On April 18, 2016 Genzyme, the specialty care global business unit of Sanofi, reported that positive new brain volume data from an ongoing clinical study of Lemtrada (alemtuzumab) will be presented at the 68th American Academy of Neurology (AAN) Annual Meeting (Press release, Genzyme, APR 18, 2016, View Source [SID:1234511027]). In addition, new data from an exploratory study will be presented which demonstrate the impact of Lemtrada on retinal nerve fibers.

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Lemtrada Investigational Brain Volume Data: Relapsing-remitting multiple sclerosis (RRMS) patients treated with interferon beta-1a in CARE-MS I and II for two years who switched to Lemtrada in the extension study experienced a reduced rate of brain volume loss over the next three years.

Median yearly brain volume loss in interferon beta-1a treated patients in year two in CARE-MS I (-0.50%) and CARE-MS II (-0.33%) was reduced in years one, two and three after switching to Lemtrada (CARE-MS I: -0.07%, -0.13%, -0.09%; CARE-MS II: 0.02%, -0.05%, -0.14%).

Lemtrada Investigational Retinal Data: A measurable improvement in retinal nerve fiber layer (RNFL) thickness was seen in 26 Lemtrada-treated RRMS patients. Over two years, the change in average RNFL thickness for all eyes was +1.5 micrometers (95% CI 0.2, 2.9; p=0.032). The observed thickening of retinal fibers may reflect protection of retinal axons in these patients.

"The Lemtrada data being presented at AAN from the ongoing extension study demonstrating slowed brain volume loss over three years are consistent with sustained effects seen in prior clinical, imaging and atrophy analyses," said Dr. Anthony Traboulsee, Associate Professor of Neurology and Medical Director of the UBC Hospital MS Clinic of Vancouver Coastal Health. "In addition, given the critical importance of neuroprotection in the treatment of MS, the retinal nerve fiber data are also exciting and support further investigation."

In clinical trials, serious side effects associated with Lemtrada included infusion-associated reactions, autoimmune disorders (such as thyroid disease, autoimmune cytopenias, and nephropathies), infections and pneumonitis. Risk management programs incorporating education and monitoring help support early detection and management of key identified and potential risks. The most common side effects of Lemtrada are rash, headache, pyrexia, nasopharyngitis, nausea, urinary tract infection, fatigue, insomnia, upper respiratory tract infection, herpes viral infection, urticaria, pruritus, thyroid gland disorders, fungal infection, arthralgia, pain in extremity, back pain, diarrhea, sinusitis, oropharyngeal pain, paresthesia, dizziness, abdominal pain, flushing, and vomiting. (See Important Safety Information below.)

About Lemtrada (alemtuzumab)
Lemtrada is approved in more than 50 countries, with additional marketing applications under review by regulatory authorities globally. Lemtrada is supported by a comprehensive and extensive clinical development program that involved nearly 1,500 patients worldwide and 5,400 patient-years of follow-up.

In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates; the difference observed in slowing disability progression did not reach statistical significance. In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates, and accumulation of disability was significantly slowed in patients given Lemtrada vs. interferon beta-1a.

The precise mechanism by which alemtuzumab exerts its therapeutic effects in MS is unknown. Alemtuzumab is a monoclonal antibody that targets CD52, a protein abundant on T and B cells. Circulating T and B cells are thought to be responsible for the damaging inflammatory process in MS. Lemtrada depletes circulating T and B lymphocytes after each treatment course. Lymphocyte counts then increase over time with a reconstitution of the lymphocyte population that varies for the different lymphocyte subtypes.

Genzyme holds the worldwide rights to alemtuzumab and has responsibility for its development and commercialization in multiple sclerosis. Bayer Healthcare receives contingent payments based on global sales revenue.

Lemtrada (alemtuzumab) U.S. Indication
LEMTRADA is a prescription medicine used to treat adults with relapsing forms of multiple sclerosis (MS). Because of its risks, LEMTRADA is generally used in people who have tried 2 or more MS medicines that have not worked well enough. It is not known if LEMTRADA is safe and effective for use in children under 17 years of age.

Do not receive LEMTRADA if you are infected with human immunodeficiency virus (HIV).

IMPORTANT SAFETY INFORMATION

LEMTRADA can cause serious side effects including:

Serious autoimmune problems: Some people receiving LEMTRADA develop a condition where the immune cells in your body attack other cells or organs in the body (autoimmunity), which can be serious and may cause death. Serious autoimmune problems may include:

• Immune thrombocytopenia, which is when reduced platelet counts in your blood cause severe bleeding that, if not treated, may cause life-threatening problems. Call your healthcare provider right away if you have any of the following symptoms: easy bruising; bleeding from a cut that is hard to stop; heavier menstrual periods than normal; bleeding from your gums or nose that is new or takes longer than usual to stop; small, scattered spots on your skin that are red, pink, or purple

• Kidney problems called anti-glomerular basement membrane disease, which can, if untreated, lead to severe kidney damage, kidney failure that needs dialysis, a kidney transplant, or death. Call your healthcare provider right away if you have any of the following symptoms: blood in the urine (red or tea-colored urine); swelling of legs or feet; coughing up blood

It is important for you to have blood and urine tests before you receive, while you are receiving and every month, for 4 years or longer, after you receive your last LEMTRADA infusion.

Serious infusion reactions: LEMTRADA can cause serious infusion reactions that may cause death. Serious infusion reactions may happen while you receive, or up to 24 hours or longer after you receive LEMTRADA.

• You will receive your infusion at a healthcare facility with equipment and staff trained to manage infusion reactions, including serious allergic reactions, and urgent heart or breathing problems. You will be watched while you receive, and for 2 hours or longer after you receive, LEMTRADA. If a serious infusion reaction happens while you are receiving LEMTRADA, your infusion may be stopped.

Tell your healthcare provider right away if you have any of the following symptoms of a serious infusion reaction during the infusion, and after you have left the healthcare facility:

• swelling in your mouth or throat

• trouble breathing

• weakness

• fast, slow, or irregular heartbeat

• chest pain

• rash

To lower your chances of getting a serious infusion reaction, your healthcare provider will give you a medicine called corticosteroids before your first 3 infusions of a treatment course. You may also be given other medicines before or after the infusion to try to reduce your chances of having these reactions or to treat them after they happen.

Certain cancers: Receiving LEMTRADA may increase your chance of getting some kinds of cancers, including thyroid cancer, skin cancer (melanoma), and blood cancers called lymphoproliferative disorders and lymphoma. Call your healthcare provider if you have the following symptoms that may be a sign of thyroid cancer:

• new lump

• swelling in your neck

• pain in front of neck

• hoarseness or other voice changes that do not go away

• trouble swallowing or breathing

• cough that is not caused by a cold

Have your skin checked before you start receiving LEMTRADA and each year while you are receiving treatment to monitor for symptoms of skin cancer.

Because of risks of autoimmunity, infusion reactions, and some kinds of cancers, LEMTRADA is only available through a restricted program called the LEMTRADA Risk Evaluation and Mitigation Strategy (REMS) Program.

Thyroid problems: Some patients taking LEMTRADA may get an overactive thyroid (hyperthyroidism) or an underactive thyroid (hypothyroidism). Call your healthcare provider if you have any of these symptoms:

• excessive sweating

• unexplained weight loss

• eye swelling

• nervousness

• fast heartbeat

• unexplained weight gain

• feeling cold

• worsening tiredness

• constipation

Low blood counts (cytopenias): LEMTRADA may cause a decrease in some types of blood cells. Some people with these low blood counts have increased infections. Call your doctor right away if you have symptoms of cytopenias such as:

• weakness

• chest pain

• yellowing of the skin or whites of the eyes (jaundice)

• dark urine

• fast heartbeat

Serious infections: LEMTRADA may cause you to have a serious infection while you receive and after receiving a course of treatment. Serious infections may include:

• Herpes viral infections. Some people taking LEMTRADA have an increased chance of getting herpes viral infections. Take any medicines as prescribed by your healthcare provider to reduce your chances of getting these infections.

• Tuberculosis. Your healthcare provider should check you for tuberculosis before you receive LEMTRADA.

• Hepatitis. People who are at high risk of, or are carriers of, hepatitis B (HBV) or hepatitis C (HCV) may be at risk of irreversible liver damage.

These are not all the possible infections that could happen while on LEMTRADA. Call your healthcare provider right away if you have symptoms of a serious infection such as fever or swollen glands. Talk to your healthcare provider before you get vaccinations after receiving LEMTRADA. Certain vaccinations may increase your chances of getting infections.

Swelling of lung tissue (pneumonitis): Some people have had swelling of the lung tissue while receiving LEMTRADA. Call your healthcare provider right away if you have the following symptoms:

• shortness of breath

• cough

• wheezing

• chest pain or tightness

• coughing up blood

Before receiving LEMTRADA, tell your healthcare provider if you:

• are taking a medicine called Campath (alemtuzumab)

• have bleeding, thyroid, or kidney problems

• have HIV

• have a recent history of infection

• have received a live vaccine in the past 6 weeks before receiving LEMTRADA or plan to receive any live vaccines. Ask your healthcare provider if you are not sure if your vaccine is a live vaccine

• are pregnant or plan to become pregnant. LEMTRADA may harm your unborn baby. You should use birth control while receiving LEMTRADA and for 4 months after your course of treatment

• are breastfeeding or plan to breastfeed. You and your healthcare provider should decide if you should receive LEMTRADA or breastfeed. You should not do both.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. LEMTRADA and other medicines may affect each other, causing side effects. Especially tell your healthcare provider if you take medicines that increase your chance of getting infections, including medicines used to treat cancer or to control your immune system.

The most common side effects of LEMTRADA include:

• rash

• headache

• thyroid problems

• fever

• swelling of your nose and throat

• nausea

• urinary tract infection

• feeling tired

• trouble sleeping

• upper respiratory infection

• herpes viral infection

• hives

• itching

• fungal infection

• joint pain

• pain in your arms or legs

• back pain

• diarrhea

• sinus infection

• mouth pain or sore throat

• tingling sensation

• dizziness

• stomach pain

• sudden redness in face, neck, or chest

• vomiting

Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of LEMTRADA.

You are encouraged to report side effects of prescription drugs to the FDA. Visit View Source or call 1-800-FDA-1088

Please click here for full U.S. Prescribing Information, including boxed WARNING and Medication Guide, for additional Important Safety Information

On April 18, 2016 Onxeo S.A. (Euronext Paris, Nasdaq Copenhagen: ONXEO), an innovative company specializing in the development of orphan oncology therapeutics, reported the final data from a study aiming to confirm the mechanism of action of Livatag, a doxorubicin loaded nanoparticle formulation based on Onxeo’s Transdrug technology in overcoming cellular resistance in hepatocellular carcinoma (HCC) (Press release, Onxeo, APR 18, 2016, View Source [SID:1234511024]). Livatag is currently being evaluated in a Phase III trial (ReLive) in patients with advanced HCC, or primary liver cancer.

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Results, presented today in a poster (Abstract #2143 / Poster #13) by Dr. Graham Dixon, PhD, Onxeo’s Chief Scientific Officer, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, demonstrated that the bio-distribution of doxorubicin Transdrug (Livatag) nanoparticles showed a preferential affinity for the liver and an increased exposure in plasma compared to free doxorubicin, together supporting the use of Livatag in the treatment of patients suffering from advanced HCC.

While evaluating the mechanism of action the study showed that the nanoparticle formulation of doxorubicin Transdrug (Livatag) entered into HCC cell lines via passive diffusion and avoided recognition by certain multi-drug resistance (MDR) proteins, (P glycoprotein 1, or Pgp) leading to major accumulation of the drug in the cells and a dramatic increase in cytotoxicity in HCC cell lines compared to free doxorubicin.

Further investigations will be performed to test if doxorubicin Transdrug (Livatag) also overcomes resistance induced by other MDR-related proteins expressed by HCC cells as well as the involvement of the Livatag nanoparticle "ion pair" in overcoming the efflux-mediated resistance.

Graham Dixon, PhD, Chief Scientific Officer of Onxeo, commented, "These are important findings as they confirm that the underlying mechanism of action of Livatag’s nanoformulation effectively accumulates doxorubicin specifically in the liver and evades tumor cell resistance mediated by multiple drug resistance MDR efflux pumps, enabling an efficacious and safe approach to cancer treatment. These results further support our ongoing Phase 3 ReLive study of Livatag for the treatment of patients with advanced HCC, for which we anticipate preliminary data readout mid-2017."

On April 18, 2016 Agendia, Inc., together with the European Organisation for Research and Treatment of Cancer (EORTC) and Breast International Group (BIG), reported results from the initial analysis of the primary objective of the Microarray In Node-negative (and 1 to 3 positive lymph node) Disease may Avoid ChemoTherapy (MINDACT) study at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 in, New Orleans, LA (Press release, Agendia, APR 18, 2016, View Source [SID:1234511023]).

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Using the company’s MammaPrint assay, patients with early-stage breast cancer who were considered at high risk for disease recurrence based on clinical and biological criteria had a distant metastasis-free survival at five years in excess of 94 percent. The MammaPrint test—the first and only genomic assay with FDA 510(k) clearance for use in risk assessment for women of all ages with early stage breast cancer—identified a large group of patients for whom five-year distant metastasis–free survival was equally good whether or not they received adjuvant chemotherapy (chemotherapy given post-surgery).

"The MINDACT trial design is the optimal way to prove clinical utility of a genomic assay," said Prof. Laura van ’t Veer, CRO at Agendia, Leader, Breast Oncology Program, and Director, Applied Genomics at UCSF Helen Diller Family Comprehensive Cancer Center. "It gives the level 1A clinical evidence (prospective, randomized and controlled) that empowers physicians to clearly and confidently know when chemotherapy is part of optimal early-stage breast cancer therapy. In this trial, MammaPrint (70-gene assay) was compared to the standard of care physicians use today, to decide what is the best treatment option for an early-stage breast cancer patient."

The MINDACT trial is the first prospective randomized controlled clinical trial of a breast cancer recurrence genomic assay with level 1A clinical evidence and the first prospective translational research study of this magnitude in breast cancer to report the results of its primary objective.

Among the 3,356 patients enrolled in the MINDACT trial, who were categorized as having a high risk of breast cancer recurrence based on common clinical and pathological criteria (C-high), the MammaPrint assay reduced the chemotherapy treatment prescription by 46 percent.Using the 70-gene assay, MammaPrint, 48 percent of lymph-node positive breast cancer patients considered clinically high-risk (Clinical-high) and genomic low-risk (MammaPrint-low) had an excellent distant metastasis-free survival at five years in excess of 94 percent.

"Traditionally, physicians have relied on clinical-pathological factors such as age, tumor size, tumor grade, lymph node involvement, and hormone receptor status to make breast cancer treatment decisions," said Massimo Cristofanilli, MD, Associate Director of Translational Research and Precision Medicine at the Robert H. Lurie Comprehensive Cancer Center, Northwestern University in Chicago. "These findings provide level 1A clinical utility evidence by demonstrating that the detection of low-risk of distant recurrence reported by the MammaPrint test can be safely used in the management of thousands of women by identifying those who can be spared from a toxic and unnecessary treatment."

MINDACT is a randomized phase III trial that investigates the clinical utility of MammaPrint, when compared (or – "used in conjunction with") to the standard clinical pathological criteria, for the selection of patients unlikely to benefit from adjuvant chemotherapy. From 2007 to 2011, 6,693 women who had undergone surgery for early-stage breast cancer enrolled in the trial (111 centers in nine countries). Participants were categorized as low or high risk for tumor recurrence in two ways: first, through analysis of tumor tissue using MammaPrint at a central location in Amsterdam; and second, using Adjuvant! Online, a tool that calculates risk of breast cancer recurrence based on common clinical and biological criteria.

Patients characterized in both clinical and genomic assessments as "low- risk" are spared chemotherapy, while patients characterized as "high- risk" are advised chemotherapy. Those with conflicting results are randomized to use either clinical or genomic risk (MammaPrint) evaluation to decide on chemotherapy treatment.

The MINDACT trial is managed and sponsored by the EORTC as part of an extensive and complex partnership in collaboration with Agendia and BIG, and many other academic and commercial partners, as well as patient advocates.

"These MINDACT trial results are a testament that the science of the MammaPrint test is the most robust in the genomic breast recurrence assay market. Agendia will continue to collaborate with pharmaceutical companies, leading cancer centers and academic groups on additional clinical research and in the pursuit of bringing more effective, individualized treatments within reach of cancer patients," said Mark Straley, Chief Executive Officer at Agendia. "We value the partnership with the EORTC and BIG and it’s a great honor to share this critical milestone."

Breast cancer is the most frequently diagnosed cancer in women worldwide.[i] In 2012, there were nearly 1.7 million new breast cancer cases among women worldwide, accounting for 25 percent of all new cancer cases in women.[ii]

On April 18, 2016 Biothera Pharmaceuticals, Inc. reported research providing new insights into the ability of the Company’s Phase 2 cancer immunotherapy to coordinate innate and adaptive immune responses and enhance the effectiveness of combination therapies (Press release, Biothera, APR 18, 2016, View Source [SID:1234511022]). The findings were presented today at the AACR (Free AACR Whitepaper) annual meeting, which is taking place in New Orleans, LA, April 16-20, 2016.

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AACR logoThe company is presenting five poster presentations at the AACR (Free AACR Whitepaper) meeting focusing on the unique therapeutic mechanisms of action for Imprime PGG, a Pathogen Associated Molecular Patterning molecule, or PAMP. Imprime PGG acts as an immunological "ignition switch" enlisting the innate immune system to enhance the therapeutic efficacy of tumor targeting, anti-angiogenic, and immune checkpoint inhibitor antibodies.

"These data provide compelling new evidence demonstrating in vivo that Imprime PGG can re-orient the immunosuppressive tumor microenvironment, especially in concert with anti-VEGF or anti-VEGF2 antibodies," said Jeremy Graff, Ph.D., Chief Scientific Officer and Senior Vice President, Research, at Biothera Pharmaceuticals. "Imprime PGG also effectively binds to dendritic cells, enhancing the maturation of these professional antigen presenting cells, which is key to enabling robust T cell-mediated anti-cancer immune responses and to driving therapeutic synergy with immune checkpoint inhibitors."

The Biothera Pharmaceuticals presentations, including three appearing in the late-breaking immunology research poster session, advance understanding of the mechanisms by which Imprime PGG augments immune responses:

Imprime PGG significantly enhances the efficacy of both anti-PD-1 and anti-PD-L1 immune checkpoint inhibitors in preclinical in vivo Fully 83% of mice treated with Imprime PGG and anti-PD-L1 antibody were tumor-free after 29 days, compared with 33% of mice treated with anti-PD-L1 antibody alone. The tumor-free mice were re-injected in the opposite flank with cancer cells and remained tumor-free, indicating the establishment of immunological memory against the tumor.
Imprime PGG also upregulates expression of the critical co-stimulatory marker CD86, as well as PD-L1, on peripheral blood mononuclear cells (PBMCs) isolated from the blood of Imprime PGG-treated colon cancer patients, providing the first evidence from human clinical trials that Imprime elicits the critical immune changes in humans previously recognized in preclinical studies.
Additionally, Imprime PGG has been shown to trigger the direct tumor-killing functions of the innate immune system, dramatically reducing metastatic burden in concert with a tumor-targeting antibody in a model of melanoma metastases to lung.
By promoting myeloid-derived suppressor cell (MDSC) differentiation and repolarization of suppressive M2 macrophages, Imprime PGG re-educates the immune microenvironment and enhances the therapeutic efficacy of anti-angiogenic antibodies.
Further, Imprime PGG enables the activation and maturation of dendritic cells – the body’s professional antigen presenting cells – to facilitate T cell-mediated, antigen-specific immune responses.
The posters will be available on the Biothera Pharmaceuticals website, as they are presented today and tomorrow.

On April 18, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) reported new data from more than 1,200 pediatric tumors across 51 cancer subtypes that were analyzed using the company’s comprehensive genomic profiling assays, FoundationOne and FoundationOne Heme (Press release, Foundation Medicine, APR 18, 2016, View Source [SID:1234511021]). The dataset reveals novel and potentially targetable genomic alterations identified during pediatric cancer clinical care that offer the possibility for new research towards novel therapeutics. In a separate but related announcement at the White House Precision Medicine Initiative Summit in February, Foundation Medicine made this data set publically available for research to motivate and accelerate development of new therapies to fight pediatric cancer.

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These findings were presented in a late-breaking poster titled "Genomic profiling of 1239 diverse pediatric cancers identifies novel discoveries across tumors" by Juliann Chmielecki, Ph.D., associate director, cancer genomics at Foundation Medicine, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 taking place April 16-20 in New Orleans.

The poster presentation details genomic profiles from 1,239 pediatric tumors (ages 0-18). Multiple studies are actively investigating clinically relevant genomic alterations in common tumor subtypes for therapeutic exploitation. As demonstrated by the identification of novel fusions and mutations, this data set offers significant discovery potential and can be used to generate hypotheses, validate rare findings, and investigate the genomic landscape of rare tumors in a pediatric population for which only small studies currently exist. Key findings include:

Genomic profiles from 51 disease subtypes representing sarcomas (26.6 percent), other blastomas (22.4 percent), brain tumors (20.4 percent), hematological malignancies (19.5 percent), carcinomas (9.8 percent) and gonadal tumors (1.4 percent).

Alterations with proven clinical actionability in pediatric cancers (BRAF V600E and ALK, NTRK1 and ABL1 fusions) were found in 3.9 percent of samples across brain, sarcoma and hematologic cases.

Three novel ALK fusions were identified in a neuroblastoma (BEND5-ALK), a soft tissue sarcoma (IGFBP5-ALK) and an astrocytoma (PPP1CB-ALK), respectively. Two novel BRAF fusions were also found in an astrocytoma (BCAS1-BRAF) and a ganglioglioma (TMEM106B-BRAF).

This large data set also challenges the paradigm of "disease-specific" alterations as previously characterized fusions involving ALK, NTRK1 and PAX3 were observed in novel diseases from which they were originally reported.

Pediatric cancers are a rare and diverse collection of diseases. Childhood cancer rates have been rising slightly for the past few decades, with The American Cancer Society estimated 10,380 children in the U.S. were diagnosed with cancer in 2015. Pediatric cancer remains the leading cause of death by disease among children. Despite advances in detection and treatment of childhood cancer, over 1,900 pediatric patients in the United States succumb to disease each year.1