Immunotherapy and niche biotech companies battle rare cancers

People in Kentucky with rare forms of cancer often face limited options for treatment. The rarity of their diseases limits the number of patients that scientists can study. The low numbers of patients mean that fewer researchers focus on their diseases, and little data is collected about their tumors. Advances in immunotherapy drugs and small biotechnology companies that choose to specialize in rare diseases have begun to create hope for patients afflicted with rare cancers.

Multiple immunotherapy drugs target DNA within cancer cells. They reduce the cells' ability to repair themselves and thereby diminish the tumor. Tumors for rare cancers sometimes have genetic components similar to tumors for more common cancers like colorectal or breast cancer. This similarity makes them vulnerable to the immunotherapy drugs designed to treat common cancers.

Additionally, small biotechnology companies are stepping into the rare cancer arena to develop niche drugs. The market space has been left relatively open by large pharmaceutical companies that tend to pursue drugs needed by large numbers of people. To encourage these large companies to apply resources to rare diseases, the Food and Drug Administration offers financial incentives for the development of treatments for diseases affecting no more than 200,000 people.

Although limited treatment options trouble people with rare diseases, they often face difficulty getting an accurate diagnosis in the first place. Physicians might not recognize their disease or fail to test for rare conditions. A person who missed treatment opportunities because of a failure to diagnose or misdiagnosis might suffer medical harm. A conversation with an attorney might be appropriate if the person wants to learn about medical malpractice. An attorney may investigate the person's care to see if it met legal standards for thoroughness. If it did not, then an attorney may prepare an insurance claim or lawsuit.

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O'Brien Batten & Kirtley, PLLC
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