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Related Concept Videos

Treatment Resistant Cancers02:56

Treatment Resistant Cancers

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Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
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Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
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Tumor Immunotherapy01:27

Tumor Immunotherapy

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Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
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Cancer Therapies02:49

Cancer Therapies

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Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
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Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
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Tumor Progression02:07

Tumor Progression

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Tumor progression is a phenomenon where the pre-formed tumor acquires successive mutations to become clinically more aggressive and malignant. In the 1950s, Foulds first described the stepwise progression of cancer cells through successive stages.
Colon cancer is one of the best-documented examples of tumor progression. Early mutation in the APC gene in colon cells causes a small growth on the colon wall called a polyp. With time, this polyp grows into a benign, pre-cancerous tumor. Further...
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Related Experiment Video

Updated: Dec 12, 2025

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies
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Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies

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Developing therapies for rare tumors: opportunities, challenges and progress.

Diana Bradford1,2, Karlyne M Reilly2, Brigitte C Widemann2

  • 1Department of Hematology/Oncology, Children's National Medical Center, Washington, DC 20010, USA.

Expert Opinion on Orphan Drugs
|August 9, 2020
PubMed
Summary

Developing effective therapies for rare tumors requires collaboration. Advances in clinical trial design and molecularly driven treatments offer new hope for these challenging diseases.

Keywords:
BASKET trialsmolecular subtypesorphan drugsumbrella trials

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Area of Science:

  • Oncology
  • Rare Diseases
  • Clinical Trials

Background:

  • Rare tumors constitute a significant portion of adult and pediatric malignancies.
  • The rarity of these tumors presents challenges in understanding pathophysiology, clinical characterization, and therapeutic development.

Purpose of the Study:

  • To review advances in rare tumor research and treatment.
  • To highlight successful collaborative efforts and novel clinical trial designs.

Main Methods:

  • Review of recent advances in rare tumor research.
  • Focus on pediatric oncology collaborative groups and molecularly driven trials.

Main Results:

  • Novel clinical trial designs and molecularly driven treatments have led to new therapy approvals for rare diseases.
  • Collaboration among researchers, patient groups, industry, and regulatory agencies is crucial.

Conclusions:

  • Continued scientific progress and interdisciplinary collaboration are essential for advancing rare tumor therapy development.
  • Examples from pediatric oncology demonstrate the success of collaborative models.