A Decade in the Making
When people think of rheumatology, they most often think of rheumatoid arthritis (RA) – and for good reason. More than 1.3 million Americans continue to suffer from this devastating disease that causes the immune system to attack its own tissue, leading to painful swelling of the joints. In addition, RA is a systemic disease affecting many organ systems. Biotech companies, like Genentech, physicians and researchers have dedicated years to developing treatments that help relieve the symptoms of RA and work to reduce joint damage caused by this disease that can significantly impact a person’s day to day life.
I began my work with the Genentech’s rheumatology team over ten years ago as part of the project development group. Since then, the program has grown rapidly, exemplifying the company’s commitment to science and patients with high unmet needs in rheumatic diseases. One particularly rewarding project that comes to mind evaluated the process in which a medicine might be administered while also assessing how we could make this experience less invasive for the patient. It’s extremely important to make this process as comfortable as possible for patients already experiencing the side-effects of their conditions.
At Genentech we understand that like many diseases, RA affects people in different ways. Not only are there multiple types of disease manifestations, there are different biologic pathways that can cause the disease. This understanding is very important when it comes to the first step – diagnosis. The practice of rheumatology is not an exact science and requires rheumatologists to rely upon many elements of their education and experience to guide them in coming to a diagnosis. There are often overlapping signs and symptoms that make this determination challenging. Because of the different pathways involved, treatment decisions between conditions are often also very different, so the correct diagnosis is key.
With this in mind, our scientists began investigating these alternative pathways, looking inside our already existing portfolio, in the hope of identifying an agent with a new and unique mechanism of action to help patients with moderate to severe RA that sometimes weren’t benefitting from the existing treatment options. This approach, while intricate and taxing, led to a new treatment option for adult patients with RA and started the rheumatology franchise at Genentech.
Since then, we’ve participated in many clinical trials that have had success, but that doesn’t mean that we’re always successful. Sometimes it takes obstacles to realize when we need to stop, reassess and come about a situation from a new angle with a fresh perspective on what didn’t work and how we can modify our approach. A study is often considered to have “failed” when the final results do not meet the intended endpoint. The reality is that we can always learn from this. In one specific circumstance, we actually identified a new patient population that could potentially benefit from the “failed” study. The key takeaway here is that we should always design trials in a rigorous fashion with the intent to demonstrate the hypothesized benefit. If a study is conducted in a highly scientific fashion, even when it misses its desired outcome, it can still be of high medical value. We use both our successes and failures in rheumatology to forge ahead.
We are currently on a quest to find biomarkers in rheumatic diseases. This brings us back to the challenge of trying to determine what therapy might be best for an individual patient. The process of “trial and error” is not desirable for patients with serious medical conditions and a personalized medicine approach is needed. We’re trying to identify markers, or characteristics, for individual patients that might help predict what therapy would be best for that specific person. This is a somewhat complex endeavor and requires coordination amongst our research and medical teams, as well as collaborations with external experts and investigators. Our ultimate hope is to be able to allow physicians to tailor specific therapies for each patient.
If we’re successful in identifying markers for patient response or non-response to therapies, this could help in determing the best therapy for a patient’s disease. This may ultimately lead to better long-term medical outcomes if patients receive the best therapy early in their disease, and reduce complications from continued disease activity due to inadequate therapy.
As part of the Genentech rheumatology team for more than ten years, I have developed a strong respect for the many people within the company who work passionately to bring medicines to the clinic. I have recognized the dedication of the treating physicians (and staff) who are willing to collaborate to help develop new therapies. Furthermore, I have the highest admiration for the many patients that participate in medical trials, realizing that they may or may not benefit medically from participation, but still volunteer in the interest of advancing science for society. It’s help from patients like these that allows the scientific community to make progress in providing what is necessary to address unmet needs.