Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering

Feeling sad to read Pat's update. Have lots of hope and prayers for Bluebird and Gene Therapy for Thal. Andy, what are your thoughts?

where can we compare two vectors side by side?

Jay,

I do not believe that is possible. As far as I have been told, Bluebird will not release specific information about it's vector.

Secrecy is dangerous! we are called subjects no problem! we are called guinea pigs no problem!
Secrecy is dangerous! we can guess and smell that it may not be a human patch no problem we need to know.
Secrecy is dangerous! it may invite other problems no problem we need to know.
Secrecy is dangerous! it denies fair competition.

Believe me Jay, I tried to get that info but they won't reveal proprietary information. I prefer the transparency that Michel Sadelain has demonstrated over the years, but I also understand Bluebird's stance.

But Andy then whats the point in being a listed multimillion setup. There is something called patents, copyrights. I expect at-least after getting through national insurance they will reveal detailed information if that's the birds ambition.

Collaboration between MSK and Weill Cornell for finding cure to Sickle cell.


Scientists to develop stem cell therapies to treat blood disorders

Scientists and transplant clinicians at the Ansary Stem Cell Institute at Weill Cornell Medical College and the Center for Cell Engineering at Memorial Sloan Kettering Cancer Center have been awarded a $15.7 million, four-year research grant from the New York State Stem Cell Science Program (NYSTEM). The scientists will translate their approach to manipulate hematopoietic stem cells to cure acquired and inherited blood disorders.

For many patients with such blood diseases, including sickle cell disease, the only hope for a cure requires transplanting normal blood stem cells. But in many instances suitable blood stem cells cannot be found or there are too few cells for transplantation. The consortium of scientists seeks to expand production of stem cells outside the body using specialized blood vessel cells – known as a vascular niche – to nurture the stem cells.

The scientists will conduct two clinical trials using this platform to expand hematopoietic stem cells. The first trial uses the vascular niche to expand umbilical cord blood stem cells for transplantation in patients with blood cancers who cannot be cured by chemotherapy or donors. The second trial aims to correct the genetic abnormality in blood stem cells from patients with sickle cell anemia and return these healthy, functioning stem cells to patients. If successful, the techniques may provide safer, broadly available stem cell transplants to thousands of patients.

“This innovative approach marries Weill Cornell Medical College’s stem cell expansion capabilities with Memorial Sloan Kettering’s robust cell engineering and gene-transfer techniques,” said Dr. Shahin Rafii, principal investigator, director of the Ansary Stem Cell Institute and a Weill Cornell professor of medicine, genetic medicine and reproductive medicine.

“We are indebted to our NYSTEM partners for their support, because this award offers the opportunity for new curative therapies for patients with blood malignancies and sickle cell disease,” said Dr. Joseph Scandura, co-principal investigator and a hematopoietic stem cell physician-scientist and scientific director of Weill Cornell’s Dr. Richard T. Silver, Myeloproliferative Neoplasm Center. Scandura is also an assistant professor of clinical medicine at Weill Cornell.

Sickle cell disease is caused by a mutation in the oxygen-carrying protein hemoglobin that distorts the size and shape of red blood cells, causing them to clump together and stick to blood vessel walls, cutting off blood and oxygen supply to vital organs. There are no FDA-approved techniques to expand blood-forming stem cells to cure this disease, and any patients who do not receive transplants of normal blood stem cells risks life-threatening complications.

“The expansion of blood-forming stem cells is a critical advance for the successful implementation of a number of genetic therapies based on gene addition or gene correction,” said Dr. Michel Sadelain, principal investigator at Memorial Sloan Kettering (MSK). “Our combined expertise in stem cell expansion and globin gene therapy for thalassemia is a strong foundation for developing a potentially curative therapy for sickle cell disease,” he said, referring to a blood disorder in which the body makes an abnormal hemoglobin.

Consortium scientists expect the vascular niche platform will generate large numbers of patients’ own blood-forming stem cells, enabling a genetic modification of their stem cells and avoiding the risks of transplanting cells from another person. These two trials will require manufacturing clinical grade human blood vessel, or endothelial cells, for intravenous infusion for clinical trials.

The NYSTEM grant supports a collaborative effort led by Rafii and Scandura at the Ansary Stem Cell Institute at Weill Cornell and Sadelain and Dr. Isabelle Rivière at MSK. Their team brings significant expertise with stem-cell engineering and clinical translation of cell therapies. Phase I clinical trials are expected to start within the next two to three years.

Direct Link: http://news.cornell.edu/stories/2015/05/scientists-develop-stem-cell-therapies-treat-blood-disorders

Thank you for sharing this catchR. 

This is very interesting after seeing SK nearly disappear for a while. 
Yet this approach seems differently - and the focus seems to be SSA.

I wish this group the best,

I am happy to see our sickle cell brothers & sisters cured as well as our thals. 

Sharmin

Speaking of Dr. Sadelain, I had the chance to talk to him personally in the international conference in abu dhabi in 2013. He is such a wonderful, humble and friendly doctor. At that time, they were testing wheather they will succeed with using immunosuppressive medication rather than the harsh chemotherapy.... He was so excited and gave me a lot of time to explain but unfortunatlly told me intermediates are not on his list at least for the time being....and I can understand why....
I really hope this study will continue and would hear any results in the near future
Manal

Information verified again on September 16, 2015. Good news is they have not extended the estimated study completion date of July 2016. Next round of verification will be in March 2016. Keep your fingers crossed till next announcement.

https://clinicaltrials.gov/ct2/show/NCT01639690

Thank you for sharing Canadian Family.  I am praying for this because of the reduced intensity preparation required as compared to blue bird. 

Thank you Canadian Family for sharing with us.

Thank you for sharing Canadian Family.  I am praying for this because of the reduced intensity preparation required as compared to blue bird.  

Hi Sharmin,

I agree with your assessment. This approach uses low intensity preparation which is much safer, I am more inclined towards this approach compared to Blue Bird's. What I am still not sure is the involvement of Dr. Michel Sadelain in these trails. Last I heard from Andy was that he has its own Bio venture and there were some tension between MSKCC and him. We also have no update from Pat Girondi for a while now. I hope silence brings good news in future for us.

I just got off the phone with Pat Girondi. They have a meeting come up soon and we should be able to get more information after that. He did tell me that the first two patients who started at SK have a greatly reduced transfusion regimen now, and a third patient in his 40's has had some gene expression but less than is needed. It may be that a higher dose is needed in older patients. Keep in mind that these are all beta thal majors and not HbE beta thals, as the early patients that Bluebird has treated are.

hello sir
its now oct. 15 ,
how much the team have proceeded, and what we can expect of the best?

I don't know if Sadelain is still involved with gene therapy at SK. Sadelain became part of a group that started a new company directing research towards gene therapy to cure cancer. This company then had a huge influx of funds from a large pharma company, which I believed created a large windfall for all involved.