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FDA Recommends Including Patients 12-18 in Adults Clinical Trials

Posted: December 31, 2016 - 1:33pm

Unfortunately, fibrolamellar falls in the nether world where it does not qualify for funding or regulation for pediatric diseases or adult diseases. Clinical trials for adults start at age 18 and some clinical trials for fibrolamellar have been held up because too many eligible patients were under 18. The FDA has just recommended for the first time that sponsors of clinical trials include patients in the 12-18 age bracket for appropriate diseases. The recommendation was published in Clinical Cancer Research: Enrolling Adolescents in Disease/Target-Appropriate Adult Oncology Clinical Trials of Investigational Agents. Here's the abstract:

"The enrollment of adolescents with cancer in clinical trials is much lower than that of younger pediatric patients. For adolescents with "adult-type" cancers, lack of access to relevant trials is cited as one of the reasons for this discrepancy. Adolescents are generally not eligible for enrollment in adult oncology trials, and initial pediatric trials for many drugs are conducted years later, often after the drug is approved. As a result, accrual of adolescents to these trials may be slow due to off-label use, prospectively collected safety and efficacy data are lacking at the time of initial approval, and, most importantly, these adolescents have delayed access to effective therapies. To facilitate earlier access to investigational and approved drugs for adolescent patients with cancer, and because drug exposure is most often similar in adolescents and adults, we recommend the inclusion of adolescents (ages 12-17) in disease- and target-appropriate adult oncology trials. This approach requires careful monitoring for any differential safety signals, appropriate pharmacokinetic evaluations, and ensuring that ethical requirements are met. Inclusion of adolescents in adult oncology trials will require the cooperation of investigators, cooperative groups, industry, institutional review boards, and regulatory agencies to overcome real and perceived barriers". Clin Cancer Res; 23(1); 1-4. ©2016 AACR

We are pleased to be able to give readers of our website this link to the entire article here, which is not available elsewhere on line. 

FibroRegistry at the White House

Posted: December 15, 2016 - 1:06pm

This past week the US House and Senate passed the 21st Century Cures Act. This provides $5 billion for the NIH for The Precision Medicine Initiative, Brain Initiative and the Moonshot on Cancer - a legislation established earlier this year aiming to make therapies more available and improving early stage prevention. Its emphasis was given to pediatric, adolescent and young adult cancers, including fibrolamellar.

On Tuesday, Elana Simon, a fibrolamellar patient/researcher and member of the Fibrolamellar Registry board, went to the White House to stand on stage as President Obama signed the 21 Century Cures Act. We are grateful for this important step forward towards hope and action!

White House Recognizes New York Teen's Cancer Research

Posted: May 12, 2015 - 7:57pm


By Amy Golod | Staff Writer May 12, 2015, at 12:01 a.m.

At the end of January, Elana Simon, a 19-year-old Harvard University freshman, was invited to the White House without knowing why. On Jan. 29, the day before President Barack Obama announced his Precision Medicine Initiative, much to Simon’s surprise, she was asked to write an introductory speech for the president’s remarks.

Simon was diagnosed with fibrolamellar hepatocellular carcinoma, a rare liver cancer, at age 12 and underwent surgery to remove the tumor. She is now in remission. Her diagnosis led her to initiate a study of this disease, which currently has no effective treatment option aside from surgery.

Elana Simon researched a rare liver cancer; she was diagnosed with it at age 12 and is now in remission.
Elana Simon, 19
Freshman, Harvard University
From New York, N.Y.

She and a team of scientists at Rockefeller University, Memorial Sloan Kettering Cancer Center and New York Genome Center identified a genetic mutation that may be the cause of fibrolamellar, which affects about 200 adolescents and young adults annually worldwide. The February 2014 issue of Science magazine published the findings.

Simon’s focus on a specific group among liver cancer patients demonstrates the precision medicine model. Obama’s initiative aims to look at people’s genes, environments and lifestyles to develop data-driven, targeted medical treatments for diseases such as cancer.

“Because the research I did was a good example of the benefits of precision medicine and [I was] approaching research from such a specific, personalized [way] … they thought it would be appropriate for me to speak about it,” Simon states.

As the White House guests settled into their seats and Simon waited for the program to begin, she had a brief, impromptu one-on-one conversation with President Obama. “It wasn’t a big media event, it was just the two of us talking, which was very exciting.”

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Simon had met President Obama at the 2014 White House Science Fair where she presented her research to him. Last year, she was the inaugural winner of the American Association for Cancer Research’s Young Champion in Cancer Research Award and a Davidson Institute Fellow.

The road to Simon’s discovery began during her sophomore year of high school at the Dalton School in New York City. With the help of her science teacher, Simon found an internship at a Mount Sinai School of Medicine lab where she was exposed to genetic sequencing while looking at data from pancreatic cancer patient samples. She learned that as people age, gene mutations develop over time. Parsing them and determining which are relevant to the cancer research is a challenge, she notes.

“The more data you have, the more power you have, and the more conclusive the discovery can be.”  — Elana Simon

Simon thought that studying fibrolamellar would be a perfect candidate for genetic sequencing because it generally afflicts young patients whose DNA has not developed as many mutations as older patients’ due to age.

She said that she and her father, who heads a laboratory at Rockefeller, always talk about science and he encouraged her to start the research. She brought up the idea to her surgeon, Dr. Michael La Quaglia, chief of the pediatric surgical service at Memorial Sloan Kettering Cancer Center. When she asked if the project would be informative, he offered to help her obtain fibrolamellar samples. Dr. La Quaglia also assigned fellows to the research, and Simon states that they became mentors.

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Even though Simon was connected to a top surgeon, she said it was difficult to procure samples. She created a YouTube video seeking patient participation, thinking this appeal might be more fruitful than contacting hospitals, many of which may be protective, competitive or unwilling to share samples. “The more data you have, the more power you have, and the more conclusive the discovery can be,” she points out. In addition, she notes patients could feel that they were helping with the research.

“I think this is how the Precision Medicine Initiative is hopefully going to be successful and change people’s attitudes toward data and sharing samples,” Simon says. “Collaborating with others is so much more powerful than working on your own.”

She started the work at Rockefeller, which commissioned New York Genome Center to produce the gene sequencing on 15 patients’ liver tumors and adjacent healthy liver samples.By the summer following Simon’s junior year, the data was ready for analysis.

Simon explains that parts of two genes typically far apart fuse together to create the abnormality. All of the tumor samples revealed the presence of this mutation, while the healthy tissue did not, leading to the possibility that it is a cause of the disease. The research at Rockefeller is ongoing, and Simon intends to resume work at the lab this summer.

In the meantime, she is pursuing computer science studies at Harvard. Simon recognizes the power of a computer in her research; she says a human alone could not have parsed the same volume of data. “Computer science is an important tool in medicine,” she adds.