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Patients suffering from rare Genetic Diseases hold Silent Protest March against Costly Treatments

An awareness programme held after the walk witnessed a discussion on the ways and means of funding the treatment of rare genetic disorders

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New Delhi, Sep 30, 2016: Patients suffering from Lysosomal Storage Disorders, or rare genetic diseases, organised a silent protest march to seek that the government frame a policy on the issue and allocate funds for their costly treatment, a release said.

Holding placards and banners to highlight their plight, over two dozen patients, and their caregivers participated in the march from Jantar Mantar to Connaught Place, organised by the Lysosomal Storage Disorders Support Society (LSDS).

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“Two committees have been formed this year to address the concerns of patients with rare diseases including LSDs but nothing concrete has emerged in terms of providing free treatment, the primary demand of patients and families,” said the release.

It said while the Delhi government formed a committee under the chairmanship of Dr. D K Tempe, Dean of Maulana Azad Medical College in February 2016 to develop a policy and funding for rare genetic diseases, another committee was constituted by the Union Health Ministry this year for developing a policy on providing medicines for treatment of rare diseases.

Maulana Azad Medical College. Source: mamc.ac.in
Maulana Azad Medical College. Source: mamc.ac.in

An awareness programme held after the walk witnessed a discussion on the ways and means of funding the treatment of rare genetic disorders.

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“Treatment for LSDs is beyond the reach of patients. Government support in terms of funding is imperative to help them lead a normal life. We urge the government to expedite the process of forming a policy and funding system for the treatment of LSDs without further delay so that patients get timely treatment,” said LSDS President Manjit Singh.

In Delhi, there are 14 known patients suffering from Gaucher disease, the most common form of LSD, said the release. The disease is an inherited genetic condition that causes fatty deposits to build up in organs and bones, as its sufferers do not have enough of an important enzyme (glucocerebrosidase) required to break down a certain type of fat molecule (glucocerebroside).

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Enzyme Replacement Therapy (ERT), the most effective treatment for LSDs, is available in India but most patients are deprived of receiving it due to the high cost. These treatments are not supported by government or insurance companies unlike in developed countries, the release said. (IANS)

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To Treat Brain Cancer Scientists Taking Polio’s Help

The result is a longer life for patients whose brain cancer returned

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A radiologist examines the brain X-rays of a patient. In a small study, patients with brain tumors were given genetically modified poliovirus, which helped their bodies attack the cancer.
A radiologist examines the brain X-rays of a patient. In a small study, patients with brain tumors were given genetically modified poliovirus, which helped their bodies attack the cancer. VOA

There’s an exciting new breakthrough in treating some types of deadly brain tumors, that uses, of all things, a polio virus. Doctors at Duke Health in North Carolina genetically altered the virus because it produces such a strong immune response in our bodies. The result is a longer life for patients whose brain cancer returned. All had glioblastoma, an aggressive and lethal type of brain cancer. Of the 61 patients in the study, 21 percent who got this new treatment had were alive three years later.

While that number is low, the survival rate for glioblastoma is normally even lower, usually, a year and a half after diagnosis. The researchers compared the study group to a group of patients drawn from historical cases at Duke. Only four percent of these patients survived three years after treatment.

Dr. Annick Desjardins, one of the authors, said not all patients respond, but if they do, they often become long-term survivors. Desjardins said, “The big question is, how can we make sure that everybody responds?”

Stephanie Hopper was the first patient in the Duke study. She was diagnosed with glioblastoma eight years ago. She had the tumor removed, but two years later, it returned. The modified virus is directly injected into the brain during surgery. After treatment, Hopper’s tumor shrunk to the point where it’s barely noticeable in her brain scans, and the tumor is continuing to shrink.

Dr. Darell Bigner is the senior author of the study which was published in the New England Journal of Medicine. He explained that by modifying the virus, it destroyed its ability to infect nerve cells and cause polio, but the virus retained the ability to kill cancer cells. In fact, the modified virus targeted the tumor cells.

Prior to the study, the researchers decided they needed a different approach to treating glioblastomas which is why they looked at experimenting with the polio virus.

There's an exciting new breakthrough in treating some types of deadly brain tumors, that uses, of all things, a polio virus. Doctors at Duke Health in North Carolina genetically altered the virus because it produces such a strong immune response in our bodies
There’s an exciting new breakthrough in treating some types of deadly brain tumors, that uses, of all things, a polio virus. Doctors at Duke Health in North Carolina genetically altered the virus because it produces such a strong immune response in our bodies. Flickr

One of the goals of a phase one trial is to find a dose that is safe. In some patients, the therapy caused their brains to swell and they experienced seizures and other bad side effects so the dose was lowered. Study participants were selected according to the size of their recurring tumor, its location in the brain and other factors designed for patient protection.

For five of the 61 patients in the trial, the cancer returned. They were treated a second time and Bigner says, “Those that we’ve been able to follow long enough have responded to the treatment the second time. That’s extremely important.” Combining the polio virus with other approved therapies is one approach already being tested at Duke to improve survival.

Also read: A One-Shot Nanoparticle Vaccine for Polio is Developed by MIT scientists

The researchers are continuing their work on treating glioblastomas and planning other studies as well. They want to test the therapy on children’s brain tumors. The therapy may also expand beyond brain tumors to include breast cancer and melanoma patient as well. (VOA)