The sky’s the limit for specialty drug innovation
April 19, 2019
In 1899, Charles Duell, commissioner at the US patent office, reportedly stated that “everything that can be invented has been invented.” Duell is surely blushing in his grave: through the 20th century and into our new millennium, invention has exploded in all domains, medicine being a shining example.
First came antibiotics, then genetic testing, and still later, complex specialty medicines that mimic, amplify, and intercept physiologic processes. Among the numerous space-age technologies that have landed on earth is next-generation sequencing, a high-speed DNA processing technique that can identify patients at risk of a variety of cancers.
The pace of these advances continues to astonish. Twenty years ago, before the advent of targeted anti-retroviral therapy, AIDS was a killer disease that incited worldwide fear, and HIV-positive patients could expect to live just 10 to 12 years following diagnosis.1 A young person beginning therapy today can expect to live to age 78, close to the average lifespan.2 Hepatitis C treatment has made equally dramatic strides. A decade ago, hep C ravaged bodies and lives like a silent time bomb. Today, direct-acting antivirals offer a path to a permanent cure—a milestone that earlier generations didn’t even dare dream about.3
Then there’s oncology, the focus of breakthrough after breakthrough. The old phrase “fight against cancer,” which assumed one either won the fight (cured the disease) or lost it (succumbed), no longer reflects reality. Sophisticated new treatments have turned cancer into a chronic disease for many patients: these medications keep the cancer in long-term remission, allowing patients to carry on—more or less—with their lives.
The drug ibrutinib, for example, has given a new lease on life to people with treatment-resistant leukemia. In a landmark trial of patients with chronic lymphocytic leukemia, 59% of patients receiving ibrutinib survived 3 years without progression of disease, compared to just 3% of those treated with a traditional comparator drug.4 Most remarkable of all? Complete responses to ibrutinib increased over time.
Even within a particular drug class, new kids on the block are outdoing their predecessors—a testament to the vigor of today’s specialty drug research. As one example of in-class progress, the ALK inhibitor alectinib significantly outperformed the older ALK inhibitor crizotinib in patients with a specific type of advanced lung cancer.5 And it goes without saying that these new targeted therapies roundly beat chemotherapy.6
If there were an Academy Awards for medicine, an emerging cancer treatment called CAR-T cell therapy would surely get an Oscar. A tour de force of modern medical science, CAR-T therapy involves adding genetic matter to a patient’s T cells (immune fighter cells), enabling them to mount a targeted attack on cancer cells. Once in the bloodstream, these genetically enhanced cells continue to multiply and do their job.
The FDA has given the nod to several CAR-T agents, and Health Canada’s recent approval of Kymriah and Yescarta—CAR-T agents used for some forms of lymphoma—has ushered the class into Canada. Canadian stakeholders are also joining forces to ramp up CAR-T research and capacity. Case in point: the BioCanRx network and its partners is funding 16 research initiatives that have the potential to cure cancer, such as made-in-Canada CAR-T.7 And that’s a good thing, because the worldwide CAR-T market is expected to grow at an annual rate of 45.6%.8 More importantly, these therapies give patients with treatment-resistant cancer a real shot at beating their disease.
On a parallel track, the science of cancer interception—stopping cancer before it even shows up—is coming into its own. For example, researchers are now working on drugs to treat smoldering multiple myeloma (SMM). Affected patients have abnormal proteins in the blood and urine that, in 5 percent of cases, progress to outright multiple myeloma.9 The next rung on the ladder, a cure for cancer, may not be so pie-in-the-sky anymore.
More than a century after Charles Duell’s misguided prognostication, there is no reason to believe medical invention will ever slow down. Quite the opposite: pharma innovation is blazing more brightly than ever, with thousands of Canadians reaping the benefits of manufacturers’ investment in targeted therapies. But is it sustainable?
References:
Longer life expectancy for HIV-positive people in North America. Accessed March 1, 2019 at https://www.catie.ca/en/treatmentupdate/treatmentupdate-200/anti-hiv-agents/longer-life-expectancy-hiv-positive-people-north
Land E. Life expectancies close to 80 years for young people starting HIV meds early. BETA, Nov. 14, 2018. Accessed March 1, 2019 at https://betablog.org/life-expectancies-close-to-80-years-for-young-people-starting-hiv-meds-early/
Hepatitis C takes 15 years off the life span, raises death risk 12-fold. Hep Mag, Oct. 1, 2013. Accessed March 1, 2019 at https://www.hepmag.com/article/death-risk-24659-138388084
Goodman J. Ibrutinib provides long-term survival benefits for patients with CLL. Cancer Therapy Advisor, May 24, 2017. https://www.cancertherapyadvisor.com/home/news/conference-coverage/american-society-of-clinical-oncology-asco/asco-2017/ibrutinib-provides-long-term-benefits-for-patients-with-cll/
Peters S et al. Alectinib versus Crizotinib in Untreated ALK-Positive Non–Small-Cell Lung Cancer. NEJM 2017; 377:829-838.
Novartis receives Health Canada approval of its CAR-T cell therapy, Kymriah™ (tisagenlecleucel). Novartis press release, Sept. 6, 2018. Accessed March 1, 2019 at https://www.novartis.ca/en/news/media-releases/novartis-receives-health-canada-approval-its-car-t-cell-therapy-kymriah
BioCanRx, and partners, announce funding to manufacture first made-in-Canada CAR-T cells. BioCanRx press release, Feb. 15, 2017. Accessed March 12, 2019 at https://biocanrx.com/biocanrx-partners-announce-funding-manufacture-first-made-canada-car-t-cells
CAR T Cell Therapy Market: Market size & Forecast, 2018-2025. Polaris Market Research. Accessed March 1, 2019 at https://www.polarismarketresearch.com/industry-analysis/car-t-cell-therapy-market
Thwarting Deadly Diseases Before They Start: The New Science of Cancer Interception. Interview with Ming Qi, MD, PhD, Senior Director of Clinical Research in Hematology and Oncology at Janssen R&D. Accessed March 1, 2019 at https://www.janssen.com/blogs/the-new-science-of-cancer-interception