US Scientists Use Gene Editing Tool to Fix Mutation in Human Embryo.

Oregon scientists use breakthrough gene editing procedure to fix birth defect in embryo. According to a CBS TV News story, August 2 2017, researchers from the Oregon Health and Science University ran an experiment that safely repaired a mutated gene in a human embryo. The experiment used a procedure is called CRIPR cas 9, which is a genetic editing tool. The scientists edited out a defective mutation of DNA in the embryo. In a next step, the DNA automatically repaired itself without the mutation. The successful experiment demonstrated that you are scientists are technically capable of repairing mutations of DNA in embryos. This sort of thing was once considered science fiction, but now it is reality. Clearly the FDA will need to weigh in on whether or when medical clinical trials might begin in the US. There are many bio ethical considerations that have to be made before this experimental method comes to the marketplace. For example this type of technology could be a way of curing certain types of disease conditions such as enabling a person with HIV to have an immune system that could prevent the onset of AIDS or perhaps this technique could be used to fight certain forms of cancer. Down the line in the far future people might consider the possibility to alter human features to their liking. Scientist in other countries such as China, South Korea and others are using the gene editing technology to suit the needs of their localities.

The FDA Approved a New Gene Therapy to Treat Blood Cancers.

CBS News reported today that the FDA has approved a new type of therapy that will treat pediatric patients with a specific form of leukemia. The therapy is called CAR – T therapy. The concept behind the therapy is that patients have blood with T cells removed from their bloodstream and sent to a factory in New Jersey. The factory uses a specific type of DNA gene therapy that enables the T cells to recognize leukemia cells. The genetically modified T cells are then infused back into the patient. So the therapy works like a living drug that is injected into the patient that goes out and attacks cancer cells. 

The drug company, Novartis is working with doctors and their patients to deliver this new type of DNA therapy. So far, the therapy is producing some remarkable results by putting the cancer into remission. 

Dr. David Agus, from USC’s Westside Cancer Center, was very excited about about the promise for this new therapy. In addition to the therapy itself, the doctor explained that Novartis had developed an innovative payment plan based on the value of the treatment. That is, if the therapy works for a certain amount of time then the patient would pay a fee. If the therapy does not work there is no charge. This innovative therapy and payment system costs about $495,000. Whereas this fee is very large, it compares to be far less than a bone marrow transplant, which costs about $800,000. Novartis said that this new therapy will be tested for other diseases in the near future starting with lymphoma and other similar cancers. Dr. Agus is hopeful that this therapy could be used to treat other diseases such as diabetes or other conditions.

FDA Expanded Approval to a Cancer Immunotherapy

CBS’s doctor David Agus said in a May 30 story of CBS This Morning, that the FDA had recently expanded the uses for the immunotherapy known as KeyTruda for additional indications or medical conditions. All cancer cells operate in a type of stealth mode that allows it to hide from immunity cells that would normally eat it. The therapy targets cancer cells that have a biomarker that enables them to switch on a gene function that says it’s OK for the immune system to eat it. The therapy was originally used to fight lung cancer and some forms of skin cancer. The new FDA approval would expand the list to treat cancer searches kidney cancer, endometrial cancer, colon cancer and a growing list of other cancers. This immunotherapy enables patients previously without hope, to get an extended life of 1 to 3 years. Dr. Agus said that the cancer always returns, because the cancer mutates into a different form that is not suitable for treatment with this immunotherapy.

Recent News in Cancer Research, 2017

 ⁃ Computer model says leading cause of cancer is due to random mutations of the DNA in cells.

During March, three scientists at John’s Hopkins research labs have released results of their mathematical model based cancer study that was published in the journal Science, concluded that the chance of getting cancer is mostly caused by random mutations of DNA. 

The top line numbers boil down to this: random mutations in DNA:66%, preventable cancers with environmental causes: 29%, genetically or heredity based cancers 5%. The scientists also said that it’s important to understand that at least three random mutations in the DNA are needed before any cancer tumor can be formed. This cancer study is a follow on study of a that occurred two years ago which was met with much controversy because of its broad scope. This current study is beginning to generate controversy as well, as scientist lineup on either side of the discussions. It’s interesting to note that the network news shows on TV did not mention the study at all.

 ⁃ Today the FDA fast tracked a cancer immunotherapy to fight a rare form of blood cancer called advanced lymphoma. 

According to Dr Jon LaPook of CBS News, there is a new experimental immunotherapy called, car-T. The immunotherapy uses a patient’s own immune cells that are removed from the body and are treated by genetic modification that re-programs the immune cells to target lymphoma cells, and then put back into the patient’s blood stream. The cancer treatment was developed by researchers at MD Anderson Cancer Center in Texas. Dr. Frederick Lock, the clinical trial director, reported results of 101 patients that had advanced lymphoma that used the new immunotherapy. He said that the immunotherapy “puts a GPS navigation on the front of the cell.” He said when immunotherapy is put back into the patient’s blood stream, they reprogrammed immuno cells destroy the advance lymphoma cells.The results: after a single treatment 39% of the patients had no evidence of cancer after 8-months. There were three treatment related deaths, and there were some challenging side effects. 

 ⁃ The future of cancer treatment: immunotherapies.

On March 12, 2017 CBS News’ Dr. Jon LaPook reported on recent developments in immunotherapy. Memorial Sloan Kettering Cancer Center is using an experimental cancer treatment called car–T to treat pediatric leukemia in a clinical trial. The young girl patient featured in Jon’s story had already failed several rounds of chemotherapy and as a last ditch effort she was enrolled in the clinical trial. About six weeks after therapy, the girl had no sign of cancer in her.

Researchers at the National Cancer Institute have been studying immunotherapy for 40 years. Dr. Steven Rosenberg in 1984, was the first person to use a person’s own immune system to fight and beat cancer. He said that we are now at a sort of a inflection point at which the knowledge learned of the last four decades will be used to offer some dramatic treatment results in a few years to come. So far immunotherapy has been successful treating cancer in the lung, bladder, melanoma. 

A new class of Immunotherapy that is available now are called checkpoint inhibitors. These treatments include products called Keytruda, Opdivo, and Tecentriq. These products target cancer in lung,kidney,bladder, melanoma, and more. Perhaps only 10% of all cancers can benefit by using immunotherapies according to Dr. Rosenburg.

So far there is not much going on in treating metastatic cancer, but scientists are hopeful that they are learning new things every day about how to treat that type of cancer.

President Trump Targets Healthcare in Today’s Speech to the Congress.

In President Trump’s speech today before members of Congress, he touched on several healthcare topics. 

Most notably, he spent time discussing the need for Congress to repeal and replace the Affordable Care Act or Obamacare. 

He also spoke about the high prices of prescription drugs that have been in the news recently . He said he is looking for a solution to bring the cost of drugs down.

President Trump also spoke about reducing regulation and fast tracking development of new miracle drugs by getting the FDA to move their regulatory processes at a faster pace. The president effectively illustrated the case of Megan Crowley, a person in the audience, who is a victim of a rare genetic condition called Pompe disease. Her father vowed to find a solution to her illness and eventually founded a biotech company with the aim of finding a drug solution that could cure his daughter’s illness. Eventually he succeeded and now Megan has lived longer than expected, and is attending college.

These healthcare topics are important subjects that members of Congress will need to pay attention to in the near term. Industry spokesperson’s are already working on strategies to respond to the president’s call to action.

Emerging Genetic Research Stories Coming This Year

According to CBS’s medical contributor Dr. David Agus on January 4, 2017, we can expect to see more developments in the use of the gene modification enzyme called CRYSPR. This technology enables scientists to alter one of the genetic letters of the 3.2 billion letters of the human genetic code. 
He said that scientists are using IVF mitochondrial replacement techniques to change defective genetic material from the mothers egg which would be replaced with donor mitochondria. 

Another Application is to have a baby get genetic material from three people to eliminate a birth defect. A recent story on CBS September 27, 2016, told a story of a doctor that did a three person fertilization in Mexico for a patient. This IVF technique can be performed in the UK and in Mexico. The story reported that the baby was born successfully in good health.

 Emerging ideas include the potential for using CRYSPR gene editing technology to prevent single gene disorders such as cystic fibrosis, Huntington’s disease and sickle cell anemia. This technology might be used to treat cancer and destroy viruses such as HIV or hepatitis.The first food using this technology is a mushroom that has already reached the market. 

Dr. Agus said that there are concerns that there’s a downside to using this type of technology as well. He mentioned that in China scientists there are working on embryos and editing the genes. And he wonders how are they are managing to do that and what are the concerns for society. He worried that perhaps they are changing hair color, eye color and other things that will make the babies look more interesting, or more attractive. He is worried that this is a slippery slope of things to come. He said that we need some kind of global leadership concerning the ethics of using this technology.

thousands of healthcare and biotech industry investors are coming to San Francisco in early January.

San Francisco will host two major Biotech industry conferences. During the week of January 9, the 35th Annual JP Morgan Healthcare Conference will likely see more than 7000 attendees at the Westin St. Francis. 

Nearby, The Biotech Showcase at the Hilton, will be host to numerous smaller biotech companies that seek to raise funding from a variety of investors. 

As in past years, Other venues will play host to numerous other related events within a short distance in San Francisco.

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