Gene therapy offers hope for untreatable cancers | DW News

Summary

A teenager in the UK has become cancer-free following a groundbreaking treatment for leukemia called base editing. This innovative therapy involves genetically engineering immune cells to target and eliminate cancerous ones. Six months post-treatment, the patient shows no signs of cancer, offering hope for the future of cancer treatment. Dr. David Liu, one of the inventors of base editing, explains how this technology can correct genetic 'misspellings' and has the potential to treat a wide range of diseases. The success of this case, led by Dr. Waseem Qasim at UCL, marks a significant step forward in the field of immuno-oncology.

Takeaways

• 🌟 A teenager in the UK has become cancer-free after a new treatment for leukemia called 'base editing'.
• 🧬 Base editing is a technique that involves altering the patient's DNA to fight diseases like cancer.
• 🛠️ The treatment involves genetically engineering new immune cells to target and eliminate cancerous ones.
• 🎓 Dr. David Liu, a Harvard professor, is one of the inventors of base editing and explains its potential in medicine.
• 🔍 Base editors are molecular machines that can convert one DNA letter to another at a specific sequence of interest.
• 💊 The treatment has the potential to correct genetic diseases and make precise changes for fighting diseases like leukemia.
• 🏥 Alyssa was the first patient to undergo this experimental treatment after conventional treatments failed.
• 🚀 Six months post-treatment, Alyssa's cancer is undetectable, offering hope for the future of cancer treatment.
• 🔬 The approach taken in Alyssa's treatment was innovative, focusing on reprogramming T cells to target her cancer without harming her own cells.
• 🌍 There are ongoing clinical trials for base editing in treating different diseases in various countries.
• 🤝 The leadership and scientific vision of Dr. Waseem Qureshi at UCL led the clinical trial for Alyssa's experimental therapy.
• 🔄 While Alyssa is currently cancer-free, there is always a chance of cancer returning, and base editing could potentially be reapplied if needed.

Q & A

What is the name of the new treatment for leukemia mentioned in the script?

The new treatment for leukemia is called base editing.

What does base editing involve in terms of altering the patient's DNA?

Base editing involves genetically engineering new immune cells that hunt down the cancerous ones by making precise single letter changes in the DNA.

How did doctors perceive Alyssa's leukemia before she underwent base editing treatment?

Doctors feared Alyssa's leukemia was incurable after all conventional treatments failed.

What was the outcome of Alyssa's treatment with base editing six months after the procedure?

Six months after the treatment, the cancer was no longer detectable in Alyssa's body.

What is the potential of base editing according to the doctors in the script?

Doctors believe that base editing technology could help fight a wide range of diseases beyond just leukemia.

Who is Dr. David Liu, and what is his connection to base editing?

Dr. David Liu is one of the inventors of base editing, a professor, and director of the American Institute of Transformative Technologies in healthcare at Harvard University.

How does base editing work according to Dr. David Liu's explanation?

Base editors are molecular machines engineered from natural components that can convert one DNA letter to a different letter of our choosing at a specific sequence of interest.

What is the significance of the approach taken in Alyssa's treatment as opposed to traditional cancer treatments?

Instead of attacking the cancer cells directly by changing their DNA sequence, the treatment made precise base edits in donor's T cells, reprogramming them to target Alyssa's cancer without harming her cells.

What is the current status of Alyssa's health after the base editing treatment?

Alyssa is currently cancer-free, although there is always a chance of cancer returning even after remission.

Are there any ongoing clinical trials using base editing for other diseases?

Yes, there are four clinical trials ongoing in four different countries using base editing to treat different diseases.

What was the role of Dr. Waseem Qureshi in Alyssa's treatment?

Dr. Waseem Qureshi led the clinical trial at UCL (University College London) and decided to try the experimental base editing therapy on Alyssa to give her the best chance of recovery.

Outlines

00:00

🧬 Breakthrough in Cancer Treatment: Base Editing

A teenager in the United Kingdom has become cancer-free after receiving a revolutionary treatment for leukemia known as base editing. This treatment involves the alteration of the patient's DNA. Doctors are optimistic about the potential of this technology for the future of cancer treatment. Alyssa, the patient, was the first to undergo this experimental procedure after conventional treatments failed. The treatment genetically engineers new immune cells to target and eliminate cancer cells. Six months post-treatment, Alyssa shows no signs of cancer. Dr. David Liu, one of the inventors of base editing and a professor at Harvard University, explains that base editors are molecular machines that can make precise changes to DNA, correcting genetic diseases or making changes to fight diseases like leukemia. The treatment's success in Alyssa's case is promising, but doctors remain cautious about using the term 'cure' due to cancer's evolving nature. The approach taken in Alyssa's treatment was innovative, focusing on reprogramming T cells to target cancer without harming healthy cells, which proved effective. The potential for base editing extends beyond this single case, with ongoing clinical trials for different diseases.

05:00

🌟 Hope for the Future: Base Editing's Broad Applications

The script discusses the broader implications of base editing in medical science. Dr. Waseem Qureshi at UCL led the clinical trial that included Alyssa, showcasing the experimental therapy as a new approach to treating cancer. The treatment's success has given hope globally, demonstrating the potential of base editing beyond just leukemia. The conversation with Professor David Liu from Harvard highlights the technology's versatility, as it is not limited to a single disease. There are currently four clinical trials underway in different countries, each targeting a different disease using base editing. The treatment's precision in making single-letter changes to DNA offers a promising tool in the medical field. The script concludes with an acknowledgment of the ongoing nature of cancer treatment and the importance of continued research and innovation, with base editing being a significant step forward.

Keywords

💡Base Editing

Base editing is a revolutionary genetic engineering technique that involves making precise changes to an organism's DNA. In the context of the video, base editing is used to treat leukemia by altering the DNA of immune cells so that they can target and destroy cancerous cells. This approach represents a significant advancement in the field of medicine, as it offers a more precise way to correct genetic errors that cause diseases, such as the leukemia mentioned in the script.

💡Leukemia

Leukemia is a type of cancer that starts in blood-forming tissue, such as the bone marrow, and results in an overproduction of abnormal white blood cells. In the video, a teenager named Alyssa is described as being cancer-free after undergoing a new treatment for leukemia. The script highlights the desperate situation where conventional treatments have failed, making the success of the base editing treatment particularly significant.

💡Immune Cells

Immune cells are a critical component of the body's defense system, playing a vital role in identifying and combating foreign substances and diseases. The video discusses how base editing is used to genetically engineer new immune cells that can specifically hunt down cancerous cells. This innovative approach leverages the body's own immune system to fight cancer, which is a central theme in the script.

💡DNA

DNA, or deoxyribonucleic acid, is the hereditary material in humans and almost all other organisms. It is composed of a series of 'letters' or base pairs that carry genetic information. In the video, base editing is described as a method to alter DNA by converting one DNA 'letter' to a different one, which can correct genetic diseases or be used to fight diseases like leukemia.

💡Genetic Engineering

Genetic engineering is a field of biotechnology that involves the direct manipulation of an organism's genes using biotechnology. The video script describes how doctors use genetic engineering to create new immune cells through base editing, which are then used to treat cancer. This process exemplifies the application of genetic engineering in medicine to develop personalized treatments.

💡Cancer-Free

Being 'cancer-free' means that there is no detectable cancer in the body. In the context of the video, Alyssa is described as being cancer-free after undergoing base editing treatment for her leukemia. This term is significant as it indicates a positive outcome of the treatment and a return to a healthy state, which is the ultimate goal of any cancer treatment.

💡Clinical Trials

Clinical trials are research studies conducted to determine the safety and effectiveness of medical interventions, such as new treatments or drugs. The script mentions that there are four clinical trials ongoing to treat four different diseases using base editing. This highlights the broad potential of the technology and its application in various medical fields.

💡Misspellings in DNA

In the video, 'misspellings' in DNA refer to genetic mutations or errors that can lead to diseases, such as certain types of cancer. Base editors are described as tools that can correct these 'misspellings' by making precise changes to the DNA sequence. This concept is crucial for understanding how base editing can be used to treat genetic diseases.

💡Remission

Remission is a period during which the signs and symptoms of a disease, such as cancer, have disappeared, even though the disease may still be present in the body. The script notes that Alyssa's cancer is no longer detectable, which indicates she is in remission. It's an important term because it signifies a positive treatment outcome, but also implies the possibility of the disease returning.

💡Immuno-Oncology

Immuno-oncology is a field of study that focuses on the interaction between the immune system and cancer. It aims to develop treatments that harness the immune system to combat cancer. The video discusses how the base editing treatment for Alyssa's leukemia is based on the principles of immuno-oncology, where immune cells are reprogrammed to target and destroy cancer cells without harming healthy cells.

💡Harvard University

Harvard University is a prestigious institution known for its research and innovation. The video features Dr. David Liu, a professor and director at Harvard, who is one of the inventors of base editing. His involvement underscores the credibility and significance of the base editing technology, as well as the role of academic institutions in advancing medical science.

Highlights

A teenager in the UK is cancer-free after a new treatment for leukemia called base editing.

Base editing involves altering the patient's DNA to treat diseases.

Alyssa was the first patient to undergo base editing after conventional treatments failed.

The procedure genetically engineers new immune cells to hunt down cancerous ones.

Six months post-treatment, Alyssa's cancer is undetectable.

Doctors believe base editing could fight a wide range of diseases.

Alyssa hopes her participation helps other children get cured through this research.

Dr. David Liu, one of the inventors of base editing, explains the technology.

Base editors are molecular machines that can convert one DNA letter to another.

Base editing can correct genetic 'misspellings' and make precise changes for fighting diseases.

Doctors used base editing to reprogram T cells to target Alyssa's cancer without harming her cells.

The treatment's success does not guarantee a lifelong cure, but it is hopeful for the future.

There's always a chance of cancer returning, even after remission.

Base editing's power lies in its ability to make precise DNA changes for various diseases.

Four clinical trials are ongoing using base editing to treat different diseases.

Dr. Waseem Quasim led the clinical trial for Alyssa's experimental therapy.

The success of Alyssa's treatment gives hope worldwide for the potential of base editing.

Transcripts

when tonight a teenager in the United

Kingdom is cancer free after undergoing

a new treatment for leukemia it's called

base editing and it involves altering

the patient's DNA doctors say that this

breakthrough has huge implications for

the future of treating cancer

[Music]

at the center of a medical breakthrough

after all conventional treatments failed

doctors feared Alyssa's leukemia was

incurable

with few options left Alyssa volunteered

to be the first patient to undergo an

experimental treatment known as base

editing The Cutting Edge procedure

involves genetically engineering new

immune cells that hunt down the

cancerous ones six months on the cancer

is no longer detectable in Alyssa's body

it is extremely exciting

um so obviously this is a new field in

medicine and it's fascinating that we

can redirect immune system to fight

cancer doctors say this treatment only

scratches the surface of what base

editing could achieve they believe the

technology could help fight a wide range

of diseases I know that I've helped

other children as well

that they might that other children

might be able to get cured because of

the research that I've been part in all

these people Melissa is still being

monitored just in case the cancer

returns

when Dr David Liu is one of the

inventors of Base editing he's professor

and director of the American Institute

of transformative Technologies in

healthcare at Harvard University I asked

him earlier to explain just how base

editing works

sure so so base editors are machines

molecular machines that we engineered

drawing from components in nature and in

our Laboratory

that convert one DNA letter to a

different letter of our choosing in the

vast genome they can be targeted to a

specific sequence of Interest

where they then rearrange the atoms in

one DNA base to instead become a

different DNA base so base editors can

correct the misspellings that cause

genetic disease

or make other kinds of precise single

letter changes that can be useful for

fighting disease such as the T Cell

leukemia that Alyssa had

so if you if you're correcting then the

the the mistakes and which that's what

we understand cancer cells to be anyway

the result of a mistake in cellular

reproduction are we talking about then

being a step closer to having a cure for

cancer

scientists and doctors are really

reluctant to use the word pure when it

comes especially to to treating cancer

because cancer by its very nature is

constantly evolving to try to evade our

medicines

um but in this case the doctors did not

simply attack the cancer by trying to

change the DNA sequence of the cancer

cells instead they took a a much more

clever approach uh one that is built on

the work of many many scientists in

immuno oncology as it's called in which

we make precise

base edits in a donor's T cells that

then reprogrammed those cells

to go after Alyssa's cancer without

harming Alyssa's cells so that approach

um proved to be very effective in in

treating Melissa and six months after

treatment

she doesn't have any detectable cancer

of course that doesn't necessarily mean

she's cured for life but we're all

hopeful that her cancer-free status will

last for the foreseeable future and we

know that when someone has had cancer it

is cancer-free or goes into remission

there's always a chance of cancer coming

back let's say that in the case of

illicit that that cancer does come back

using this new treatment this new

technology would you be able then to

once again go in and change just a small

portion of her DNA to once again

turn off the new cancer are we talking

about maybe having to go in maybe

several times each time the cancer is

able to regenerate itself

well the the most powerful and perhaps

the most exciting aspect of Base editing

is that it is simply a way to make

precise single letter changes in the DNA

of human cells or any other kind of cell

for that matter it's not limited to

being applied to any one disease and in

fact there are four clinical trials

ongoing right now to treat four

different diseases in four different

countries using face editing in the case

of Alyssa she already went through a

treatment a more standard treatment for

her cancer that proved unsuccessful so

she's already lived through the

unfortunate experience of receiving a

cancer treatment that then results in

the cancer coming back and thanks to the

the leadership and scientific vision of

Dr Waseem quasim at UCL University of

College London he's the one who led the

clinical trial to say okay perhaps we

should try this experimental new therapy

that he designed on Alyssa to give her

the best chance of recovering yeah well

it's definitely given people all around

the world a reason for for Hope moving

forward and a lot of that has to do with

the work that you have been doing we

appreciate your time and your insights

tonight Professor David Liu from Harvard

thank you for your interest thank you

thank you