
  • Targeted CDK4 inhibition is being evaluated as a treatment for refractory germ cell malignancies.
  • The phase 1 group regularly opens new first-in-humans phase 1 trials that are focused a wide range of novel targets involved in tumor cell signaling, angiogenesis, metastasis/migration and anti-apoptosis. The phase 1 group is also evaluating therapeutic strategies that target inflammatory cells and stroma within the tumor microenvironment.

Research Programs


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