The motorcycle-riding, guitar-playing Collins announced Tuesday he will step down by the end of the year from his job as chief of the research agency, having served more than a dozen years under three presidents. While Dr. Collins saved their lives. Accolades for Collins flowed in from the scientific community as soon as news of his impending departure was announced. And the praise from politicians was distinctly bipartisan. Richard Burr R-N. Starting in the s, every biomedical advance, from in vitro fertilization to fetal tissue and stem cell research to the cloning of Dolly the sheep resulted in intense political fights and blaring headlines.
But after that doubling, a stagnant NIH budget caused cutbacks in university research, creating controversy of its own , which Collins had to manage. Controversy comes with the territory. America, a science funding advocacy group. Both Woolley and McKinney said they are confident there are plenty of good candidates to lead NIH, although neither would name any. Julie Rovner, who has covered health care for more than 30 years, offers insight and analysis of policies and politics in her regular HealthBent columns.
But it ended up benefiting — and becoming a must-attend event for — scientists at all career stages. Back in the U. N Engl J Med. Posted In: News. Posted on November 8th, by Dr. Posted In: Director's Album - Photos. Posted on November 4th, by Dr. For many people struggling with depression, antidepressants and talk therapy can help to provide relief. Her improvement has lasted now for more than a year.
Researchers led by Andrew Krystal, UCSF, Weill Institute for Neurosciences, attempted in the latest work to translate this valuable lead into improved care for depression.
Their results were published recently in the journal Nature Medicine [1]. Krystal and colleagues, including Chang and Katherine Scangos, who is the first author of the new study, began by mapping patterns of brain activity in the patient that was associated with the onset of her low moods. There, it continuously monitors brain activity and can deliver electrical stimulation via two leads, as shown in the image above [2].
Researchers found they could detect and predict high symptom severity best in the amygdala, as previously reported. The next question was where the electrical stimulation would best relieve those troubling brain patterns and associated symptoms.
It will be important to learn whether this precision approach to DBS is broadly effective for managing treatment-resistant depression and perhaps other psychiatric conditions. It will take much more study and time before such an approach to treating depression can become more widely available.
Also, it is not yet clear just how much it would cost. But these remarkable new findings certainly point the way toward a promising new approach that will hopefully one day bring another treatment option for those in need of relief from severe depression. Nat Med. Posted on November 2nd, by Dr. Leave a Comment. Collectively, up to 30 million Americans, many of them children, are born with one of the approximately 7, known rare diseases.
Most of these millions of people also share a common genetic feature: their diseases are caused by an alteration in a single gene. Many of these alterations could theoretically be targeted with therapies designed to correct or replace the faulty gene. But there have been significant obstacles in realizing this dream. The science of gene therapy has been making real progress, but pursuing promising approaches all the way to clinical trials and gaining approval from the U.
Another challenge is economic: for the rarest of these conditions which is most of them , the market is so small that most companies have no financial incentive to pursue them.
To overcome these obstacles and provide hope for those with rare diseases, we need a new way of doing things. Its aim: optimize the gene therapy development process and help fill the significant unmet medical needs of people with rare diseases.
The goal of the Consortium is to reduce the cost of gene therapy protocols and increase the likelihood of success, making it more attractive for companies to invest in rare diseases and bring treatments to patients who desperately need them. Fortunately, there is already some precedent.
This pilot project has helped to develop adeno-associated viruses AAVs , which are small benign viruses engineered in the lab to carry a therapeutic gene. They are commonly used in gene therapy-related clinical trials of rare diseases. Since the launch of PaVe-GT two years ago, the project has helped to introduce greater efficiency to gene therapy trials for rare disease.
The BGTC will now continue to advance in-depth understanding of basic AAV biology and develop better gene therapies for rare and also common diseases. The consortium aims to develop a standard set of analytic tests to improve the production and functional assessment of AAVs and therapeutic genes.
Such tests will be broadly applicable and will bring the needed manufacturing efficiency required for developing gene therapies for very rare conditions. The BGTC also will work toward bringing therapies sooner to individuals in need.
To start, BGTC-funded research will support four to six clinical trials, each focused on a distinct rare disease. Through these trials, the BGTC will chart a path from studies in animal models of disease to human clinical trials that cuts years off the development process.
This will include exploring methods to streamline regulatory requirements and processes for FDA approval of safe and effective gene therapies, including developing standardized approaches to preclinical testing.
This work promises to be a significant investment in helping people with rare diseases. Today, only two rare inherited conditions have FDA-approved gene therapies.
The hope is this investment will raise that number and ultimately reduce the many significant challenges, including health care costs, faced by families that have a loved one with a rare disease. In fact, a recent study found that health care costs for people with a rare disease are three to five times greater than those for people without a rare disease [2].
These families need help, and BGTC offers an encouraging new way forward for them. NIH news release, October 27, Tisdale, A.
Orphanet J Rare Dis 16,
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