BioMedWire Editorial Coverage: Recent developments in gene therapy hold out hope for the treatment of a wide range of diseases, including fatal lung cancer.
Genprex Inc. (NASDAQ: GNPX) (GNPX Profile) is focused on using gene therapy to tackle lung cancer, developing new drugs that can be used alongside existing treatments. Pfizer Inc. (NYSE: PFE) has created a state-of-the-art research center focused on gene therapy treatments, which it uses for illnesses including cancer. Gilead Sciences Inc. (NASDAQ: GILD) is using biotechnology to tackle a range of challenging diseases, including HIV and hepatitis. Sarepta Therapeutics Inc. (NASDAQ: SRPT) is developing 20 different genetic treatments for a wide range of diseases. Merck & Company Inc. (NYSE: MRK) has treatments in development for both prostate and lung cancer, offering further hope for those battling potentially fatal diseases.
- Despite multiple available treatments, lung cancer usually remains deadly.
- Gene therapy provides a different way of treating this disease, altering the cancer’s cells.
- Bringing these treatments to market involves a complicated process that must be carefully managed.
Battling Lung Cancer
Despite huge public awareness and a decrease in smoking, lung cancer remains one of the most common types of cancer and is a major killer in many countries. An estimated 2.09 million people were affected by lung cancer worldwide in 2018, and 1.76 million of them died. While prevention has improved, survival rates remain low once the disease takes root.
Survival rates for late-stage lung cancer have improved little in recent decades, and better treatments are the dream of all involved with the disease, from researchers and doctors to patients and their loved ones. In a bid to beat the cancer, companies are now turning to new technologies such as gene therapy. But the road to success is a long, challenging one.
Tackling cancer has become a huge issue for modern governments, charities and health-care businesses. Companies such as Genprex Inc. (NASDAQ: GNPX) have put the whole focus of their operations on tackling the disease. Whether aimed at curing the disease or extending the lives of patients, these treatments are varied and often innovative. But the very nature of cancer, in which a body’s own cells destroy it, makes this difficult.
A number of approaches are commonly recommended by cancer experts. These include surgery, removing cancerous tissue in hopes of stopping it from spreading; radiotherapy, in which radiation is used to contain and kill cancerous cells; and chemotherapy, in which drugs are deployed. All can be effective in the right circumstances, and they are often at their most effective when combined.
In efforts to advance the battle against cancer, companies and researchers often gather at events such as the Sachs Annual Immuno-Oncology BD&L and Investment Forum held earlier this year, at which companies such as Genprex presented information about their progress. The cross-fertilization of ideas is as invaluable as the crossover of treatments, allowing researchers to learn from each other’s progress and to seek the funding they need to bring treatments to market.
Genprex has been successful in securing this vital funding, obtaining monies from a variety of sources, including a successful IPO and a $10 million private placement. This success has allowed the company to press forward with research in one of the boldest new forms of treatment: gene therapy.
Gene Therapy for Cancer
Imagined in the 1970s and first carried out in the 1980s, gene therapy is one of the newest frontiers in medicine. The therapy involves the delivery of nucleic acids, the tiny building blocks of life, into human cells. This can rewrite the genetics of cells, removing parts that are causing ill health and strengthening those that fight disease.
Gene therapy can be used against cancer in a variety of ways. Tumor suppressor genes can be introduced to rapidly reproducing cancerous cells, slowing down or halting their spread. Adenoviruses can be used to destroy cancer cells. Enzymes can even be introduced into cells to make them more susceptible to chemotherapy drugs, improving the effectiveness of existing treatments.
The details of treatments vary and are often very specific. For example, Genprex’s drug candidate Oncoprex(TM) uses electrically charged nanoparticles to deliver its treatment directly to cancer cells, avoiding healthy cells. Once taken into a cancer cell, Oncoprex’s TUSC2 gene creates a protein that can restore damaged functions in a cell.
Repairing a cell might sound like a bad idea when that cell is cancerous, but the problem with cancer cells stems from the fact that they are broken. If the cells were healthy to begin with, they wouldn’t damage the body that produces them. Oncoprex restores pathways in the cell that lead to natural cell death, while interrupting those that lead the cell to replicate. This means that cancer cells stop spreading so fast and start dying.
A number of companies have emerged with the aim of applying gene therapy to specific diseases. For example, Genprex specializes in tackling non-small cell lung cancer through its developmental drug Oncoprex. But these treatments don’t exist in isolation, and while each company’s developments often result in the profits that come from hard work, they also strengthen the knowledge base available as the company works to find a cure.
Commercializing Cancer Treatments
For many in medicine, commercialization is a dirty word, dealing as it does with the financial value placed on human lives. But commercialization of cancer drugs represents far more than this. It represents the entire process of testing a treatment and ensuring that it can be effectively replicated and safely used.
This process is complex, and it is vitally important that a company understands and controls the steps involved if the process is to succeed. This leads to the public announcement of commercialization plans to demonstrate a company’s work.
For example, Genprex recently published its milestones for the development of Oncoprex over the next year. These include working out details of a newly optimized manufacturing method, completing the development of the drug’s delivery system, and recruiting both sites and patients to take part in clinical trials of the drug. As Oncoprex is designed to work in combination with other cancer treatments, this will involve contracting for multiple trials to establish its safety and effectiveness not just alone but alongside other treatments such as immunotherapy.
Any such program is an attempt to manage uncertainties. What sites will be available for testing? How many patients will be willing to participate? What will the results of the trials be? By planning for these questions and the steps involved in tackling them, a company such as Genprex can create a smoother path to market for its treatments and hasten the arrival of better care for patients.
Developing Drugs and Beyond
Genprex is not alone in its efforts to find effective treatments for some of the world’s terminal diseases.
One of the world’s leading biopharmaceutical companies, Pfizer Inc. (NYSE: PFE) is heavily involved in developing cancer treatments, working in areas such as metastatic hormone-sensitive prostate cancer. Like Genprex, Pfizer is exploring the potential of gene therapy, and it is dedicating substantial resources to the challenge. The company recently announced the investment of half a billion dollars in a state-of-the-art gene therapy facility in North Carolina, where it will work on highly specialized, one-time gene therapies using custom-made, recombinant, adeno-associated viruses to deliver cures.
Gilead Sciences Inc. (NASDAQ: GILD) has been turning its biopharmaceutical expertise to dealing with a variety of diseases, including HIV and hepatitis C. The company recently invested heavily in Galapagos, a company developing innovative small-molecule medicines. By combining expertise across the two companies, Gilead Sciences will be able to develop more innovative medicines. “We are excited to close this unique agreement, which will generate both long-term strategic value and mutual, immediate benefits,” said Daniel O’Day, Chairman and chief executive officer of Gilead. “The collaboration reflects Gilead’s intent to grow our innovation network through diverse and creative partnerships.”
Sarepta Therapeutics Inc. (NASDAQ: SRPT) is also working on treatments for a range of diseases, with more than 20 gene therapies in its development pipeline to tackle ailments including muscular dystrophy. Like Genprex, Sarepta has been presenting its findings at conferences, increasing awareness of its achievements and providing opportunities to seek funding. The company is currently seeking accelerated approval of its golodirsen injection to treat Duchenne muscular dystrophy, a process that has highlighted the complications of commercialization. After the FDA raised unexpected concerns, the company is moving to address these and so bring its treatment to patients as quickly as possible.
A global health-care company with more than 125 years in the business, Merck & Company Inc. (NYSE: MRK) has more than 30 treatments currently going through development and review. Among these are several cancer treatments, such as one for prostate cancer, which has recently shown good results in testing. Merck is bringing hope to lung cancer patients through its KEYTRUDA treatment. The company has been carrying out research into the treatment’s effectiveness against non-small cell lung cancer, evaluating its use both on its own and in combination with other therapies. The results show a strong foundation for the treatment of cancer.
Like other diseases, lung cancer faces a growing range of treatments, not least from gene therapy. Through these treatments, and rigorous processes to bring them to market, the lives of patients are being put in safe hands.
For more information on Genprex, visit Genprex Inc. (NASDAQ: GNPX)
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