With hundreds of promising immunotherapies currently in development--like ONCR-001, the lead oncolytic virus candidate from Oncorus--it’s more important than ever for patients with cancer to consider participating in clinical trials. According to the Cancer Research Institute, only about 3 percent to 6 percent of eligible individuals take part in clinical trials for cancer therapies. This low participation rate slows down the clinical development process significantly, and means that valuable new treatments can take much longer to reach the people who need them.For patients who are thinking about participating in an immunotherapy clinical trial, the Cancer Research Institute offers some helpful information on how these treatments differ from other therapeutic approaches, and what prospective trial participants should look for. One of the most important things to understand is that immunotherapy is frequently used as a complement to or in combination with other treatments. This means that immunotherapy trials often require that patients be treated prior to or concurrently with the present standard of care. However, because immunotherapy does not have the same toxic side effects as conventional chemotherapies, participating in an immunotherapy trial may involve a lower degree of toxicity.In addition, prospective participants should be aware that, while immunotherapies are safe and have demonstrated significant potential for curing “incurable” cancers, there is still much that remains to be discovered about them. This means that immunotherapy trials may require a more intensive level of participation, such as more visits over a longer period, or a greater frequency of certain procedures. Furthermore, immunotherapies typically take longer to have an effect than other therapies.Patient participation in clinical trials is a key cornerstone of innovation to advance new therapies. Well-designed studies offer patients excellent care while allowing access to standard regimens in addition to the investigational product being studied.
Color blindness prevents over 10 million Americans from accurately distinguishing among certain primary colors, and at present there is no treatment or cure. The genetic disorder stems from a mutation on the X chromosome. Not only can it cause serious complications in everyday life, making tasks such as night driving more difficult, but it can also prevent people from pursuing certain career paths, such as aviation and electrical work. In early 2015, the medical research startup Avalanche Biotechnologies partnered with the University of Washington to improve upon a new gene therapy technique originally developed by Jay and Maureen Neitz, the lead researchers at the university’s Neitz Color Vision Lab. The couple has dedicated decades to advancing human health, and in 2009, they succeeded in curing color blindness in squirrel monkeys by surgically introducing new genes into the retina cells responsible for facilitating color vision. The team was eager to use their findings to help correct human vision, but the risk associated with their early-stage technique necessitated the development of a nonsurgical approach. In 2012, Jay and Maureen Neitz met Avalanche founder and then CEO Thomas Chalberg at a scientific conference. Dr. Chalberg’s firm had recently begun to optimize and commercialize a nonsurgical technique allowing for the delivery of genes via injection into the eye’s gelatinous vitreous layer. The three immediately set about collaborating to develop a genetic therapy for color blindness.
In 2015, Avalanche Biotechnologies, Inc., announced a partnership with the University of Washington in Seattle to develop genetically based treatments for color blindness. Using its proprietary platform, Ocular BioFactory, the Bay Area company collaborated with the university to explore innovations in the treatment of red-green color blindness, often categorized as CVD, or color vision deficiency. To further the project, two color vision experts from the university were scheduled to join the company’s scientific advisory panel. Avalanche also established a public education website, ColorVisionAwareness.com. The academic-corporate partnership sought to expand on UW research on gene therapies for color blindness, already detailed in a peer-reviewed article in the scientific publication Nature.About 10 million Americans are affected by color blindness, with males far outnumbering females. In fact, about 8 percent of males exhibit some form of color vision deficiency. Avalanche’s innovative BioFactory technology makes it possible to specifically target the human retina to deliver genetically modified virus components, which offer the opportunity to pioneer in the treatment of color blindness and other ophthalmologic conditions.
Due to the procedural rigor required by the medical and scientific communities, biotechnology companies face a unique set of challenges on the fundraising circuit. Here are three tips to help biotechnology entrepreneurs acquire funding for their business ventures. Focus on the science: In biotechnology, investors typically want to hear about the specific scientific proposition underlying the company. Entrepreneurs should always bring data to investor meetings and be honest about what they do and do not know. Balance youthful energy with experience: As with almost any other business, experienced leaders can add a great deal of value to a business. However, energy, passion, and youthful enthusiasm also plays a large role in a company’s ability to grow, recruit talent, and develop innovative solutions. Investors usually like to see both aspects in a biotechnology company.Research your investors: In many cases, biotechnology investors like to focus their activities on specific niches, including early stage, reformulations, and single asset plays. Consequently, entrepreneurs should research their investors and avoid wasting resources on deals that are unlikely to happen.
As any entrepreneur will tell you, raising money is an indispensable part of all stages of a company’s life cycle. Due to the unique demands of biotechnology, entrepreneurs in this sector face a number of unique fundraising challenges. To overcome fundraising obstacles, biotechnology entrepreneurs have begun using crowdfunding more and more in recent years. A popular alternative to more traditional funding sources, such as universities, crowdfunding relies on social influence and networking to raise money. While crowdfunding is commonplace for most tech entrepreneurs, biotechnology entrepreneurs with an academic background may not feel as comfortable in this arena. Nevertheless, crowdfunding has the potential to help entrepreneurs grow their companies on their own terms.Another useful fundraising source for biotechnology entrepreneurs involves corporate incubators, which support small biotech companies. Although some incubators ask for an ownership stake in the company, many do not require any type of equity investment. Entrepreneurs using this funding method benefit from state-of-the-art research facilities, as well as networking opportunities with fellow entrepreneurs.
An innovative cancer treatment that utilizes the body’s own immune system to fight cancer cells, immunotherapy has emerged as one of the most promising treatments of the past several years.
Unlike targeted therapies, which target a specific pathway thought to be involved in cancer growth, the goal of cancer immunotherapy is to activate the body's immune system. As they grow, many tumors gain the ability to suppress the immune system by turning off nearby T-cells. A new class of immunotherapies called checkpoint inhibitors help "release the brakes" of the immune system, by allowing T-cells to operate efficiently. A number of checkpoint inhibtors, including those against CTLA4, PD-1, and PD-L1, are now avaialble, and many others are being developed.
Another class of cancer immunotherapies being developed are the oncolytic viruses. These are viruses that have been genetically engineered to target and kill cancer cells. In addition to directly attacking cancer cells, oncolytic therapies activate the immune system by sensitizing and activating T-cells against the tumor. Imlygic, developed by BioVex and Amgen, is the first oncolytic virus to be available, approved by FDA and EMA in Q4-2015. Results have been very promising, particularly in combination with checkpoint inhibitors like CTLA-4 and PD-1.
Scientific advancements are helping people with colorblindness to see the world more vividly.Researchers are working on several fronts to combat color blindness, a genetic condition that primarily affects men, reducing color vision to as few as two colors. People with colorblindness typically have photopigments in their eyes that are less sensitive to certain colors and cannot translate those hues to the brain. Husband-and-wife researchers Jay and Maureen Neitz of the University of Washington have used retinal surgery and gene therapy to treat color blindness in squirrel monkeys. Their method, which is being developed for humans as the result of a partnership with the company Adverum (formerly Avalanche Biotechnologies), replaces a defective gene with one that functions correctly. Currently, there are no treatments available to help people with color blindness. Although some claim that spectacles can help, they have been tested in clinical trials, and no available data supports their use.
