UAMS Researcher Awarded $1.75 Million Grant to Study New Therapies for Metastatic Melanoma

Alan Tackett, Ph.D., was awarded a National Cancer Institute grant of $1.75 million to support his ongoing research into new therapies for metatatic melanoma.

Alan Tackett, Ph.D., was awarded a National Cancer Institute grant of $1.75 million to support his ongoing research into new therapies for metatatic melanoma.

| LITTLE ROCK — Alan Tackett, Ph.D., a cancer researcher at the University of Arkansas for Medical Sciences (UAMS), has received a five-year $1.75 million grant from the National Cancer Institute (NCI) to identify new tumor targets in the treatment of metastatic melanoma.

Tackett is a professor in the UAMS College of Medicine Department of Biochemistry and Molecular Biology and serves as associate director of basic research in the UAMS Winthrop P. Rockefeller Cancer Institute.

Although melanoma is less common than other forms of skin cancer, it is the most deadly form of the disease. About 96,480 Americans – including 760 Arkansans – are estimated to be diagnosed with melanoma of the skin in 2019. While not all of these cases will metastasize, or move to other areas of the body, for those that do, treatment options are limited and often unsuccessful.

In recent years, a type of immunotherapy known as immune checkpoint inhibition has shown unprecedented success in the treatment of metastatic melanoma. Immunotherapy harnesses the body’s natural immune system to seek and destroy cancer.

However, while immunotherapies, such as the drugs Keytruda and Opdivo, are successful for many patients, others fail to receive the same positive outcome.

“While these new therapies show great promise for many people, approximately half of metastatic melanoma patients do not respond to immune checkpoint inhibitors. My laboratory is focused on understanding why some patients do not respond to these immunotherapies, so we can use that information to turn these patients into responders” said Tackett, who holds the Scharlau Family Endowed Chair for Cancer Research at UAMS.

To accomplish this task, Tackett uses a sophisticated approach called proteomics that allows his team to measure molecular changes in melanoma cells at the atomic level.

In 2016, Tackett received funding from the National Institutes of Health to implement a National Resource for Proteomics at UAMS, which provides a biomarker discovery platform to researchers at UAMS, as well as those in 23 other states and Puerto Rico.

“By defining pathways active in certain types of melanomas, we can identify new targets for drug development that could increase responsiveness to immune checkpoint inhibitors and thereby save the lives of thousands of patients each year,” said Tackett, whose research has been highly funded by the National Institutes of Health for his entire career.

To accomplish its research, Tackett’s team works closely with UAMS surgeons, oncologists and pathologists to obtain fresh tumor samples removed during surgery at UAMS Medical Center.

“Getting fresh tissue out of surgery and into our lab very quickly is key. This gives us the ability to sort it into individual cell populations and analyze it in ways that are difficult to do with older, fixated tissue samples,” he said.

New patients at the UAMS Winthrop P. Rockefeller Cancer Institute are asked in the early stages of treatment about their interest in donating a portion of their surgically removed tumor, or other specimen such as blood or urine, to benefit research.

The procurement of donated tumor tissue does not require any type of additional procedure, but makes use of a portion of the tissue that was removed during the normal surgical process.

“The research environment at UAMS is ideal for these types of studies as basic scientists work seamlessly with clinicians to move research from the lab to the clinic as quickly as possible,” said Tackett.

This federal grant will bolster the Cancer Institute’s ongoing efforts to receive National Cancer Institute Designation, which requires the institution achieve at least $20 million in annual direct cost research funding from an approved list of funding agencies.

To achieve designation, cancer centers undergo a highly competitive assessment process that demonstrates an outstanding depth and breadth of research in three areas: basic laboratory, patient/clinical and population-based. The designation brings with it many benefits, including expanded access to federal funding for researchers and improved access to clinical trials for patients.


UAMS is the state's only health sciences university, with colleges of Medicine, Nursing, Pharmacy, Health Professions and Public Health; a graduate school; hospital; a main campus in Little Rock; a Northwest Arkansas regional campus in Fayetteville; a statewide network of regional campuses; and seven institutes: the Winthrop P. Rockefeller Cancer Institute, Jackson T. Stephens Spine & Neurosciences Institute, Harvey & Bernice Jones Eye Institute, Psychiatric Research Institute, Donald W. Reynolds Institute on Aging, Translational Research Institute and Institute for Digital Health & Innovation. It is the only adult Level 1 trauma center in the state. UAMS has 2,727 students, 870 medical residents and five dental residents. It is the state's largest public employer with more than 10,000 employees, including 1,200 physicians who provide care to patients at UAMS, its regional campuses, Arkansas Childrens Hospital, the VA Medical Center and Baptist Health. Visit www.uams.edu or www.uamshealth.com. Find us on Facebook, Twitter, YouTube or Instagram.

###


June 14, 2019

Kevin Raney, Ph.D.

Kevin Raney, Ph.D.

Professor and Chair Department of Biochemistry and Molecular Biology UAMS College of Medicine Research Interest Statement The Raney laboratory is interested in the mechanisms and functions of nucleic acid enzymes called helicases. Helicases play central roles in all aspects of DNA and RNA metabolism; therefore they are key enzymes in maintaining genomic stability. Genetic mutations…


UAMS-Developed Cytophone Detects Melanoma in Earliest Stages, Could Prevent Fatal Disease Spread

Vladimir Zharov, Ph.D., Ds.C. (center) is pictured with collaborators (left to right) Laura Hutchins, M.D.; James Y. Suen, M.D.; Ekaterina I. Galanzha, M.D., Ph.D., D.Sc.; Eric Siegel, M.S.; Issam Makhoul, M.D.; Azemat Jamshidi-Parsian, M.S.; Mustafa Sarimollaoglu, Ph.D; and Aayire C. Yadem, M.S.

Vladimir Zharov, Ph.D., Ds.C. (center) is pictured with collaborators (left to right) Laura Hutchins, M.D.; James Y. Suen, M.D.; Ekaterina I. Galanzha, M.D., Ph.D., D.Sc.; Eric Siegel, M.S.; Issam Makhoul, M.D.; Azemat Jamshidi-Parsian, M.S.; Mustafa Sarimollaoglu, Ph.D; and Aayire C. Yadem, M.S.

| LITTLE ROCK — A University of Arkansas for Medical Sciences (UAMS) research team led by Vladimir Zharov, Ph.D., D.Sc., has demonstrated the ability to detect and kill circulating tumor cells (CTCs) in the blood using a noninvasive device called Cytophone that integrates a laser, ultrasound and phone technologies.

This device is 1,000 times more sensitive than other methods at detecting of CTCs in the blood of patients with melanoma, a deadly form of skin cancer.

The Cytophone also has shown the ability to detect CTCs even when the tumor is not identifiable on the skin, either because too small (known as the T0 or TX stage) or after surgical removal, and then to destroy them without harming surrounding blood cells.

The team’s findings titled “In Vivo Liquid Biopsy using Cytophone Platform for Photoacoustic Detection of Circulating Tumor Cells in Melanoma Patients” were published in the June 12 issue of Science Translational Medicine, a prestigious scientific journal published by the American Association for the Advancement of Science.

“The only methods that are available to detect CTCs are mainly based on drawing blood from the patient. An average blood sample taken from a patient consists of only a few milliliters, which may or may not contain any CTCs. In contrast, the Cytophone can monitor a person’s entire five-liter blood supply, potentially locating every CTC in it. No needle is used, and no blood is removed,” said Zharov, a professor in the UAMS College of Medicine Department of Otolarynology-Head and Neck Surgery.

The portable Cytophone platform is based on a principle called in vivo photoacoustic flow cytometry, a technology that uses laser pulses to penetrate through intact skin and into blood vessels to monitor circulating abnormal cells and other disease-associated biomarkers. The published research describes the first demonstration of noninvasive detection of CTCs directly in the bloodstream of melanoma patients. When a melanoma CTC passes the laser beam into the vessels, the laser pulses heat the natural melanin nanoparticles in these cells. The fast thermal expansion of these nanoparticles then generates a sound that is detected using an ultrasound transducer attached to the skin.

The research team demonstrated that the advanced software with fast signal processing algorithms makes Cytophone data tolerant to skin pigmentation and motion that led to  the identification of CTCs in 96% of the patients in between 10 seconds and 60 minutes without generating false positives in the controls at the established thresholds and current detection limit of five CTCs in five liters of blood.

Researchers found that the device not only discovered CTCs in advanced stage patients, but also revealed the presence of CTCs in the patients with early Stage 2 disease.

“With the Cytophone, we can listen to the laser-triggering sound from each individual cell in the body. CTCs could be one of the best early metastasis markers, because obviously only viable CTCs can create deadly spread of the disease,” said Zharov, who also serves as director of the Arkansas Nanomedicine Center and holds the Josephine T. McGill Chair in Cancer Research at UAMS.

This technology also has demonstrated the ability to destroy the detected CTCs, resulting in a large drop in CTC numbers and preventing spread of the disease to other parts of the body, known as metastasis.

Thus, the Cytophone may be able to serve as a theranostic platform by integrating both diagnostic and therapeutic capabilities using the same laser to detect and kill the cancer cells right in the bloodstream.

The Zharov team also demonstrated the Cytophone performance for the identification of cancer-related blood clots, which is the second leading cause of death among cancer patients.

“We are developing the robust, easy-to-use portable and wearable Cytophone versions with advanced small lasers, which will be available for cancer clinics across the country to start a multi-center clinical trial involving more melanoma patients. Our goal is to determine whether Cytophone-based early diagnosis combined with destroying CTCs is effective as a stand-alone treatment or in combination with conventional therapies in preventing or at least inhibiting metastasis,” said Zharov.

Zharov’s team first also demonstrated the ability to detect non-pigmented CTCs by injecting a cocktail of magnetic and gold nanoparticles with a special biological coating into the bloodstream. Breast cancer-related clinical trials are in progress that takes into account successful preclinical trials using this technology previously published in the journal Nature Nanotechnology.

Other applications for the Cytophone in label-free mode could include detection of sickle cells to prevent sickle cell crisis, detection of clots to prevent stroke, and selection of the most effective drug through monitoring of circulating disease-associated markers count decrease.

Zharov’s research is supported by the National Institutes of Health, including an R01 grant from the National Cancer Institute, and the National Science Foundation, among others.

Collaborators on the research were Ekaterina I. Galanzha, M.D., Ph.D., D.Sc.; Yulian A. Menyaev, Ph.D.; Aayire C. Yadem, M.S.; Mustafa Sarimollaoglu, Ph.D.; Mazen A. Juratli, M.D., Ph.D.; Dmitry A. Nedosekin, Ph.D.; Stephen R. Foster, M.D.; Azemat Jamshidi-Parsian, M.S.; Eric R. Siegel, M.S., Issam Makhoul, M.D., Laura Hutchins, M.D.; and James Y. Suen, M.D. Additional UAMS clinic team members who supported this trial include N. Harris and A. Trammel.


UAMS is the state's only health sciences university, with colleges of Medicine, Nursing, Pharmacy, Health Professions and Public Health; a graduate school; hospital; a main campus in Little Rock; a Northwest Arkansas regional campus in Fayetteville; a statewide network of regional campuses; and seven institutes: the Winthrop P. Rockefeller Cancer Institute, Jackson T. Stephens Spine & Neurosciences Institute, Harvey & Bernice Jones Eye Institute, Psychiatric Research Institute, Donald W. Reynolds Institute on Aging, Translational Research Institute and Institute for Digital Health & Innovation. It is the only adult Level 1 trauma center in the state. UAMS has 2,727 students, 870 medical residents and five dental residents. It is the state's largest public employer with more than 10,000 employees, including 1,200 physicians who provide care to patients at UAMS, its regional campuses, Arkansas Childrens Hospital, the VA Medical Center and Baptist Health. Visit www.uams.edu or www.uamshealth.com. Find us on Facebook, Twitter, YouTube or Instagram.

###


June 11, 2019

Maurizio Zangari, M.D.

zangari

Professor Department of Hematology Oncology UAMS College of Medicine Director of Myeloma Bone Disease Research Research Interest Statement My research focuses on the clinical and preclinical aspects of bone disease. I first described the bone anabolic properties of proteasome inhibitors in myeloma patients and the effects on the PTH axis. Specifically, I investigated the effect…


Myeloma Patient in Remission, Grateful for Additional Time

Linda Reins of Tulsa, Oklahoma, and her husband Robert recently learned she is in complete remission.

Linda Reins of Tulsa, Oklahoma, and her husband Robert recently learned she is in complete remission. “Dr van Rhee has given me time and hope,” she said.

| Linda Reins, 73, of Tulsa, Oklahoma, is no stranger to cancer. In 1985, when she was 40, her local gynecologist’s office floored her with the first diagnosis.

“They told me, ‘You’re pregnant and oh, you also have cervical cancer,’ said Reins, who had two teenaged sons at the time. “The next day, my husband at the time said ‘I can’t handle this; I’m out of here’ and left me.”

Reins had a full hysterectomy shortly after her daughter, now 33, was born and she didn’t require any additional treatment.

She has since remarried, and she and husband Robert, an executive vice president with a bank, will celebrate their 20th anniversary this year.

Two years after her diagnosis of cervical cancer, Reins battled breast cancer, defeating it with a lumpectomy.

In early 2018, while being treated for a broken hip in Tulsa, a full-body CT scan revealed a lesion in the ball of her hip.

“The doctor told me ‘You have multiple myeloma and it is incurable,’” Reins said. “And I cried. We had never heard of this type of cancer.”

In retrospect, the location of the rare blood disease was a blessing.

“The cancer was contained to just the ball of my hip, they took it out and replaced it,” said Reins, who recently retired from the University of Tulsa as an administrative assistant following a long career with a private oil and gas exploration and production company.

Not long after her diagnosis, she discovered a friend of hers in Tulsa was a 14-year myeloma survivor of myeloma. They had dinner on a Friday evening and Reins’ friend, a patient of Frits van Rhee, M.D., Ph.D., urged her to seek treatment at the UAMS Myeloma Center. Her friend emailed Dr. van Rhee that night.

“The next day, which was a Saturday, Dr. van Rhee called and talked to me for at least 40 minutes,” Reins said during a recent checkup at UAMS.

Linda and her husband did a lot of research looking for the best place to have her cancer treated. UAMS with its long-term survival rate was at the top of her list. It is difficult to decide to leave home and be treated in another state, she said, but after speaking with Dr. Van Rhee, she decided UAMS is where she should be.)

She began seeing van Rhee, who diagnosed her myeloma as aggressive, high risk. She underwent two stem cell transplants, the latest one in February. She learned during her recent visit she is in remission.

“I am very grateful to Dr. van Rhee,” she said of the center’s clinical director. “He is remarkable and one of the smartest and caring people I know. No matter how busy the man is, I truly believe he looks at everything in each patient’s chart.”

She added that the protocols used at the center and the way treatment is structured are remarkable and lifesaving.

“If you really want to have a good chance of extending your life,  the place to go is the UAMS Myeloma Center,” she said.

The center does two tests that are unique to UAMS.

“One is the genome test and the other one shows what type of myeloma you have; whether it’s aggressive or regular,” Reins said. “Those are the two most important tests you can have.”

There were rough patches throughout her treatment. She contracted a rare fungal infection in her lungs following her second transplant and was treated by Mary Burgess, M.D., an infectious disease specialist and assistant professor in the Department of Internal Medicine in the College of Medicine.

“My husband and I are very thankful we had the opportunity to come to the UAMS Myeloma Center and are even more grateful to have Dr. van Rhee as my physician,” Reins said.

“My experience at UAMS was made much easier thanks to the professional team assisting Dr. van Rhee, the exemplary staff people working in Infusion 4, and the process used by the center in its treatment protocol.”

“I have never met anyone there who wasn’t kind or helpful; he has surrounded himself with wonderful and knowledgeable people.”

“I have been so blessed,” said Reins, today a grandmother of six. “And to hear the words ‘complete remission’ was really, really nice. Dr. van Rhee has given me time and hope.”


UAMS, Governor Celebrate Clinical Trials Day

Betty Fortner, a cancer survivor and clinical trial participant, briefly told her story to Gov. Asa Hutchinson and UAMS and ACRI research leaders during a photo opportunity for Clinical Trials Day.

Betty Fortner, a cancer survivor and clinical trial participant, briefly told her story to Gov. Asa Hutchinson and UAMS and ACRI research leaders during a photo opportunity for Clinical Trials Day.

| A celebration of Clinical Trials Day was a chance for Gov. Asa Hutchinson to shake hands with UAMS researchers and hear the story of Betty Fortner, a cancer survivor and clinical research advocate.

Appearing with Gov. Asa Hutchinson are (l-r), Pamela Christie, Kristin Zorn, M.D., Clare Nesmith, M.D., Betty Fortner, Laura James, M.D., Sherry Courtney, M.D., Hutchinson, Barry Brady, Amy Jo Jenkins, Thomas Burrow, M.D., David Avery, Rohit Dhall, M.D., and Stacie Jones, M.D.

Appearing with Gov. Asa Hutchinson are (l-r), Pamela Christie, Kristin Zorn, M.D., Clare Nesmith, M.D., Betty Fortner, Laura James, M.D., Sherry Courtney, M.D., Hutchinson, Barry Brady, Amy Jo Jenkins, Thomas Burrow, M.D., David Avery, Rohit Dhall, M.D., and Stacie Jones, M.D.

During a photo shoot at the state Capitol on May 20, Clinical Trials Day, Fortner briefly became the center of attention when Hutchinson asked about her story.

“I told him I spit in a cup, which started the process of me learning I had the BRCA mutation,” Fortner said.

Inherited mutations of the BRCA1 and BRCA2 genes increase the risk of female breast and ovarian cancers, as well as other cancers.

Hutchinson, who listened closely along with several research leaders from UAMS and Arkansas Children’s Research Institute (ACRI), asked about her children. She said her test results have led to genetic testing by other members of her family – some finding they also have the BRCA mutation.

Fortner, of Hot Springs, was invited to the Capitol event by her doctor, UAMS’ Kristin Zorn, M.D., director of the Division of Gynecologic Oncology in the UAMS College of Medicine.

Darri Scalzo, UAMS research compliance officer, and Laura James, M.D., director of the UAMS Translational Research Institute, help celebrate Clinical Trials Day.

Darri Scalzo, UAMS research compliance officer, and Laura James, M.D., director of the UAMS Translational Research Institute, help celebrate Clinical Trials Day.

The governor’s Clinical Trials Day proclamation was initiated by the UAMS Translational Research Institute, which helps researchers conduct clinical trials. The proclamation includes a brief history of Clinical Trials Day, which celebrates the first randomized clinical trial – the famous study in 1747 that determined citrus fruit could prevent scurvy.

Clinical Trials Day is celebrated around the world, recognizing clinical research professionals and volunteer participants for their contributions to the medical advances achieved through clinical trials.

John Kilgore, a research study participant, joined the Clinical Trials Day celebration.

John Kilgore, a research study participant, joined the Clinical Trials Day celebration.

UAMS’ celebration of Clinical Trials Day included information booths in its hospital lobby and Winthrop P. Rockefeller Cancer Institute lobby, both high-traffic public areas. Research staff from the Translational Research Institute, Cancer Clinical Trials and Research Affairs office and Office of Research Compliance handed out snacks, including citrus fruit, and provided information about clinical trials at UAMS. ACRI, a UAMS affiliate, sponsored a similar event.

UAMS Chancellor Cam Patterson, M.D., MBA, officially kicked off the Clinical Trials Day celebration in the hospital lobby, expressing his appreciation for researchers and the participants who make clinical trials possible.

Thanks to advances in genetic testing, Fortner was able to catch her ovarian cancer early and is a five-year survivor. She reads a lot of medical information, and her appreciation for clinical research has led her to volunteer for two clinical trials.

She was ineligible for one because she is in remission, but she was able to participate in a study on the benefits of eating healthier and exercising to prevent a recurrence of ovarian cancer.

“Clinical trials not only help those of us in remission, but also hopefully in the future when someone gets a diagnosis like this, there will be new ways to help treat it,” Fortner said.


May 30, 2019

Welcome Hayley Bierman

Bierman

Dear Patients, It is my pleasure to inform you that Hayley Bierman has accepted the position as an Advanced Nurse Practitioner in my clinic. Hayley has worked in the Winthrop P. Rockefeller Cancer Institute’s Infusion 4 clinic since 2012 providing care to our patients undergoing chemotherapy and stem cell transplantation.  Patients and caregivers can reach…


Undergrads Explore Cancer Research Careers at UAMS Workshop

INBRE workshop organizer Tom Kelly, Ph.D., gives instructions about the day's activities.

INBRE workshop organizer Tom Kelly, Ph.D., gives instructions about the day’s activities.

| As Hendrix College junior John Pablo-Kaiser considers his career options, cancer research is high on the list.

“We’ve made leaps and bounds in what we know about cancer, but there is so much left to learn. I really think of it like a new frontier,” he said.

A biochemistry for pre-med major, Pablo-Kaiser recently took advantage of the opportunity to explore the field of cancer research during a daylong workshop at UAMS sponsored by Arkansas INBRE (IDeA Networks of Biomedical Research Excellence).

Participants engage in a small-group discussion with thyroid cancer researcher Aime Franco, Ph.D.

Participants engage in a small-group discussion with thyroid cancer researcher Aime Franco, Ph.D.

The Arkansas INBRE program supports research in public and private four-year colleges across Arkansas by building research capacity and raising awareness about career opportunities in biomedical research. It is supported by the National Institutes of Health (NIH) Institutional Development Award (IDeA), which was established to broaden the geographic distribution of NIH funding for biomedical and behavioral research.

Lawrence Cornett, Ph.D., associate vice chancellor for research at UAMS, serves as principal investigator and director of Arkansas INBRE.

Conducted May 22 in the UAMS Library, the Bench to Bedside Cancer Research Workshop welcomed 15 Arkansas undergraduates interested in pursuing a career in medicine and/or biomedical research. The students, who ranged from incoming sophomores to recent graduates, represented eight colleges and universities in Arkansas and two out of state.

Catherine Peppers, a Conway resident and biology major at Smith College in Northampton, Massachusetts, chose to participate because of her interest in the medical field.

Antino Allen, Ph.D., discusses his radiation therapy research with workshop participants.

Antino Allen, Ph.D., discusses his radiation therapy research with workshop participants.

“My mom told me about the workshop and recommended it to me. I thought it would be a good way to learn more about the field,” said Peppers, who plans to attend medical school after graduation.

The first Arkansas INBRE-sponsored workshop, held in 2018, focused on opioid addiction and drew positive reviews from participants.

“The students who attended our first workshop thought it was a valuable experience. We decided to offer the workshop again with a focus on cancer research, giving students the chance to find out about different career paths available to them in the field,” said Cornett.

About 12 UAMS scientists engaged in clinical or basic cancer research took part in the interactive workshop by presenting case studies and participating in a panel discussion and round table conversations.

“This is your chance to interact with some of UAMS’ leading scientists and learn what it takes to become a cancer researcher,” workshop organizer Tom Kelly, Ph.D., told the students. Kelly is an associate professor in the UAMS College of Medicine Department of Pathology.

Topics of discussion included everything from the long-term effects of radiation and chemotherapy on the brain to the ways in which the body’s immune system can be used to fight melanoma and other cancers.

Students also had the opportunity to eat lunch with graduate students and learn about programs offered by the UAMS Graduate School.


May 23, 2019

Rangaswamy Govindarajan, M.D., M.R.C.P.(U.K.)

Rangaswamy Govindarajan, M.D., M.R.C.P.(U.K.)

Professor of Medicine Division of Hematology/Oncology Chief, Section of Hematology/Oncology, CAVHS Quality Officer, Cancer Service Line UAMS Winthrop P. Rockefeller Cancer Institute Clinical Research Interest Statement Dr. Govindarajan’s clinical interests are in the fields of gastrointestinal oncology and sarcoma. He treats patients with malignancy involving the entire GI tract, bone and soft tissue sarcomas. He…


Commentary by UAMS’ van Rhee Featured in Blood Journal

“Significant progress has been made in recent years in the field of multicentric Castleman disease,” Frits van Rhee wrote in a recent commentary in Blood, a publication by the American Society of Hematology, noting several advancements.

“Significant progress has been made in recent years in the field of multicentric Castleman disease,” Frits van Rhee wrote in a recent commentary in Blood, a publication by the American Society of Hematology, noting several advancements.

| Frits van Rhee, clinical director of the UAMS Myeloma Center, weighed in on a new combination therapy for treating newly diagnosed Castleman disease patients in the April 18 issue of Blood, a publication by the American Society of Hematology.

In the commentary, “Storming the Castle with TCP,” van Rhee and coauthor Katie Stone, then-director of the Immunotherapy Lab at the Myeloma Center, examined a report on the new treatment for newly diagnosed idiopathic multicentric Castleman disease (iMCD) patients.

Castleman  is a rare, incurable disease of lymph nodes that can cause fever, weight loss, fatigue and nausea.

The treatment used oral thalidomide, cyclophosphamide, and prednisone (TCP) to attack the disease through multiple mechanisms of action. It was reported by Lu Zhang and others in, “Phase 2 study using oral thalidomide-cyclophosphamide-prednisone for idiopathic multicentric Castleman disease.” Zhang wrote the TCP regimen showed promising results and safety and while more research is necessary, it is an important treatment option, particularly when siltuximab is unavailable.

“Significant progress has been made in recent years in the field of multicentric Castleman disease,” van Rhee wrote, noting several advancements. They include the establishment of the Castleman Disease Collaborative Network (CDCN), which classified MCD into distinct clinic-pathological entities followed by formal Castleman disease diagnostic and treatment guidelines, based on the severity of the disease, which spans a wide spectrum.

Treatment using siltuximab, while approved in both the United States and Europe, is not a cure, van Rhee wrote, and must be used lifelong because relapses have been reported when the therapy is stopped. It is also expensive and is not available in all parts of the world, he added.

Meanwhile, the new TCP regimen offers an all-oral and less cost-prohibitive treatment option in areas where access to siltumximab is limited.

“It is exciting to see a widely available, new treatment option for iMCD emerge; however many questions remain,” he wrote, adding that the progression of the rare, complex disease is still poorly understood.

“Ultimately, a thorough understanding of the disease is needed to develop more effective and better-targeted therapies for all iMCD patients,” van Rhee concluded.

Visit http://www.bloodjournal.org/content/133/16/1697 to read the article.

 

 


Next page