Sickle Cell Disease and Voxelotor

According to results of phase 3 study, Voxelotor increased hemoglobin levels and reduced markers of hemolysis among sickle cell disease patients. Although two medications approved by the FDA are available (hydroxyurea and L-glutamine [Endari, Emmaus Life Sciences]), chronic medical complications and early death in persons with sickle cell disease remain a substantial burden. In particular, chronic organ dysfunction has become a leading cause of death in adults with sickle cell disease in the United States.

Voxelotor (GBT440, Global Blood Therapeutics) — a deoxygenated sickle hemoglobin polymerization inhibitor — reversibly binds with hemoglobin to help stabilize the oxygenated hemoglobin state. In a previous phase 1/phase 2 trial, voxelotor demonstrated favorable pharmacokinetics and dose-dependent increases in affinity of hemoglobin and oxygen with low-grade toxic effects.

For the current phase 3, international, multicenter, double-blind HOPE trial,  274 patients with sickle cell disease in a 1:1:1 ratio were assigned to receive once-daily voxelotor at a dose of 1,500 mg (n = 90; median age, 24 years; range, 12-59) or 900 mg (n = 92; median age, 24 years; range, 12-59), or placebo (n = 92; median age, 28 years; range, 12-64).

Most of the patients were black (n = 183) and 159 were female. A majority in each group had homozygous hemoglobin S and had experienced at least two vaso-occlusive crises within the past month. About two-thirds were receiving hydroxyurea.

The percentage of patients who demonstrated an increase in hemoglobin level of more than 1 g/dL at 24 weeks served as the study’s primary endpoint. Change in hemoglobin level from baseline to week 24, markers associated with hemolysis, and the incidence rate of vaso-occlusive crises served as secondary endpoints.

Median follow-up was 42.3 weeks (range, 0.1-73.3) in the 1,500-mg voxelotor group, 38.1 weeks (range, 4-72.4) in the 900-mg voxelotor group, and 37.2 weeks (range, 8.1-72.9) in the placebo arm.

Results of the intention-to-treat analysis showed hemoglobin response at 24 weeks among a significantly higher percentage of patients in the 1,500-mg voxelotor group (51%; 95% CI, 41-61) than the placebo group (7%; 95% CI, 1-12). One-third of patients (33%; 95% CI, 23-42) in the 900-mg voxelotor group responded to the treatment by week 24.

Researchers observed a higher percentage of responses among patients in the 1,500-mg voxelotor group than the placebo group regardless of concurrent hydroxyurea use or anemia severity at baseline.

Adjusted mean change in hemoglobin level from baseline to week 24 in the intention-to-treat analysis was 1.1 g/dL (95% CI, 0.9-1.4) in the 1,500-mg voxelotor group, 0.6 g/dL (95% CI, 0.3-0.8) in the 900-mg dose group and –0.1 g/dL (95% CI, –0.3 to 0.2) in the placebo group.

Fewer patients in the 1,500-mg and 900-mg groups experienced worsening anemia between baseline and 24 weeks than in the placebo group. Additionally, the 1,500-mg group demonstrated significantly greater reductions in indirect bilirubin levels and percentage of reticulocytes than the placebo group.

Grade 3 or higher adverse events occurred among 26% of patients in the 1,500-mg and placebo groups and 23% of patients in the 900-mg group. Investigators determined most adverse events were unrelated to treatment.

The absence of an increased incidence rate of vaso-occlusive crisis with voxelotor despite significant increases in the hemoglobin level suggests that voxelotor raises hemoglobin levels without negatively affecting blood viscosity. Longer-term follow-up is needed to further characterize the effect of voxelotor on the incidence of vaso-occlusive crisis.

The hemoglobin response and reduction in hemolysis observed with an orally administered, once-daily medication with side effects that minimally affect lifestyle may make voxelotor a promising advancement in the management of sickle cell disease if approved by the FDA.

Explore more about hematology at World Hematology 2019 at Rome, Italy, this July. Grab Grab 25% off on your registrations until June 25, 2019. To avail offer, Please do drop us an email at: worldhematology@pulsusglobal.com | worldhematology@pulsusmeet.com

Source: HemOnc Source

Ph+ALL

Philadelphia Chromosome positive acute lymphoblastic leukemia (Ph+ALL) is a rare subtype of the most common childhood cancer, acute lymphoblastic leukemia (ALL). Like ALL, Ph+ ALL is a cancer of a type of white blood cell called lymphocytes. It has a well-known mutation in its genetic code that fuses two genes together (the BCR and ABL genes) that do not normally fuse together. This BCR-ABL gene, also known as the Philadelphia Chromosome, can cause the white blood cells to become cancerous. While this Philadelphia chromosome is rare in pediatric ALL, it is much more common in adult ALL. It’s also the main cause of another much more common adult leukemia called chronic myelogenous leukemia (CML).

The signs and symptoms of Ph+ ALL are the same as standard ALL, which include: enlargement of the liver or spleen, enlarged lymph nodes, paleness, fevers, bruising, weight loss, bone pain, and abnormal blood cell counts. These symptoms are nonspecific and can occur in more common viral illnesses, as well. 

Historically, pediatric Ph+ ALL patients had been very difficult to cure with standard chemotherapy. Survival rates were only around 30 percent, compared to survival rates of most pediatric ALL patients of more than 85 percent. It was not until recently, when a new class of drugs that directly target the Philadelphia Chromosome were developed, that that survival rates doubled to about 70 percent.

These drugs are called tyrosine kinase inhibitors. The most well-known drug in this class is called imatinib (or Gleevec). Using imatinib, in combination with chemotherapy, is now the recognized standard-of-care treatment for kids with Ph+ ALL. In fact, the next large US clinical trial that will attempt to improve the outcomes in children with Ph+ ALL will use imatinib in combination with chemotherapy.

Research is going into developing new types of drugs to directly target the Philadelphia Chromosome. Other research is also focused on trying to use alternative therapies to cure those 30 percent of patients who don’t respond to treatment with imatinib (or similar drugs in its class).

Explore more about Hematology this July at Rome, Italy. For more information, PS: WORLD HEMATOLOGY 2019

Source: https://www.stbaldricks.org/blog/post/what-is-philadelphia-chromosome-positive-all

Platelet Decoys

According to pre- clinical findings,  A novel antiplatelet therapy showed potential in reducing blood clot risk and preventing cancer metastasis.

The therapy involves modification of human platelets to create “decoys” that can bind to certain cells but will not carry out other normal platelet functions, such as those associated with clotting.

The reversibility and immediate onset of action are major advantages of these ‘platelet decoys,’ and it is envisioned to be useful as a potential antimetastatic therapy and periprocedural antithrombotic agent.

Explore more such interesting works in hematology at Rome, Italy, this July. To know more, PS: http://bit.ly/2xaW3zT

Source: https://www.healio.com/hematology-oncology/hematology/news/online/%7Bd060c4cd-40d2-4b79-b36d-f00075fd81d8%7D/platelet-decoys-may-lower-blood-clot-risk-prevent-cancer-metastasis

World Hematology 2019

World Hematology 2019 welcomes the oncologists, haematologists, immunologists, pathologists, research scholars, industrial professionals and student delegates from biomedical and healthcare sectors to be a part of it.

It has many interactive scientific sessions on different types of Blood disorders and Cancers as well as various diagnostic and therapeutic advancements in this field. It is an initiative to unite the diverse research and business communities working in this field under one roof to explore every single aspect of Oncology and Hematology. 

The conference is focussed on the following topics and sessions:

• Advancements in Hematopoietic cell transplantation
• Anticipating the development of Paediatric Hematology/ Oncology
• Recent trends in diagnosing and treating Hematology/ Oncology indications
• Emerging trends in Cancer Care
• Therapeutic advancements and many more….

Explore hematology, oncology with the experts in the field in world’s leading international conference on hematology and oncology, PS: World Hematology 2019

High-dose irradiation before HSCT and risk for malignant neoplasms

Study results shows that, survivors of allogeneic hematopoietic cell transplantation who underwent high-dose total body irradiation incurred nearly 8 times the risk for subsequent malignant neoplasms compared to general population.

Results showed the association to be strongly dose- and fractionation-dependent, and total body irradiation doses for nonmyeloablative conditioning posed no increased risk above that of myeloablative chemotherapy-only based regimens.

“The big takeaway is that people need to be aware of this risk,” Scott Baker, MD, director of the Fred Hutch survivorship program at Fred Hutchinson Cancer Research Center, said in a press release. “For those patients who received high-dose radiation, they need to be especially vigilant about following all the standard cancer prevention and screening recommendations. And younger patients, especially women, should talk to their doctor about starting screening for some cancers, such as breast, at an earlier age than recommended for this general population.”

In the retrospective study, Baker and colleagues evaluated 4,905 patients (57.4% men; 91.8% white) who underwent HSCT between 1969 and 2014 and had survived at least 1 year without subsequent malignant neoplasms. Patients had undergone the transplants for hematologic malignancies (n = 4,500) or nonmalignant conditions (n = 405). Median age at time of transplantation was 34.5 years (range 0.3-78.9),

Risk factors assessed by the researchers included sex, race, age at first transplant, diagnosis for transplant, stem cell source, regimen (including dose and fractionation) and development of graft-versus-host disease as a time-dependent covariate.

Eleven percent of the HSCT recipients (n = 499) developed a total of 581 subsequent malignant neoplasms at a median 10.3 years (range, 1-39.7) after HSCT.

With a median follow-up of 12.5 years (range, 1-42.11) among 1,709 surviving patients,  observed a 22% cumulative incidence of subsequent malignant neoplasms at 30 years post-HSCT.

Compared with SEER population rates matched for age, sex and calendar year, results showed a 2.8-fold (95% CI 2.6-3.1) increase in the standardized incidence ratio of subsequent malignant neoplasms.

The highest SIRs were for subsequent malignant neoplasms in the bones (28.8), oral cavity (13.8), skin (7.3), central nervous system (6) and endocrine organs (4.9).  (EARs) for subsequent malignant neoplasms per 1,000 person-years were seen in cancers of the breast (EAR = 2.2; 95% CI, 1.4-3), oral cavity (EAR = 1.5; 95% CI, 1.2-2) and skin (EAR = 1.5; 95% CI, 1.5-2).

Survivors exposed to either unfractionated (600 cGy-1,200 cGy-1,200 cGy) or high-dose fractionated (1,440 cGy-1750 cGy) total body irradiation had the highest incidence of subsequent malignant neoplasms. Those who received low-dose total body irradiation (200 cGy-450 cGy) demonstrated risk comparable to that of chemotherapy alone, albeit still twice that of the general population.

Patients aged 20 years and younger at the time of transplant had the highest risk for subsequent malignant neoplasms compared with age-matched members of the general population, although this risk decreased with longer follow-up in relation to transplant.

Patients aged 50 and older at the time of transplant showed sustained elevated SIRs over time, although follow-up beyond 20 years for these patients could not be accessed. Those aged 20 years or younger at the time of transplantation had a 2.28-times (95% CI, 1.31-3.96; P = 0.003) higher risk for subsequent malignant neoplasms than patients aged 50 years and older.

Risk for subsequent malignant neoplasms also appeared significantly higher among recipients of cord blood (HR = 3.02, 95% CI, 1.29-7.09) and peripheral blood stem cells (HR = 1.55; 95% CI, 1.16-2.07) vs. bone marrow, including after adjustment for acute and chronic GVHD.

“The ultimate goal for the transplant world is to find effective, nonradiation conditioning regimens, especially for children,” Baker said in the press release.

Get to know more about Hematology/ Oncology at World Hematology 2019 

Source: https://www.healio.com/hematology-oncology/bone-marrow-transplantation/news/online/%7B87098c14-0bcd-4c14-b775-c99998be6f5c%7D/high-dose-irradiation-before-hsct-increases-risk-for-subsequent-malignancies

Dogs detect human Cancers

Research suggests that dogs can detect many types of cancers in humans.

Like many other diseases, cancers leave specific traces, or odor signatures, in a person’s body and bodily secretions. Cancer cells, or healthy cells affected by cancer, produce and release these odor signatures.

Depending on the cancer type, dogs are able to detect these signatures in a person’s:

• skin
• breath
• urine
• feces
• sweat

Dogs can detect these odor signatures and, with training, alert people to their presence. People refer to dogs that undergo training to detect certain diseases as medical detection dogs.

They detect some substances in very low concentrations, as low as parts per trillion, which makes their noses sensitive enough to detect cancer markers in a person’s breath, urine, and blood.

The fact that dogs can detect cancer has significant benefits for humans. Using dogs to detect and diagnose cancer is a low-risk, noninvasive method.

Medical detection dogs present few side effects and may offer advantages because they are mobile, can begin work quickly, and can trace an odor to its source.

They also have the potential for use in patient care settings or laboratories to identify cancer in tissue samples from people with suspected cancers.

Dogs’ abilities may also help with developing machines that can reliably detect odor signatures from cancer, such as electronic noses.

However, research is still underway and the effectiveness and reliability of canine cancer detection requires further research.

Explore more such interesting information by joining us at World Hematology 2019, this July at Rome, Italy

Source: https://www.medicalnewstoday.com/articles/323620.php

Fast track designation to leronlimab for breast cancer

The FDA granted fast track designation to leronlimab for use in combination with carboplatin for the treatment of patients with CCR5-positive metastatic triple-negative breast cancer.

Leronlimab (PRO 140, CytoDyn Inc.) is an investigational humanized IgG4 monoclonal antibody that blocks CCR5, a cellular receptor believed to play key roles in tumor invasion and metastasis.

Agents that block CCR5 have been shown to block tumor metastases in laboratory and animal models of aggressive breast cancer and prostate cancer.

“This [fast track designation] is an important acknowledgement of the potentially paradigm-shifting therapy option in metastatic triple-negative breast cancer,” Richard Pestell, MD, PhD, vice chairman and chief medical officer of CytoDyn, said in a company-issued press release. “Currently, there are no enduring treatment options for [patients with metastatic triple-negative breast cancer] and we thank the FDA for recognizing the potential of leronlimab [in this setting].”

Enrollment is underway for a study designed to evaluate the agent for patients with metastatic triple-negative breast cancer.

CytoDyn expects to submit a biologics license application to the FDA this year for leronlimab as part of combination therapy for patients with HIV.

The CCR5 receptor also may play a key role in modulating immune cell trafficking to sites of inflammation.

CytoDyn is conducting a phase 2 study of leronlimab to support the concept that the CCR5 receptor on engrafted cells is key for the development of acute graft-versus-host disease. The FDA previously granted orphan drug designation to leronlimab for the prevention of GVHD.

Explore more about oncology and research in the field at: http://bit.ly/2xaW3zT

Source: https://www.healio.com/hematology-oncology/breast-cancer/news/online/%7B1bd93db0-7445-455b-bbd1-d2700e3e8cf1%7D/fda-grants-fast-track-designation-to-leronlimab-for-metastatic-triple-negative-breast-cancer

Biological age and breast cancer risk

Biological age, estimated by measuring DNA methylation, appeared to be a predictor for breast cancer risk, according to study results.

“[When looking] at a group of people who are all the same age, some may be perfectly healthy while others are not,” Jacob K. Kresovich, PhD, MPH, a postdoctoral fellow in the molecular and genetic epidemiology group of the National Institute of Environmental Health Sciences, said in a press release. “That variability in health may be better captured by biologic age than chronologic age.”

Because chronological age is a leading risk factor for breast cancer, Kresovich and colleagues hypothesized that biological age acceleration may be associated with increased breast cancer risk.

They measured baseline blood DNA methylation in 2,764 women enrolled in the Sister Study, including 1,566 women who developed breast cancer within years after baseline blood draw.

Researchers used three established methylation-based biological “clocks” and defined biological age acceleration for each woman by comparing estimated biological age with chronological age.

Results showed a significant association between biological age acceleration and increased risk for breast cancer (HR = 1.15; 95% CI, 1.07-1.23).

Explore more about hematology oncology at World Hematology 2019, this July at Rome, Italy. For more info, PS: https://hematology.cmesociety.com/

Source: https://www.healio.com/hematology-oncology/breast-cancer/news/online/%7B2d7e000a-e544-4b24-b435-f054012c680e%7D/older-biological-age-may-increase-breast-cancer-risk

Ibrutinib plus R-CHOP improves survival among younger patients with aggressive lymphoma subtype

The addition of ibrutinib to standard R-CHOP chemotherapy improved EFS, PFS and OS among patients aged younger than 60 years with untreated nongerminal center B-cell diffuse large B-cell lymphoma, according to results of a randomized, phase 3 study.

The combination, however, appeared associated with increased toxicity and resulted in worse outcomes among patients aged older than 60 years.

“The interaction between age and toxicity was surprising [because] it was not observed within the context of the phase 1 trial that we have published,” Anas Younes, MD, chief of the lymphoma service and Steven Greenberg chair at Memorial Sloan Kettering Cancer Center. “At the present time, there is no obvious biological explanation, but this will be addressed through planned extensive correlative studies. R-CHOP plus ibrutinib is not commonly given to patients with other types of lymphoma.”

DLBCL accounts for as many as 40% of all lymphoma cases, making it the most common form of lymphoma in the world. A previous phase 1 study appeared to demonstrate the safety of ibrutinib (Imbruvica; Pharmacyclics, Janssen) in combination with R-CHOP chemotherapy — which consists of rituximab (Rituxan; Genentech, Biogen) plus cyclophosphamide, doxorubicin, vincristine and prednisone — among patients with untreated B-cell lymphoma, including DLBCL.

Younes and colleagues specifically investigated whether ibrutinib improves the efficacy of R-CHOP in patients with untreated nongerminal center B-cell or activated B-cell diffuse large B-cell lymphoma.

Researchers randomly assigned 838 patients (median age, 62 years; range, 19-88; 53.3% men) to R-CHOP with ibrutinib at 560 mg daily (n = 419) or with placebo (n = 419) in a 21-day cycle for six or eight cycles, depending upon institutional guidelines.

Three-quarters of the patients (75.9%) had the activated B-cell DLBCL subtype.

EFS among the intent-to-treat population and patients with the activated B-cell subtype served as the study’s primary endpoint. Secondary endpoints included PFS, OS and safety.

Median follow-up was 34.8 months.

Results showed that the addition of ibrutinib to R-CHOP did not improve EFS compared with placebo and R-CHOP in the intent-to-treat population (HR = 0.93; 95% CI, 0.72-1.2) or the activated B-cell population (HR = 0.94; 95% CI, 0.7-1.27).

Ibrutinib also did not increase PFS (HR = 0.91; 95% CI, 0.71-1.18), OS (HR = 0.99; 95% CI, 0.71-1.38) or rates of overall response (89.3% vs. 93.1%) and complete response (67.3% vs. 68%) among the intent-to-treat population.

Explore more about hematooncology this July at Rome, Italy. For more info, PS: http://bit.ly/2xaW3zT

Source: https://www.healio.com/hematology-oncology/lymphoma/news/online/%7Bf698cfd3-4f65-4bdb-91c0-50c860dcd560%7D/ibrutinib-plus-r-chop-improves-survival-among-younger-patients-with-aggressive-lymphoma-subtype

CAR T-cell therapy bb2121 shows promising efficacy, manageable toxicity in multiple myeloma

A chimeric antigen receptor T-cell therapy targeting B-cell maturation antigen produced high response rates with manageable toxicity among patients with heavily pretreated relapsed or refractory multiple myeloma, according to results of a phase 1 study. “The adverse events noted in our study were very manageable, and we saw low rates of cytokine release syndrome and neurotoxicity,” Noopur Raje, MD,director of the Center for Multiple Myeloma at Massachusetts General Hospital and professor of medicine at Harvard Medical School.

“[The toxicity] was manageable to the extent that we are now thinking [forward to] outpatient settings. Response rates were high, but we did not see a plateauing of responses in very late-stage multiple myeloma.”

The CAR T-cell therapy bb2121 (Celgene, Bluebird Bio) has shown promise in preclinical studies for treatment of multiple melanoma, for which new therapies have prolonged survival but failed to prevent relapse.

Raje and colleagues reported results from the first 33 patients (median age, 60 years, range, 37-75; men, n = 21) with relapsed or refractory multiple myeloma who received bb2121 in the dose-escalation (n = 21) and dose-expansion (n = 12) phases of the open-label, multicenter trial. All patients had received at least three previous lines of therapy that included a proteasome inhibitor and an immunomodulatory agent or were double refractory. All patients in the dose-escalation cohort and two patients in the dose-expansion cohort had tumor B-cell maturation antigen (BCMA) expression of 50% or greater.

After undergoing lymphodepletion with fludarabine and cyclophosphamide, patients received bb2121 as a single infusion at doses of 50 x 10⁶, 150 x 10⁶, 450 x 10⁶ or 800 x 10⁶ CAR-positive T cells in the dose-escalation phase and 150 x 10⁶ to 450 x 10⁶ CAR-positive T cells in the expansion phase.

Safety served as the primary endpoint. Data cutoff occurred 6.2 months after the last infusion.

Results showed an objective response rate of 85% (95% CI, 68.1-94.9), with 15 patients (45%) achieving a complete response, six of whom have since relapsed.

Median PFS was 11.8 months (95% CI, 6.2-17.8).

Sixteen patients with partial or complete responses appeared negative for minimal residual disease ( 10^-4 nucleated cells).

All 33 patients experienced adverse events, including 28 (85%) who experienced a grade 4 event.

The most common grade 4 hematologic adverse events included neutropenia (n = 26), leukopenia (n = 13), thrombocytopenia (n = 10) and lymphopenia (n = 9). Grade 3 hematologic adverse events included anemia (n = 15), leukopenia (n = 6) and thrombocytopenia (n = 5).

Get to know more about hemonc at: http://bit.ly/2xaW3zT

Source: https://www.healio.com/hematology-oncology/myeloma/news/online/%7Bda1d1336-ff71-467d-a93d-3af4e6294cd5%7D/car-t-cell-therapy-bb2121-shows-promising-efficacy-manageable-toxicity-in-multiple-myeloma

Waldenström macroglobulinemia progression

Researchers at Dana-Farber Cancer Institute have developed a classification system to determine whether a patient with asymptomatic Waldenström macroglobulinemia has a low, intermediate or high risk of developing symptomatic disease.

“This study is part of the efforts conducted by the Center for Prevention of Progression of Blood Cancers (CPOP) at Dana-Farber that aim to understand how blood cancers progress over time from early precursor stages,” Irene Ghobrial, MD, director of CPOP and the Michele & Steven Kirsch Laboratory for Waldenström’s Research, said in a press release. “We also try to identify biomarkers that predict cancer progression and provide these patients with early therapeutic interventions.”

Waldenström macroglobulinemia is a rare form of non-Hodgkin lymphoplasmacytic lymphoma of the bone marrow marked by production of monoclonal immunoglobulin M (IgM) protein —which gathers in the blood, weakens circulation and can cause complications.

Researchers studied 439 patients with asymptomatic Waldenström macroglobulinemia (median age at diagnosis, 61 years; range, 26-91; 62.2% men) who had been diagnosed and observed at Dana-Farber from 1992 to 2014 to determine risk factors for progression to symptomatic disease.

Progression to symptomatic Waldenström macroglobulinemia that required chemotherapy served as the primary endpoint.

Median follow-up was 7.8 years.

Seventy-two percent of patients (n = 317) progressed to symptomatic disease during the 23-year study period.

Median time to progression from diagnosis of asymptomatic disease to symptomatic disease was 3.9 years (95% CI, 3.2-4.6), and the probability of progression within 2 years of diagnosis was 30.8% (95% CI, 26.7-35.3).

Independent predictors of disease progression included IgM of 4,500 mg/dL or greater, bone marrow lymphoplasmacytic infiltration of 70% or greater, 2-microglobulin of 4 mg/dL or greater, and albumin levels less than 3.5 g/dL.

Using these four values as continuous measures, researchers trained and cross-validated a proportional hazards model to evaluate progression risk. The risk model stratifies patients into three groups: high risk (median time to progression [TTP], 1.8 years), intermediate risk (median TTP, 4.8 years) and low risk (median TTP, 9.3 years).

Get to know more about hematology/ oncology at World Hematology 2019

Source: https://www.healio.com/hematology-oncology/lymphoma/news/online/%7B0b7ddb76-ddb0-4e07-ae63-b557fd802b25%7D/new-model-identifies-risk-for-waldenstrm-macroglobulinemia-progression

FDA grants orphan drug status to AUTO3 for ALL

The FDA has granted orphan drug status to AUTO3, a cell-based immunotherapy for the treatment of acute lymphoblastic leukemia (ALL).

AUTO 3 (Autolus Therapeutics) is a programmed T-cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that are independently optimized for single-target activity.

“By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies,” according to a press release from the manufacturer.

“We believe that AUTO3 has the potential to be a best-in-class therapy in pediatric ALL by addressing antigen escape, a common cause of relapse in these patients,” Christian Itin, PhD, CEO of Autolus, said in a press release. “AUTO3 may also provide an improved safety profile over currently marketed CAR T therapies with low levels of severe (cytokine release syndrome) and neurotoxicity observed in clinical studies.”

AUTO3 is currently being tested in pediatric patients with ALL as part of the AMELIA clinical trial and in patients with diffuse large B cell lymphoma in the ALEXANDER clinical trial.

Data from the American Cancer Society suggest there will be 5,930 new cases of ALL and approximately 1,500 deaths in 2019. Sixty percent of cases will be in patients aged younger than 20 years.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

Get to know more about hematology/ oncology by joining us at Rome, Italy, this July, PS: World Hematology 2019  

Source: https://www.healio.com/hematology-oncology/leukemia/news/online/%7Ba0cbd1ae-c5f8-4907-b397-d467ab36ad31%7D/fda-gives-orphan-drug-status-to-auto3-for-all

Calcium and Cancer Risk

Latest research has reported that excess intake of calcium from supplements has been linked to an increased risk for cancer-related deaths.

The report found no mortality benefits associated with any reported dietary supplement use among nearly 31,000 adults in the National Health and Nutrition Examination Survey.

They found that excess calcium consumption was associated with increased risk for cancer-related deaths. Calcium supplements were specifically implicated in the excess of mortality, according to the investigators, with a rate ratio of 1.53 (95% confidence interval, 1.04-2.25) for intakes of 1,000 mg/day versus no intake.

Explore more about oncology/ hematology at World Hematology 2019, this July, at Rome, Italy. For more info, PS: https://hematology.cmesociety.com/

Source:https://www.mdedge.com/internalmedicine/article/199202/preventive-care/calcium-supplement-use-linked-cancer

Do you know?

The FDA has granted fast track designation to Annamycin for treatment of patients with relapsed or refractory acute myeloid leukemia.

Annamycin (Moleculin Biotech) is a liposome-formulated anthracycline designed to eliminate cardiotoxicity and avoid multidrug resistance mechanisms associated with other approved anthracyclines.

The agent is being evaluated in separate phase 1 and phase 2 trials in the United States and Europe.

Studies in animal models showed the agent to be noncardiotoxic. Trials that included patients with leukemia showed the agent was associated with fewer dose-limiting toxicities than typically experienced with doxorubicin, according to Moleculin.

Explore more about hematology/ oncology at: https://hematology.cmesociety.com/

Source: https://www.healio.com/hematology-oncology/leukemia/news/online/%7B59c6cf34-2eec-4605-a347-f477d91a927d%7D/fda-grants-fast-track-designation-to-annamycin-for-relapsedrefractory-acute-myeloid-leukemia

Track 19 : Hematology & Oncology: Case Reports

Case reports are detailed reports that has all the signs, symptoms, diagnosis, treatment and follow up of the patients. These case reports serve as an evidence for medicine, scientific and educational purposes. Case reports of hemotology or oncology gives the information of preventive or therapeutic interventions as these generally require stronger evidence. Thus, a case report has all the details of a disease or a disorder of an individual in medical field. Case reports give the demographic profile of the patient suffering from disease, references of any unusual or unique incidences, etc.

Do submit your abstracts in the following link: Abstract-Submission

Do you know?

Do you know that one pint of blood is capable of saving 3 lives and people suffering from sickle cell disease need up to 4 pints or units of blood every single month to survive.

Know more such interesting facts and work on hematology, oncology at: World Hematology 2019

Do you Know?

Are you curious to know, why red blood cells are shaped like breath-mint disks with a dent in the middle?

The answer to this query seems interesting, this is because, the breath-mint design allows the cells to twist through capillaries and the tiniest blood vessels. A sphere or cube is less flexible and might get stuck. Also, the dents in the cells add to the surface area, allowing more oxygen and carbon dioxide to pass in and out of the cell.

To know more interesting facts about hematology/ oncology, PS:https://bit.ly/2KAr8Bq

Join us this July, to unleash the enigmas in hem/ onc, PS: http://medicines.doctor/haematology5

World Hematology 2019

Here we have our media partners who support us in making our event. Do join us at Rome, Italy to unravel the conundra in hematology/ oncology. For more details, PS: http://medicines.doctor/haematology5

Alcohol consumption and Multiple Myeloma

Research has shown that high alcohol consumption impairs the immune system and, in turn, increases cancer risk.  However, low alcohol consumption improves insulin sensitivity, which, in turn, decreases risk of diabetes and other obesity-related disorders and thus, indirectly, may decrease risk of multiple myeloma.

Source: https://www.mdedge.com/hematology-oncology/quiz/7735/multiple-myeloma/alcohol-consumption-and-multiple-myeloma?channel=250

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For the first time in 27 years, the FDA has proposed amending mammography screening regulations. On March 27, 2019, the FDA proposed policy changes to modernize mammography services. “Among the proposed amendments to improve communication and medical decision making is the addition of breast density information to the mammography lay summary letter provided to patients and to the medical report provided to their referring health care professionals. Mammograms of dense breasts—breasts with a higher proportion of fibroglandular tissue compared to fatty tissue—can be difficult to interpret because the dense tissue can obscure signs of breast cancer and lower the sensitivity of the image. Dense breasts have also been identified as a risk factor for developing breast cancer,” according to an FDA news release.

The proposed amendments also seek to enhance information provided to health care professionals by proposing to codify three additional categories for the assessments of mammograms, including adding an important category titled “known biopsy proven malignancy,” which would help identify for health care professionals those cases where cancer being mammographically evaluated for therapy are already known and identified. The proposed amendments would also modernize mammography quality standards and better position FDA to enforce the MQSA regulations and take action when violations are found.

Source: https://www.mdedge.com/hematology-oncology/quiz/7746/fast-facts-friday/fast-facts-friday-march-29-2019?channel=59610