Category Archives: Medical/Research

Children’s Cancer Is Unprofitable and Ignored

An estimated 2,000 children die of cancer each year, and the overall incidence of childhood cancer has been slowly increasing since 1975. Despite significant advances against certain pediatric cancers, including acute lymphoblastic leukemia, there are still some types of cancer for which there are few or no effective treatments. As John London found out, new drug development in the field is slow, often lagging way behind adult treatments, and few compounds are designed specifically for children. “I was on my own, as many parents are,” London says. “The medical community had no interest.”

That is in large part due to a practical reason: Childhood cancers make up less than 1 percent of all cancers diagnosed each year, according to the American Cancer Society. That 1 percent is not much of a market for drugmakers, who rack up an estimated $1.4 billion in out-of-pocket costs while bringing a novel drug to market. They would never recoup that treating the 700 children diagnosed with neuroblastoma annually, or the 100 diagnosed with diffuse intrinsic pontine glioma, a deadly brain tumor.

“The big elephant in the room is the cost of this type of research,” says Raphaël Rousseau, director of pediatric oncology drug development at pharmaceutical giant Roche. Combined with the small potential market, that’s led very few pharmaceutical companies to invest in developing drugs for pediatric cancer. Merck has one ongoing pediatric oncology trial. Pfizer is testing preclinical therapies only. Novartis leads the pack, with seven drugs in clinical trials for children’s cancer.

Where Big Pharma is absent, government has stepped in. Most pediatric clinical trials are operated by the National Cancer Institute’s (NCI) Children’s Oncology Group (COG), which runs approximately 40 to 50 therapeutic trials across the country at any one time, according to Peter Adamson, chairman of the organization and a pediatric oncologist at the Children’s Hospital of Philadelphia. Yet even with federal funding, pediatric cancer research receives only a fraction of the money that adult cancer research gets, and it’s decreasing. In 2013, the NCI invested $185.1 million from a $4.79 billion budget in pediatric cancer research, the lowest amount since 2009.

“The options we have now to be explored are really blossoming, but the funds available to do the studies that need to be done are shrinking,” says Richard O’Reilly, chairman of pediatric oncology at Memorial Sloan Kettering Cancer Center, in Manhattan. ‘We don’t want future generations to look back on this time and ask, ‘What the hell were they doing?’”

Read more…

Tim Duncan donates $247,000 to fund ground-breaking San Antonio Cancer Genome Research Project

San Antonio Spur, Tim Duncan, through the Tim Duncan Foundation and Blackjack Speed Shop, has made a landmark donation of $247,000 to the San Antonio 1000 Cancer Genome Project, a Texas not-for-profit 501 (c) (3), Open Science research project launched in 2012 by START. Based in San Antonio, START’s mission is to accelerate the development of new anticancer drugs through Phase I research. With four clinic sites on three continents (the US, Spain and China), START is the largest provider of Phase I testing and treatment for patients with advanced cancer. START’s Phase I researchers can uniquely claim direct, hands-on involvement with the clinical development of 18 FDA approved anti-cancer drugs that are now in use by oncologists around the world.

The San Antonio 1000 Cancer Genome Project is a one-of-a-kind cancer genome research project that has theunprecedented support of more than 200 surgeons, oncologists, and cancer researchers from a broad spectrum of affiliations. This unparalleled collaboration is enabling researchers to step beyond the institutional barriers that currently fragment and piecemeal cancer research, and makes the San Antonio 1000 Cancer Genome Project the largest community-wide undertaking of its kind.

Through the support and involvement of these participating surgeons, START researchers are collecting fresh tumor tissue from 1000 patients with the 10 most common cancers in San Antonio. The project then aims to perform whole genome sequencing on both the cancerous and normal tissue from each patient. Then, for the first time anywhere, researchers will link this genetic information to the patient’s clinical outcomes. Most significantly, all data will be made available publicly at no cost to researchers worldwide. The project’s promise to make all data freely available is built on the belief that no single investigator or institution has all of the answers and encourages the involvement of outsiders who might bring fresh ideas to the urgent and complex problem of gene abnormalities and cancer.

Like most people, Tim Duncan has been touched personally by cancer, and hopes for a cure for cancer in his lifetime. Duncan says he was drawn to the San Antonio 1000 Cancer Genome Project, “because the information collected will ultimately be shared.”

“The idea that a group of cancer researchers were willing to set aside egos, to make the information freely available and work strictly for the greater good of curing cancer was exactly the type of cancer project I was willing to support,” Duncan said.

Read More…

Lymphoseek Approved for All Solid-Tumor Cancers

Approval of the Lymphoseek system for detecting sentinel lymph nodes has been extended to cover all solid-tumor cancers, its manufacturer said Wednesday.

The FDA is also permitting the radiolabeled tracer system to also now be used with or without lymphoscintigraphy, according to Navidea Biopharmaceuticals.

Previously, Lymphoseek had been approved in conjunction with melanomas, breast cancers, and head and neck tumors.

The product uses a technetium-99 labeled tracer to identify lymph nodes serving areas near primary tumors, allowing oncologists to select for excision and analysis those nodes most likely to harbor emigrating cancer cells. The tracer is called tilmanocept, and it binds to CD206 receptors in lymph nodes.

Navidea said the expanded approval was based on data collected in the company’s melanoma, breast cancer, and head-and-neck cancer trials. “An integrated analysis of data from all three studies showed positive diagnostic performance of Lymphoseek across the solid tumor types studied,” the firm said in announcing the new approval.

The FDA has requested a postmarketing study to be performed in pediatric cancer patients, to be completed by 2018, the firm added.

Source

Fighting Cancer By Fixing Our Cells’ Hard Drives

It’s pretty much standard practice in cancer research to search for genes that could serve as leads for new tumor-fighting drugs. But Cigall Kadoch, a 29-year-old assistant professor of pediatric oncology at the Dana-Farber Cancer Institute (yes, that means she’s already on the tenure track at Harvard) is following a new lead for anti-cancer medicines: exploring the genes that regulate the molecular structure of DNA itself.

Kadoch will be on stage at the Forbes Under 30 Summit on October 21, as part of a panel on the future of medicine, called Tomorrow’s Medicine Today.

A quick review of basic biochemistry: DNA is a molecular ladder in which a code made of molecules called bases (adenine, thymine, cytosine, and guanine or A, T, G, C) create recipes for all of the proteins that make up every part of the body, as well as instructions for when to make them.

But as with a computer’s hard drive, the code isn’t all that matters. The physical structure that reads the code and translates it for the body matter, too.

This DNA-reading structure, called chromatin, is constantly being modified as a way of determining what bits of DNA code get read. And the chromatin remodeling complex, which makes these modifications, is itself controlled by genes written into the DNA.

Kadoch, as a graduate student at Stanford, found that genes related to a chromatin remodeling complex called BAF, were, when mutated, the cause of a rare pediatric cancer: synovial sarcoma, usually found around major joints. Approximately 800 new cases occur in the U.S. each year.

Now, she has a 12-person team in her Boston lab looking for new drugs targeted at BAF, which might help not only kids with synovial sarcoma but also other patients. Kadoch guesses that 25% of cancers might be caused in part by BAF-related mutations. “It’s an amazing thing going from leading your own efforts to leading with a whole group behind you,” she says.

Original Article

Doctors Helping Kids Fight Cancer

Dr Andrew Pendleton makes his rounds. The Pediatric Cancer Specialist at Memorial Health says for these kids, its not just about the medicine, but their attitude.

“The most important thing is hope,” explains Dr Andrew Pendleton. “The 4 letter word hope. All the majority of patients will be cured depending on the type of cancer, but there is always reason for hope.”

Hope, and good medicine, is what helps keep a smile on these youngsters faces. No easy task considering what they are going through. But Pendleton says kids actually make better patients than adults.

“I think its easier for me to work with them because they are pure, they didn’t cause it, whatever we do will be beneficial,” said Dr Pendleton.

“Children do not complain about things,” said Dr Pendleton. “They never lived through life and know what normal life is. To them this is normal life, they adjust, they adapt, they thrive.”

“People ask me all the time how can you do this Doctor?” explains Pendleton. “How can you not do this? The patients, the children didn’t deserve anything they get. They go through life, not just the steps of life, eating breathing, going through the day. They live life, they play when they feel good they smile, they bring life to us, they bring a joy to us.”

Joy, and a cure. That’s what Dr Pendleton and everyone here want to bring to these families.

“Hope. All the reason to hope. No reason not to hope for a better future,” said Pendleton.

Despite the success rate and growing number of children with cancer, the national funding has been cut almost 30% in the past few years.

Read More…

Boston doctor in forefront of pediatric cancer research

Dr. Charles Roberts has a photograph of Mary Eisnor hanging in his office so he never forgets what he’s fighting for.

Mary, whose family lives in Bridgewater, died in 2010 at the age of 8 of hepatoblastoma, a form of liver cancer.

Roberts, an associate professor in the department of pediatrics at Harvard Medical School and in pediatric oncology at Dana-Farber Cancer Institute, attended Mary’s funeral.

“Every time we lose a patient to cancer it makes us want to go back and work that much harder,” Roberts said.

Roberts spends about 80 percent of his time running a research lab and 20 percent taking care of the kids who stand to benefit from that research.

But we are only just beginning to understand how the human body works at the genetic level, Roberts said.

“It’s an incredibly complex choreography,” he said.

Roberts compared cancer to typos in a book – only the stakes are much higher.

“The mistakes that happen in genes are causing kids to die,” Roberts said.

Roberts said childhood cancer research could absolutely benefit from more funding.

Pharmaceutical companies generally don’t focus on childhood cancer because there is not a large return on their investment, he said. That is due to the fact that – fortunately – childhood cancer is relatively rare, Roberts said.

Research companies also tend to be a little reticent to test drugs on children, so drugs tend to be tested on adults first, but the people developing treatment protocols for children are pediatric oncologists familiar with the unique needs of young patients, Roberts said.

Read More…

‘Dream come true’: Tumor Paint BLZ-100 gets go-ahead for first U.S. trial

A new protein-linked dye derived from scorpion venom that lights up cancer cells so surgeons can precisely target brain tumors will get a trial run in the U.S., Blaze Bioscience Inc. officials announced Thursday.

Food and Drug Administration officials have approved an investigational new drug application, or IND, for Tumor Paint BLZ-100, a molecule discovered and first developed by researchers at Fred Hutchinson Cancer Research Center, Seattle Children’s Hospital and the University of Washington.

“I think it really is a dream come true – to be outdone only by seeing these cancers light up in patients,” said Dr. Jim Olson, a Fred Hutch pediatric brain cancer expert who pioneered the notion of targeting tumors with fluorescent dye to help surgeons distinguish healthy cells from malignancies.

Read More…

Is a Phase I Clinical Trial Right for You?

You’re probably familiar with the term “clinical trial.” And if you or someone you care about is a patient at Memorial Sloan Kettering, it’s even more likely you’ve heard the phrase before, since these studies have played an integral role in the fight against cancer.

By definition, a clinical trial is a research study that tests a new medical approach in a group of people to make sure it is safe and effective. All of the drugs used to treat people with cancer today were developed through a series of carefully constructed clinical trials.

We conduct one of the largest clinical research programs in the world. In 2013, our physicians led more than 1,200 clinical research studies for adult and pediatric cancers.

“We learn from all of these studies, which build upon each other and ultimately lead to new discoveries and improved treatments for patients,” says medical oncologist Paul Sabbatini, Deputy Physician-in-Chief for Clinical Research at MSK.

The first step in testing a new drug in humans is called a phase I trial. These studies offer eligible patients the chance to try an innovative treatment that in most cases has some preclinical data suggesting it might have activity against a particular type of cancer.

In this Q&A, Dr. Sabbatini sheds light on how phase I clinical trials are conducted at MSK and how to determine whether patients may be eligible for one. Stay tuned to our blog next month for a second post, in which he will demystify common misconceptions surrounding clinical trials.

Read Q&A…

Fight for compassionate use reform goes to Washington

A proposed new law could make it easier for terminally ill patients to get access to potentially life-saving drugs. The process, known as expanded access, or compassionate use, was a topic at this year’s annual Childhood Cancer Summit in Washington, D.C., Friday.

The summit is the yearly meeting of the Childhood Cancer Caucus, co-founded in 2009 by Central Texas Congressman Michael McCaul, in order to better direct Congressional resources and efforts to the cause of preventing pediatric cancer.

McCaul was also a friend of Austin attorney Andrea Sloan, 45, who waged a brave, public battle for compassionate use in 2013 while in the final stages of ovarian cancer. After months of requests, an anonymous drug company ultimately granted Sloan compassionate use of a drug, but her disease had progressed quickly and she passed away on Jan. 1, 2014.

McCaul said he is ready to introduce new legislation in Congress in Sloan’s honor that would keep other patients from having to wait so long for their requests to be granted—time they do not have.

Read More…