by Mona Jhaveri - June 8, 2020

When it comes to finding a cure for cancer, most people subscribe to the prevailing social contract that informally, if not subconsciously, binds citizens to elite research universities, government agencies, and major pharmaceutical companies driving the process. The informal, unofficial social contract goes something like this:

Contribute to cancer research and your contribution will buy you and your loved ones progress against cancer development, yielding cancer-fighting treatments you someday may need.

In our minds, science and medicine “works.” We assume FDA-approved medicines are readily achieved through clinical cancer research. Yet, the institutions informally controlling the discourse around this social contract do not discuss much with the public about (a) the time this takes (b) the cost it demands (c) the inefficiency of the process, (d) the actual cancer statistics against investment in university research primarily (e) health equity disaggregated by ethnicity or socioeconomic category (f) data on the actual research we fund, (g) the relationship between research advances and actual drugs developed, (h) the role of the National Cancer Institute and other major players in the industry, and (i) all the hundreds of good ideas developed through the research process that never make it through proof of concept stages due to lack of funding.

So too, cancer prevention research, disease progression, and cancer disparities is not sufficiently discussed, despite, arguably, greater cancer awareness in patient advocacy groups. For the past fifty years, since President Nixon declared war on health disparities and on cancers in the United States through the National Cancer Act, we have been believing in, and unwittingly accepting the social contract to make this “cure” available should we or our loved ones become diagnosed with say pancreatic cancer, childhood cancer, skin cancer, ovarian cancer, blood cancer, colorectal cancer, lung cancer, brain cancer, bladder cancer, pediatric cancer, or types of cancer where rare tumors exist.

The coronavirus outbreak and spread of the covid-19 pandemic has only made the war on cancer more complicated, limiting access to health care at cancer centers and common cancer screening tests. And with the economy downturn, it will likely also impact research funding, slowing down advances in cancer research even more.

In fact, we really need to speed up progress at many levels of research development such that when we hold scientific meetings we get excited about tangible progress. While we are understanding more and more about cancer screening, the genetic make-up of cancer cells or cancer biology, patient immune response to different innovations, improving cancer data sharing, cancer diagnosis, cancer news around causes of cancer, and oncology research, a new national cancer strategy is needed.

Unfortunately, the call to action “research for a cure” has been misleading Americans who wish to speed up cancer research and see progress in our war on cancer. This is because the research that runs through the research infrastructure we have deployed does not end in cures for cancer patients. Rather, our approach generates knowledge of the disease, for the most part, that once-in-a-while leads to a discovery – or potential cure to save lives with clinical trial results that would still need to be validated and commercialized before anyone is to ever benefit. Expanding research grants alone is not enough.

The process of “translating” these “once-in-a-while” research ideas to treat or prevent cancers into real solutions is complex, costly, time consuming, highly risky and fraught with insurmountable challenges. As a result, many innovations that hold promise to cure are simply not developed, and this is to the detriment of our global society. At present, the public – the ultimate client and consumer of these products – is by and large unaware of the suboptimal process the cancer industry employs.

Translating Research Into Cures

This first step in translating discoveries into cancer treatment and cures for patients with cancer begins with “protecting” an invention that has often been developed through government research funding. This phase is distinct from a “cancer drug” for specific cancers or tumor types, that is an outcome far upstream in the process. Protecting an invention such as precision medicine for a specific cancer type or tumor cells is done so that others cannot use the invention. This is done through the issuance of patents.

Patents are essentially a license provided by the government that gives the inventor a right or “title” for a set period to exclude others from making, sharing data, using or selling the invention. Most inventions require the filing of many patents that protect various aspects of the invention, such as the structure of your chemical or biological entity, the route of how it will be administered to patients, and the diseases it will be used to treat. The “intellectual property suite” is then filed in countries and regions all over the world. Usually, well before an invention has proven safety or efficacy in reducing a cancer burden, substantial investments in patent protection involving tens to hundreds of thousands of dollars over a period of years is required. All of this is unknown to the public, yet is supported by taxpayer resources.

Initial patents are often filed by the institutions that house the research laboratories, through their offices of “technology transfer.” Under the Bayh-Dole Act, universities have the right to license federally funded innovations and generate licensing revenue. The purpose of the Bayh-Dole Act was to incentivize universities to support the commercialization of discoveries resulting from their in-house research.

While some universities rake in substantial licensing revenue, most are simply not equipped to market these assets to the biotech industry – the key intermediary in translating basic research into cancer cures through further development of patented technologies. Universities also struggle to incentivize and support their own students and staff to spin out biotech companies for the purposes of commercializing their inventions, as a means to address what is widely understood as an inefficient system.

In fact, counterintuitively, the skill sets required to advance an innovation is not shared by academic researchers. Drug development draws on multidisciplinary teams with scientific know-how as well as expertise in intellectual property, FDA regulations, market competition, readiness and penetration. This “skill set gap” between academic researchers and other experts in the chain leading to medical cures, is a major reason as to why great ideas born in an academic research lab are slow to progress into useful solutions for the public.

That said, even if optimally structured teams are assembled for a biotech company to launch and carry out its product development activities, there is a second real issue that precludes progress: lack of funding. Funding opportunities are limited to support the development of early stage biotech innovations.

Typically, the healthcare sector of the investment community provides key funding for biotech innovations in exchange for equity in the company. These include angel investors, family offices, and venture capital firms. However, most investors shy away from companies with assets that are considered “early stage” where risks for failure are high. Specifically, investors do not want to advance capital into companies where products are still proving their principle, or have not been tested in humans for key parameters such as safety and efficacy. Investors would rather wait until innovations have been “de-risked” before they support a fledgling company.

Unfortunately, since there are so few funding streams to support early stage biotechs, too many promising innovations linger in the “valley of death” – a metaphorical funding bottleneck where great ideas go to die because they lack the requisite funds to move forward. At present, the public has little understanding that the valley of death exists and is a major bottleneck to generating a range of cancer cures and better drug targeting.

Speeding Up Progress

The way to speed up progress in cancer research is to address the disconnects and breakdowns in the product-development process. Under the current structure for product development, a first step in advancements in cancer research is for research organizations/universities to more ably lead the effort by increasing the capacity of staff and students in the key areas of biotech commercialization. At present this is lacking. This would include: knowledge of patents, the FDA regulatory process, and better understanding of how products in development become market ready. Further, university technology transfer offices need to actively seek out biotech companies that are interested and capable of advancing discoveries born in their university labs.  The role of technology transfer must involve building strategic relationships with those who can actively commercialize.

For example, the SPARK program – https://sparkmed.stanford.edu/ – is a unique partnership between university and industry experts. SPARK provides a cost-effective model to generate proof of concept using out-of-the-box academic approaches combined with industry standards. Importantly, SPARK provides access to specialized knowledge and technical expertise regarding drug and diagnostic development, dedicated core laboratory facilities, and sources of funding to support translational efforts. Sixty-two percent of Spark projects are in clinical trials or have been licensed to new or existing companies or transferred to industry, a case study found, a much higher proportion than occurs with academic research discoveries in general. The model has spread to more than 60 universities and colleges in 22 countries, with many more to go.

Finally, access to funding to help early stage innovations move forward is equally critical. While there are few equity based investment mechanisms for nascent innovations, there are only a handful of “non-dilutive” or grant funding programs in biotech.  These include, Small Business Innovative Research (SBIR) grants and grants from state economic development programs. At present there are very few philanthropic grant systems for biotech, surprising given that biotech industry revenues in 2020 are over $113 Billion.

Sound Affects is another, up-and-coming option for early stage biotech to raise non-dilutive funds. Sound Affects is a 501(c)3 charitable crowdfunding platform for biotech in the oncology space. Sound Affects partners with the music industry to help bridge the divide between biotech entrepreneurs working on healthcare solutions and the public that is seeking progress in the war on cancer and cancer health disparities. Through the Sound Affects platform, individuals can give their dollar to help advance the development biotechnologies that would otherwise disappear in the valley of death.

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