Remembering Harold R. Roberts, MD
Harold R. Roberts, MD, a leader in the field of thrombosis and hemostasis and two-time American Society of Hematology (ASH) award recipient, passed away on September 9, 2017, at age 87. He was formerly the Sarah Graham Keenan Professor of Medicine and Pathology and Laboratory Medicine at the University of North Carolina (UNC) School of Medicine, where he founded the UNC Center for Thrombosis and Hemostasis in 1978 and served as director for 20 years.
Dr. Roberts received multiple honors from ASH, including the Henry Stratton Medal in 2001 for outstanding contributions to hematology and the Mentor Award in 2007. These awards recognize a long career of basic science research – in 1965, Dr. Roberts co-developed the first highly purified concentrate of blood clotting factor VIII, which was later commercialized by Baxter-Hyland – and of supporting the development of young scientists interested in hematology.
His other awards include the French International Prize for Research in Hemophilia, the Kenneth Brinkhous Award for Excellence in Clinical Research from the National Hemophilia Foundation, and both the Distinguished Career Award for Contributions to Hemostasis and the Grant Medal from the International Society on Thrombosis and Haemostasis (ISTH).
He is survived by two sons and two grandchildren. Dr. Roberts was preceded in death by his wife, Marilyn, earlier this year.
Sources: University of North Carolina School of Medicine news release; International Society on Thrombosis and Haemostasis news release, September 12, 2017.
Remembering Tessa Holyoake, PhD
Tessa Holyoake, PhD, a world-renowned expert in chronic myeloid leukemia (CML) and a member of ASH, passed away on August 30, 2017, at 54 years old. She was a professor of experimental hematology at the Institute of Cancer Sciences at the University of Glasgow and director of the Paul O'Gorman Leukaemia Research Centre.
Dr. Holyoake was the first to identify the existence of cancer stem cells in CML in 1999 during a research fellowship in Vancouver, Canada. She later demonstrated the resistance of those stem cells to CML-specific therapies such as imatinib and worked to identify key CML stem cell survival pathways.
Her accolades include the Scottish Health Awards Cancer Care Award in 2009, the Lord Provost of Glasgow Health Award in 2011, the Scottish Alba Saltire Society Fletcher of Saltoun Award in 2015, and the Rowley Prize by the International CML Foundation in 2017. She became a fellow of the Academy of Medical Sciences in 2013.
Dr. Holyoake is survived by her husband, mother, sister, and brother.
Source: University of Glasgow news release, August 31, 2017.
CDC Awards LSU $15 Million to Reduce Cancer Burden in Louisiana
The Centers for Disease Control and Prevention awarded a $15 million grant to be distributed over five years to the Louisiana State University (LSU) Health New Orleans School of Public Health to support its cancer registry, as well as programs in cancer education, early detection, and comprehensive control.
The grant will help fund the LSU Health New Orleans' Comprehensive Cancer Control Program, which focuses on preventable cancers. A portion of the grant will support the Louisiana Tumor Registry – a statewide, population-based cancer registry that collects detailed information about patients to guide policies for cancer prevention, early detection, diagnosis, treatment, prognosis, and survivorship with the aim of reducing the state's cancer burden and disparities in cancer care and survival.
"While Louisiana has one of the highest death rates from cancer in the country, the cancer control programs at the LSU Health New Orleans School of Public Health are working across the state to help Louisianans prevent cancers," said Donna Williams, DrPH, director of Louisiana Cancer Prevention and Control Programs and associate dean for public health practice and community engagement at the LSU School of Public Health.
Source: Louisiana State University press release, August 8, 2017.
PCORI Provides $18 Million to Fund Two Studies on Sickle Cell Disease
The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors announced $18 million in funding to support two sickle cell disease (SCD) studies being conducted at the Carolinas Medical Center and the Children's Hospital of Philadelphia. Each group of researchers will evaluate ways to manage the transition of patients with SCD from pediatric to adult care.
Researchers at Carolinas Medical Center will receive $9.8 million to study whether the use of peer support and educational programming could help improve outcomes related to emergency department visits and hospital admissions for young adults with SCD. Researchers at the Children's Hospital of Philadelphia and Northwell Health Cohen Children's Medical Center will receive $8.5 million to compare three approaches for improving quality of life for young patients with SCD as they transition to adulthood: engagement with community health workers, mobile apps to help manage their condition, and enhanced usual care.
"The good news is that young people with this serious disease are more likely than ever to live well into adulthood," said PCORI Executive Director Joe Selby, MD, MPH. "The comparative clinical-effectiveness research that PCORI funds can give these patients, their families, and clinicians the information they need to make better-informed decisions about their care options."
Source: Patient-Centered Outcomes Research Institute news release, September 12, 2017.
FDA Provides Funding to Support Development of PRTX-100 for ITP
The U.S. Food and Drug Administration's (FDA) Office of Orphan Products Development awarded a $403,000 grant to Protalex, Inc., to support future clinical development of PRTX-100 as a treatment option for immune thrombocytopenia (ITP). PRTX-100 is a highly purified form of staphylococcal protein A (an immunomodulatory protein known to modify aspects of the human immune system) that is administered as a short intravenous infusion.
Two phase I/II dose-escalation studies are enrolling patients with ITP in the U.S. and Europe to assess outcomes with PRTX-100.
The FDA's Orphan Products Clinical Trials Grants Program encourages the clinical development of new drugs for rare diseases or conditions for which no other therapy exists or for which the candidate drug will be superior to an existing therapy. PRTX-100 previously received orphan-drug designation for ITP in the U.S. and Europe.
Source: Protalex, Inc., press release, August 17, 2017.
Jackson Laboratory Researcher Awarded More Than $2 Million to Study the Role of RNA Structure in Gene Regulation
With the help of a $2,059,618 grant provided over five years from the National Institute of General Medical Sciences, Zhengqing Ouyang, PhD, an assistant professor at the Jackson Laboratory in Farmington, Connecticut, will build a research program to characterize the roles of RNA structure in post-transcriptional gene regulation.
Dr. Ouyang said his goal "is to provide a precise genomic blueprint for clinical diagnoses and prognoses, as well as develop new and more effective therapies for cancer and other diseases."
His laboratory has developed computational tools to reveal chromatin conformation, RNA structure, and regulatory networks. With the research funding, the group plans to establish a new path to understanding genome-wide regulation of gene expression.
Source: Jackson Laboratory press release, August 21, 2017.
UC San Diego Researchers Receive Funding to Study ROR1 Inhibitor in B-Cell Malignancies
Researchers from the University of California (UC) San Diego School of Medicine received an $18.2 million grant from the Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine (CIRM) to support a phase Ib/IIa clinical trial of cirmtuzumab (an anti-ROR1 antibody) for the treatment of B-cell malignancies.
Thomas Kipps, MD, PhD, professor of medicine and deputy director of research at the UC San Diego Moores Cancer Center, and colleagues will examine the safety and efficacy of cirmtuzumab plus ibrutinib. The new agent was developed in Dr. Kipps' laboratory through funding from CIRM's HALT leukemia grant, which was awarded to Dennis Carson, MD, principal investigator, and Catriona Jamieson, MD, PhD, deputy director of the Sanford Stem Cell Clinical Center and director of stem cell research at Moores Cancer Center.
Source: University of California San Diego press release, August 24, 2017.
Michael Pulsipher Receives NHLBI Funding to Study Severe Aplastic Anemia
Michael Pulsipher, MD, of the Children's Center for Cancer and Blood Diseases at Children's Hospital Los Angeles (CHLA), has received a $1.3 million grant from the National Heart, Lung, and Blood Institute to fund a pilot study that will compare outcomes between young patients with newly diagnosed severe aplastic anemia (SAA) who received either unrelated-donor blood and marrow transplantation or immunosuppressive therapy. The study is a joint effort between the Pediatric Blood and Marrow Transplantation Consortium at CHLA and the North American Pediatric Aplastic Anemia Consortium.
Dr. Pulsipher noted that immunosuppressive therapy can lead to a response in 70 to 80 percent of patients, but nearly one-third of patients do not respond and require transplant, usually from an unrelated donor. "If the trial shows that unrelated-donor transplant is better than immune suppression, as preliminary data suggest, it will lead to better long-term survival, changing the current standard of care for young patients with SAA," he said.
Source: Children's Hospital Los Angeles press release, September 6, 2017.
The Leukemia & Lymphoma Society Pledges to Help Patients Affected by Recent Hurricanes
As part of a hurricane-relief program, The Leukemia & Lymphoma Society (LLS) announced that it will commit up to $1 million to help patients with cancer who were affected by Hurricanes Harvey, Irma, and Maria. Patients with a confirmed blood cancer diagnosis who live in declared disaster areas are eligible to receive $500 to help with transportation to treatment, replacement of lost or damaged medications, and other needs directly related to the hurricanes.
"Living with a blood cancer is extremely challenging; a national disaster adds a new level of danger and stress," said Louis J. DeGennaro, PhD, president and chief executive officer of LLS.
Visit lls.org/hurricane-relief-program for more information on eligibility.
Source: The Leukemia & Lymphoma Society news release, September 15, 2017.
Five Researchers Awarded Albany Medical Center Prize for Gene-Editing Tool
The Albany Medical Center announced the winners of its annual medicine and biomedical research prize. This year's awardees (pictured below) were recognized for their roles in the creation of the CRISPR-Cas9 gene-editing system.
The $500,000 prize has been awarded annually since 2001 to scientists who have altered the course of medical research. Past recipients include eight Nobel Laureates. Announcing the award, Vincent Verdile, MD, dean of Albany Medical College, noted, "Rarely has such a recent discovery transformed an entire field of research, as CRISPR has in biological research. Its implications for biological processes, including human health and disease, are promising and quite profound."
Emmanuelle Charpentier, PhD, Max Planck Institute for Infection Biology, Germany 
Jennifer Doudna, PhD, University of California, Berkeley 
Luciano Marraffini, PhD, Rockefeller University, New York City 
Francisco J.M. Mojica, PhD, University of Alicante, Spain 
Feng Zhang, PhD, Massachusetts Institute of Technology, Cambridge
