Hematologic disorders comprise a vast category of diseases that affect millions of Americans and have a significant impact on the lives of individuals, their loved ones and the U.S. healthcare system overall. Although some types of blood disorders can be managed to prevent symptoms or complications, others lead to a poor prognosis because the disease relapses or is refractory to treatment.
Given recent advances in understanding the underlying biology of blood disorders and the development of new drug discovery technology, the biopharma industry is making important strides in discovering and delivering scientific innovations for various types of blood diseases. Genentech, which has a track record of more than two decades of delivering breakthrough medicines that have redefined treatment paradigms across various blood disorders, believes every person deserves effective treatment options at all stages of their treatment journey.
“In the area of hematologic cancer, we are developing innovative immunotherapies and other novel medicines with the hope of helping patients live longer lives and improve their overall treatment experience by reducing some of the burdens and access issues they face. I’m proud to lead a team that is pioneering new approaches to personalize care and address the diverse needs of people with blood diseases.”
Ginna Laport, Vice President, Global Head of Lymphoma/CLL Development.
Raising the bar in lymphoma
Diffuse large B-cell lymphoma (DLBCL), the most aggressive type of non-Hodgkin’s lymphoma (NHL), poses a treatment challenge — both for patients who have been diagnosed for the first time and haven’t yet received treatment, and for those who have received previous lines of therapy but whose disease has relapsed or is refractory to treatment. The standard of care, although highly effective, has not changed in two decades and 40% of patients relapse after the standard treatment or never respond.
With the goal of providing therapies that can improve outcomes for both groups of patients, Genentech researchers are advancing the clinical development of different classes of innovative medicines. These include antibody-drug conjugates (ADC) that deliver chemotherapy directly to malignant cells and novel T-cell engaging bispecific antibodies.
Another type of NHL for which there continues to be unmet need is follicular lymphoma (FL). When this indolent (slow-growing) type of NHL relapses or doesn’t respond to treatment, it becomes increasingly difficult to treat and patients often have a poor prognosis. T-cell engaging bispecific antibodies may provide a much-needed therapeutic option for these difficult-to-treat cancers due to their unique design that brings the T cell (a type of immune cell) in close proximity to the target B cell (which can be healthy or malignant), which activates the release of cancer cell-killing proteins from the T cell.
Exploring further areas of unmet need in hematologic cancers
Multiple myeloma, the third most common type of blood cancer, remains an incurable disease characterized by multiple relapses, with an overall five-year survival rate of about 55%. As part of Genentech’s ongoing development of T-cell engaging bispecific antibodies, Genentech researchers are also evaluating these molecules in multiple myeloma with the hope of better understanding the impact this class of medicines could have in this evasive cancer.
Improving the treatment of rare blood diseases
Beyond hematologic cancers, Genentech is focused on delivering medicines that could transform the treatment of rare blood disorders, specifically hemophilia A. The current standard of care for this genetic bleeding disorder is intravenous injections of factor VIII replacement therapy two to three times per week, taken for life, to control or prevent bleeding episodes. The development of antibodies to factor VIII replacement therapy is a key challenge: As many as 30% percent of people with severe hemophilia A develop factor VIII inhibitors. Inhibitors block replacement factor VIII and significantly increase the risk of mortality. Genentech scientists have already delivered a bispecific factor IXa- and factor X-directed antibody to treat hemophilia A and are building on that pioneering work to deliver this medicine to patients with and without inhibitors, including infants.
Genentech scientists are relentlessly focused on pursuing bold therapeutic ideas in the hopes of improving outcomes for people with hematologic cancers and rare blood diseases, rethinking what is possible when it comes to standards of care and the patient treatment experience.
To learn more, visit Genentech’s Hematology Hub.
 American Society of Clinical Oncology. Multiple Myeloma: Statistics. Cancer.Net. [Internet; cited 2022 June]. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/statistics.