Pfizer Scientists Are Doubling Down To Conquer Blood Disorders

Blood Disorders

Blood disorders affect millions of people worldwide today. Because of this, this December, experts in the field of blood disorders from all over the world will assemble in New Orleans for the 64th Annual Meeting of the American Society of Hematology (ASH), an organisation dedicated to curing blood disorders.

Pfizer is stepping up its efforts to address unmet needs in blood cancer, sickle cell disease, and other hematologic diseases by presenting more than 30 abstracts at ASH from across its haematology portfolio. Pfizer has recently received Breakthrough Therapy designation from the U.S. Food and Drug Administration for its investigational multiple myeloma treatment, for which it is especially excited to share data from a robust clinical trial program. This treatment is intended to treat multiple myeloma that has relapsed or become resistant to treatment.

This is Pfizer Oncology’s 12th designation as a “Breakthrough Therapy,” which is intended to expedite the development and regulatory review of a drug for serious or life-threatening illnesses and when preliminary clinical evidence suggests that the drug may significantly outperform currently available therapies on a clinically significant endpoint.

Pfizer will discuss significant findings in sickle cell disease along with oncology advancements, including the most recent addition to a growing corpus of real-world evidence. The potential of next-generation therapies to lessen complications and enhance red blood cell health will also be presented using fresh data.

Pfizer has a promising pipeline of preclinical and clinical assets that could change the way sickle cell disease is treated thanks to its extensive knowledge of rare blood disorders and its recent acquisition of Global Blood Therapeutics, a company established to treat sickle cell disease. Two of the pipeline drugs have the potential to be best-in-class and have received the FDA’s Orphan Drug and Rare Pediatric Disease designations.

Additionally, Pfizer will present data on haemophilia at the congress, continuing the company’s long history in the field, which goes beyond the era of recombinant proteins and includes the ongoing research and development of gene therapies for haemophilia A and B.

“At Pfizer, we’re applying decades of scientific expertise and in-depth understanding of blood disorders to identifying new targets, applying novel technologies and accelerating development to address areas of significant unmet need.”

Chris Boshoff, Chief Development Officer Of Oncology And Rare Diseases At Pfizer.

Pfizer is working to address the very high unmet demand for blood cancer through the development of novel new therapies as well as initiatives aimed at significantly enhancing health equity.

Some blood cancers can be treated, however many are currently incurable.

White blood cell growth and function are impacted by blood malignancies, which frequently develop in the bone marrow, the place where new blood cells are made. Although there are more than 100 different types of blood cancer, leukaemia, lymphoma, and myeloma are the most common types. Some move more slowly than others, and vice versa. Myeloma is one of the blood malignancies that still has no cure, despite recent medical advancements.

People who are diagnosed with multiple myeloma still have a median life expectancy of just over five years despite the disease being originally reported in the 1840s. Multiple myeloma is a fatal condition that causes plasma cells to proliferate uncontrollably in the bone marrow and can cause skeletal devastation. The term “multiple myeloma” refers to the proliferation of clonal malignant plasma cells, which typically occurs in multiple locations. The majority of patients who complete their initial course of treatment will eventually have a disease relapse. The time in remission shortens and the range of available treatments is reduced with each relapse.

Pfizer is presenting data at the ASH Annual Meeting that advance the field of hematology, including information on the use of a novel cancer immunotherapy known as bispecific antibodies (BsAbs) to treat multiple myeloma.

BsAbs that target immune cells attach to and interact with both immune cells and tumour cells. One arm binds specifically to antigens on cancer cells, such as the highly expressed B-cell maturation antigen (BCMA) on the surface of multiple myeloma cells. T-cells are attracted to one another by the other arm’s binding to them, triggering an immune response. As long as BCMA is expressed on the tumour cells, these immune engagers allow the patient’s own immune system to kill cancer cells, regardless of the cytogenetic risk group.

“Agents targeting BCMA are emerging as the next wave of breakthrough in multiple myeloma”, “This is an exciting area of innovative research, and we are well-positioned to be competitive in this space.”

Chris Boshoff, Chief Development Officer Of Oncology And Rare Diseases At Pfizer.

While this is happening, the firm has been enhancing its line-up of six blood cancer medications that have received FDA approval. Trillium Therapeutics, a Canadian pharmaceutical company that developed a number of promising clinical-stage blood cancer immunotherapy candidates, was acquired by Pfizer last year. The portfolio of Trillium includes SIRP-CD47 biologics, which are intended to improve patients’ innate immune systems’ capacity to recognise and eliminate cancer cells.

Identifying health inequalities and creating an industry baseline to track progress on diversity and participation in clinical trials

Reaching health equity is a major obstacle in many different illness areas. For instance, sickle cell disease is a chronic illness that primarily affects persons of sub-Saharan African heritage. It can also afflict people of Hispanic, South Asian, Southern European, and Middle Eastern descent.

The incidence and prognosis of multiple myeloma are disproportionately higher in ethnic and racial minority groups, who also tend to be older populations. But these populations are still underrepresented in research and clinical trials on cancer.

“An important factor to consider when translating research into effective medicines is the diversity of the real-world patient population. Clinical trials often fail to capture that diversity — a failure that needs correcting.”

Dany Habr, MD, Oncology Chief Medical Affairs Officer At Pfizer And Co-author Of A Paper About The Importance Of Diversity Of Participants In Multiple Myeloma Studies.

Habr and co-author Massimo Corsaro use a recent review of 112,293 patients enrolled in 230 oncology trials that resulted in FDA approvals over the course of the previous ten years in their research. Compared to 76.3% White participants, only 3.1% were Black and 6.1% were Hispanic. Based on the incidence of cancer in the US, that represents less than 22% and 44% of their projected representation, respectively.

The median age of participants in 177 multiple myeloma treatment trials was 62, according to a separate analysis, whereas the median age of patients with multiple myeloma as a whole is 70. Only 5 of these trials included participants over 75 who had results reported.

Habr will participate in the industry’s theatre symposium at ASH, “Pursuing Scientific Breakthroughs for Patients with Hematologic Malignancies,” along with Pfizer’s Suneet Varma, Global Oncology and US President, and Sriram Krishnaswami, Vice President and Development Head, Multiple Myeloma. This symposium will feature discussion on Pfizer’s research in blood cancer, its vision for delivering innovation, and for addressing healthcare disparities in the treatment of multiple my

Pfizer has committed to addressing both informational and practical barriers to diverse clinical trial participation. In order to serve as an industry benchmark against which to gauge future development, Pfizer has published a 10-year retrospective analysis of ethnic, racial, age, and gender diversity in its past clinical trials.

Through its Diversity in Clinical Trials Center of Excellence programme, the company has been asking patient advocacy organisations for input on the design of its clinical trials, conducting clinical trials in regions with a diverse pool of potential participants, and providing educational materials that are suitable for different ages and cultures in an effort to increase public confidence in clinical research.

In order to better understand the obstacles to treatment and care, Pfizer has also been working with the communities of sickle cell patients and advocacy groups.

“Listening to patients’ voices is critical for understanding and addressing disparities in care”, “Our partnerships with the blood disorders community are key to this mission, and we look forward to continuing to work together at ASH and beyond.”

Dany Habr

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