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What do different cancer drugs do?
What do different cancer drugs do?

The Hindu

time27-06-2025

  • Health
  • The Hindu

What do different cancer drugs do?

The story so far: A major new investigation by the Bureau of Investigative Journalism published by The Hindu has revealed that cancer drugs shipped around the world to more than 100 countries have failed quality tests. But what are these drugs and how are they used? Cancer is a disease characterised by uncontrolled cell growth. It is caused by mutations in genes disrupting or inhibiting the normal ways in which cells divide or die. This causes cancerous cells to develop – and when a mass of cancerous cells grows, it can form a tumour. Chemotherapy drugs disrupt this process, and these medicines have transformed the landscape of cancer treatment, improving outcomes for millions of patients. The six types of drug in our investigation – cisplatin, oxaliplatin, cyclophosphamide, doxorubicin, methotrexate and leucovorin – are all chemotherapy treatments. While there are differences in exactly how these drugs work, most share a common goal: to target and kill cancer cells while minimising harm to normal cells. What is cisplatin? Cisplatin is a platinum-based drug that emerged in the 1960s from an unexpected discovery. Researchers found that a platinum-containing compound blocked the growth of bacteria, leading to the realisation that it could potentially be used to combat cancer. Cisplatin works by binding to the DNA of cancer cells, preventing them from growing and dividing. The drug is particularly effective against testicular, ovarian, bladder and certain lung cancers. Its introduction has significantly improved survival rates for many patients, whether used alone or in combination with other therapies. However, like all potent medications, cisplatin comes with side effects, including sickness, kidney damage and hearing issues. Cisplatin also suppresses the function of the bone marrow, where new blood cells are produced. The resulting reduction in white blood cells has severe effects on patients' immune systems, leaving them vulnerable to infection. What is oxaliplatin? Oxaliplatin, like cisplatin, is a platinum-based drug. It also works by binding to and damaging the DNA of cancer cells. It is used mainly to treat the more advanced stages of colorectal cancer and following surgery for the removal of colon cancer, to wipe out remaining malignant cells. Oxaliplatin comes with broadly similar side effects to cisplatin. What is cyclophosphamide? Cyclophosphamide is another key chemotherapy drug, often used to tackle disease including breast cancer, some types of leukemia (a type of blood cancer), sarcoma (cancer of the connective tissues) and advanced lymphoma (cancer of the lymph nodes). Cyclophosphamide disrupts the DNA of cancer cells. This limits their ability to replicate and mutate. It also causes a drop in white blood cells and can also cause pain when passing urine due to inflammation of the bladder. What is doxorubicin? Often referred to as the 'red devil' due to its striking colour and severe side effects, doxorubicin was derived from a type of soil bacteria called Streptomyces, and was initially explored as an antibiotic. Doxorubicin is effective against several types of cancer, including breast cancer, leukaemia, lymphoma and sarcoma. It works by interfering with the cancer cell's DNA and disrupting the replication process. It is a staple in chemotherapy protocols, despite potential side effects including heart damage, risk of infection, skin changes and hair loss. What is methotrexate? Methotrexate is very effective against various cancers, including leukaemia, lymphoma and certain types of tumour. By inhibiting the protein responsible for creating new DNA molecules, it effectively stifles cancer cell growth. Methotrexate is often given in high doses, followed byleucovorin to help protect healthy cells from damage. What is leucovorin? Leucovorin, also known as folinic acid (not to be confused with folic acid), is a form of vitamin B9. It differs from the rest of the drugs that were tested in the Bureau's investigation in that it is not technically considered a chemotherapy drug, but a companion to chemotherapy. It was first introduced in the 1950s and was designed to manage the side effects of methotrexate, for which it is still used today. Meriem Mahdi is with The Bureau of Investigative Journalism.

Acousia Therapeutics Reaches 50% Patient Enrollment Milestone in Phase 2 PROHEAR Study
Acousia Therapeutics Reaches 50% Patient Enrollment Milestone in Phase 2 PROHEAR Study

Associated Press

time29-04-2025

  • Health
  • Associated Press

Acousia Therapeutics Reaches 50% Patient Enrollment Milestone in Phase 2 PROHEAR Study

Acousia Therapeutics GmbH announced that it has successfully enrolled 50% of patients in its ongoing Phase 2 clinical trial, thePROHEAR Study, evaluatingACOU085 (INN: bimokalner)for the prevention ofcisplatin-induced hearing lossin patients withtesticular cancerundergoing chemotherapy. Cisplatin is a cornerstone in modern oncology and is used annually in more than 500,000 cancer patients worldwide. Its introduction in the late 1970s revolutionized testicular cancer treatment, improving the 5-year survival rate (from just 5% to 98%). However, its life-saving efficacy is often overshadowed by severe side effects, withpermanent, bilateral hearing lossamong the most debilitating. Studies indicate that up to80% of patientstreated with cisplatin may experience clinically-relevant hearing loss. ThePROHEAR Studyis aplacebo-controlled, randomized, double-blind, split-body trialin which participants receiveACOU085, a Kv7.4 channel activator, in one ear and aplaceboin the contralateral ear. The study is currently being conducted at13 leading German ENT university clinicsand is still actively recruiting. 'This marks a significant milestone on Acousia's journey to offer a treatment for the prevention of permanent hearing loss caused by cisplatin,' saidProfessor Hubert Löwenheim, Chair of the Department of Otolaryngology - Head & Neck Surgery at Tuebingen University and Scientific Supervisor of the study. 'Preventing this often overlooked side effect is critical for cancer patients. Notably, all PROHEAR participants who have received ≥300 mg/m² of cisplatin to date have developed ototoxicity. This is higher than reported in most, if not all, previous studies.' The PROHEAR Study is on track to complete enrollment by the end of thesecond half of 2025. For more information, ACOU085 (INN: bimokalner)is a first-in-class, small-molecule, etiology-agnostic otoprotective drug-candidate delivered using standard transtympanic administration of a proprietary, slow-release gel formulation. Ototoxic hearing loss is a typical, severe, and permanent side effect of cisplatin treatment and is a consequence of irreversible damage to the sensory cells in the inner ear, the so-called outer hair cells (OHCs). ACOU085 modulates a biologically validated target, the KCNQ4-encoded Kv7.4 potassium channel of the OHCs, and has demonstrated significant potential to reduce cisplatin-induced hearing loss and preserve outer hair cells from ototoxicity in preclinical models.

Opinion - NIH funding cuts imperil lifesaving drugs and US prosperity
Opinion - NIH funding cuts imperil lifesaving drugs and US prosperity

Yahoo

time23-02-2025

  • Health
  • Yahoo

Opinion - NIH funding cuts imperil lifesaving drugs and US prosperity

In 1972, testicular cancer was a leading killer of young men — almost uniformly fatal. Today, it has a 90 percent survival rate. The drug responsible for this miraculous reversal, Cisplatin, would never have been discovered without 'indirect funding' from the National Institutes of Health — resources used for all the essential services needed to support clinical studies. Like Cisplatin, every groundbreaking cancer treatment you've ever heard of started the same way — not in a corporate boardroom, but in a university lab where NIH indirect funding pays the staff, keeps the computers humming, and medical equipment functioning. All of this may abruptly end, given the Trump administration's plan to retroactively slash NIH indirect support to 15 percent of grants. For decades, research institutions have negotiated the rate with NIH, ranging between 25 to 70 percent. This drastic cut will deter universities from accepting NIH research awards. Universities, including my own, often supplement NIH grants by more than two times the amount with their own institutional funds, and that is at the more generous negotiated rates. It's clear that the administration's characterization of indirect costs as 'waste,' 'bureaucracy' or mere 'overhead' reflects a dangerous misunderstanding of the value of scientific research. The NIH funds basic 'high-risk and pre-commercial research' that no private capital venture or pharmaceutical company would invest in due to the long horizon for finding cures. Across the board — from Alzheimer's disease to cancer and heart disease — NIH's funding of basic research has turned out to be a spectacularly successful investment. NIH spurs awe-inspiring scientific breakthroughs and powers our economy. A recent study found that NIH supported 354 of 356 drugs approved from 2010 to 2019. That means that most of the lifesaving medications we take for granted today would not have been possible without the NIH. It is estimated that every dollar in publicly funded basic research yields $8.38 after eight years. There are few government investments that can match the health and economic benefits of NIH funding. Capping indirect funding will decimate biomedical research, which has been the engine of the U.S. economy and the envy of the world. This rash overreach will cost the jobs of thousands of highly trained researchers and those who support their work with little prospect for relocation or retraining. It will leave patients enrolled in clinical trials without access to the cutting-edge treatments already supported by their tax dollars. This myopic decision, ostensibly made to save costs, will endanger our country's health, safety and prosperity. What's more, the White House plan is flagrantly unlawful. NIH has existing contracts with 2,500 hospitals, medical institutions and universities in all 50 states. These are binding contracts that cannot be revoked without proof of violation and absent due process. A basic principle of contract law is that contracts cannot be changed or rescinded retroactively. In fact, when the first Trump administration tried to do the same thing in 2017, Congress enacted specific protections for NIH-funded contracts. The partnership between the federal government and a decentralized network of research universities has its origins in the Manhattan Project which had to rely on foreign-trained scientists. Insufficient scientific expertise became even more concerning in the 1960s after the Soviet Union's launch of the first satellite to orbit the Earth signaled that we were falling behind in the space race. The response was to develop the current system: supporting universities to develop independent research capacities, rather than having all projects run out of Washington. Our decentralized network of research universities is a vital component in driving scientific innovation and safeguarding our national security. Not only does our ability to train our own scientists make us self-sufficient, but it also provides a tremendous resource for the Department of Defense, which directs 50 percent of its basic research to universities. By reliably supporting a consistent state of scientific readiness, our research network is able to pivot quickly towards addressing new threats, whether occurring naturally or a biological attack. A federal court issued a temporary restraining order pausing the new rate cut in litigation filed by 22 state attorneys general. Universities and national academic associations just filed another lawsuit. The ongoing litigation is likely to succeed, but the damage caused by this capricious and reckless action is immediate and devastating for medical centers across the country, which have already budgeted for (and incurred obligations based on) the specific indirect cost rates that have been negotiated and formalized with the NIH. The NIH funding model has made the U.S. the world's science superpower and our universities the premier destination for students from every nation. Slashing NIH funding will not save money, but it will unravel a biomedical system that has powered our economy and protected our population for generations. Congress stopped these dangerous cuts to NIH in 2017 and needs to hear scientists' voices again to protect medical research today and in the future. Lawrence O. Gostin is a distinguished professor of Law at the O'Neill Institute for National and Global Health Law at Georgetown University and Director of the World Health Organization Center on Global Health Law. Jennifer S. Bard is a professor of Law at the University of Cincinnati. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

NIH funding cuts imperil lifesaving drugs and US prosperity
NIH funding cuts imperil lifesaving drugs and US prosperity

The Hill

time23-02-2025

  • Health
  • The Hill

NIH funding cuts imperil lifesaving drugs and US prosperity

In 1972, testicular cancer was a leading killer of young men — almost uniformly fatal. Today, it has a 90 percent survival rate. The drug responsible for this miraculous reversal, Cisplatin, would never have been discovered without 'indirect funding' from the National Institutes of Health — resources used for all the essential services needed to support clinical studies. Like Cisplatin, every groundbreaking cancer treatment you've ever heard of started the same way — not in a corporate boardroom, but in a university lab where NIH indirect funding pays the staff, keeps the computers humming, and medical equipment functioning. All of this may abruptly end, given the Trump administration's plan to retroactively slash NIH indirect support to 15 percent of grants. For decades, research institutions have negotiated the rate with NIH, ranging between 25 to 70 percent. This drastic cut will deter universities from accepting NIH research awards. Universities, including my own, often supplement NIH grants by more than two times the amount with their own institutional funds, and that is at the more generous negotiated rates. It's clear that the administration's characterization of indirect costs as 'waste,' 'bureaucracy' or mere 'overhead' reflects a dangerous misunderstanding of the value of scientific research. The NIH funds basic 'high-risk and pre-commercial research' that no private capital venture or pharmaceutical company would invest in due to the long horizon for finding cures. Across the board — from Alzheimer's disease to cancer and heart disease — NIH's funding of basic research has turned out to be a spectacularly successful investment. NIH spurs awe-inspiring scientific breakthroughs and powers our economy. A recent study found that NIH supported 354 of 356 drugs approved from 2010 to 2019. That means that most of the lifesaving medications we take for granted today would not have been possible without the NIH. It is estimated that every dollar in publicly funded basic research yields $8.38 after eight years. There are few government investments that can match the health and economic benefits of NIH funding. Capping indirect funding will decimate biomedical research, which has been the engine of the U.S. economy and the envy of the world. This rash overreach will cost the jobs of thousands of highly trained researchers and those who support their work with little prospect for relocation or retraining. It will leave patients enrolled in clinical trials without access to the cutting-edge treatments already supported by their tax dollars. This myopic decision, ostensibly made to save costs, will endanger our country's health, safety and prosperity. What's more, the White House plan is flagrantly unlawful. NIH has existing contracts with 2,500 hospitals, medical institutions and universities in all 50 states. These are binding contracts that cannot be revoked without proof of violation and absent due process. A basic principle of contract law is that contracts cannot be changed or rescinded retroactively. In fact, when the first Trump administration tried to do the same thing in 2017, Congress enacted specific protections for NIH-funded contracts. The partnership between the federal government and a decentralized network of research universities has its origins in the Manhattan Project which had to rely on foreign-trained scientists. Insufficient scientific expertise became even more concerning in the 1960s after the Soviet Union's launch of the first satellite to orbit the Earth signaled that we were falling behind in the space race. The response was to develop the current system: supporting universities to develop independent research capacities, rather than having all projects run out of Washington. Our decentralized network of research universities is a vital component in driving scientific innovation and safeguarding our national security. Not only does our ability to train our own scientists make us self-sufficient, but it also provides a tremendous resource for the Department of Defense, which directs 50 percent of its basic research to universities. By reliably supporting a consistent state of scientific readiness, our research network is able to pivot quickly towards addressing new threats, whether occurring naturally or a biological attack. A federal court issued a temporary restraining order pausing the new rate cut in litigation filed by 22 state attorneys general. Universities and national academic associations just filed another lawsuit. The ongoing litigation is likely to succeed, but the damage caused by this capricious and reckless action is immediate and devastating for medical centers across the country, which have already budgeted for (and incurred obligations based on) the specific indirect cost rates that have been negotiated and formalized with the NIH. The NIH funding model has made the U.S. the world's science superpower and our universities the premier destination for students from every nation. Slashing NIH funding will not save money, but it will unravel a biomedical system that has powered our economy and protected our population for generations. Congress stopped these dangerous cuts to NIH in 2017 and needs to hear scientists' voices again to protect medical research today and in the future. Lawrence O. Gostin is a distinguished professor of Law at the O'Neill Institute for National and Global Health Law at Georgetown University and Director of the World Health Organization Center on Global Health Law. Jennifer S. Bard is a professor of Law at the University of Cincinnati.

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