Breaking News
July 21, 2018 - Species diversity can have positive and negative impacts on disease transmission
July 21, 2018 - Genome research suggests presence of enteric fever in medieval Europe
July 21, 2018 - Risk of Sensory Deficits Drops With Rising Gestational Age
July 21, 2018 - Mum’s sleep matters—the effect of sleep on an unborn baby
July 21, 2018 - UC San Diego researchers awarded two grants for investigating stem cell-based therapies
July 21, 2018 - Cellular ‘garbage disposal’ may actually work on some of the proteins to neuronal development
July 21, 2018 - More Pregnant Women Having Heart Attacks
July 21, 2018 - Acne Breakouts | NIH News in Health
July 21, 2018 - Change health messaging to focus on potential impact to help stop the next pandemic
July 21, 2018 - Frailty associated with poor survival rates in young heart patients
July 21, 2018 - New discovery could save millions of lives from fatal fungal infections
July 21, 2018 - OBD presents latest data on the use of EpiSwitch™ in predicting patient response to immunotherapy and identifying lymphoma subtypes
July 21, 2018 - Childhood adversity increases susceptibility to addiction via immune response
July 21, 2018 - Scientists identify potential target for the treatment of binge eating
July 21, 2018 - Whole-brain LIPUS therapy improves cognitive dysfunction in mice simulating dementia, Alzheimer’s
July 21, 2018 - Digital media use raising risk of ADHD symptoms among the young
July 21, 2018 - Phase 3 study of tanezumab in patients with osteoarthritis pain meets all three co-primary endpoints
July 21, 2018 - Restoring mitochondrial function to reverse aging-related skin wrinkles, hair loss in mice
July 21, 2018 - SP PennTech introduces RW-500 rotary vial washer for biotech, pharmaceutical applications
July 21, 2018 - Researchers to study molecular mechanisms behind susceptibility of males to autism
July 21, 2018 - Fourth Published Clinical Trial Confirms Long-Term Safety of Niagen Supplementation at High Doses and Shows Potential for Improvement in Liver Health
July 21, 2018 - Greening vacant urban land reduces feelings of depression for surrounding residents
July 21, 2018 - Parents say intense gun violence in PG-13 movies appropriate for teens 15 and older
July 21, 2018 - Collaborative study to assess effects of exercise training for cognitive deficits in MS
July 21, 2018 - FAU researchers find possible cause of Parkinson’s disease in the patients’ immune system
July 21, 2018 - Protective qualities of ‘good cholesterol’ reduce after menopause
July 21, 2018 - Researchers develop new way to uncover hidden breast cancer tumors
July 21, 2018 - FDA approves first drug for treatment of adult AML patients with specific genetic mutation
July 21, 2018 - Top AI companies join hands to discover novel drugs for DMD
July 21, 2018 - Ferring announces FDA approval of ZOMACTON for injection in four new pediatric indications
July 20, 2018 - Researchers design proteins that can self-assemble into complex structures
July 20, 2018 - AVITA Medical expands management team to support launch of RECELL device to treat burns
July 20, 2018 - FDA Approves Tibsovo (ivosidenib) for Relapsed or Refractory Acute Myeloid Leukemia with an IDH1 Mutation
July 20, 2018 - Developmental screening and surveillance rates remain low, new study suggests
July 20, 2018 - TGen opens tissue donation portal to advance DIPG research
July 20, 2018 - Health impact of highly processed summertime staples
July 20, 2018 - Exergaming can improve health in overweight and obese children, study shows
July 20, 2018 - Postmenopausal factors may impact heart-protective qualities of ‘good cholesterol’
July 20, 2018 - MRI and blood test combination results in improved prostate cancer diagnosis
July 20, 2018 - Update Health Professional and Consumer on Recent Recalled Products
July 20, 2018 - Researchers trace Parkinson’s damage in the heart
July 20, 2018 - Wearable device designed to measure cortisol in sweat
July 20, 2018 - Scientists demonstrate a new regulation mechanism for skeletal muscles
July 20, 2018 - Exposure to mobile phone radiation may negatively impact memory performance in adolescents
July 20, 2018 - SUSU scientists find alternative method to treat post-traumatic stress disorder syndrome
July 20, 2018 - Gestational diabetes may increase offspring’s heart disease risk
July 20, 2018 - New vaccine could protect unborn babies from Zika virus
July 20, 2018 - Researchers find high mercury and methylmercury concentrations in traditional Tibetan medicine
July 20, 2018 - Brief Safety Plan Intervention in ER Can Cut Suicidal Behavior
July 20, 2018 - The Mount Sinai Hospital receives accreditation as geriatric emergency department
July 20, 2018 - Toward a better understanding of Parkinson’s disease
July 20, 2018 - Med school communications office wins four national awards | News Center
July 20, 2018 - Professional baseball players with faster hand-eye coordination may have better batting performance
July 20, 2018 - Study looks into mechanisms that control sleep and wakefulness
July 20, 2018 - Scientists identify melanoma biomarkers that could help tailor immunotherapy treatments
July 20, 2018 - Research reveals long-term efficacy of drug used to treat common cause of kidney failure
July 20, 2018 - Timing of dinner associated with breast and prostate cancer risks
July 20, 2018 - Health Tip: Performing the Heimlich Maneuver
July 20, 2018 - Nearly all adolescents have eating, activity or weight-related issues
July 20, 2018 - Sage launches new web-based tool that helps explore curated genomic analyses of Alzheimer’s
July 20, 2018 - High-performance porous polymeric material for chromatography applications
July 20, 2018 - New molecule shows great promise for future treatment of many cancers
July 20, 2018 - New research project investigates alternative treatments for eye infections
July 20, 2018 - Immune T cells are built to react as fast as possible, shows study
July 20, 2018 - ZHX2 protein could offer a new treatment strategy for kidney cancer
July 20, 2018 - EKF’s Quo-Lab POC HbA1c analyzer meets international quality targets for diabetes testing
July 20, 2018 - Health burdens of very high risk drinking are potentially large, study reveals
July 20, 2018 - Using miniature drug-filled nanocarriers to target headaches and tumors
July 20, 2018 - Researchers uncover cause for progression of prostate cancer to incurable stage
July 20, 2018 - Studies highlight issues regarding black lung, opioid overdose, police violence and more
July 20, 2018 - AbbVie submits supplemental NDA to FDA for venetoclax to treat acute myeloid leukemia
July 20, 2018 - Researchers are one step closer to developing eye drops to treat age-related macular degeneration (AMD)
July 20, 2018 - Patients maintain muscle mass five years after surgically induced weight loss
July 20, 2018 - AMSBIO introduces new, powerful CRISPR/Cas9 gene editing kits
July 20, 2018 - PureTech Health collaborates with Roche to advance oral administration of antisense oligonucleotides
July 20, 2018 - Analysis reveals disparities in cancer death rates among minority groups
July 20, 2018 - Dr Maddy Parsons receives Royal Microscopical Society Life Science Medal
July 20, 2018 - Study finds link between DNA methylation and non-alcoholic fatty liver disease
July 20, 2018 - Military personnel with head trauma and football players with suspected CTE show similar brain changes
July 20, 2018 - Vidac Pharma Announces Initiation of Phase 2b Clinical Trial of VDA-1102 Ointment in Patients with Actinic Keratosis
First-of-its-kind cancer drug effective in 93% of pediatric patients

First-of-its-kind cancer drug effective in 93% of pediatric patients

image_pdfDownload PDFimage_print

A first-of-its-kind drug targeting a fused gene found in many types of cancer was effective in 93 percent of pediatric patients tested, researchers at UT Southwestern’s Simmons Cancer Center announced.

Most cancer drugs are targeted to specific organs or locations in the body. Larotrectinib is the first cancer drug to receive FDA breakthrough therapy designation for patients with a specific fusion of two genes in the cancer cell, no matter what cancer type. The research appears in The Lancet Oncology.

“In some cancers, a part of the TRK gene has become attached to another gene, which is called a fusion. When this occurs, it leads to the TRK gene being turned on when it’s not supposed to be and that causes the cells to grow uncontrollably. What’s unique about the drug is it is very selective; it only blocks TRKreceptors,” said lead author Dr. Ted Laetsch, Assistant Professor of Pediatrics and with the Harold C. Simmons Comprehensive Cancer Center.​

Larotrectinib, targets TRK fusions, which can occur in many types of cancer. While the TRK fusions occur in only a small percentage of common adult cancers, they occur frequently in some rare pediatric cancers, such as infantile fibrosarcoma, cellular congenital mesoblastic nephroma, and papillary thyroid cancer, said Dr. Laetsch, who leads the Experimental Therapeutics Program (ETP) in the Pauline Allen Gill Center for Cancer and Blood Disorders at Children’s Health in Dallas.

“Every patient with a TRK fusion-positive solid tumor treated on this study had their tumor shrink. The nearly universal response rate seen with larotrectinib is unprecedented,” Dr. Laetsch said.

Among them was 13-year-old Briana Ayala of El Paso, who aspires to a career in fashion design. In 2016, Briana was found to have a rare tumor in her abdomen wrapped around her aorta, the largest artery in the body.

Surgeons in her hometown said it would be too dangerous to operate, so her family brought Briana to Children’s Health in Dallas, where UT Southwestern Professor of Surgery Dr. Stephen Megison had to remove portions of her aorta while removing most of the tumor.

But the cancer started to grow again and no further treatments were available.

Dr. Laetsch sent her tumor for genetic testing and found that Briana’s cancer had the TRK fusion, meaning the new drug might help.

Briana enrolled in the phase 1 clinical trial of larotrectinib and began taking the drug twice a day. Within weeks her pain and the swelling in her abdomen diminished, and scans showed her tumors had shrunk significantly.

Nearly two years later, Briana is back in school and playing with her dog, Goofy, and the family’s seven parakeets. She’s also been able to pick up her sketch pad and her dreams of a New York City fashion career.

“These are the kind of amazing responses we’ve seen with larotrectinib,” said Dr. Laetsch, “and this is why I’m so excited about it.”

The results of the larotrectinib trial in adult patients – a 75 percent response rate – were published last month in the New England Journal of Medicine.

The TRK-fusion mutation can be present in many types of cancers, including lung, colon, thyroid, and breast cancer, as well as certain pediatric tumors. TRK, short for tropomyosin receptor kinase, is a gene that plays a key role in brain and nervous system development and has a limited role in nervous system functions such as regulating pain in later life.

Larotrectinib belongs to a class of molecules known as kinase inhibitors, which work by cutting back on the enzymatic activity of a key cellular reaction. The selectivity of the drug means it does not cause the severe side effects associated with many traditional cancer treatments, and none of the patients with TRK fusions had to quit the study because of a drug-induced side effect.

Equally important, the response was long-lasting for most patients. “For some of the targeted drugs in the past, many patients responded initially, but then resistance developed quickly. To date, the response to this drug seems to be durable in most patients,” said Dr. Laetsch, who investigates the use of tumor molecular profiling to guide therapy in UT Southwestern’s Pediatric Hematology and Oncology Division.

A next step in the research is a clinical trial involving a similar drug for those patients who developed resistance. Dr. Laetsch will be the national leader for that clinical trial in children.

Source:

http://www.utsouthwestern.edu/newsroom/articles/year-2018/pediatric-cancer-drug.html

Tagged with:

About author

Related Articles