Archive for March, 2015

Designation supports the advancement of CTL019 to help address the unmet need of patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL) The filing was submitted by the University of Pennsylvania's Perelman School of Medicine which is conducting the CTL019 Phase I/II clinical trials Novartis and Penn have exclusive global collaboration to research, develop and commercialize CAR T cell therapies for the investigational treatment of cancers

Basel, July 7, 2014 - Novartis announced today that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy status to CTL019, an investigational chimeric antigen receptor (CAR) therapy for the treatment of pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The Breakthrough Therapy filing was submitted by the University of Pennsylvania's Perelman School of Medicine (Penn) which has an exclusive global agreement with Novartis to research, develop and commercialize personalized CAR T cell therapies for the treatment of cancers.

This is the fifth Breakthrough Therapy designation for Novartis, continuing the company's trajectory as a leader in developing innovative therapies to help treat diseases in which there remains significant unmet medical need[1],[2],[3],[4]. Novartis' Zykadia(TM) (ceritinib, previously known as LDK378), for the treatment of anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC), is one of the first medicines to receive an FDA approval following earlier receipt of Breakthrough Therapy designation by the FDA[5].

"This Breakthrough Therapy designation underscores the potential of CTL019 as a life-saving therapy for patients with relapsed/refractory ALL, who are in desperate need of new treatment options," said David Epstein, Division Head, Novartis Pharmaceuticals. "Novartis welcomes increased dialogue with the FDA and a potentially expedited review to streamline the development of CTL019 and hopefully bring this promising therapy to patients as quickly as possible."

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. The designation includes all of the fast track program features, as well as more intensive FDA guidance. It is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met[6].

"This is a major milestone as we are now one step closer in helping address the high unmet needs of this patient population," said Carl H. June, M.D., Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in the Perelman School of Medicine and director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania. "We are excited about the strength of the positive early data seen in pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia and look forward to building upon these findings as we continue advancing the CTL019 clinical program in Phase II trials."

Novartis recently established the Cell and Gene Therapies Unit under the leadership of Usman Azam, Global Head, to bring an intense focus on advancing innovative cell-based therapies, including the development of CARs. Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program, CTL019.

About CTL019 CTL019 uses CAR technology to reprogram a patient's own T cells to "hunt" cancer cells that express specific proteins, called CD19. After they have been reprogrammed, the T cells (now called CTL019) are re-introduced into the patient's blood; they proliferate and bind to the targeted CD19+ cancer cells and destroy them.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world.

About Acute Lymphoblastic Leukemia Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children, representing approximately 25% of cancer diagnoses among children younger than 15 years[7]. It can also occur in adults. ALL is a type of cancer in which the bone marrow makes too many abnormal white blood cells (lymphocytes). ALL usually gets worse quickly if it is not treated and can be fatal within a few months; therefore it is critical for patients to start treatment soon after diagnosis. Patients with relapsed ALL experience ALL cells returning in the marrow and a decrease in normal blood cells following their remission. Patients with refractory ALL still have leukemia cells in their bone marrow following treatment[8].

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Novartis personalized cell therapy CTL019 receives FDA ...

The Sims 3 | Perfect Genetics Challenge #1 - Azkadellia Settles In
I take on the Perfect Genetics Challenge for The Sims 3! Azkadellia Ideal settles in to her new home and a new job! Challenge Rules: --------- Perfect Genetics Challenge - http://modthesims.info/t...

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Quick Medical Genetics - Aicardi syndrome
This is a lecture about the genetic disease Aicardi syndrome for trainees and medical professionals. Lecture by Philip M. Boone, MD, PhD. References: http://www.omim.org/entry/304050 http://www.nc...

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Ponderlust | Ep. 5: Genetics

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Gene therapy for CCALD
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SuperCells at Centre for Life
Exhibition at Centre for Life, Newcastle. Exhibition presented by the Stem Cell Network and produced by the Sherbrooke Museum of Nature and Science (Quebec, Canada); in partnership with the...

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Stem Cell Injection Treatment Stem Cell Therapy
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Knee arthritis 3 years after stem cell therapy by Harry Adelson, N.D.
Donna from Colorado describes her outcome three years after stem cell therapy for her arthritic knee by Harry Adelson, N.D. http://www.docereclinics.com.

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My Conversation with Ralph Snyderman, Father of Personalized Medicine (Pt 2)
In Part 2 of this video series, Ralph Snyderman MD and Pat Salber MD (@docweighsin) discuss the challenge of engaging the unengaged patient. Just telling folks what they need to do to stay...

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My Conversation with Ralph Snyderman, Father of Personalized Medicine (Pt 1)
Ralph Snyderman MD, Chancellor Emeritus of Duke University has been called the Father of Personalized Medicine. Who better to help us understand what #39;s going on in this exciting field. In this...

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Teaching Independent Transfers for Spinal Cord Injury Video: Jennifer Hastings | MedBridge
Watch the first chapter FREE: https://www.medbridgeeducation.com/courses/details/teaching-independent-transfers-for-spinal-cord-injury Instructor: Jennifer Hastings, PT, PhD, NCS In this...

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The Prolotherapist: Fox Morning Blend, March 10, 2015
Ross Hauser, MD discusses hip arthritis on Fox 4 Morning Blend in southwest Florida. If you have hip arthritis, or other hip pain, we would love to help you. We treat much more than hips too!...

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David Kekich - Regenerative Medicine (Part 3/6)
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What types of conditions does regenerative medicine treat?
Dr. Trevor Bullock of Regen Orthopedics describes the types of injuries and conditions that can be helped by regenerative medicine. These include a number of chronic conditions: arthritis,...

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With a little help from my friends fundraising concert | Frankston TV
We would like to thank all the artists who performed at the fundraising concert for Wayne Higgins on Sunday the 15th of March. EVERYONE, including Glenn Shorrock, Mike Rudd, Andrew Wishart,...

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Measuring the concentration of leukemia cells in patient bone marrow during the first 46 days of chemotherapy should help boost survival of young leukemia patients by better matching patients with the right intensity of chemotherapy. St. Jude Children's Research Hospital investigators led the research, which appears in the March 20 edition of the journal Lancet Oncology.

The findings stem from a study of 498 children and adolescents with acute lymphoblastic leukemia (ALL) enrolled in a St. Jude-led protocol between 2000 and 2007. The clinical trial was the first to use measurement of residual leukemia cells -- or minimal residual disease (MRD) -- in bone marrow to help guide therapy. St. Jude pioneered MRD measurement as a tool to guide leukemia treatment.

"This analysis shows that MRD-directed therapy clearly contributed to the unprecedented high rates of long-term survival that patients in this study achieved," said first and corresponding author Ching-Hon Pui, M.D., chair of the St.Jude Department of Oncology. Overall, 93.5 percent of patients were alive five years after their cancer was diagnosed. "MRD proved to be a powerful way to identify high-risk patients who needed more intensive therapy and helped us avoid over-treatment of low-risk patients by reducing their exposure to chemotherapy," Pui said.

Researchers hope the findings will expand use of MRD measurements to guide leukemia treatment in children and adults.

The technique might also help identify patients who could be cured with less intensive chemotherapy, Pui said. Overall long-term survival was 97.9 percent or better for 244 patients in this study classified as low risk based on a variety of factors including their age at diagnosis and MRD of less than 1 percent on day 19 of treatment. "Given the excellent outcome, it will be important to determine if treatment can be further reduced in this subgroup of patients," Pui said.

In countries with limited resources, Pui said the findings suggest that results of MRD on day 19 can be used to reduce treatment-related deaths by identifying patients who will likely be cured with low-intensity chemotherapy. "This study demonstrates these patients have an extremely low risk of relapse," he said.

The study showed that measuring MRD just twice during remission induction therapy -- at day 19 and day 46 -- rather than multiple times during the more than two years of treatment was sufficient to guide treatment of most pediatric ALL patients. That will help save money and protect patients from the discomfort and risks associated with bone marrow aspiration for MRD testing. MRD measurements should continue, however, to guide treatment of patients with detectable MRD on day 46 of treatment. That is a level of 0.01 percent or more, which translates into one leukemia cell in 10,000 normal cells.

MRD was not a perfect predictor of relapse risk. Cancer returned in 26 of the 430 patients with undetectable MRD when treatment ended after 120 weeks. Researchers are working to develop even more sensitive methods for tracking treatment response in order to identify those at risk for having their cancer return.

Overall, researchers showed that regardless of other risk factors, including age at diagnosis or the initial white blood cell count, patients with an MRD level of 1 percent or more on day 19 of therapy were far less likely than other young leukemia patients to be alive and cancer-free 10 years later. Having detectable leukemia cells on day 46 of treatment was also associated with lower survival.

MRD levels on days 19 and 46 led to the reclassification of 50 patients from low risk to a higher risk leukemia that warranted more intensive therapy. Researchers credited the change with boosting survival.

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Measuring treatment response proves to be a powerful tool for guiding leukemia treatment

New York, March 20 (IANS): High stress levels can have a critical impact not only on the surface, making our skin age, but also on a molecular level, when stressed cells cannot cope with the pressure and perish much faster than the ones which can.

In a new research report released on Thursday, scientists at the University of California, Berkeley, analysed blood stem cells and found that the cell's ability to repair damage in the mitochondria, their power source, was critical to their survival.

Researchers tried to "relax" these stressed-out cells by slowing down the activity of their mitochondria.

"We found that by slowing down the activity of mitochondria in the blood stem cells of mice, we were able to enhance their capacity to handle stress and rejuvenate old blood. This confirms the significance of this pathway in the aging process," Xinhua news agency quoted Danica Chen, an assistant professor with the Department of Nutritional Sciences and Toxicology.

This pathway lies mainly in the multitude of proteins that need to be folded properly for the mitochondria to function correctly. When the folding goes awry, the mitochondrial unfolded-protein response, or UPRmt, kicks in to boost the production of specific proteins to fix or remove the misfolded protein.

Researchers found that certain proteins known as SIRT7 help cells cope with the stress of unfolding the proteins in the mitochondria, helping those with higher levels of SIRT7 survive longer by making them "unwind". But the levels of SIRT7 decrease as people age.

"The protein level decreases as years go by," Chen said. "But if we increase this protein in blood stem cells, we can make them live longer. Cells in general don't just die suddenly; they are submitted to high stress levels and lose their functions with age."

Chen does not want to encourage the thought that she and other researchers have found the "fountain of youth", but more of a new path for study.

"We still don't know if this would work on other kinds of stem cells, such as pancreatic stem cells or heart cells, and we don't have any expertise with those tissues, so we would be very happy to collaborate with other laboratories to tackle the matter," she said.

The study, published on Thursday in the Science journal, is expected to help researchers gain more insight into the aging process, and even slow it down.

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Cambridge stem cell scientists searching for new cystic fibrosis treatments have grown "mini-lungs" in a laboratory.

The millimetre-wide cell clusters were created using stem cells derived from the skin of patients with the devastating lung disease.

They are the latest in a line of 3D "organoids" produced to mimic the behaviour of specific body tissues, following "mini-brains" for studying Alzheimer's disease and "mini-livers" to model diseases of the liver.

Dr Nick Hannan, led the team from Cambridge University.

He said: "In a sense, what we've created are 'mini-lungs'.

"While they only represent the distal (outer) part of lung tissue, they are grown from human cells and so can be more reliable than using traditional animal models, such as mice.

"We can use them to learn more about key aspects of serious diseases - in our case, cystic fibrosis."

Cystic fibrosis occurs when the movement of water to the inside of the lungs is reduced, causing a build up of thick mucus that leads to a high risk of infection.

The scientists reprogrammed ordinary skin cells to create stem cells that could be transformed into lung tissue.

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Stem cell "mini-lungs" created in Cambridge University lab

Stem cell therapy and me1
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In this image provided by the National Human Genome Research Institute, a NHGRI researcher monitors a DNA sequencing machine at the NIH in Bethesda, Md.(AP Photo/National Human Genome Research Institute, Maggie Bartlett)

A breakthrough gene-editing process developed in 2012 could potentially be used to eradicate genetic diseases in humansor make a person more intelligent or attractive. The Crispr-Cas9 or "DNA scissors" technique involves making DNA-altering changes to sperm, eggs, or embryos that could then be inherited by future generations.

For example, negative mutations could be replaced with "corrected" DNA strings, Bloomberg reports. The technique is relatively easy for anyone who knows about molecular biology, and it's already been tested in mice, rats, and monkeys, the New York Times reports.

But a group of 18 biologists, including a Crispr-Cas9 inventor, cautions the dangers of genome-editing in a new study and warns "scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans" until the consequences "are discussed among scientific and governmental organizations." In a similar essay last week, several scientists called for a moratorium on gene-editing research that "could be exploited," as "an ethical breach could hinder a promising area of therapeutic development." Scientists around the world won't be forced to abide by the request, but the biologists hope to use their "moral authority" to bar experiments in parts of the world where lab research isn't well regulated.

Similar requests have been agreed to in the past; however, scientists tell Nature that research papers about created human embryos with edited genomes have already been submitted for publication.

Ethicists say editing human DNA to boost intelligence or beauty should never be attempted, but there are also potential dangers as the process can change genes apart from the ones intended.

"You could exert control over human heredity with this technique, and that is why we are raising the issue," an expert says. (Britain is the first nation that will allow three-parent babies.)

This article originally appeared on Newser: Scientists: Let's Halt Gene-Editing in Humans

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Scientists want to halt gene editing in humans

Planet Bizzaro Ep Seventeen Genetic Gas
Zoomer and his pals find out Zargon has plagued their food supply with genetically engineered bugs, so Zoomer uses his own genetic engineering to create a super fruit that ends up creating...

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International Centre for Genetic Engineering and Biotechnology
ICGEB: A brief overview and introduction by Mauro Giacca, Director-General, and Researchers in Trieste, Italy.

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