gene therapy
sa smith | April 14, 2015One of my favorite stories is the tale of a GMO virus deployed to treat Hemophilia B:
Gene therapy for Hemophilia B
... Hemophilia B is a disease in males caused by point mutations/deletions/etc in the clotting Factor IX gene. If you dont make Factor IX, you wont clot properly, and will have all of the health issues we associate with hemophilia.
... The scientists in this paper took six Hemophilia B patients, and treated them with an Adeno-Associated Virus-8 (two at low, medium, and high doses of virus) that contained a functional copy of the Factor IX gene.
Four of them of them bumped up…
sa smith | April 8, 2015You all know me.
There are two things I really love:
Studying HIV
Using viruses for gene therapy
One would think I would be over-the-moon about the FDA approving human trials for a gene therapy to stop HIV. HIV! Gene therapy! YAY!!
With HIV Cure as the Goal, Gene Therapy Research Expands
When this line of research initially emerged, I WAS super excited:
GMO in GMOs used to make GMO cells to treat HIV
Go read that.
Now, just to be clear, that was in no way a 'functional cure'. No one in that small trial was 'functionally cured'. But I still thought it was a great, creative step in a positive…
sa smith | December 9, 2014Five kids in the first trial.
Then eleven.
Now thirty (ultimately 39):
Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia
Slowly but surely, HIV genetically modified to genetically modify relapsed acute lymphoblastic leukemia (ALL) patients T-cells are prolonging (saving?) kids lives.
Ive written about this treatment a couple times before:
‘Dismal prognosis’ with leukemia? Nothing a GMO virus cant fix.
GMO virus vs B-Cell Acute Lymphoblastic Leukemia: Why is this not a standard therapy yet??
Basically, scientists get HIV to teach cancer patients Cytotoxic T-cells how to…
carlyo | November 13, 2014The ‘Nifty Fifty (times 4)’, a program of Science Spark, presented by InfoComm International, are a group of 200 noted science and engineering professionals who will fan out across the Washington, D.C. area in the 2014-2015 school year to speak about their work and careers at various middle and high schools.
Meet Nifty Fifty Speaker Dr. Jordan Green
As a researcher, Jordan Green, Ph.D. is on the front lines of creating innovative technologies and therapeutics that can directly benefit human health.
Specifically, this involves employing techniques in cellular engineering and nanobiotechnology…
sa smith | March 27, 2014You all might have heard about 'delta32' or 'delta-CCR5' people in association with HIV infection.
People who naturally, by chance, have deletions and mutations in the CCR5 gene of their DNA dont make functional CCR5 proteins. It doesnt appear to be 'a big deal', and people who have this particular mutation seem to live normal lives. But the absence of the CCR5 protein means that in these people, HIV does not have one of its favorite co-receptors (note: there are others! CCR5 is just HIVs favorite!). Thus people with this phenotype are resistant to HIV infection.
That is SUPER for them... but…
sa smith | February 24, 2014WHY IS THIS NOT STANDARD THERAPY YET?
Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia
I wrote about this last year:
‘Dismal prognosis’ with leukemia? Nothing a GMO virus cant fix.
In the previous study, scientists took cytotoxic T-cells from five B-ALL patients, and infected them with a genetically modified virus. This GMO virus had a genome that essentially contained a 'cheat sheet' for teaching those CTLs how to kill B-ALL cancer cells. Four out of the five B-ALL patients recovered enough that they could get a bone marrow transplant.
In…
sa smith | February 18, 2014Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial
1. Start with HIV-1s 'country cousin', Equine Infectious Anemia Virus (EIAV). EIAV is a lentivirus, like HIV, so it is a good gene therapy virus. Lentiviruses are a bit more trustworthy than the gamma retroviruses (not as likely to cause insertional mutagenesis/cancer), plus the scientists made some modifications to the LTRs to keep them from accidentally over-expressing nearby genes.
2. Scoop out EIAVs genomic guts. Replace them with…
sa smith | January 23, 2014This therapy isnt perfect, but its a cool start!
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
'Choroideremia' is a kind of blindness caused by deletion of a gene called REP1. Because REP1 is on the X-chromosome, there is no 'second chance gene' on the other chromosome in men. They have no functional REP1, so, men with this deletion get choroideremia.
This makes treating choroideremia with gene therapy attractive because we know what is going on. It is not an overly complex network-- its a deleted gene that is causing the trouble. We…
sa smith | March 25, 2013Using a genetically modified HIV-1 to genetically modify leukemia patients T-cells to teach them how to kill the cancer?
YAWN!
Thats childs play, at this point.
Lets give those GMO viruses a real challenge.
Lets get them to fight a real bastard form of leukemia-- B-cell Acute Lymphoblastic Leukemia (B-ALL) in adults.
In kids, B-ALL has high cure rates (>80% cured).
In adults, things are more difficult (38% are alive 5 years post-diagnosis). Things get much more difficult if the adult has relapsed (7% alive 5 years post-diagnosis).
Can a GMO HIV-1 do better than 7% in relapsed adult B-ALL…
oracknows | March 15, 2013It's been a long time, been a long time, been a long lonely, lonely, lonely, lonely time.
Besides indulging my taste for shamelessly working lyrics from Led Zeppelin and other classic bands into my post, what am I talking about? Simple. Things have been way too serious lately. I mean, just look at yesterday's post. Sure, I framed it semi-humorously, with The Evil Skeptics infiltrating the screening of the Burzynski sequel, but part of the reason that I did that is because the subject matter is so unrelentingly depressing. That's why when a reader sent me a link to the victim target subject of…
dr. dolittle | February 18, 2013 Image of beagle from www.dogbreedinfo.com/beagle.htm
Diabetes is characterized by high blood sugar. The cause of high blood sugar differs for people with type 1 versus type 2 diabetes. For type 1 diabetics, the pancreas produces little or no insulin, the hormone responsible for lowering blood sugar. For type 2 diabetics, tissues in the body are not responsive to insulin, termed insulin resistance, resulting in persistently elevated blood sugar. Muscle tissue is the main site of glucose disposal in the body and therefore, the main site of insulin's action.
Researchers from Universitat…
sa smith | February 4, 2013This is one of the cool ideas not included in the 1800 human gene therapy trials because its still in animal models, not humans:
Preclinical Therapy of Disseminated HER-2+ Ovarian and Breast Carcinomas with a HER-2-Retargeted Oncolytic Herpesvirus
If Ive said it once, Ive said it 1000 times: 'Cancer' is not a monolithic entity. We have not 'cured cancer' because there are lots and lots and lots of different kinds of cancers.
Some ovarian cancers and some breast cancers have a protein upregulated, HER-2. Scientists have capitalized on this observation to create anti-tumor drugs that focus on…
sa smith | January 31, 2013I wish I wish I wish this article was open access! ARG!!
Gene therapy clinical trials worldwide to 2012 - an update
This article is a great review of gene therapy as a therapy, and how it has evolved from an experiment that killed children to an accepted therapy that is saving and improving lives.
Here is a list of all the diseases that have been/are/will be treated with gene therapy:
http://www.ncbi.nlm.nih.gov/pubmed/23355455
These are trials in humans-- this list doesnt include all of the therapies that are still in the tissue culture/animal model segments of the pipeline. Can anyone…
sa smith | January 28, 2013Hey remember how I told you all scientists are using herpes, the regular-ol much hated cold-sore virus, to treat/cure cancers?
The approach has left us with miraculous effects... or no effects at all.
I mean, half of the melanoma patients lived A LOT LONGER than they were supposed to, several of them ending up with no evidence of disease... But that also means that half werent helped at all.
HSV-1 couldnt help a child with rhabdomyosarcoma... when it was 'supposed' to work so well.
What happened?
Of course that question has a collection of complex answers, and there are lots of scientists…
sa smith | January 9, 2013Whoda thought that injecting viruses into peoples hearts would be not only fun, but good for their health! I just wrote about this little guy that can turn regular heart muscle cells into pacemaker cells (in guinea pigs), and here is another cool study hot off the presses:
Long-Term Follow-up Assessment of a Phase 1 Trial of Angiogenic Gene Therapy Using Direct Intramyocardial Administration of an Adenoviral Vector Expressing the VEGF121 cDNA for the Treatment of Diffuse Coronary Artery Disease
There is a cell protein-- VEGF, vascular endothelial growth factor-- that convinces your body to…
sa smith | January 2, 2013When youre trying to cure a genetic disease with a genetically modified virus, you dont have to get a perfect score.
You dont have to cure everyone in the trial.
You dont even really have to cure them-- Just make their lives a little easier, making it so that they can skip a few invasive procedures, or make it so that you can back off on some more aggressive traditional therapies.
Teeny-tiny wins are still WINS.
Not because we will take what we can get, but because we can modify those tiny wins and turn them into bigger and better wins.
Take Hemophilia B, for example:
The scientists in this…
sa smith | December 17, 2012Im seriously, you guys.
ANY MEDICAL CONDITION YOU CAN THINK OF: There is a scientist, somewhere, trying to use viruses to treat/cure that condition.
The latest:
Direct conversion of quiescent cardiomyocytes to pacemaker cells by expression of Tbx18
Lots of people, either through age or genetic defect, need pacemakers.
Pacemakers, while functional, come with a list of difficulties-- from the insertion process to making sure the damn battery works, to all the bacteria that could hitch a ride on the pacemaker to cause all kinds of complications.
But we are kinda stuck. I mean, we cant just make…
sa smith | December 11, 2012A lot of folks have been forwarding me this story from the New York Times--
In Girl’s Last Hope, Altered Immune Cells Beat Leukemia
It is a FANTASTIC story about a little girl who had been sick from leukemia for two years, wasnt responding to conventional therapies, and her parents chose to try alternative medicine. Not magic lotions and potions from soulless snake-oil salesmen-- A GMO virus, made in part with HIV-1.
What, exactly, her physicians used, I do not know-- This is a MSM piece, not a peer-reviewed paper, but Im guessing they used something like what I wrote about last year:
Oh,…
sa smith | October 10, 2012Lets play a game.
Think of a human disease. Any disease. Viral, bacterial, genetic, acquired, anything.
Im pretty sure that no matter what disease you just thought of, there is a scientist, somewhere, trying to use a virus to cure/treat that disease.
As I was doing my rounds on PubMed, looking for cool new research, I stumbled upon this paper:
Canalostomy as a Surgical Approach for Cochlear Gene Therapy in the Rat.
I couldnt find this article online, so I searched PubMed for more info on using GMO viruses to treat deafness.
NOH MAH GAWD. There are SO MANY papers! A handful:
Cochlear…
sa smith | September 25, 2012So what do you do after you use a genetically modified virus to treat-->functionally cure Hemophilia B?
Why, use a genetically modified virus to treat-->hopefully functionally cure Hemophilia A, of course!
Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A
Though they are both 'hemophilia', hemophilia A and B are different creatures. Likewise, the way virologists are going about treating these two illnesses are different.
Hemophilia B is when someone (usually a male) has mutations in their clotting Factor IX…