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World Blood Cancer Day on May 28th, 2016

 

The World Blood Cancer Day, which was initiated by the German Bone Marrow Donor Center (“Deutsche Knochenmarkspenderdatei”, DKMS) to show solidarity with blood cancer patients, will take place on May 28th.

 

What is Blood Cancer?

Various malignancies which affect the blood cell production or function, bone marrow and/or lymphoid system are referred to as blood cancer. All blood cancers have in common that the diseased cells proliferate uncontrollably, often with fatal consequences for patients. According to the cell types involved, blood cancer is diagnosed as leukemia (blood), lymphoma (lymph nodes) or myeloma (plasma). Leukemia is further classified according to the course of the disease as either acute or chronic leukemia and according to the cell origin as either lymphatic or myeloid leukemia.

 

Current Standard of Care Treatments

Currently available treatments for blood cancer include amongst others chemotherapy, radiation therapy and stem cell transplantation (SCT). However, if the disease is already in an advanced stage, chemotherapy and radiation can in many cases only prolong a patient’s life. Hence, there is still a high unmet medical need for alternative and more effective treatments. For many blood cancer patients, a stem cell transplantation is the only potentially curative treatment. Transplantation of allogeneic (donor-derived) blood stem cells is a widely accepted medical procedure to restore normal blood cell production (hematopoiesis) in patients treated for blood or lymphatic cancers, or otherwise suffering from defective blood formation or immune disorders.

 

Today, approximately 30,000 patients worldwide receive allogeneic stem cell transplants every year, but a matching donor is not found for every patient. However, approximately 35-50% of all blood stem cell transplant patients develop severe forms of acute or chronic Graft versus Host Disease (GVHD), a potentially fatal complication where donor-derived T cells (immune cells) attack the normal tissues of the patient. Half of these patients do not respond adequately to standard first-line therapy. As of today there is no approved second-line treatment available. Respective new therapies and safer ways to reduce the risk of GVHD and to allow for transplantation for patients, even if there is no matching donor, would be a tremendous achievement.

 

We strongly believe that the World Blood Cancer Day 2016 is a perfect event to raise awareness for this indication by highlighting some of our clients’ clinical programs to improve the treatment of blood cancers with innovative and unique approaches. Please find below just some examples of companies we think have the potential to change the treatment environment for patients suffering from this terrible disease.


Pioneers Committed to Changing the Current Treatment Paradigm:
 

 

 

Please contact us:

 

Dr. Cora Kaiser

cora.kaiser@mc-services.eu

+49 89 210228 38

Apogenix’ lead immuno-oncology candidate APG101 is in development for the treatment of solid tumors and malignant hematological diseases, such as myelodysplastic syndromes (MDS). MDS is a bone marrow disorder that is characterized by ineffective hematopoiesis and can lead to severe anemia. In most cases, the anemia is treated with blood transfusions that eventually result in an iron overload, which can damage the liver and other organs. In addition, these patients are at risk for developing acute myeloid leukemia. Currently, there is no approved drug for the treatment of low and intermediate-I-risk MDS patients, and only few clinical trials have rendered positive results. In MDS, activation of the CD95 receptor by the CD95 ligand inhibits the development of red blood cells. APG101 blocks the CD95 ligand, thereby enabling the maturation of erythrocyte precursor cells to functional erythrocytes. Interim results of a phase I trial with APG101 in low and intermediate-I-risk MDS patients reveal an increase in erythrocyte precursor cells and a strong trend toward reduction in transfusion frequency after treatment with APG101. Final results of the clinical trial are expected in the first half of 2016.

ERYTECH’s (Euronext Paris: ERYP) initial focus is on the treatment of blood cancers, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), by depriving tumors of nutrients necessary for their survival. ERYTECH has recently filed for European Marketing Authorization for its lead product candidate, ERY-ASP (trade name GRASPA®), following positive efficacy and safety results from its completed Phase II/III pivotal clinical trial in Europe in children and adults with relapsed or refractory ALL. ERYTECH also has an ongoing Phase I clinical trial of ERY-ASP in the United States in adults with newly diagnosed ALL, and a Phase IIb clinical trial in Europe in elderly patients with newly diagnosed AML, each in combination with chemotherapy. The EMA and the U.S. Food and Drug Administration (FDA) have granted orphan drug designations for GRASPA for the treatment of ALL, AML and pancreatic cancer.

Kiadis Pharma (Euronext Amsterdam and Brussels: KDS) is focused on cell-based immunotherapy products for the treatment of blood cancers and inherited blood disorders. The Company’s products have the potential to address the risks and limitations connected with allogeneic hematopoietic stem cell transplantation (HSCT), namely graft-versus-host disease (GVHD), cancer relapse, opportunistic infections and limited matched donor availability. In April 2016, the Company reported positive Phase II results with its lead product ATIR101™ in patients with blood cancer. In that study, zero patients developed grade III-IV GVHD although family members were used as donors that were only half-matched to the patient. The data confirmed that the use of ATIR101™ in such a protocol significantly reduces Transplant Related Mortality while Overall Survival is significantly improved. ATIR101™ has been granted Orphan Drug Designations both in the US and Europe and will enter Phase III later this year.

Medigene (FSE: MDG1) concentrates on the development of personalized T cell-based immunotherapies. An antigen-tailored Dendritic Cell (DC) vaccine is currently evaluated in a company-sponsored Phase I/II clinical trial in acute myeloid leukemia (AML), which entered Phase II in April 2016. In addition, Medigene's DC vaccine technology for treating acute myeloid leukemia (AML) is used in an ongoing investigator-initiated Phase I/II trial at LMU Hospital Munich and in compassionate use patients at Oslo University Hospital. Medigene also got a US patent granted (see press release) for the method of making DC vaccines for leukemia patients post SCT from family member donors. Medigene has a second platform in development and currently prepares first clinical trials for T-cell receptor modified T cells in which the patient’s own T cells get armed with tumor- specific T cell receptors. The receptor-modified T cells are then expanded in vitro and provided back to the patients in high numbers that are able to combat large amounts of tumor. Further information about Medigene's DC-Vaccines and TCRs can be found respectively at: https://vimeo.com/123005832 and https://vimeo.com/123007480.

MorphoSys’ (FSE: MOR) proprietary candidate MOR208 is a monoclonal antibody for the treatment of B cell malignancies, such as non-Hodgkin’s lymphoma (NHL) or chronic lymphocytic leukemia (CLL), targeting B lymphocytes via the CD 19 antigen. MOR208 has been engineered to possess significantly enhanced antibody-dependent cell-mediated cytotoxicity, thus improving a key mechanism for tumor cell killing. MOR208 is currently being evaluated as monotherapy in a phase IIa trial in relapsed/refractory patients with four different subtypes of NHL, including diffuse large B cell lymphoma (DLBCL) and in a combination trial with Lenalidomide in patients with relapsed/refractory DLBCL. Additionally, MorphoSys’s partner the Ohio State University is evaluating MOR208 in combination with Lenalidomide in an investigator-sponsored phase II trial in CLL patients. Preliminary data showed that MOR208 is well tolerated and demonstrates encouraging single-agent activity. Based on these promising results, MorphoSys will commence a second phase II combination trial of MOR208 in diffuse chronic lymphocytic leukemia (CLL) in the near future. In addition, MorphoSys aims to start a pivotal study of MOR208 monotherapy in DLBCL in 2017.

Xenikos’ lead candidate, T-Guard™, is currently in clinical phase I/II testing in Europe for second-line treatment of life-threatening graft versus host disease (GVHD) following transplantation of allogeneic (donor-derived) blood stem cells in patients with advanced blood cancer. Preliminary results from this study showed strong clinical responses and a doubling of the 6-months overall survival rate compared to historical controls, with a well-manageable side effect profile without severe infusion reactions. T-Guard™ consists of a combination of two toxin-loaded anti-T-cell antibodies and shows promise as a therapeutic tool for safely and swiftly resetting the body’s immune system in T-cell-mediated diseases such as acute GVHD. Once injected into the body, T-Guard™ specifically identifies and eliminates adult T cells, with a strong preference for the activated ones. The particular combination of immunotoxins used to construct T-Guard™ was designed to provide a unique blend of synergistic efficacy, narrow specificity and multiple, gentle mechanisms of action. T-Guard™ has been granted Orphan Drug Designation in both the EU and U.S.

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