FDA Grants Breakthrough Status to Immunotherapy Combination for Pancreatic Cancer

The gist: A new pancreatic cancer treatment combines two drugs, known as CRS-207 and GVAX Pancreas. This combo treatment boosts a patient’s own immune system to fight cancer. It has been tested in volunteer patients in clinical trials, and has shown promising results for people with metastatic pancreatic cancer. The U.S. Food and Drug Administration (FDA) has now granted breakthrough therapy designation for CRS-207 plus GVAX Pancreas, meaning that review and approval will be accelerated so that the drug can more quickly reach patients in the U.S., outside of clinical trials.

“The FDA today granted breakthrough therapy designation to the combination of two immunotherapies — CRS-207 and GVAX Pancreas — for the treatment of metastatic pancreatic cancer, according to Aduro BioTech, the combination treatment’s manufacturer.

“The combination of GVAX, an irradiated, granulocyte-macrophage colony–stimulating factor vaccine, and CRS-207, an immunotherapy vaccine containing live-attenuated Listeria monocytogenes bacteria, induces a potent innate and T-cell–mediated immune response.

“ ‘We are extremely pleased to receive Breakthrough Therapy Designation and the high degree of FDA collaboration toward advancement of our program that it confers,’ Stephen T. Isaacs, chairman, president and CEO of Aduro, said in a press release. ‘This designation underscores the potential of our combination immunotherapy approach to make a difference in the lives of patients with pancreatic cancer, which remains a very difficult cancer to treat. We are encouraged by our phase 2 results and look forward to completing enrollment in our phase 2b ECLIPSE trial by end of 2015.’ “


FDA Grants Breakthrough Designation to Personalized Immunotherapy for ALL

“The FDA today granted breakthrough therapy designation for CTL019, an investigational personalized immunotherapy, for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia, according to a press release issued by Penn Medicine.

“CTL019 (Novartis), developed by the University of Pennsylvania, is the first personalized cellular therapy for the treatment of cancer to receive this classification.

“In early-stage clinical trials conducted at the Hospital of the University of Pennsylvania and Children’s Hospital of Philadelphia, 89% of patients with ALL who were not responding to conventional therapies achieved complete remission after treatment with CTL019.

“ ‘Our early findings reveal tremendous promise for a desperate group of patients, many of whom have been able to return to their normal lives at school and work after receiving this new, personalized immunotherapy,’ Carl H. June, MD, director of translational research in the Abramson Cancer Center of the University of Pennsylvania. ‘Receiving the FDA’s Breakthrough Designation is an essential step in our work with Novartis to expand this therapy to patients across the world who desperately need new options to help them fight this disease.’ ”

Editor’s note: This story describes a new leukemia treatment called CTL019, which boosts a patient’s own immune system to fight cancer. CTL019 treatment is personalized for each patient, since it involves altering a patient’s immune system cells to attack tumor cells. It has been tested in volunteer patients in clinical trials, and has shown promising results for adults and children with relapsed or refractory acute lymphoblastic leukemia (ALL). The U.S. Food and Drug Administration (FDA) has now granted breakthrough therapy designation for CTL019, meaning that review and approval will be accelerated so that the drug can more quickly reach patients outside of clinical trials.


ASCO 2014: Highlights for People Dealing with Melanoma


Every year, new cancer treatment insights are shared at the American Society of Clinical Oncology (ASCO) Annual Meeting. Here are some of the most notable recent developments in melanoma treatment, gleaned from researchers’ presentations at ASCO last month: Continue reading…


Blinatumomab Receives Breakthrough Therapy Designation for ALL

“The FDA has granted a breakthrough therapy designation to blinatumomab for the treatment of adult patients with Philadelphia-negative relapsed/refractory B-precursor acute lymphoblastic leukemia (ALL), according to Amgen, the company developing the drug.

“The breakthrough therapy designation was based on results from a phase II trial of 189 patients. As of January 2014, 43% (n = 82) of patients achieved a complete remission (CR) or CR with partial hematological recovery (CRh). These results were presented at the 2014 ASCO Annual Meeting and at the 19th Congress of the European Hematology Association.

” ‘There is a high unmet need for new medicines to treat relapsed and refractory ALL patients, who have very few treatment options,’ Sean E. Harper, MD, executive vice president, Research and Development, Amgen, said in a statement. ‘The results from the phase II trial evaluating blinatumomab in adult patients with relapsed or refractory ALL are encouraging and provide a strong basis for a regulatory filing later this year and potential approval in this serious disease.’ ”

Editor’s note: Blinatumomab is a new drug that is being tested as a potential treatment for people with acute lymphoblastic leukemia (ALL) that is resistant to treatment (refractory) or that has returned after treatment (relapsed). Based on promising research results, blinatumomab has received a Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA), specifically for adults with relapsed or refractory B-precursor ALL that is Philadelphia-negative. This means that the FDA finds the drug to be very promising compared to currently available treatments, and will accelerate the approval process that would ultimately permit U.S. oncologists to prescribe the drug outside of a clinical trial.


AstraZeneca Seeking to Get Cancer Drugs to Patients Early

“British patients could benefit from AstraZeneca’s promising new cancer drugs well before they complete clinical testing, the Telegraph can reveal.

“Pascal Soriot, chief executive, told the Telegraph he was looking to make two of Astra’s most eye-catching cancer drugs widely available to patients as soon as possible.

“Britain’s recently launched early access to medicines scheme, which aims to get drugs to patients years earlier than the normal regulatory process allows, is among the programmes in Mr Soriot’s sights. The US, France and Japan have also established pathways to fast-track promising new drugs to patients ahead of final regulatory approval.”


Clovis Oncology Receives Breakthrough Therapy Designation for CO-1686 for the Treatment of Second-line EGFR Mutant Non-small Cell Lung Cancer (NSCLC) in Patients with the T790M Mutation

Clovis Oncology, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the Company’s investigational agent CO-1686 as monotherapy for the treatment of second-line EGFR mutant NSCLC in patients with the T790M mutation. The Breakthrough Therapy designation was granted based on interim efficacy and safety results from an ongoing Phase 1/2 study of CO-1686. CO-1686 is the Company’s novel, oral, targeted covalent (irreversible) inhibitor of mutant forms of the epidermal growth factor receptor (EGFR) for the treatment of non-small cell lung cancer in patients with initial activating EGFR mutations as well as the dominant resistance mutation T790M.”

Editor’s note: CO-1686 is a targeted therapy drug that is meant to treat patients whose tumors have mutations in the EGFR gene, as detected by molecular testing. Some tumors with EGFR mutations become resistant to first-line treatment and develop a new mutation called T790M. Evidence shows that non-small cell lung cancer (NSCLC) patients with T790M may benefit from second-line treatment with CO-1686.The drug has received a Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA). This means that the FDA finds CO-1686 to be very promising compared to currently available treatments for T790M-mutated NSCLC, and will accelerate the approval process that would ultimately permit oncologists to prescribe the drug outside of a clinical trial.


Induction of Systemic Immunity Following Treatment of Tumors with PV-10 Reported by Moffitt Cancer Center Researchers at American Association for Cancer Research Annual Meeting

“Provectus Biopharmaceuticals, Inc. (OTCQB: PVCT, http://www.pvct.com), a development-stage oncology and dermatology biopharmaceutical company, announced today that a poster presentation detailing significant decrease in melanoma cells in patients’ injected tumors 7-14 days after intralesional PV-10 treatment that was accompanied by similar decrease in uninjected bystander tumors was presented April 6 by researchers from the Moffitt Cancer Center at the American Association for Cancer Research Annual Meeting in San Diego, CA. These clinical and pathologic changes were accompanied by increases in important immune cell populations detected in the patients’ peripheral blood.”

Editor’s note: This story is about a new treatment called PV-10 that is injected directly into melanoma tumors. A clinical trial to test the drug in people with melanoma has shown promising results. The manufacturers of the drug have submitted an application for “breakthrough therapy” designation from the U.S. Food and Drug Administration (FDA) to expedite the FDA-approval process and rapidly make the drug available to many more melanoma patients in the U.S.


Cancer Drug Tafinlar Receives Breakthrough Therapy Designation for Lung Cancer

The application for dabrafenib (Tafinlar) as a treatment for certain lung cancer cases has been given a boost with the U.S. Food and Drug Administration (FDA) designating it a breakthrough therapy. Tafinlar is being investigated as a therapy for patients with non-small cell lung cancer (NSCLC) who have a mutation called BRAF V600E in the BRAF gene and have received at least one previous round of chemotherapy. In a recent clinical trial, Tafinlar exhibited antitumor activity in such patients. The breakthrough therapy designation provides increased drug development guidance from the FDA and accelerated approval for drugs that treat serious or life-threatening conditions and that provide a substantial improvement over currently available treatments. Tafinlar is already approved for use in certain types of skin cancer.


Compassionate Drug Access: A Real Option for Cancer Patients?


A recent New York Times article tells the story of one woman’s quest to gain access to an experimental drug to treat her deadly cancer. Her story is familiar to many of us who have heard similar tales; a cancer patient runs out of treatment options, but with the help of proactive oncologists is able to receive a new, investigational drug; that is, a drug not yet approved by the U.S. Food and Drug Administration (FDA). This last-resort treatment approach is known as compassionate use or, as the FDA prefers to call it, expanded access. The U.S. National Library of Medicine explains: Continue reading…