What things are we have a expect gene therapy drugs?

Nowadays gene therapy has left a dream and gene therapy has approaching to fact. But everybody wants find a new treatment for genetically disease so that the person can take a price of the required size million dollar.

Last month the Food and Drug Administration (FDA) found a new gene that causes blindness. This experiments ended with $850,000 and the experiments finished lower than many analysts expected. The researching team spent $425,000 per eye. The study cured patients in four years. Only about 1000 to 2000 people in the United States accepted cure in their body. Also we have super expensive treatment for rare patients. Some people who have conditions they have a gene that we called RPE65. the gene may causes blindness getting to worse. In some cases , people will be complete blindness. Spark Therapeutics called this drug: Luxturna. Companies and insurances involve each other to still turn profit.

Treatments

In 2018 gene therapy will build on scientists and drug companies. “Some of the more exciting developments last year concerned successful product development and pricing in the commercial arena. I expect that to move forward some more,” said Dr. Sanjeev Gupta , a professor of medicine and pathology at Albert Einstein College of Medicine and Montefiore Health System. In the cancer field, the FDA allowed CAR T-cell therapy for certain children and young people with one type of acute lymphoblastic leukemia (ALL). This is type of immunotherapy that consists repairing immune cells such as “T cells” to rise their capability to invade cancer. The FDA allowed CAR T-cell therapy for a cure of type of B-cell lymphoma that this cell happens in adults.

Scientist emphasizing this sentence: “major progress in treating cancers [that occur in the blood],”

Gupta said: “we’ll hopefully see more such progress for solid tumors.”

We can see progress another fields is gene editing. Gene editing is capability to changes in a cell’s DNA progression. “Gene editing with CRISPR technologies is becoming more and more exciting,” Gupta said. “Several companies are competing in gene editing — the Sangamo trial for Hunter syndrome was an early example — but we’ll be seeing more trials of gene editing.” Gupta added. Scientists have a genome editing tool which allowed scientists to add/remove and change a part of DNA sequence. Scientists at Harvard University newly found a technique until slow advanced form of deafness in mice. In this conditions genetic mutation occurs in people. Scientists try the treatment in people and this condition may not be long.

Gupta expected to see development genes another area. Also this way being able to control active gene that scientists used the way to cure a disease without changing DNA.

Disease in front of gene therapy

Any disease maybe is a potential target for gene therapy. Some treatments is frequent in hospital. A treatment blindness which FDA allowed is not accidental.

“Eye conditions are amenable to gene therapy for various reasons,” said Gupta, “including easy access to the eye and treatment outcomes that are more readily evaluated than in some other cases.”

One of the complex genetic diseases as Hunter Syndrome consists many organ systems and occurs bad damage inside body. Blood disease are easier purposes because we can be remove and genetically repaired and can be put back their body.

The leukemia one of disease that cured by CAR T-cell therapy. “Hemophilia A and B are major targets that many companies are actively pursuing,” said Gupta. Hemophilia is a genetic disturbance that causes to the blood clot.

“Also, we should see developments in sickle cell disease or beta thalassemia,” added Gupta, “where discrete mutations may be edited to correct abnormal hemoglobin production that causes rapid destruction of red blood cells and anemia.”

Gupta thinks muscular dystrophy one of the targets in future that will be cure.

Gene therapy is more accessible

Gene therapy remarkable achievement that would help a couple thousand people. When one person who is blind what happens , have a blood clotting or have a cancer can’t influence the treatment?

Genetic techniques are still new. “In the short term, the costs will likely be high,” said Gupta. “Eventually, with economies of scale, pricing should come down as it usually does.”

Drug companies have to cover their costs of gene therapy. You can compare a pill when a person take the pill every day for rest of their life. Drug companies need every day to charge about 10000 dollar for drugs.

David Mitchell, researcher and president of the advocacy group Patients for Affordable Drugs, told MIT Technology Review that drug companies will charge “what they think they can get away with.”

Gene therapy has finally moved out of the realm of science fiction… and this time, it may be here to stay.

But with price tags edging toward the million-dollar mark, will anyone but the mega-wealthy be able to afford these new treatments?

Last month, the Food and Drug Administration (FDA) approved a new gene therapy treatment for a rare, inherited form of blindness. The cost? $850,000 — lower than the $1 million that many analysts expected. Still, that’s $425,000 per eye. For a drug that doesn’t fully restore vision. And that’s only known to work for up to four years in some patients.

Only around 1,000 to 2,000 people in the United States may benefit from this one-time treatment. Like other super-expensive treatments for uncommon diseases, the rarity of this disorder partially explains its high cost. People with this condition have a particular gene, known as RPE65, that causes their vision to deteriorate. In some people, it may cause complete blindness.

The drug, called Luxturna, was developed by Philadelphia-based Spark Therapeutics. As the manufacturer and insurance companies wrestle with how to make this drug affordable while allowing the company to still turn a profit, the gene therapy field continues to move ahead. Treatments on the horizon What happens in 2018 for gene therapy will build on what scientists and drug companies accomplished last year.

“Some of the more exciting developments last year concerned successful product development and pricing in the commercial arena. I expect that to move forward some more,” said Dr. Sanjeev Gupta, a professor of medicine and pathology at Albert Einstein College of Medicine and Montefiore Health System.

In the cancer arena, the FDA approved CAR T-cell therapy for certain children and young adults with one type of acute lymphoblastic leukemia (ALL).

This type of immunotherapy involves genetically modifying a person’s immune cells — T cells — to boost their ability to attack cancer. The FDA also approved a CAR T-cell therapy for the treatment of a type of B-cell lymphoma that occurs in adults. With scientists achieving this “major progress in treating cancers [that occur in the blood],” Gupta said “we’ll hopefully see more such progress for solid tumors.”

Another area that will continue to see progress is gene editing, the ability to make precise changes in a cell’s DNA sequence.

“Gene editing with CRISPR technologies is becoming more and more exciting,” Gupta told Healthline. “Several companies are competing in gene editing — the Sangamo trial for Hunter syndrome was an early example — but we’ll be seeing more trials of gene editing.”

CRISPR-Cas9 is a genome editing tool that allows scientists to add, remove, or alter specific sections of DNA, not just in people but in other organisms as well. Scientists at Harvard University recently used this technique to slow a progressive form of deafness in mice.

The genetic mutation that causes this condition also occurs in people, although only in a small number of families — so it may not be long before scientists try this treatment in people. Another area that Gupta expects to see developments in the near future is gene expression regulation. Being able to control which genes are active may give scientists a way to treat diseases without having to alter the DNA. Diseases ripe for gene therapy While any disease is a potential target for gene therapy, some treatments are easier to achieve in the clinic. It’s no coincidence that one of the first gene therapies approved by the FDA is a treatment for blindness.

More complex genetic diseases, such as Hunter syndrome, involve multiple organ systems and the damage occurs deeper inside the body than the eye. Blood diseases are also easier targets because blood cells can be removed, genetically modified, and then put back into a person’s body. This includes the leukemia treated by the recently approved CAR T-cell therapy. But there are other potential treatments.

“Hemophilia A and B are major targets that many companies are actively pursuing,” said Gupta. Hemophilia is a genetic disorder that impairs the blood’s ability to clot.

Gupta thinks muscular dystrophy may be another major target for gene therapy in the near future. Making gene therapy more accessible .Even gene therapies that would help only a couple thousand people would be a remarkable achievement.

But what happens when people who are blind, have a blood clotting disorder, or have cancer can’t afford the treatment?

Gene therapy techniques are still fairly new, so we may see a drop in price once the research and development for these drugs becomes more commonplace.

“In the short term, the costs will likely be high,” said Gupta. “Eventually, with economies of scale, pricing should come down as it usually does.”

Part of the high cost of gene therapy is the one-off treatment. Drug companies have to recover their development costs in a single payment.Compare this to a pill taken every day by a person for the rest of their life — the high costs spread out over this longer period are much less steep.

The question, though, is how much will the price drop?

Drug companies still charge tens of thousands of dollars for drugs that need to be taken every day. And let’s not forget the $100,000 hepatitis treatment that debuted last year. David Mitchell, founder and president of the advocacy group Patients for Affordable Drugs, told MIT Technology Review that drug companies will charge “what they think they can get away with.”

Spark Therapeutics has been conversing with insurance companies about the blindness treatment. One insurer has accepted to pay for luxturna therapy. In past decade gene therapy has worked in clinic.

“We must remain vigilant to make treatments accessible,” said Gupta. “These wonderful achievements of science have resulted from work supported by taxpayer funds over a long period of time. The future of gene therapy is bright, but we must make it brighter by making it affordable for each and every individual.”

References : healthline