For sickle cell disease and beta thalassemia using gene editing. So far very promising results in curing the disease with one application. Conditionally approved in UK, with Europe and US FDA review ongoing. Remarkable how life-changing it is for people who have lived with chronic episodes of debilitating pain their entire lives to suddenly be free from it. Cost, about 2 million, and access will be a big factor with the economics of a single treatment versus a lifetime of non-curing treatments. UK first to approve CRISPR treatment for diseases: what you need to know
A single infusion of a gene-editing medicine may control inherited high LDL cholesterol. This is an example of a recently tested CRISPR modification that can gene edit to permanently fight high cholesterol.
Yes, moving into a large disease indication will test how successful curative treatments will be in crowded space with multiple therapeutic options
Cancer treatments aren't far behind. It is what the covid vaccine people were focused on before covid. Hopefully volums will drop price as it treats many more patients
While not gene editing, gene therapy for hematological cancers have already been approved, like Yescarta and Kymriah, and proven to be highly effective as a last option for patients with lymphomas. It's a good question on volume and cost. Most of the programs target rare diseases with few good options for patients with the exception I-boy noted above which would have higher volume.
I am VERY skeptical of these types of treatments and claims. On one hand you have a potential cure and on the other hand you have a potential zombie apocalypse/Planet of Apes scenario.
The efficacy endpoints are pretty clear, patients no longer have painful episodes caused by the disease. Is your fear of new health technology due to lack of understanding how they work?
Seems pretty straightforward. A person has a genetic propensity to high cholesterol. For a rare variant it can be deadly. There is also a rare variant where people have hardly any LDL and those people don’t get heart disease and they are otherwise healthy. There are drugs that can correct for this, and there are some in testing in pill form that are more effective than statins and have no known side effects. The genetic approach accomplishes the same thing, except permanently. If I had the hypercholestolia or whatever it’s called, I’d gladly take the genetic alteration vs likely die early due to clogged arteries. Down the road it could also be a treatment for normal people with high cholesterol, but I suspect that will be many years in the future.
F’in with genetics to combat cholesterol seems a tad bit risky. My 90 year old mother, who was a RN, has had high cholesterol for the last 50 years. No meds. She has zero blockages and can still kick my butt. I am a hard no on the risk for reward here. Now, sickle cell, Parkinson’s or other bad diseases, I support the risk for reward.
In actuality, it's the patients, parents and advocacy groups pushing for treatments, especially for rare diseases where there are few options. For people living with SCD, this is a life-changer. Here's an article on a patient in the trial, to give you that perspective: https://www.washingtonpost.com/science/2023/04/28/sickle-cell-disease-treatment-gene-therapy/ There are known and unknown risks with any novel treatment. Unfortunately, no one has a magic crystal ball to predict every outcome and there's always a risk to benefit to consider based on the testing before a clinical trial, and patients are well-informed before taking part in trials. For gene therapy trials in particular there is a mandatory 15-yr follow up for patients, due to potential for cancer, although the risk is very low. However, to just generically say something bad may happen some day, is quite the self-defeat and taking that perspective we would never have treatments for any disease.
that is not what I am saying at all. I just have trepidation about messing with Genes/DNA. For terminal diseases and life debilitating illnesses I support, always with caution.
