Heather Myler, Ph.D.
Bioanalytical Community Chair, American Association of Pharmaceutical Scientists (AAPS)
While innovative technologies continue to emerge, we anticipate the convergence of best-in-class therapeutics with pioneering therapies that will introduce truly transformative treatment options for patients. Combined with advances in biomarker technology, physicians can diagnose patients earlier and create customized treatment plans specific to each patient’s condition. However, this shift requires a change in how the industry approaches research, diagnostics, and patients.
By evaluating a patient’s genetic makeup, scientists and physicians can target more effective, individualized prevention and treatment strategies.
“Drug discovery and development is transitioning toward targeting subgroups of patients with specific disease markers by using real-world evidence to address knowledge gaps and offer alternatives to conventional study designs and treatment regimen,” says Eric Faulkner, vice president and executive director of the Precision and Transformative Medicine Center of Excellence at Evidera.
These changes break through a fundamental challenge for the biopharmaceutical industry. Historically, patients often have had access to treatment options that reduce symptoms without eradicating the underlying disease, but that paradigm is changing. Artificial intelligence and data analytics are accelerating the realization of customized and sustained therapies that allow scientists and physicians to leverage a combination of strategies that surpass traditional treatment options and address previously life-threatening diseases.
Cell and gene therapies are rapidly expanding into the biopharmaceutical space to address unmet patient needs, with significant future growth anticipated. Spinraza, an anti-sense oligonucleotide (gene) therapy and first treatment for spinal muscular atrophy (SMA), was approved by the U.S. Food and Drug Administration (FDA) in 2016 after demonstrating remarkable improvements in motor function and survival in clinical trials. This is a true breakthrough, as SMA is the leading genetic cause of infant deaths worldwide.
For oncology patients, who comprise a broad cross-section of the population, the impact of the changing paradigm is telling.
“Kymriah is a personalized immuno-oncology treatment and first FDA-approved CAR T-cell therapy for patients under the age of 25 with acute lymphoblastic leukemia that is relapsed (went into remission and came back) or refractory (did not go into remission with other treatments),” says Shefali Kakar, AAPS member and vice president and global head of Oncology PK Sciences at Novartis. “A patient’s own white blood cells are collected, isolated, and genetically engineered to express receptors that target CD19 on the surface of B cells. These engineered CAR T-cells are then transfused back into the patient, where they recognize and attack CD19-expressing cancerous cells.”
But even the most sophisticated and advanced technologies have their challenges. “Some cancer cells use protective mechanisms, including the PD-1 pathway, to hide from T-cells, reducing the ability of T-cells to attack PD-1 ligand expressing cancer cells,” says Pierre Jolicoeur, executive director of vaccine sciences at PPD Laboratories. “Melanoma and non-small cell lung cancer have large amounts of PD-L1 on their surface, resulting in tumor growth and metastasis. Immune checkpoint inhibitor therapies that block this protective mechanism allow the immune system to destroy them.”
By leveraging research conducted over the years, we have established a foundation that offers great promise for the future.
“Decades of scientific discoveries have enabled a deeper understanding of how tumor cells grow, survive, and develop drug resistance, which has fueled tangible advances in the development of many novel therapies,” says Meena Subramanyam, AAPS member and vice president and global program leader at Takeda. “The concept of harnessing the immune system to eliminate tumors that have been resistant to all prior forms of therapy is a significant breakthrough, because it allows the combination of immunotherapies with conventional treatments to achieve a cure in patients.”
The continued growth into this new era requires a wide range of entities spanning research, development, and commercialization to the health care professionals who administer the therapies and the patients who receive them.
“Complex innovations and the ability to implement them effectively demand an extensive ecosystem,” says Patrick Bennett, AAPS member and vice president of business strategies at PPD Laboratories. “This requires connectivity between academia, government, instrument and technology vendors, industry groups, pharmaceutical and health care companies, commercial laboratories, and research organizations.”
That type of connectivity often is achieved by membership in nonprofit associations like the American Association of Pharmaceutical Scientists (AAPS), an organization dedicated to advancing the capacity of pharmaceutical scientists to develop products that improve global health by offering scientists and physicians an opportunity to share their research and address challenges.
While the compilation of newly available technologies is challenging, the health care industry is connected like never before, and scientists, physicians, and patients are working together toward a common goal.
“We are at an exciting time where our understanding of cancer biology, from genetic mechanisms to tumor microenvironments, and developments in technology, such as nanomedicine, artificial intelligence, and genome editing, are converging to enable entirely new approaches to cancer diagnosis and therapy,” says Jennifer West, Fitzpatrick University Professor of Engineering at Duke University.
The rules of the road are in constant flux and it is imperative that health care providers be integrated, flexible, and data- and outcome-driven, and draw on cross-functional expertise to navigate the changing landscape. Connection and collaboration are key to the pace at which scientists and physicians can identify the best treatment options and ultimately provide patients with life-changing results.
Heather Myler, Ph.D., Bioanalytical Community Chair, American Association of Pharmaceutical Scientists (AAPS), [email protected]