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There’s No ‘I’ in Drug Development

By Get Science Team, original article here

Scientific collaborations between researchers, organizations and universities can help yield better results for patients.

In the wake of his son Beau’s untimely death from brain cancer in 2015, Vice President Joe Biden launched the “Cancer Moonshot” initiative to accelerate cancer research. In a visit to Duke University right after the announcement of the initiative, Biden explained that his aim was to cut through red tape and enable greater cooperation in the search for cancer treatments. Biden joked that he wasn’t smart enough to be a scientist, but that after four decades in Washington, he was very good at bringing people together and marshalling federal resources.

That very philosophy—that people must use their different and complementary talents to create an ecosystem of health care innovation—is why pharmaceutical companies often work with academia, patient advocacy groups, biotech firms and other industry partners to help make a bigger impact on patients’ lives. In fact, as part of the Cancer Moonshot goal of accelerating immunotherapy research, a number of pharmaceutical companies shared a U.S. National Institutes of Health (NIH) grant that totaled $215 million.

But collaboration is also crucial to the scientific research process on an even more basic level, as researchers learn from each other’s work in order to move their fields forward. Nowhere is this truer than when “basic” scientific research—which focuses on, for example, fundamental questions about a biological system and how it works—is translated into research that applies those learnings to a real-life situation. A recent review article in The New England Journal of Medicine concluded that “bridging the gap between promising laboratory observations and the development of effective therapies remains risky and expensive, with fewer than 1 in 10,000 early translational programs successfully achieving Food and Drug Administration approval.” By bringing together institutions and companies in collaborations, Pfizer hopes to increase the chances of success and catalyze health innovation.

Read on for three examples of how scientific collaborations work.

A Breast Cancer Treatment Breakthrough

British scientist Sir Paul M. Nurse gained fame after being awarded the 2001 Nobel Prize in medicine, but his journey to this recognition in Sweden started in 1987, when he identified CDK (cyclin-dependent kinase), a protein that regulates different phases of the cell cycle.

He couldn’t have done it without the pioneering work of American scientist Leland Hartwell, who discovered more than 100 genes that coordinate the four-stage cell cycle, including the version of CDK found in yeast. Meanwhile, Nurse’s fellow Brit, Tim Hunt, was studying sea urchin eggs when he found cyclins, the proteins that “turn on” the function of CDK. This is a prime example of the collaboration inherent in the translation of basic to applied research.

Hartwell and Hunt shared the Nobel Prize with Nurse for discovering these key molecules that regulate how cells multiply, thereby giving clues as to what goes awry as cancerous cells reproduce.

In the public realm, the NIH had provided more than $41 million to support Hartwell’s research.

In the private realm, Pfizer researchers discovered a selective inhibitor of specific CDKs (4 and 6), and began working to see if it could help delay the progression of cancer. Pfizer provided material and funding to labs at the UCLA Geffen School of Medicine and the Revlon/UCLA Women’s Cancer Research Program to test the compound’s efficacy.

Today, tens of thousands of patients have been treated with CDK 4/6 inhibitors and participate in ongoing clinical trials involving academia, the government and the pharma industry to evaluate their use in earlier stage and different types of cancer.

A New Partnering Model for Academia and Industry

While pharmaceutical companies are highly skilled at shepherding potential therapeutic compounds through the lengthy process of determining whether such compounds may ultimately be safe and effective medicines, the work of understanding basic biological mechanisms and disease pathways often also happens in university labs.

For its part, Pfizer created the Innovative Target Exploration Network (ITEN) model to generate novel drug targets in emerging areas of science that could complement research within Pfizer’s core areas of expertise. “We’re really looking for specific projects that we know have a strong strategic fit within our organization,” says Uwe Schoenbeck, Pfizer’s head of external research and development innovation. “Ideally, you would like to achieve something together that neither of us could do alone.”

As an early example of that model, partners from the University of Cambridge and the University of Oxford in the UK are focusing on a potential therapeutic pathway that degrades certain disease-causing proteins by using specific enzymes. And the University of Texas Southwestern is focusing on mouse forward genetics to identify novel drug targets in cancer and metabolic disease. Each collaboration will be managed by a researcher from Pfizer who will liaise with principal investigators at the respective universities.

A Lupus Patient Care Innovation

When singer Selena Gomez came forward to her fans about receiving a kidney transplant to help treat her lupus, they responded in full force. Gomez has more than 100 million Instagram followers, currently the most in the world, and her appeals to raise money have brought in about $500,000 to the Lupus Research Alliance, according to the organization’s president and CEO, Kenneth Farber.

Lupus, an autoimmune disease that most often strikes women between the ages of 15 and 44, can cause damage throughout the body. Symptoms vary widely, which makes managing the disease challenging. The Lupus Research Alliance is the world’s leading private funder of lupus research, powered by donations like those from Gomez and her fans.

Recently, the Alliance announced the first clinical trials via its Lupus Clinical Investigators Network. In partnership with Pfizer, a study called Validation in Lupus of an Electronic Patient Reported Outcomes Tool (VALUE) will test a custom smartphone app that will let lupus patients report their symptoms in real time, rather than intermittently at the doctor’s office. More frequent and accurate reporting by patients can give better information to their healthcare providers, and involve them more in their own treatment.

The app was designed with input from Pfizer, lupus patients, AMPEL BioSolutions and Tata Consultancy Services Ltd. It was built using an open-source framework introduced by Apple, called Apple ResearchKit.

As these examples show, when it comes to medical advancements, shooting for the moon truly takes a village.