6 Amazing Medical Breakthroughs Marking 2025.. Discover Them

SadaNews - Science continues to advance, particularly in the medical field. The past year saw numerous breakthroughs that have opened new doors of hope for millions around the world.

Earlier this month (December 2025), the World Health Organization officially announced the approval of GLP-1 treatments for managing obesity in adults, marking another significant step in a class of medications that has already transformed healthcare worldwide.

Rare Disorder

In May, scientists were able to achieve their dream of treating rare disorders in children, through the case of child K.G. Muldon, who was born with a severe deficiency in the enzyme Carbamoyl Phosphate Synthetase 1. This is an extremely rare disorder that prevents the body from metabolizing protein correctly, threatening to cause gradual brain damage.

By using Base Editing technology, an extremely precise form of gene editing that allows for the replacement of a single letter in DNA with another, a team at the Children’s Hospital of Philadelphia and Penn Medicine developed a customized treatment in less than seven months.

The child then received, over three sessions, billions of gene editing tools targeting the liver to correct the mutation. He is now growing normally, according to Forbes magazine.

Treatment Restores Hearing in Deaf Children

Regarding the realm of the deaf, most of us may not have heard of the OTOF gene, which plays a crucial role in our ability to hear as it produces the Otoferlin protein that enables sound to travel from the ear to the brain.

However, some people are born with a defect in this gene, causing them to suffer from profound deafness.

Therefore, scientists from Regeneron developed a gene therapy that uses a harmless virus (AAV vector) to deliver a healthy copy of the gene to the ear cells, enabling them to produce the protein.

A study published in October showed that the treatment resulted in significant improvement in hearing for 11 out of 12 children, with three of them reaching normal hearing levels.

In this context, Russell Lunsford from Ohio State University clarified that "once the faulty gene is replaced, hearing loss in these children can be reversed... this is the power of gene therapy."

New Hope for Huntington’s Disease

Moreover, gene therapy has also shown progress in the field of complex neurodegenerative diseases.

In September, astonishing results emerged in the treatment of Huntington's disease, caused by a single genetic mutation that leads to the accumulation of toxic protein in neurons, a devastating disease that results in dementia, paralysis, and early death.

A recent trial involved injecting gene therapy into a part of the brain called the striatum, which is the most affected area by the disease, to disrupt the toxic protein. Results indicated that the treatment reduced disease progression by 75% after three years.

Although experts like Roger Barker from the University of Cambridge emphasized the need for more data before FDA approval, they considered this "the beginning of a new wave of gene therapies targeting the root of the defect in these diseases."

Head and Neck Cancer

Similarly, the treatment of head and neck cancer witnessed the greatest breakthrough ever, following numerous failures in clinical trials.

During a meeting of the American Society of Clinical Oncology last May, the incredible success achieved by the immune treatment Pembrolizumab was discussed.

Doctors showed that administering the treatment as a first line before surgery and chemotherapy and radiation increases the chances of eradicating cancer cells and preventing their return, especially in patients with high levels of the PD-L1 protein.

In this regard, Kevin Harrington from the Institute of Cancer Research in London confirmed that "13% of patients saw a 90% reduction in tumor size after just two doses of immunotherapy before surgery."

Researchers are now working to enhance the effectiveness of the initial treatment to reduce the need for intensive surgical procedures and treatments later on.

Cholesterol and Heart

For individuals with genetic factors that lead to high cholesterol and triglycerides, the current option is to take medications like statins for life.

However, a new study published last month in the New England Journal of Medicine showed that gene editing could provide a new option.

In a phase one clinical trial, a treatment based on CRISPR/Cas9 technology successfully disabled the ANGPTL3 gene in liver cells, leading to reduced cholesterol and triglyceride levels within two weeks, with the reduction lasting for 60 days, opening the door to the possibility of developing a "one-dose" treatment for heart diseases.