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Ian MacDonald, MSc, MD, talked about the future of gene therapy trials in choroideremia, a single-gene disorder that primarily affects the eye.

Within the relatively short time that ipilimumab and vemurafenib have been commercially available, phase II data for the investigational agents nivolumab and MK-3475, for the combination of dabrafenib and trametinib, and for adoptive cell therapy strongly suggest even further improvements in treatment outcomes.

A new compound shows potential as a novel targeted therapy for retinoblastoma. While studies are still in the preliminary stages, the compound known as sd-rxRNA was able to penetrate all cell layers of the retina and was taken up by tumor cells within 24 hours of intravitreal administration, said Michael Byrne, PhD.

Phase III study of RPE65 for Leber’s congenital amaurosis under way, among other research

Phase III study of RPE65 for Leber’s congenital amaurosis under way, among other research

A dose-adjusted regimen of etoposide, doxorubicin, and cyclophosphamide with vincristine, prednisone, and rituximab (DA-EPOCH-R) obviated the need for radiotherapy in patients with primary mediastinal B-cell lymphoma in a single-group, phase II, prospective study.

The Wnt signaling pathway was demonstrated as a promising target in cancer stem cell therapy through a series of talks highlighting the promise and efficacy of a new generation of inhibitors that are making their way into the clinic.

After receiving breakthrough therapy designation from the FDA for the treatment of two B-cell malignancies earlier this year, ibrutinib has received an additional breakthrough designation.

The compound LDK378, a highly selective inhibitor of ALK, has been granted "Breakthrough Therapy Designation" by the FDA for the treatment of patients with ALK-positive metastatic non-small cell lung cancer.

Although the presence of an EGFR mutation is a predictive marker for response to EGFR tyrosine kinase inhibitor therapy in patients with non-small cell lung cancer, the mutation is not a prognostic factor.

A trial comparing axitinib to sorafenib as first-line therapy for metastatic renal cell carcinoma showed a statistically significant improvement in progression-free survival for the drug; however, the difference failed to meet the phase III trial’s prespecified significance level of 0.025.

Axitinib did not demonstrate superiority over sorafenib as first-line therapy for patients with metastatic renal cell carcinoma, on the endpoint of progression-free survival.

Tivozanib did not show a significant difference in overall survival when compared with sorafenib in patients with renal cell carcinoma who received up to one prior line of therapy excluding targeted agents.

Combination targeted therapy did not significantly extend progression-free survival compared with single-agent bevacizumab in patients with advanced renal cell carcinoma.

The investigational agent ibrutinib was granted "Breakthrough Therapy Designations" by the FDA as a singular therapy for a pair of B-cell malignancies: relapsed or refractory mantle cell lymphoma and Waldenström's macroglobulinemia.

As therapy based on cell-signaling pathways has become a priority in cancer research, so has the concept of designing clinical trials that can better target patient populations more likely to benefit from a particular regimen.

The combination of whole-brain radiation therapy and the EGFR inhibitor erlotinib showed a promising response rate and was well tolerated in a new phase II trial of patients with brain metastases from non-small-cell lung cancer.

Local ablative therapy plus continuation on the same tyrosine kinase inhibitor could be utilized as a treatment alternative in metastatic non-small cell lung cancer.

Relapsed or refractory chronic lymphocytic leukemia and acute lymphoblastic leukemia patients have shown durable responses of almost 2 years to a novel T-cell engineering technique developed at the University of Pennsylvania Perelman School of Medicine in Philadelphia.

A unique targeted therapy, quizartinib, was able to clear leukemia cells from the bone marrow in more than 33% of patients with an aggressive form of acute myeloid leukemia marked by a mutation in the FLT3 gene.

Claire E. Dearden, MD, Consultant Hematologist, Head of CLL Clinic, Royal Marsden Hospital, London, UK, highlights the pharmacotherapeutic strategies for the management of B- and T-cell PLL, noting the clinical outcomes associated with the diseases, and briefly touches on the future of PLL therapy.

The combination of temsirolimus plus bevacizumab was not superior to interferon plus bevacizumab as first-line therapy for patients with clear cell metastatic renal cell carcinoma.

A phase III study of axitinib as a front-line therapy for metastatic renal cell carcinoma did not meet its primary endpoint of demonstrating a longer median progression-free survival than sorafenib.

Stereotactic body radiation therapy is being compared to standard surgery in a phase III trial in high-risk operable patients with early-stage non–small-cell lung cancer.

Pazopanib appears to be another good option for first-line therapy of metastatic renal cell carcinoma along with sunitinib.

Maintenance therapy with both gemcitabine and erlotinib improved progression-free survival in patients with non–small-cell lung cancer who were initially treated with cisplatin-gemcitabine induction chemotherapy, according to a new phase III trial.

WARNING: IMMUNE-MEDIATED ADVERSE REACTIONS YERVOY (ipilimumab) can result in severe and fatal immune-mediated adverse reactions due to T-cell activation and proliferation. These immune-mediated reactions may involve any organ system; however, the most common severe immune-mediated adverse reactions are enterocolitis, hepatitis, dermatitis (including toxic epidermal necrolysis), neuropathy, and endocrinopathy. The majority of these immune-mediated reactions initially manifested during treatment; however, a minority occurred weeks to months after discontinuation of YERVOY. Assess patients for signs and symptoms of enterocolitis, dermatitis, neuropathy and endocrinopathy and evaluate clinical chemistries including liver function tests (LFTs) and thyroid function tests at baseline and before each dose. Permanently discontinue YERVOY and initiate systemic high-dose corticosteroid therapy for severe immune-mediated reactions.

Patients with refractory non-small cell lung cancer had a statistically significant improvement in PFS when treated with targeted therapy duo versus a single agent.

Three-fourths of patients with advanced hepatocellular carcinoma associated with hepatitis C infection achieved disease control with antibody therapy directed at a T-cell antigen.

A recent study suggests patients may be selected for therapy based on the number of EGFR gene copies, and evaluated for clinical benefit based on the severity of the rash that often develops.