Alexandra Guadagno

The Food and Drug Administration (FDA)’s approval of Turalio (pexidartinib), a CSF1R inhibitor drug for patients with certain types of tenosynovial giant cell tumors, may provide patients who are ineligible for surgical resection with a significantly improved response and clinical benefit.

Tenosynovial giant cell tumors are a rare, locally aggressive neoplasm that is driven by an overexpression of colony-stimulating factor 1 (CSF1), a genetic anomaly which secretes a protein into the blood that controls various white blood cell functions and plays the important role of controlling immune and inflammatory processes. This genetic abnormality can attract inflammatory cells to that area in the joints, according to Dr. William D. Tap, lead investigator of the ENLIVEN study and chief of the Sarcoma Medical Oncology Service at Memorial Sloan Kettering Cancer Center in New York City.

“This is a disease that we have really only begun to truly understand within the last 10 years from a scientific standpoint, and from a clinical standpoint really within the last five to eight years,” said Tap. “Because of that, (tenosynovial giant cell tumor) has been very much neglected by the medical community in our understanding of it and how to approach the treatment of it. That is one of the areas of the greatest unmet medical need, as this condition has been affecting people for long periods of time but there haven’t been great medical treatments for it.”

The condition can occur anywhere in the body and be painful and debilitating for patients. The non-malignant tumor can occur in young or otherwise healthy people, and average presentation usually occurs in a person’s thirties or forties, Tap told 

®. Symptoms include pain, inflammation, stiffness, functional impairment, swelling, joint destruction, joint instability and decreased range of motion.

Tenosynovial giant cell tumor represents two distinct clinical presentations: localized (or nodular) and diffuse. Diffuse disease in tenosynovial giant cell tumors is highly irregular tumor shape and does not grow circumferentially, making it difficult to treat with surgery and often resulting in a difficult patient journey. A typical patient with diffuse disease is likely to need over 750 days off throughout their disease, intermittent physical therapy for years or decades and an arsenal of braces, compression socks, ice machines and other anti-inflammatory tools. Ninety percent of patients with tenosynovial giant cell tumors present with localized disease, according to research presented at the FDA’s May Oncologic Drugs Advisory Committee (ODAC), which voted 12 to 3 in favor of the agency approving pexidartinib to treat adult patients with tenosynovial giant cell tumors.

The primary treatment standard for tenosynovial giant cell tumors is surgical resection; however, those with diffuse disease — widely spread throughout the joint or joints – are not always amenable to surgery. Prior to the approval of pexidartinib, there were no FDA-approved systemic therapies for tenosynovial giant cell tumors.

“The diffuse type (of symptomatic tenosynovial giant cell tumors) can be very difficult because it can be very infiltrated throughout the joints and can often be very difficult from a surgical standpoint to truly remove all of the disease,” said Tap.

“Surgeons will say it’s almost like ‘operating in mud’ and patients may need to go through large procedures to try to remove as much as possible,” he added. “There is a percentage of the diffuse type that is cured with surgery alone, but we also know that there is a high relapse rate in the diffuse type of the TCGT. Depending on the literature it’s quoted anywhere from about 40% to 55% of the time that the disease will come back.”

In extreme cases, patients with the diffuse subtype of tenosynovial giant cell tumor may require more surgeries, which can result in morbidity of the joints and in some cases, amputation, according to Tap.

Turalio showed promising phase 1 activity in patients with tenosynovial giant cell tumors. When a CSF1 receptor (CSF1R) inhibitor such as Turalio is administered in patients with tenosynovial giant cell tumors, this can prevent CSF1R activation and CSF1R-mediated signaling in these cells. This can reduce inflammation, improve T-cell infiltration and antitumor T-cell immune responses to inhibit tumor cells from multiplying.

“The thought that this CSF1 gene is upregulated in this process really brought in the idea of targeting that with drugs such as (Turalio), which can be very strong inhibitors of the CSF1 receptor,” said Tap. “So, the thought is that it can block the recruitment of these inflammatory cells that actually have the receptor on their surface for this CSF1 chemoattractant.”

CSF1R inhibitors can help potentially slow down the process and improve patient symptoms and tumor mass, sometimes within weeks, he added.

The ODAC found that the drug’s “efficacy (is) clearly established by robust tumor response rate and clinically meaningful improvement in function and disease symptoms, as measured by clinical and patient reported outcomes.”

Turalio is indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumors associated with severe morbidity or functional limitations and unresponsive to treatment with surgery.

The FDA’s approval of Turalio was based on data from the phase 3 ENLIVEN trial — a global, double-blind, randomized, placebo-controlled study of Turalio in patients with advanced tenosynovial giant cell tumors, whose tumors were ineligible for surgical removal as it could cause more harm than benefit. Overall, 120 patients were analyzed in the ENLIVEN trial, 61 of which were given Turalio and 59 patients who were given a placebo.

Turalio resulted in significantly improved overall response rate (the percentage of patients with a reduction in tumor burden of a predetermined amount for a designated minimum time period) and clinical benefit in terms of functional outcomes. Compared with placebo, overall response rates by tumor volume score — which calculates tumor volume as percentage of the entire synovium (the soft tissue that lines the spaces between joints) – were 55.7% for patients who received Turalio vs 0% for those who received the placebo. The study showed improved range of motion by 15.1% with Turalio compared to 6.2% with placebo; meanwhile, worst stiffness reduced by 2.45% and .28%, respectively, and pain response improved by 31.1% and 15.3%. Thus, Turalio may offer a relevant treatment option for select patients with tenosynovial giant cell tumors.

Researchers observed less than 40% change in exposure in subjects with renal impairment and no effect of mild and moderate hepatic impairment upon exposure to the drug.

However, severe liver toxicity was observed in some patients: Two serious liver toxicity cases (one requiring a liver transplant and one associated with death) have been observed with Turalio across the non-tenosynovial giant cell tumors development program.

Liver toxicities were more frequent with Turalio. There were two cases of nonfatal, serious liver toxicity and eight patients discontinued Turalio due to hepatic side effects; four were serious, nonfatal side effects with increased bilirubin (an orange-yellow substance made during the normal breakdown of red blood cells, which travels through the liver and is eventually emitted out of the body), one lasting approximately seven months. Other, less severe side effects associated with Turalio included hair color changes, vomiting, fatigue, dysgeusia (a distortion or loss of taste) and periorbital edema (swelling around one or both eyes).

“This is really important for patients and prescribing clinicians to know because this is not something that should be used in every patient and really should be only considered in very specific clinical situations after discussion has been had about the potential of this liver toxicity, even though it’s very rare,” said Tap.

breaking news report on the FDA’s approval.

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The Food and Drug Administration’s approval of Turalio for tenosynovial giant cell tumors may offer significantly improved clinical benefit for select patients with these rare, benign tumors.

What Patients With Tenosynovial Giant Cell Tumors Should Know About Turalio

A routine dentist appointment led to a cancer diagnosis for tennis star Nicole Gibbs. 

The 26-year-old player for the Orange County Breakers of World TeamTennis went for teeth whitening in the spring. That’s when her new dentist discovered a small growth on the roof of her mouth, Gibbs said in an interview this week with ESPN.

“I mentioned this to my primary doctor at least five years ago; she told me it was nothing to worry about, a common bone growth that didn't seem to be growing rapidly or doing anything of concern,” Gibbs said. “My dentist at the time felt the same.” Gibbs also said she didn’t feel any pain or discomfort.

However, this dentist told her to get a biopsy, which she got the next day. The results revealed she had microcystic adnexal carcinoma, a rare tumor that occurs on the 

Gibbs had surgery to remove the tumor on May 17. Unfortunately, because of complications, she missed her chance to qualify for Wimbledon. Her recovery involved going home from the hospital with a feeding tube that remained in place for just over three weeks.

“Cancer was initially my nightmare, but it weirdly turned into a dream-come-true diagnosis because it made me confront so many things in my life,” Gibbs said to ESPN.

The former NCAA singles and doubles champion is currently ranked No. 137 in the world. Gibbs is now cancer-free and returned to competitive tennis in early July at a tournament in Hawaii. She told ESPN she will be playing in several tournaments over the next few weeks before going to New York for US Open qualifying in the middle of August.

Photo via Instagram, courtesy of Nicole Gibbs @gibbsyyyy

Kevin “Racks” Roster, Jr., a World Series of Poker Player, used the California medical aid-in-dying law to end his suffering from sarcoma, a rare cancer, on July 26.

 A New York native and resident of New Jersey, Roster dedicated his last weeks to educating the public about sarcoma and peaceful dying options for terminal patients. He originally planned to use the new New Jersey medical aid-in-dying law that took effect on Aug. 1, but his doctors told him that he probably wouldn’t live long enough to see the law passed.

The 36-year-old moved to California to access its End of Life Option Act — a law that permits terminally ill adult patients with the capacity to make informed medical decisions to be prescribed an aid-in-dying medication. He died peacefully in his sleep shortly after taking the medication, surrounded by his wife, caretaker and best friend, at his apartment in Rancho Cordova, according to a press release.

After receiving the sarcoma diagnosis in 2017, Kevin dedicated his final days to raising awareness about the disease and the End of Life Option Act through his blog, Twitter, YouTube and by playing semi-professional poker while wearing sarcoma-education gear.

He also had partnered with Compassion & Choices, which is an organization intended to “improve care, expand options and empower everyone to chart their end-of-life journey,” to convince lawmakers in his home state of New York — and across the United States &mdash; to authorize medical aid in dying as an option for adults that are mentally capable and terminally ill to peacefully end their painful suffering.

He told everyone he had stage 4 terminal brain cancer, but it was all a lie. 

For years John Looker had kept up the charade of claiming to have 

 — the same cancer that took the life of 

. He became a “cancer celebrity” and star fundraiser for Pelotonia, a charity event in Ohio that raises millions of dollars for cancer research at the James Cancer Hospital and Solove Research Institute. He was revealed to  be a fraud after suspicious members realized his symptoms were not lining up with his alleged diagnosis.

Ohio Attorney General Dave Yost announced on July 29 a settlement with Looker, who was accused of misrepresenting a cancer diagnosis to solicit and then misuse charitable donations for a cancer organization and scholarship fund. Under the settlement, Looker agreed to pay $1,800 in restitution and a $2,000 civil fine. Looker has also agreed to not hold any position with a charitable organization in Ohio.

On July 31, Pelotonia discussed its new regulations in light of the situation, which will require participants to sign a release guaranteeing their statements are true. 

“John was at one time a beloved member of the Pelotonia community. In Pelotonia’s earliest days, he told people he was fighting brain cancer and this new organization had absolutely no reason to disbelieve him then,” stated the release. “He invested fully in an elaborate persona that fooled all of us for years, including his friends, medical professionals, the media and those closest to him. He would get sick on training rides and attribute it to side effects of chemotherapy. He would ask friends to drive him to treatment appointments. When someone was newly diagnosed, he would reach out to them and share his story. And through it all, he appeared to have a positive outlook which inspired those around him.”

An example of a 2016 social media post by Looker soliciting donations. Photo credit: Instagram via 

The cancer and dancing communities have rallied around Dorian Gramajo of Virginia — a Norfolk business owner, dance teacher and competitor &mdash; to show their support after he received a diagnosis of 

Some of that support has been exhibited through the GoFundMe page set up for the Gramajo

family. Within months of learning about his cancer, hundreds of people had donated almost $40,000.

Gramajo and his wife, Tracy, are currently at Duke University Hospital in North Carolina. Tracy Gramajo said that her husband began experiencing back pain in March, which they initially thought was just an injury from dancing but turned out to be cancer.

“It’s been life changing for us very quickly,” Tracy told WTKR News Channel

3. “He has had internal bleeding and external bleeding, just been through the ringer in the hospital. He has had transfusions and dialysis and all kinds of crazy stuff that you never would have imagined he have to go through.” The family is moved by the community’s support. “He is really touched; he gets really emotional about it,” added Tracy Gramajo. “It's definitely something that is helping him pull through this.”

While Dorian Gramajo is most appreciative of the financial donations and emotional support, he is still in need of a bone marrow donor. According to WTKR, there is no one currently in the 

A new contest will launch on UK Channel 4 as celebrities attempt to swim the English Channel to raise money for Stand Up to Cancer (SU2C). 

“Sink or Swim” will feature celebrities who either can’t swim or don’t swim well as they  make their best attempt at navigating the English Channel.

Although the air date for the reality show has yet to be released, it will feature a host of celebrities and non-swimming athletes, according to 

The stars will not be without guidance as they attempt to face their fears and push themselves past their limits; they will be coached by Olympic silver medalist Keri-Anne Payne, adventurer Ross Edgley (the first person to swim the whole way around Great Britain) and Paralympic champion Ellie Simmonds.

In each episode of “Sink or Swim,” the celebrities will participate in a qualifying swim-off to move on to the next level. In addition to raising money for SU2C, Channel 4 hopes the challenge will inspire “non-swimmers of any age to take to the water at a time when one in five British adults cannot swim.”

From today’s top performers and athletes to medical aid in dying, here’s what is making headlines in the cancer space this week.

Friday Frontline: Cancer Updates, Research and Education on August 2, 2019

In conjunction with World Lung Cancer Day — recognized on Aug. 1 – 

 has highlighted a variety of facts that can help patients assess risk for the disease.

The earlier lung cancer is detected, the better chance a person has of surviving the disease. Knowing the risk factors and statistics will help you to understand how you can minimize your possibility of developing the disease.

Lung cancer is the second most common cancer for both men and women in the United States, according to statistics reported by the American Lung Association (ALA). However, worldwide, lung cancer is the most common cancer, with approximately 2.09 million new diagnoses each year, and is responsible for the most cancer deaths, at around 1.76 million, according to the World Health Organization.

Tobacco is the leading risk factor for developing lung cancer, accounting for approximately 80% of lung cancer deaths. “While smoking tobacco is the leading risk factor for lung cancer, it is not the only one,” advised 

. “A family history of lung cancer and exposure to certain carcinogens also increase the likelihood of developing the disease.”

Some of the other risk factors are out of our control, such as race and/or gender. Women and black people worldwide are subject to a higher rate of mortality from lung cancer. “While the age-adjusted death rate for lung cancer is higher for men (46.7 per 100,000 persons) than for women (31.9 per 100,000 persons),” according to the ALA, “it is similar for blacks (40.0 per 100,000 persons) and whites (39.2 per 100,000 persons) overall. However, black men have a far higher age-adjusted lung cancer death rate as compared to white men, while black and white women have similar rates.” This could be due to disparities in access to care, as mortality rates in the poorest counties were found to be 40% higher for male lung and liver cancers during 2012 to 2016.

Lung cancer death rates declined 48% from 1990 to 2016 among men and 23% from 2002 to 2016 among women. The mortality rates among women are only projected to increase. In a 2018 study published in 

, researchers found that lung cancer mortality rates among women are 

World Lung Cancer Day 2019: Facts & Figures

The United States Senate passed legislation this Tuesday, voting 97-2 to permanently fund the 9/11 Victim Compensation Fund (VCF).

 The VCF covers health care costs of first responders, volunteers and survivors who were hurt in the attacks and were expected to run out of money by the year 2020. The legislation would extend funding to 2092. It was a bipartisan effort and President Donald J. Trump is expected to sign.

Senate Minority Leader Chuck Schumer, a New York Democrat, said, "Righteousness sometimes — sometimes – in this mangled town sometimes prevails."

Onlookers in the Senate Gallery included former “Daily Show” host Jon Stewart — a huge advocate for continued support of the VCF – and first responders that were affected by the attacks that took place on Sept. 11, 2001. Stewart and other onlookers stood and applauded when the vote surpassed the required threshold. Several senators, including New York Democrat Senator Kristen Gillibrand, who helped lead the endeavor, joined in the ovation from the Senate floor, reported 

Gillibrand said afterwards at a news conference, "Today is not a celebration. It’s a deep sigh of relief."

Gillibrand introduced Stewart as a “hero.” After a few jokes about the title, Stewart echoed her sentiments about the legislation passing. "This has been the honor of my life,” he said. “We can never repay all that the 9/11 community has done for our country. Today, they can exhale... There've been too many funerals. Too many hospices. These families deserve better.”

According to the US Centers for Disease Control and Prevention (CDC), an estimated 400,000 people are believed to have been exposed to toxic contaminants, suffered injury or trauma in Manhattan during the attacks.

A Northern California federal judge, U.S. District Judge Vince Chhabria, cut down a verdict tying Roundup weed killer to cancer by $55 million.

 The company faces thousands of similar claims in the United States.

California resident Edwin Hardeman was awarded more than $80 million in a March jury verdict for his case associating his 

 to regular use of Roundup on his residential properties. Judge Chhabria said that the $75 million in punitive damages awarded to Hardeman by the six-person jury was excessive, compared with the $5.3 million in other damages, reported the 

. Judge Chhabria said that $20 million in punitive damages — approximately four times the compensatory damages awarded to Hardeman by the jury – was more suitable under U.S. Supreme Court guidelines.

Hardeman’s trial was the second of three trials in which an individual has been awarded a settlement linking Roundup to cancer. Bayer said on Monday that Judge Chhabria’s action in reducing the damages is a step in the right direction. Bayer still plans to appeal the verdict, as the jury’s decision opposes the scientific evidence and conclusions made by many worldwide health officials and regulators regarding the safety of Roundup.

 survivor Darla Jaye was so excited about her last day of treatment at Harris Health System in Houston, Texas that she broke the "cancer free" bell while ringing it so powerfully.

"I couldn't even fathom being done, I was so overwhelmed yesterday," she said to 

. "Guess I didn't know how strong I was!"

Jaye, who said she first received her diagnosis of breast cancer in February and had a fast-growing tumor. She originally thought she would have to endure chemotherapy, but her treatment plan ended up being 30 rounds of radiation. After her final session this past week, she excitedly broke the “cancer-free” bell in celebration.

"Even though I kind of knew what was coming, you don't really know when you lay down on a radiation bed getting ready for treatment," she said. "I am very, very grateful,” added Jaye, who credits her doctors at Harris Health System for saving her life.

“The now-widespread tradition was introduced in 1996 at MD Anderson,” says the MD Anderson website, “to symbolize the restoration of balance, harmony and life energy.”

A recent study emphasized the need for custom-tailored skin cancer prevention programs to protect a wider, more diverse population. 

the goal of more comprehensive skin cancer prevention programs is to universally reduce incidences of new skin cancer occurrences.

"These findings highlight the importance of tailoring free skin cancer screening events for non-white and younger populations," said Dr. Adam Friedman, interim chair of the Department of Dermatology at the GW School of Medicine and Health Sciences and director of the Supportive Oncodermatology Clinic at the GW Cancer Center and senior author on the study. "While free screening events are important, we also have to think about comprehensive, community-based solutions that reach broader demographic populations than skin cancer screenings alone."

 and highlights the need for widespread, consistent skin cancer prevention guidelines. For example, the sun safety practices advocated for attendees at various skin cancer screening events differ from the general public, according to findings published by researchers from the George Washington University (GW) Cancer Center.

"This study also highlights the importance of reaching non-white populations with skin cancer prevention messages," added Friedman. "We have to address the myth that skin cancer only affects fair-skinned individuals. Skin cancer does not discriminate and therefore we need to encourage sun safety practices among all individuals."

A group of Miami children are doing their part to help fundraise for breast cancer research by setting up a pink lemonade stand and encouraging others to do the same, according to 

Brynne Rhodes’ mother was battling breast cancer in 2016 and wanted to do something to help find a cure for her mother. She took it upon herself and set up a pink lemonade stand to raise money for the Breast Cancer Research Foundation. Now, Rhodes has issued a challenge for her peers, requesting kids all over the United States do the same during this summer and take on the “Pink Lemonade Stand Challenge.”

A group of children in Miami have accepted her challenge, including Blaire Vazquez, age 6, and her book club are preparing to sell a whole lot of pink lemonade for a cause that hits particularly close to home for Vazquez.

“Cancer is out everywhere, my aunt has cancer, we want to end cancer,” said Blaire Vazquez.

Rosemary Carrera — Vazquez’s aunt – said, “I was diagnosed after my first mammogram when I turned 40.  I had no symptoms, no family history and it progressed quickly.” Carrera says her sister (Blaire Vazquez’s mother), Natalie Vazquez, has been a big support for her during her cancer journey and is her biggest champion.

“It definitely takes a village to go through it and we’re doing it,” said Natalie Vazquez. She has also been helping her daughter and friends with their lemonade stand signs, providing aprons and other supplies to help them set up their stand and take on the challenge.

The children will be at the Publix Supermarket across from the Miami Falls Shopping Center today, June 26, at 6:30 p.m., selling pink lemonade for breast cancer. All of the money they raise will go to the Breast Cancer Research Foundation.

If you are interested in getting your child involved, visit their website, pinklemonadestandchallenge.com.

Photo credit: @pinklemonadestandchallenge via Instagram.

From landmark Senate legislation to the Pink Lemonade Stand Challenge, here’s what is making headlines in the cancer space this week.

Alexandra Guadagno

What Patients With Tenosynovial Giant Cell Tumors Should Know About Turalio

Friday Frontline: Cancer Updates, Research and Education on August 2, 2019

World Lung Cancer Day 2019: Facts & Figures

Friday Frontline: Cancer Updates, Research and Education on July 26, 2019

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